Bernie:
We have brought the world together for three days.
I am personally moved by the support from the entire state from the governor on down.
I declare this meeting adjourned.
Tuesday, September 23, 2008
Laurie Zoloth, Northwestern University
Stem cell debates have their own weather . . .they created their own storm and always took place on a certain landscape.
The rest of the world had its own debate . . . but ours was predicated on this notion of "moral authority". The Bush doctrine.
The debate in stem cell science was driven by the same arguments that drove so many debates --notably the one about the economy that has brought us to a crisis.
The American taxpayer dollar is less and less available to scientists . . . several arguments have made their way into our consciousness--all wrong.
1. A free market is inherently just. No regulation is the best regulation.
2. When the wealthy are happy, the poor are happy. Policies that get some people rich will also help those at the bottom
3. You can just bank on an infinitely expandable future in every area.
4. Sheer individual determination can get you what you desire.
5. We need to be accommodating.
All wrong. The American economic catastrophe happened because liberals have been too accommodating . . . we need to stand up for what we believe: the enlightenment, the scientific method, the power of reason. The community has its own place alongside individual determination. The future is not infinitely expandable. There are many policies that benefit the wealthy that also harm the poor, and de-regulation of the mortgage industry is one of them. A free market has built-in inequities that will eventually harm it as a whole as well as cause damage to many of its members.
We have not defended and fought effectively for these ideas.
You, the advocates, made the research possible by forcing the issue. When the election is over and the science goes forward, it will be time to take on the real moral issues, and the advocates will need to be ready to take on those issues. Who gets cured? Who pays for those cures? Who makes those decisions?
It's almost the end.
The rest of the world had its own debate . . . but ours was predicated on this notion of "moral authority". The Bush doctrine.
The debate in stem cell science was driven by the same arguments that drove so many debates --notably the one about the economy that has brought us to a crisis.
The American taxpayer dollar is less and less available to scientists . . . several arguments have made their way into our consciousness--all wrong.
1. A free market is inherently just. No regulation is the best regulation.
2. When the wealthy are happy, the poor are happy. Policies that get some people rich will also help those at the bottom
3. You can just bank on an infinitely expandable future in every area.
4. Sheer individual determination can get you what you desire.
5. We need to be accommodating.
All wrong. The American economic catastrophe happened because liberals have been too accommodating . . . we need to stand up for what we believe: the enlightenment, the scientific method, the power of reason. The community has its own place alongside individual determination. The future is not infinitely expandable. There are many policies that benefit the wealthy that also harm the poor, and de-regulation of the mortgage industry is one of them. A free market has built-in inequities that will eventually harm it as a whole as well as cause damage to many of its members.
We have not defended and fought effectively for these ideas.
You, the advocates, made the research possible by forcing the issue. When the election is over and the science goes forward, it will be time to take on the real moral issues, and the advocates will need to be ready to take on those issues. Who gets cured? Who pays for those cures? Who makes those decisions?
It's almost the end.
Owen Hughes, Pfizer
Law and regulation are what happen to us when we lose trust.
So, there's been a loss of trust from stakeholders on so many sides of this issue. Academics, business people,
He's talking about patents and the strange mess we're probably about to find ourselves in. The bush policy which limited NIH support defined an area where funding cannot go; the burden it imposed on everybody trying to do both kinds of work was pure overhead.
And how does it work when the government marches in and takes control of what you created partially using their money (this is called a reach through) -- a catastrophe . . . but only avoidable with fully understood and clear regulations, like at the NIH.
So now if a state tries to protect its investment, the law is not well understood. The CIRM is operating as a part of the CA government, and its policies have the force of law. Nobody quite knows yet how it will play out . . . and it will get more complicated if the NIH decides to enter the funding arena. The trigger points for the reach through events will be different for feds and states, and we don't clearly know what they are.
You'll have a federal agency, a state agency, private investors, and maybe foreign nationals all with a financial stake in how it plays out.
Time now to overcome the vacuum in which we've been operating during the Bush administration. Won't be simple.
So, there's been a loss of trust from stakeholders on so many sides of this issue. Academics, business people,
He's talking about patents and the strange mess we're probably about to find ourselves in. The bush policy which limited NIH support defined an area where funding cannot go; the burden it imposed on everybody trying to do both kinds of work was pure overhead.
And how does it work when the government marches in and takes control of what you created partially using their money (this is called a reach through) -- a catastrophe . . . but only avoidable with fully understood and clear regulations, like at the NIH.
So now if a state tries to protect its investment, the law is not well understood. The CIRM is operating as a part of the CA government, and its policies have the force of law. Nobody quite knows yet how it will play out . . . and it will get more complicated if the NIH decides to enter the funding arena. The trigger points for the reach through events will be different for feds and states, and we don't clearly know what they are.
You'll have a federal agency, a state agency, private investors, and maybe foreign nationals all with a financial stake in how it plays out.
Time now to overcome the vacuum in which we've been operating during the Bush administration. Won't be simple.
Alta Charo, UMadison, Wisconsin
A look backward in time . . . the politics go all the way back to Roe V Wade in 1973, which led to special legislative rules involving fetuses, which led to same for embryos.
1980 - 1992 were years in which all science was suspended because there was no regulatory board to review proposals. The Reagan and Bush I administrations simply didn't have a review board. Simple solution.
During Clinton, we got a review board . . . and an advisory panel which took 9 months to come out with ethical guidelines, which were basically a plagiarized version of what already existed in the UK.
Then the Democrats were routed from the congress in 1994, and Clinton got very timid about this area. Embryo research was specifically mentioned in the Contract With America. The Dickey amendment was passed in 1995 and is still the law.
In 1998 when we first got stem cells to work on, we had to focus on lines because of that amendment.
In 2001 we had only funding for existing lines so that the gov't couldn't be somehow accused of enticing people to kill embryos.
The result is that we have no rules -- no funding from the feds means that there are no federal rules.
Instead we got state rules along with state funds. When states give you money they have to give you rules as well to say how you can spend it. So now we have a patchwork of funds and rules across the nation that was abandoned by its own federal government. There are also players from around the rest of the globe at work in our country.
So now we have to figure out what the rules are in which people will figure out how to collaborate. We can assume that whoever wins in Nov, we will have federal funds and that also means federal rules . . . but . . . federal law does not eliminate or necessarily override state rules . . . and it doesn't do anything at all to privately funded efforts.
How the hell are we going to come up with a coordinated set of rules . . .it will all be a legislative and bureaucratic slog. It will take a very long time. And the Dickey amendment will still be in force under the congress manages to do its thing.
We may see a dismal parade of scientists traipsing from agency to agency, trying to figure out under what rules they have to operate . . .
So, let's say the scientists somehow navigate that arena and then approach the FDA for permission to carry out their trials. In the 60's and 70's we loved the slowness of the FDA because it kept American women from taking thalidomide as they did in Canada and the UK. During the 80's and 90's there was pressure to hurry up and get things done a lot faster. Then about 5 or 6 years ago there was a slew of drugs subjected to intense intention from the public and the policy community . . . and the slowdown became the right way to go again.
So here we are with translational research in stem cell therapy. The FDA is in Mode Caution, not Mode Aggression. They want to know how confident we are that we're making safe decisions. How pure must your lines be? How many undifferentiated cells are in your transplants? Do we need medical records from the original donors of embryos from 9 years ago? Do we need to maintain immuno-suppressed animals for months and months and months to be sure that tumors are not forming? Will we be forced repeatedly to start over because our animals die of infections before our data is collected?
Okay, let's say that all that gets dealt with. Woo hoo, clinical trials. Uh oh. We now have a HUGE demand, lots of it from people who believe that their condition is going to kill them before they can get the drug. Think about thousands of people demanding to take part in clinical trials . . .ACT UP is the model.
There will somehow have to be access to treatments that have not been tested yet. They did that with the Aids patients -- but this is a biologic and not a drug. And biologics are much more complex and detaile--not to mention expensive- to manufacture than drugs . . . but we're going to have to figure out how to do it. The reason is that Americans are always ready to believe that the newest thing is the best.
But, let's say that all that gets dealt with. Woo hoo, new therapies. Uh oh. We now have the greatest new thing since sunlight in every headline, and that will mean a ton of people ready to buy it who HAVE NO healthcare. Big new struggle.
For 35 years the conversation about stem cell research has focused on the moral status of the embryo . . . let's get over it already.
For my money, the ethical issues are just beginning.
Great talk.
1980 - 1992 were years in which all science was suspended because there was no regulatory board to review proposals. The Reagan and Bush I administrations simply didn't have a review board. Simple solution.
During Clinton, we got a review board . . . and an advisory panel which took 9 months to come out with ethical guidelines, which were basically a plagiarized version of what already existed in the UK.
Then the Democrats were routed from the congress in 1994, and Clinton got very timid about this area. Embryo research was specifically mentioned in the Contract With America. The Dickey amendment was passed in 1995 and is still the law.
In 1998 when we first got stem cells to work on, we had to focus on lines because of that amendment.
In 2001 we had only funding for existing lines so that the gov't couldn't be somehow accused of enticing people to kill embryos.
The result is that we have no rules -- no funding from the feds means that there are no federal rules.
Instead we got state rules along with state funds. When states give you money they have to give you rules as well to say how you can spend it. So now we have a patchwork of funds and rules across the nation that was abandoned by its own federal government. There are also players from around the rest of the globe at work in our country.
So now we have to figure out what the rules are in which people will figure out how to collaborate. We can assume that whoever wins in Nov, we will have federal funds and that also means federal rules . . . but . . . federal law does not eliminate or necessarily override state rules . . . and it doesn't do anything at all to privately funded efforts.
How the hell are we going to come up with a coordinated set of rules . . .it will all be a legislative and bureaucratic slog. It will take a very long time. And the Dickey amendment will still be in force under the congress manages to do its thing.
We may see a dismal parade of scientists traipsing from agency to agency, trying to figure out under what rules they have to operate . . .
So, let's say the scientists somehow navigate that arena and then approach the FDA for permission to carry out their trials. In the 60's and 70's we loved the slowness of the FDA because it kept American women from taking thalidomide as they did in Canada and the UK. During the 80's and 90's there was pressure to hurry up and get things done a lot faster. Then about 5 or 6 years ago there was a slew of drugs subjected to intense intention from the public and the policy community . . . and the slowdown became the right way to go again.
So here we are with translational research in stem cell therapy. The FDA is in Mode Caution, not Mode Aggression. They want to know how confident we are that we're making safe decisions. How pure must your lines be? How many undifferentiated cells are in your transplants? Do we need medical records from the original donors of embryos from 9 years ago? Do we need to maintain immuno-suppressed animals for months and months and months to be sure that tumors are not forming? Will we be forced repeatedly to start over because our animals die of infections before our data is collected?
Okay, let's say that all that gets dealt with. Woo hoo, clinical trials. Uh oh. We now have a HUGE demand, lots of it from people who believe that their condition is going to kill them before they can get the drug. Think about thousands of people demanding to take part in clinical trials . . .ACT UP is the model.
There will somehow have to be access to treatments that have not been tested yet. They did that with the Aids patients -- but this is a biologic and not a drug. And biologics are much more complex and detaile--not to mention expensive- to manufacture than drugs . . . but we're going to have to figure out how to do it. The reason is that Americans are always ready to believe that the newest thing is the best.
But, let's say that all that gets dealt with. Woo hoo, new therapies. Uh oh. We now have the greatest new thing since sunlight in every headline, and that will mean a ton of people ready to buy it who HAVE NO healthcare. Big new struggle.
For 35 years the conversation about stem cell research has focused on the moral status of the embryo . . . let's get over it already.
For my money, the ethical issues are just beginning.
Great talk.
John McNeish, Pfizer
John is the executive director of regenerative medicine for Pfizer.
I really believe stem cell technology is at the tipping point -- and whenever you approach this kind of event it's very important to bring all the parties together.
We've been using stem cells as tools for drug discovery for 15 years now -- and my little sound bite is: better cells mean better drugs.
We've tried to take the lead in recognizing regenerative therapies, as well as cancer. We think that not so far in the distant future cells will actually be therapies, and that stem cells can be used to deliver therapies, to get drugs to where we need them to go.
Then there are combination therapies, in which your cell does both jobs -- delivers a molecule and acts as a healant itself.
iPS is all about making time go backwards . . . we have a lot of work to do with iPS cells and their derivatives.
Somewhere between 10 and 15% of all drugs brought into safety trials fail because of arrhythmia . . . so if we can make cardiomyocytes (heart cells) we could use them to prevent all those failures
They've done testing to show that cells in vitro behave exactly as they do in vivo under the influence of hundreds of drugs. This means that what happens to a living cell in the lab is exactly the same, functionally and chemically, as what happens to it inside a living body.
Small molecules modify stem cell fate . . .this gives us hope that you can identify drug-like molecules that will get stem cells to do what you want them to do in vivo.
He's talking about directed differentiation and tons and tons of testing in the lab followed by more of the same in animal models . . .
Reuters quote: "Pfizer quietly launches stem cell unit" He laughs at this. We are not hiding this, whatever they think. Couldn't they just have said "Pfizer launches stem cell unit?"
His speech rate is double what would be considered normal; probably he's going faster than usual because they got started a bit late and are trying to stay on schedule so people can make their planes.
I really believe stem cell technology is at the tipping point -- and whenever you approach this kind of event it's very important to bring all the parties together.
We've been using stem cells as tools for drug discovery for 15 years now -- and my little sound bite is: better cells mean better drugs.
We've tried to take the lead in recognizing regenerative therapies, as well as cancer. We think that not so far in the distant future cells will actually be therapies, and that stem cells can be used to deliver therapies, to get drugs to where we need them to go.
Then there are combination therapies, in which your cell does both jobs -- delivers a molecule and acts as a healant itself.
iPS is all about making time go backwards . . . we have a lot of work to do with iPS cells and their derivatives.
Somewhere between 10 and 15% of all drugs brought into safety trials fail because of arrhythmia . . . so if we can make cardiomyocytes (heart cells) we could use them to prevent all those failures
They've done testing to show that cells in vitro behave exactly as they do in vivo under the influence of hundreds of drugs. This means that what happens to a living cell in the lab is exactly the same, functionally and chemically, as what happens to it inside a living body.
Small molecules modify stem cell fate . . .this gives us hope that you can identify drug-like molecules that will get stem cells to do what you want them to do in vivo.
He's talking about directed differentiation and tons and tons of testing in the lab followed by more of the same in animal models . . .
Reuters quote: "Pfizer quietly launches stem cell unit" He laughs at this. We are not hiding this, whatever they think. Couldn't they just have said "Pfizer launches stem cell unit?"
His speech rate is double what would be considered normal; probably he's going faster than usual because they got started a bit late and are trying to stay on schedule so people can make their planes.
Don Reed
The best part of being an advocate is the incredible people you get to hang out with!
Governor Arnold Schwarzenegger's FAX number
916 558 3160
The BEST advocacy tool is a one-sentence political letter. Here's one you could send to the governor.
Please veto SB 1565 because it threatens California's Stem Cell program.
This bill is on the CA floor right now. If the gov. signs it and it becomes law, the gains of Prop 71 will be lost. The people on the ICOC are the best -- all of them there because they are fearless and dedicated. The guy you just heard is one. We have champions, leaders, and people who have given their hearts and souls to this cause.
The bill would add bureaucrats to the ICOC to overtake the decision-making process, and they're pretending the reason for this change would be to make sure the uninsured would have access to future cures.
When my son was paralyzed and I wanted to get a law passed, I imitated a NY police officer who was shot. His name was Paul Richter, and Governor Pataki told him that if he could get every congressmember in NY to sign on, he would get $15 added to every traffic ticket. (Pataki later reneged on this promise and put the money into the general fund.)
We did a letter campaign that consisted of one and two-sentence letters and fought our heads off to get $1.5 million/year. One of our scientists was funded and invented a petrie dish, the first new design in a century. We funded Hans Keirstead's research that goes on to this day. We had to add this sentence into CA law:
Research involving human embryonic stem cells from any source . . . shall be legal.
Bob Klein put $3.1million--his life savings--into the campaign for Prop 71. We learned not to ever, ever debate. Use the same words . . . over and over, never waste your time arguing. Example:
Do you support stem cell research? (If yes, sign here. If no, move on.)
George Lakoff gave us this sentence. American families deserve access to the best medical care science can provide.
Catholics support stem cell research by a 72% majority embryonic stem cell research.
When you see an article in the paper that you like, call that person and thank them. Offer to be a resource, and you've made a friend.
Don speaks in a crotchety-old-man voice: If God had decreed that a certain person should die of chicken pox it would be a sin to subvert that by a vaccination. Rev. Timothy Wright, Yale, 250 years ago.
Don lives on words form Chris Reeve: One day Roman and I will stand up from our chairs and walk away from them forever.
Mark Noble gets up to ask: What do you want us to do? (Mark is collaborating with Stephen Davies on his terrific work with astrocytes)
Don says do your work; I don't presume to know what you should do. Danny says, lock arms with us. Come with us to where we have to go and be ready to explain this research in layman's terms.
Jackie from the national association of biology teachers: Students turn into advocates when they learn what's at stake . . . what can we teachers do to help eliminate the controversy so they stop debating and lose their fear of it? Danny says, right now we're going to the high schools to help them learn about this research. I think that there needs to be more of an effort . . . we're a little late in the game, to be honest, but we'll be in touch with you as we gear up. Don says go to CAMR Advocacy and Americans for Cures, both of which have clear and lucid explanations -- like this one:
embryonic stem cells are like cash, they can be spent anywhere and buy anything. Adult stem cells are like gift certificates--you can only use them in certain stores.
Question: (Actually a suggestion for the biology teachers) follow the stories and make sure everything you read can be found in multiple publications and is well-sourced.
Governor Arnold Schwarzenegger's FAX number
916 558 3160
The BEST advocacy tool is a one-sentence political letter. Here's one you could send to the governor.
Please veto SB 1565 because it threatens California's Stem Cell program.
This bill is on the CA floor right now. If the gov. signs it and it becomes law, the gains of Prop 71 will be lost. The people on the ICOC are the best -- all of them there because they are fearless and dedicated. The guy you just heard is one. We have champions, leaders, and people who have given their hearts and souls to this cause.
The bill would add bureaucrats to the ICOC to overtake the decision-making process, and they're pretending the reason for this change would be to make sure the uninsured would have access to future cures.
When my son was paralyzed and I wanted to get a law passed, I imitated a NY police officer who was shot. His name was Paul Richter, and Governor Pataki told him that if he could get every congressmember in NY to sign on, he would get $15 added to every traffic ticket. (Pataki later reneged on this promise and put the money into the general fund.)
We did a letter campaign that consisted of one and two-sentence letters and fought our heads off to get $1.5 million/year. One of our scientists was funded and invented a petrie dish, the first new design in a century. We funded Hans Keirstead's research that goes on to this day. We had to add this sentence into CA law:
Research involving human embryonic stem cells from any source . . . shall be legal.
Bob Klein put $3.1million--his life savings--into the campaign for Prop 71. We learned not to ever, ever debate. Use the same words . . . over and over, never waste your time arguing. Example:
Do you support stem cell research? (If yes, sign here. If no, move on.)
George Lakoff gave us this sentence. American families deserve access to the best medical care science can provide.
Catholics support stem cell research by a 72% majority embryonic stem cell research.
When you see an article in the paper that you like, call that person and thank them. Offer to be a resource, and you've made a friend.
Don speaks in a crotchety-old-man voice: If God had decreed that a certain person should die of chicken pox it would be a sin to subvert that by a vaccination. Rev. Timothy Wright, Yale, 250 years ago.
Don lives on words form Chris Reeve: One day Roman and I will stand up from our chairs and walk away from them forever.
Mark Noble gets up to ask: What do you want us to do? (Mark is collaborating with Stephen Davies on his terrific work with astrocytes)
Don says do your work; I don't presume to know what you should do. Danny says, lock arms with us. Come with us to where we have to go and be ready to explain this research in layman's terms.
Jackie from the national association of biology teachers: Students turn into advocates when they learn what's at stake . . . what can we teachers do to help eliminate the controversy so they stop debating and lose their fear of it? Danny says, right now we're going to the high schools to help them learn about this research. I think that there needs to be more of an effort . . . we're a little late in the game, to be honest, but we'll be in touch with you as we gear up. Don says go to CAMR Advocacy and Americans for Cures, both of which have clear and lucid explanations -- like this one:
embryonic stem cells are like cash, they can be spent anywhere and buy anything. Adult stem cells are like gift certificates--you can only use them in certain stores.
Question: (Actually a suggestion for the biology teachers) follow the stories and make sure everything you read can be found in multiple publications and is well-sourced.
Jeff Sheehy, AIDS Research Institute
I need to remark on something . . . HIV/AIDS has not really focussed on stem cells. i want to say that what Bernie Siegel and Bob Klein are doing here is getting us out of our individual silos of specific diseases and into the playing field.
I've become friends with all these people who represent a whole collection of conditions and diseases . . . I've always been focused on AIDS and AIDS and AIDS. This is the best.
For Danny . . . we in CA won, but that's not the end. We do feel that we own the stem cell space. 12 of our 29 board members have relationships with patient advocacy groups. Patient advocates are co-chairs of the standing committees. We're funding stem cell research, but are we really moving the science forward in a significant way? Are we getting results.
At a recent meeting, a member who had been following Geron's application for FDA permission gave an eloquent presentation about where the data was missing.
For me it was one of those moments where the scientists seemed to be talking down to us as advocates . . . what we all need to understand is that working as partners with advocates is the most effective way for research to go forward.
We ought to be stopping everything right now in CA and going after those FDA issues. We need to break some NiH patterns here.
Here's a question . . . who is going to get to be in a trial, and who is going to decide?
There's going to be a question of whether placebo trials are needed.
When I think of clinical trials, I think about my friend Jeff Getty, who got a baboon bone marrow transplant that he didn't expect to survive.
I know that if I don't risk my life its over
He felt that participating in risky science is necessary; what he learned through direct experience was that suppressing the immune system drained tthe sea in which hiv swims . . . and his act made it normal for aids patients to get organ transplants. a lot of people are alive because jeff decided to risk his life.
How is it okay for someone else to make a decision for patients?
science is not always linear; the results of Jeff's again were not what anybody expected
i've heard endlessly about the death of Jesse Gelsinger from a gene therapy trial that went wrong
Trials go wrong all the time, but the response is not then to fold up and go home--it's heartbreak.
We can't not do trials because we might fail. we need trials sooner rather than later, and we must be ready for inevitable failures.
I go back to 1996 after Jeff got his transplant . . . within that year, cocktail drugs were introduced and dying aids patients were literally rising from their beds.
I've become friends with all these people who represent a whole collection of conditions and diseases . . . I've always been focused on AIDS and AIDS and AIDS. This is the best.
For Danny . . . we in CA won, but that's not the end. We do feel that we own the stem cell space. 12 of our 29 board members have relationships with patient advocacy groups. Patient advocates are co-chairs of the standing committees. We're funding stem cell research, but are we really moving the science forward in a significant way? Are we getting results.
At a recent meeting, a member who had been following Geron's application for FDA permission gave an eloquent presentation about where the data was missing.
For me it was one of those moments where the scientists seemed to be talking down to us as advocates . . . what we all need to understand is that working as partners with advocates is the most effective way for research to go forward.
We ought to be stopping everything right now in CA and going after those FDA issues. We need to break some NiH patterns here.
Here's a question . . . who is going to get to be in a trial, and who is going to decide?
There's going to be a question of whether placebo trials are needed.
When I think of clinical trials, I think about my friend Jeff Getty, who got a baboon bone marrow transplant that he didn't expect to survive.
I know that if I don't risk my life its over
He felt that participating in risky science is necessary; what he learned through direct experience was that suppressing the immune system drained tthe sea in which hiv swims . . . and his act made it normal for aids patients to get organ transplants. a lot of people are alive because jeff decided to risk his life.
How is it okay for someone else to make a decision for patients?
science is not always linear; the results of Jeff's again were not what anybody expected
i've heard endlessly about the death of Jesse Gelsinger from a gene therapy trial that went wrong
Trials go wrong all the time, but the response is not then to fold up and go home--it's heartbreak.
We can't not do trials because we might fail. we need trials sooner rather than later, and we must be ready for inevitable failures.
I go back to 1996 after Jeff got his transplant . . . within that year, cocktail drugs were introduced and dying aids patients were literally rising from their beds.
Danny Neumann
I'm gonna do something sacreligious here in the state of Wisconsin (puts on a University of Michigan hat) --
I left that UN conference so empowered . . . I went home to Ann Arbor and saw new buildings going up on the campus that I knew were meant to house labs.
Then I went on the internet.
And my heart went into my stomach. Along with North Dakota and a few other dark states, Michigan was outlawing stem cell research. So I was sitting at a Pistons game next to my friend Andy Meisner and I said we needed to do something.
We met with people at MSU and Wayne State, and we created a group called Michigan Citizens for Research and Cures. Everybody who had a stake in the outcome was part of this group.
We needed to go out and educate the citizens what this research was about--because our legislature was beyond reaching. The Republican senate would not even have a floor debate. So, we created a ballot initiative. We wanted Michigan to be a leader; we have some of the best researchers in the world. We went out to get signatures--needed 380,000 and got more than 500,000.
However.
Our state is dominated by the Right to Life. Our economy is in terrible shape.
It's not going to be enough to win on November 4th, but that is not going to be enough. We're going to be out on the streets educating the people about what the deal is.
In WA DC, here's what we need to do. Be like our opponents and put egos aside. They speak with one voice, they mobilize their troops, stay on message, leave the infighting somewhere out of sight. We need to do that.
The Republican platform now has a complete ban on human embryonic stem cell research. That's their goal.
I'm here for my 5-year old daughter, who I hope will never have to suffer from things that could have been cured, if only we worked a little harder.
I left that UN conference so empowered . . . I went home to Ann Arbor and saw new buildings going up on the campus that I knew were meant to house labs.
Then I went on the internet.
And my heart went into my stomach. Along with North Dakota and a few other dark states, Michigan was outlawing stem cell research. So I was sitting at a Pistons game next to my friend Andy Meisner and I said we needed to do something.
We met with people at MSU and Wayne State, and we created a group called Michigan Citizens for Research and Cures. Everybody who had a stake in the outcome was part of this group.
We needed to go out and educate the citizens what this research was about--because our legislature was beyond reaching. The Republican senate would not even have a floor debate. So, we created a ballot initiative. We wanted Michigan to be a leader; we have some of the best researchers in the world. We went out to get signatures--needed 380,000 and got more than 500,000.
However.
Our state is dominated by the Right to Life. Our economy is in terrible shape.
It's not going to be enough to win on November 4th, but that is not going to be enough. We're going to be out on the streets educating the people about what the deal is.
In WA DC, here's what we need to do. Be like our opponents and put egos aside. They speak with one voice, they mobilize their troops, stay on message, leave the infighting somewhere out of sight. We need to do that.
The Republican platform now has a complete ban on human embryonic stem cell research. That's their goal.
I'm here for my 5-year old daughter, who I hope will never have to suffer from things that could have been cured, if only we worked a little harder.
Dan Perry, Alliance for Aging Research
We're going to focus now on stem cell advocacy 2.0. You guys here are on the front lines; we need a pooled collective knowledge . . . you're going to hear from this outstanding panel, which includes Danny Neumann, Jeff Sheehy, and Don Reed.
(It's getting toward the end of the conference . . . lots of people seem to have left because these sessions are getting more and more thinly attended)
I'm head of a group that advocates for biomedical research into aging.
Two years, 3 months and 8 days from today, the first member of the baby boom generation is going to turn 65. The boomers are going to morph into the seniors.
In Jan 2011 we go from 6,000 people every day turning 65 to 10,000 people every day turning 65. It's going to be a "silver tsunami" of people getting type 2 diabetes, cancer, heart disease, parkinson's, dementia . . . the size of the bill for health care of this generation is going to be hard to fathom
The congress is now considering $700B to bail out wall street. But we spent $707 in one year on the 5 top diseases of aging -- and that's with no boomers at all in the mix.
The election . . . even with a president that wholeheartedly supports regenerative medicine, we're going to have a huge fight on our hands and you're going to need every tool you can get your hands on. Here's Danny Neumann, whom I've had the pleasure of hearing at the United Nations and all over the place.
(It's getting toward the end of the conference . . . lots of people seem to have left because these sessions are getting more and more thinly attended)
I'm head of a group that advocates for biomedical research into aging.
Two years, 3 months and 8 days from today, the first member of the baby boom generation is going to turn 65. The boomers are going to morph into the seniors.
In Jan 2011 we go from 6,000 people every day turning 65 to 10,000 people every day turning 65. It's going to be a "silver tsunami" of people getting type 2 diabetes, cancer, heart disease, parkinson's, dementia . . . the size of the bill for health care of this generation is going to be hard to fathom
The congress is now considering $700B to bail out wall street. But we spent $707 in one year on the 5 top diseases of aging -- and that's with no boomers at all in the mix.
The election . . . even with a president that wholeheartedly supports regenerative medicine, we're going to have a huge fight on our hands and you're going to need every tool you can get your hands on. Here's Danny Neumann, whom I've had the pleasure of hearing at the United Nations and all over the place.
Questions for Journalists
Mark Noble: one of the things that I've come to insist on is that if a journalist comes to talk with me or visits my lab, I have to see what they write before they print.
Our press releases get misquoted.
It's infuriating to spend huge amounts trying to communicate and failing.
The idiots at UPI recently reported that we repaired a damaged human spinal cord--instead of a damaged rat spinal cord.
From my point of view as a scientist who interacts frequently with journalists, the issue is accuracy, and we need for you to be willing to interact with us in such a way as to guarantee that accuracy.
When we ask for permission to review your work pre-publication . . .say yes.
Journalist: first the question is, have you ever made a mistake in your work?
Answer: I've never had to retract a data point.
J: Well that's good, but I'm going to respectfully ask that you drop the idiot label.
Think about whether it would be a good idea to have us be required to call our sources and ask if they liked what we wrote . . . I have actually had scientists try to exert editorial control over stories I've written when all I wanted was to ask for a fact check. We've been burned by scientists, too.
Journalist 2: That's often a matter of policy and not the writer's decision. I work on a magazine where we do fact-check. There's a system in place where a 2nd reporter calls every source back to make sure we got the story right. And we would never allow any of them to read the entire piece. They might get a little paragraph, but never the whole thing. There is not going to be perfect accuracy and we shouldn't expect it. We're better off with some quantity of mistakes than we would be if we tried to print.
Journalist 3: I'm much more likely to work with someone who has given me a story if they say, here's my cell phone, here's my contact info, I know you're on deadline than if they demand it.
Journalist 4: I'm different from these guys in that I depend on scientists to review what I've written . . . but I have the luxury of writing long and complicated pieces that make this process possible. You should know that we take accuracy as often as you do, because the worst feeling in the world is printing something wrong, something you didn't really understand, something you wanted so much to get right.
Same Questioner: Then let me suggest to you all that you start off by saying to the scientist that you're going to ask for a fact check.
Journalist: Well then hold to your end of the bargain . . . don't look at a quote and say, wow, I don't like the sound of that . . . let's change it. You can say, no, that's not what I said if it wasn't what you said.
Moderator steps in to say it would be cool if we had some studies to show what readers take away from stories . . . which is not details usually, but maybe if you're lucky 2 main ideas. Focus on the gist.
New questioner: Recently I had an experience with being the single source of a story that had been perpetuated around the world . . . I was never asked for more details as more and more papers just printed what Reuters had said.
Journalist: This has to do with the economics of publishing. Lots of places pay Reuters for just that privilege -- to copy and paste what they put up. (Mentions that his experience as a writer is to find his words copied and pasted all over the web without attribution . . . ) Also wonders if we really do need to have 6 different people processing what is not that complicated a story.
Journalist 2: It sounds like you were wondering why other reporters didn't pick up on the original story . . .nobody likes to write the story someone else has written. We want to write our own story . . . (scientist says that he kind of thought he would get a chance to explain or answer questions) Nah.
Journalist 3: A trend I've seen is more and more newspapers talking about axing AP or Reuters or other wire services . . . which may not be a good thing. The history of journalism used to be that the competition was all about being there first . . . but now there is also a "we're not first, but we can tell this story better" -- not all stories are going to be considered dead meat.
New Questioner: How do you handle it when people insist that there are two sides when there is really only one? Must we have an equal balance?
Journalist: the tradition in journalism is to tell both sides . . . I think we've finally figured out that we really don't have to find the one guy who will give us a quote that contradicts the main thrust of our story.
Journalist 2: agree -- the insistence on balance is fading.
Question: Is it true that editors title stories? I had a story in which the editor mishandled the content outrageously.
Journalist: Yep. And it does cause problems.
Our press releases get misquoted.
It's infuriating to spend huge amounts trying to communicate and failing.
The idiots at UPI recently reported that we repaired a damaged human spinal cord--instead of a damaged rat spinal cord.
From my point of view as a scientist who interacts frequently with journalists, the issue is accuracy, and we need for you to be willing to interact with us in such a way as to guarantee that accuracy.
When we ask for permission to review your work pre-publication . . .say yes.
Journalist: first the question is, have you ever made a mistake in your work?
Answer: I've never had to retract a data point.
J: Well that's good, but I'm going to respectfully ask that you drop the idiot label.
Think about whether it would be a good idea to have us be required to call our sources and ask if they liked what we wrote . . . I have actually had scientists try to exert editorial control over stories I've written when all I wanted was to ask for a fact check. We've been burned by scientists, too.
Journalist 2: That's often a matter of policy and not the writer's decision. I work on a magazine where we do fact-check. There's a system in place where a 2nd reporter calls every source back to make sure we got the story right. And we would never allow any of them to read the entire piece. They might get a little paragraph, but never the whole thing. There is not going to be perfect accuracy and we shouldn't expect it. We're better off with some quantity of mistakes than we would be if we tried to print.
Journalist 3: I'm much more likely to work with someone who has given me a story if they say, here's my cell phone, here's my contact info, I know you're on deadline than if they demand it.
Journalist 4: I'm different from these guys in that I depend on scientists to review what I've written . . . but I have the luxury of writing long and complicated pieces that make this process possible. You should know that we take accuracy as often as you do, because the worst feeling in the world is printing something wrong, something you didn't really understand, something you wanted so much to get right.
Same Questioner: Then let me suggest to you all that you start off by saying to the scientist that you're going to ask for a fact check.
Journalist: Well then hold to your end of the bargain . . . don't look at a quote and say, wow, I don't like the sound of that . . . let's change it. You can say, no, that's not what I said if it wasn't what you said.
Moderator steps in to say it would be cool if we had some studies to show what readers take away from stories . . . which is not details usually, but maybe if you're lucky 2 main ideas. Focus on the gist.
New questioner: Recently I had an experience with being the single source of a story that had been perpetuated around the world . . . I was never asked for more details as more and more papers just printed what Reuters had said.
Journalist: This has to do with the economics of publishing. Lots of places pay Reuters for just that privilege -- to copy and paste what they put up. (Mentions that his experience as a writer is to find his words copied and pasted all over the web without attribution . . . ) Also wonders if we really do need to have 6 different people processing what is not that complicated a story.
Journalist 2: It sounds like you were wondering why other reporters didn't pick up on the original story . . .nobody likes to write the story someone else has written. We want to write our own story . . . (scientist says that he kind of thought he would get a chance to explain or answer questions) Nah.
Journalist 3: A trend I've seen is more and more newspapers talking about axing AP or Reuters or other wire services . . . which may not be a good thing. The history of journalism used to be that the competition was all about being there first . . . but now there is also a "we're not first, but we can tell this story better" -- not all stories are going to be considered dead meat.
New Questioner: How do you handle it when people insist that there are two sides when there is really only one? Must we have an equal balance?
Journalist: the tradition in journalism is to tell both sides . . . I think we've finally figured out that we really don't have to find the one guy who will give us a quote that contradicts the main thrust of our story.
Journalist 2: agree -- the insistence on balance is fading.
Question: Is it true that editors title stories? I had a story in which the editor mishandled the content outrageously.
Journalist: Yep. And it does cause problems.
Mark Johnson MIlwaukee Journal Sentinel
He's a fill-in for someone who couldn't make it, says that he has an unusual job for a reporter, which is to write about ideas.
(How sad that this is unusual ~~)
He's the guy who has to write about things like stem cells that are complex and take actual thought to grok . . . he would not have predicted how much of a public appetite there is for really geeky stories.
He ended up covering stem cells this year because there was one story he was especially interested in. His editors didn't take a lot of convincing to take it on; he's pretty sure that there's a false conception among researchers and scientists that people won't be interested in what they're doing.
The interest in this goes beyond the sexy political debate, though. His experience is that people really do want to understand this, and in the last year he's become convinced that there's nothing he can't learn if the people will just sit down and talk with him. This year he's immersed himself in stem cells ~~ every night when he used to be reading novels he's now reading scientific papers on stem cells.
His message to the scientists here is that the interest in this story is about the science itself . . .we don't have to focus on the fight. The science is fascinating, and we shouldn't be afraid to get that story out in case it makes things more difficult.
He has faith in the intelligence of his readers, and believes that keeping the readers well-informed is the reason journalism exists.
(How sad that this is unusual ~~)
He's the guy who has to write about things like stem cells that are complex and take actual thought to grok . . . he would not have predicted how much of a public appetite there is for really geeky stories.
He ended up covering stem cells this year because there was one story he was especially interested in. His editors didn't take a lot of convincing to take it on; he's pretty sure that there's a false conception among researchers and scientists that people won't be interested in what they're doing.
The interest in this goes beyond the sexy political debate, though. His experience is that people really do want to understand this, and in the last year he's become convinced that there's nothing he can't learn if the people will just sit down and talk with him. This year he's immersed himself in stem cells ~~ every night when he used to be reading novels he's now reading scientific papers on stem cells.
His message to the scientists here is that the interest in this story is about the science itself . . .we don't have to focus on the fight. The science is fascinating, and we shouldn't be afraid to get that story out in case it makes things more difficult.
He has faith in the intelligence of his readers, and believes that keeping the readers well-informed is the reason journalism exists.
David Wahlberg, Wisconsin State Journal
I was thinking of the good old days when it was just embryonic and adult stem cells. That was a pretty simple story to write because you had people who held positions on one or the other . . . now we have scnt and blastomeres, we have cord blood cells, iPS cells, direct re-programming --
It's confusing for a reporter and very confusing for the people we're writing for. These things come up like flashes in the pan and it's very difficult to know what to focus on and what to leave alone.
It's also the case the papers have fewer and fewer specialized reporters . . . we're trying to find ways to bring people to our website with video and slideshows and becoming more of a social networking place . . . my bottom line is that it's already really hard and going to get even harder to communicate well.
It's confusing for a reporter and very confusing for the people we're writing for. These things come up like flashes in the pan and it's very difficult to know what to focus on and what to leave alone.
It's also the case the papers have fewer and fewer specialized reporters . . . we're trying to find ways to bring people to our website with video and slideshows and becoming more of a social networking place . . . my bottom line is that it's already really hard and going to get even harder to communicate well.
Rick Weiss, Center for American Progress
He wrote for the WA Post for 15 years, and a great many of those stories were about stem cells.
I would make the point that stem cells have been the seminal iconic story of science journalism of our time . . .both good and bad. What's that story about? Science and progress, the possibility of cures, a chance to let readers know how research is done, how genes and cells work
At the same time it's an epic moral story about the beginnings of life . . if we can even talk about that--because as a great scientist once told me, in truth life actually began only once, and that was billions of years ago ( I LOVE that)
And of course it's a great political story, a philosophical and religious story.
Then there are the hype stories and the people trying to sell us a bill of goods. A researcher once told me that he knew stem cells could not cure alzheimers but he would say the opposite because people needed a fairy tale.
We have senators standing up talking about embryos being dismembered, when we all know that 5-day-old embryos don't have any members to be dissed . . .
So, how is this going to be play out now? Science journalism is shrinking with the newspapers.
I would make the point that stem cells have been the seminal iconic story of science journalism of our time . . .both good and bad. What's that story about? Science and progress, the possibility of cures, a chance to let readers know how research is done, how genes and cells work
At the same time it's an epic moral story about the beginnings of life . . if we can even talk about that--because as a great scientist once told me, in truth life actually began only once, and that was billions of years ago ( I LOVE that)
And of course it's a great political story, a philosophical and religious story.
Then there are the hype stories and the people trying to sell us a bill of goods. A researcher once told me that he knew stem cells could not cure alzheimers but he would say the opposite because people needed a fairy tale.
We have senators standing up talking about embryos being dismembered, when we all know that 5-day-old embryos don't have any members to be dissed . . .
So, how is this going to be play out now? Science journalism is shrinking with the newspapers.
Matt Herper, Forbes Magazine
Shows a couple of very strange images . . . a cyclops sheep (with one eye) and some glowing mice. They're from stories he wrote about stem cell research . . . which used to be what people would ask for.
2 years ago when we would send a story to our editors about stem cells, it would come back and the comment would be "where's the politics?"
Now we're writing about the science and the business . . . the stories are more about the same kinds of hurdles we face with other technologies that haven't yet delivered. It's going to be harder to write about without the politics, and harder to get the stories out.
2 years ago when we would send a story to our editors about stem cells, it would come back and the comment would be "where's the politics?"
Now we're writing about the science and the business . . . the stories are more about the same kinds of hurdles we face with other technologies that haven't yet delivered. It's going to be harder to write about without the politics, and harder to get the stories out.
Angry Dad's Challenge
Man gets up to say that he took his 5-year-old son who was profoundly disabled to Dr. Rader (?) and has been helped in a miraculous way.
People take jabs at these doctors who do these treatments -- he saved my son. Very articulate, very angry parent, trembling in this long, passionate testimony. I'm Catholic . . .the cells that helped my son came from aborted fetuses.
Medra.com, offered in the Dominican Republic. I have the eegs, mris, you can clearly see the changes year from year. he was on a trach, and a feeding tube.
Later someone tells him that the problem is that the doctor does not want to establish protocols and make it possible for what he's doing to become established in terms of techniques and methods. Another man says that he knows they haven't standardized things . . . which is exactly the problem. He doesn't care. This doctor healed his child and he doesn't like to hear him denounced as a snake oil salesman.
Very high emotion here. People trembling and shouting and trying to make themselves understood.
After the meeting ends, the crowd around the scientists and the last speaker whose son was helped is thick it takes us 30 minutes to give it up and leave for lunch.
People take jabs at these doctors who do these treatments -- he saved my son. Very articulate, very angry parent, trembling in this long, passionate testimony. I'm Catholic . . .the cells that helped my son came from aborted fetuses.
Medra.com, offered in the Dominican Republic. I have the eegs, mris, you can clearly see the changes year from year. he was on a trach, and a feeding tube.
Later someone tells him that the problem is that the doctor does not want to establish protocols and make it possible for what he's doing to become established in terms of techniques and methods. Another man says that he knows they haven't standardized things . . . which is exactly the problem. He doesn't care. This doctor healed his child and he doesn't like to hear him denounced as a snake oil salesman.
Very high emotion here. People trembling and shouting and trying to make themselves understood.
After the meeting ends, the crowd around the scientists and the last speaker whose son was helped is thick it takes us 30 minutes to give it up and leave for lunch.
Dr. Graham Creasey, Stanford University Medical Center
Talking about Amanda Barksdale (?) a paralyzed skier who came to him in 2000 for a bladder issue (if you're a ski instructor and you become wet at the top of the mountain, you'll be frozen before you get to the mountain)
Recently she came to me and said she was going to India to get a stem cell transplant. I strongly advised her not to go. How could she make an informed decision?
We all need so much more information about these offshore trials.
Amanda allowed me to share some of the data we collected about her condition.
She's been to India 3 times in the last year or so, and is going back again in 4 months. She gets a variety of stem cell injections and physical therapy. The results have been very difficult to quantify; one big problem is that we just don't have good outcome measures. Amanda herself is convinced that she is getting return -- but the measures we have don't show it in a significant or lasting sense. (He has slides up to show exactly what sensory changes they tried to measure; check the webcast)
Later someone asks what Amanda believes to have been the benefits she's gained . . . Dr. Creasey answers that the main thing she says is that it's added hope to her vocabulary.
So, if you're thinking about getting an experimental trial for a spinal cord injury, here are some questions to ask:
1. how safe is it? if they say 100%, don't go any further
2. what benefits can be expected?
3. what evidence is there for those benefits?
4. what is the study design? is there a control group? is there a chance that i'll be in it?
5. if i take part in this study, will i be excluded from other studies later?
6. what does it cost, and if this is research, should i be paying?
7. has this particular intervention been reviewed by other people?
all these are reasonable questions, and deserve reasonable answers.
There is now a partnership of associations developed to create some standard outcome measures, so that we will have some common language and strictly defined measuring sticks to know what outcomes actually are. One reason this matters is that it will help us build a network of people ready to participate in a network of clinical trials-- a huge win if it happens.
Recently she came to me and said she was going to India to get a stem cell transplant. I strongly advised her not to go. How could she make an informed decision?
We all need so much more information about these offshore trials.
Amanda allowed me to share some of the data we collected about her condition.
She's been to India 3 times in the last year or so, and is going back again in 4 months. She gets a variety of stem cell injections and physical therapy. The results have been very difficult to quantify; one big problem is that we just don't have good outcome measures. Amanda herself is convinced that she is getting return -- but the measures we have don't show it in a significant or lasting sense. (He has slides up to show exactly what sensory changes they tried to measure; check the webcast)
Later someone asks what Amanda believes to have been the benefits she's gained . . . Dr. Creasey answers that the main thing she says is that it's added hope to her vocabulary.
So, if you're thinking about getting an experimental trial for a spinal cord injury, here are some questions to ask:
1. how safe is it? if they say 100%, don't go any further
2. what benefits can be expected?
3. what evidence is there for those benefits?
4. what is the study design? is there a control group? is there a chance that i'll be in it?
5. if i take part in this study, will i be excluded from other studies later?
6. what does it cost, and if this is research, should i be paying?
7. has this particular intervention been reviewed by other people?
all these are reasonable questions, and deserve reasonable answers.
There is now a partnership of associations developed to create some standard outcome measures, so that we will have some common language and strictly defined measuring sticks to know what outcomes actually are. One reason this matters is that it will help us build a network of people ready to participate in a network of clinical trials-- a huge win if it happens.
Wise Young
What can we do to maximize the benefits and minimize the harm from medical tourism. One week ago I gave a 30 minute interview on Al Jazeera television watched by 30 million people. Arabs routinely leave their countries for medical treatment when they have serious health problems. This is legitimate medical tourism . . . within the USA we travel to other states for the best care.
Two weeks ago I was in India . . . the head of a hospital there told me that their primary market was medical tourists, mostly from Africa and the Middle East. This one hospital system gets over a million patients a year. One of the things we worry about is how this is stealing from the local care.
For the 10% of medical tourism that interests us here . . . we have this strange artificial situation that's developed because of our current political environment.
But there are also medical tourists who just can't get non-esc treatments here in the USA.
People should be careful not to assume that those who travel overseas are "desperate" . . . the typical case who comes to me is a young man, 10 or 15 years after a sci . . . he comes to me and says, "what is the best therapy?"
If I tell them not to go anywhere, they'll seek the answer from someone else. They've made up their minds that they want to try something . . . they're determined, they're impatient, they just don't want to wait anymore. It's not desperation.
We all know the problems. Overseas you don't know what you're getting, and if something goes wrong you have no recourse.
We should understand that no matter what we do or say, there will be people who make this choice--until we offer therapies.
When I announced that we would be doing clinical trials with umbilical cord blood trials with lithium in China, hundreds and hundreds of people from the USA came and asked me if they could come to China to be in them. It's outrageous
The regulatory situation in China is even worse than here-- seven layers of bureaucracy vs just the FDA. Based on this, I've decided to not wait for a change in administration . . . we're just going to go ahead and do it.
We got the CDRPA out of mothballs last spring, we attached it to an omnibus bill, we -- well, we spent 6 years of lobbying in Washington to get a measly clinical trials bill through our legislature. So it shouldn't surprise anybody that people look at that and say, wow . . . why don't I just spend a thousand bucks and go to China for this treatment.
What we don't want to do is leave this to ad hoc centers . . . trials need to be carefully designed and held here in the USA.
We have to monitor patients before and after they go. We have to do everything we can to educate our communities.
A website is a one-sided picture. I now spend 8 hours a day on this website. It's a place where people can discuss and present multiple sides of the story . . . which is absolutely critical for the evolution of this process . . . we as scientists and clinicians must be on these websites spending time on these websites, talking to the community and informing the discussion.
Medical tourism is here to stay. We have to understand how to maximize its benefit and minimize its harm.
Two weeks ago I was in India . . . the head of a hospital there told me that their primary market was medical tourists, mostly from Africa and the Middle East. This one hospital system gets over a million patients a year. One of the things we worry about is how this is stealing from the local care.
For the 10% of medical tourism that interests us here . . . we have this strange artificial situation that's developed because of our current political environment.
But there are also medical tourists who just can't get non-esc treatments here in the USA.
People should be careful not to assume that those who travel overseas are "desperate" . . . the typical case who comes to me is a young man, 10 or 15 years after a sci . . . he comes to me and says, "what is the best therapy?"
If I tell them not to go anywhere, they'll seek the answer from someone else. They've made up their minds that they want to try something . . . they're determined, they're impatient, they just don't want to wait anymore. It's not desperation.
We all know the problems. Overseas you don't know what you're getting, and if something goes wrong you have no recourse.
We should understand that no matter what we do or say, there will be people who make this choice--until we offer therapies.
When I announced that we would be doing clinical trials with umbilical cord blood trials with lithium in China, hundreds and hundreds of people from the USA came and asked me if they could come to China to be in them. It's outrageous
The regulatory situation in China is even worse than here-- seven layers of bureaucracy vs just the FDA. Based on this, I've decided to not wait for a change in administration . . . we're just going to go ahead and do it.
We got the CDRPA out of mothballs last spring, we attached it to an omnibus bill, we -- well, we spent 6 years of lobbying in Washington to get a measly clinical trials bill through our legislature. So it shouldn't surprise anybody that people look at that and say, wow . . . why don't I just spend a thousand bucks and go to China for this treatment.
What we don't want to do is leave this to ad hoc centers . . . trials need to be carefully designed and held here in the USA.
We have to monitor patients before and after they go. We have to do everything we can to educate our communities.
A website is a one-sided picture. I now spend 8 hours a day on this website. It's a place where people can discuss and present multiple sides of the story . . . which is absolutely critical for the evolution of this process . . . we as scientists and clinicians must be on these websites spending time on these websites, talking to the community and informing the discussion.
Medical tourism is here to stay. We have to understand how to maximize its benefit and minimize its harm.
Robin Smith of Neostem
As you all know, medical tourism is leaving your home to get medical care that's not offered where you live. In Britain and in Canada, people used to travel to the USA for care that they didn't want to wait for and could afford.
Right now people are leaving the USA to get health care elsewhere, either because it's cheaper (90% less in some cases) or for "the magic cure" -- 750,000 last year.
The magic cure can cost as much as 40 or 50,000 bucks . . . people who can't really afford it are going abroad to try. They're holding church fundraisers. It's projected that within 5 years, 10 million Americans will go abroad for treatments.
If you could go abroad for 2 weeks with your spouse and get lasik surgery, everything paid for including travel expenses . .. would you do it? How would you know if it was legit? What would you do if things went sideways? That's the problem.
The internet has made it so easy for information to get out . . .there's so much hope and so much promise, but we must help people make informed decisions. At the very least they have to know what questions to ask. What proof that the therapy works? What will happen post-op? What costs are not covered?
Right now people are leaving the USA to get health care elsewhere, either because it's cheaper (90% less in some cases) or for "the magic cure" -- 750,000 last year.
The magic cure can cost as much as 40 or 50,000 bucks . . . people who can't really afford it are going abroad to try. They're holding church fundraisers. It's projected that within 5 years, 10 million Americans will go abroad for treatments.
If you could go abroad for 2 weeks with your spouse and get lasik surgery, everything paid for including travel expenses . .. would you do it? How would you know if it was legit? What would you do if things went sideways? That's the problem.
The internet has made it so easy for information to get out . . .there's so much hope and so much promise, but we must help people make informed decisions. At the very least they have to know what questions to ask. What proof that the therapy works? What will happen post-op? What costs are not covered?
Doug Sipp of RIPKEN
I'm going to keep my comments very short today;
he's talking about something he saw; he witnessed a doctor telling a woman who asked if he could help her . . . the doctor told her he would take her back to Korea and make her walk again.
Five years later he heard a clinician tell a woman looking for a cure for her father's ALS that there was nothing he could do.
There are places in China, Brazil, Mexico, Thailand, all around the world where people are making claims on websites that we don't have any way of knowing are justified . . . they're not backed up by published evidence of rigorous trials.
he's talking about something he saw; he witnessed a doctor telling a woman who asked if he could help her . . . the doctor told her he would take her back to Korea and make her walk again.
Five years later he heard a clinician tell a woman looking for a cure for her father's ALS that there was nothing he could do.
There are places in China, Brazil, Mexico, Thailand, all around the world where people are making claims on websites that we don't have any way of knowing are justified . . . they're not backed up by published evidence of rigorous trials.
Tommy Thomson
Former Secretary of HHS, former governor of Wisconsin.
I didn't like your crack about Sarah Palin . . .
Here's news from somebody who was there at the beginning. I learned about stem cells in December of 1998, when Jamie Thomson made the world stand still for a moment
(lots of bombast here . . . speaking style is very politician, boisterous, with significant pauses and heavy thrusts on certain words . . . he's in a pretty politically unfriendly environment here but talking as if that's not the case)
No secretary has ever gone through what I've gone through. Nobody thought we'd get hit on 9/11
or SARS
or anthrax
or failure of flu vaccine
In August 2001, prior to 9/11, president Bush called me over and asked me to have lunch with him. We went into his office off the oval office, and he and karl rove and I sat down. I think he had a peanut butter sandwich. He asked us to debate stem cell research. Karl, I know you're against it, and Tommy, you're for it. Tell me why.
So we did.
I turned to the president and said that he could double the money for the NIH without allowing research on esc, you'll always be remembered as the guy who stopped it.
Why, asked Bush
Because every American has some family member suffering from something. And each one of them has some inner hope that escr will find a cure. (He's shouting like a preacher!)
And embryonic stem cell gives that person hope (almost in a whisper)
And that's why you have to find a way.
And I'm absolutely certain that if that lunch had not taken place, you would not have had the 78 lines . . . granted, you now only have 21 lines . . . and we have $650M ready to be used, $40M annually, but the point is there is no barrier.
(BARF)
The point is that regenerative medicine is here to stay. I just talked to the head of the NIH, who said that the most promising thing right now is that scientists are taking the diseased cells of sick people and studying the markers in their cells . . . and they're going to turn what they learn into cures.
My family has suffered from cancer, mostly breast cancer.
As a presidential candidate, I advocated for finding a cure for cancer by 2025. We have a chance in this room and on this campus to get that done.
Science is great. We can't put boundaries around science because if we don't do it here, they'll do it in CA, NY, London, and China. Let's do it everywhere.
The proudest moment of my life was about 20 months ago when my daughter, who had been able to salvage a single egg before her mastectomy. It was fertilized by her husband, and her sister carried that fetus in her womb because she couldn't. I got to hold that baby in my arms.
Whoever is elected president, it is our duty to convince them of the potential and the possibilities and the opportunities. That's why I got up at 4 am and drove here 4 hours to give you this message. Because it's not about red or blue, Republican or Democrat, it's about getting this done.
Sustained applause.
I didn't like your crack about Sarah Palin . . .
Here's news from somebody who was there at the beginning. I learned about stem cells in December of 1998, when Jamie Thomson made the world stand still for a moment
(lots of bombast here . . . speaking style is very politician, boisterous, with significant pauses and heavy thrusts on certain words . . . he's in a pretty politically unfriendly environment here but talking as if that's not the case)
No secretary has ever gone through what I've gone through. Nobody thought we'd get hit on 9/11
or SARS
or anthrax
or failure of flu vaccine
In August 2001, prior to 9/11, president Bush called me over and asked me to have lunch with him. We went into his office off the oval office, and he and karl rove and I sat down. I think he had a peanut butter sandwich. He asked us to debate stem cell research. Karl, I know you're against it, and Tommy, you're for it. Tell me why.
So we did.
I turned to the president and said that he could double the money for the NIH without allowing research on esc, you'll always be remembered as the guy who stopped it.
Why, asked Bush
Because every American has some family member suffering from something. And each one of them has some inner hope that escr will find a cure. (He's shouting like a preacher!)
And embryonic stem cell gives that person hope (almost in a whisper)
And that's why you have to find a way.
And I'm absolutely certain that if that lunch had not taken place, you would not have had the 78 lines . . . granted, you now only have 21 lines . . . and we have $650M ready to be used, $40M annually, but the point is there is no barrier.
(BARF)
The point is that regenerative medicine is here to stay. I just talked to the head of the NIH, who said that the most promising thing right now is that scientists are taking the diseased cells of sick people and studying the markers in their cells . . . and they're going to turn what they learn into cures.
My family has suffered from cancer, mostly breast cancer.
As a presidential candidate, I advocated for finding a cure for cancer by 2025. We have a chance in this room and on this campus to get that done.
Science is great. We can't put boundaries around science because if we don't do it here, they'll do it in CA, NY, London, and China. Let's do it everywhere.
The proudest moment of my life was about 20 months ago when my daughter, who had been able to salvage a single egg before her mastectomy. It was fertilized by her husband, and her sister carried that fetus in her womb because she couldn't. I got to hold that baby in my arms.
Whoever is elected president, it is our duty to convince them of the potential and the possibilities and the opportunities. That's why I got up at 4 am and drove here 4 hours to give you this message. Because it's not about red or blue, Republican or Democrat, it's about getting this done.
Sustained applause.
Greg Simon of Faster Cures
I can see the NIH from the roof of my house in Bethesda . . . (laughter)
When I look at the future of stem cell research, I ask the question WWSPD, and then do the opposite . . . (what would sarah palin do)
Talking about the efforts --all failed-- to redirect the Mississippi. We've been trying to redirect the river of stem cell research in this country for the last 8 years. In this analogy Bush and Coburn are the levies, and the research is the river.
We have a big problem, aside from all that. We live in a world that is not safe for collaboration . . . we must make some changes.
NIH--use money to reward collaboration
Researchers with deals with business
Diseases know no borders
We can't have every state building a moat around its knowledge
We can't afford to have every state or nation repeat what's already known
I talked with leaders of business and industry; I asked them what they had learned from the patients they're trying to help.
(dead silence)
That was their answer.
We need to hear you, advocates, patients, caregivers.
What a rabble-rouser! :)
When I look at the future of stem cell research, I ask the question WWSPD, and then do the opposite . . . (what would sarah palin do)
Talking about the efforts --all failed-- to redirect the Mississippi. We've been trying to redirect the river of stem cell research in this country for the last 8 years. In this analogy Bush and Coburn are the levies, and the research is the river.
We have a big problem, aside from all that. We live in a world that is not safe for collaboration . . . we must make some changes.
NIH--use money to reward collaboration
Researchers with deals with business
Diseases know no borders
We can't have every state building a moat around its knowledge
We can't afford to have every state or nation repeat what's already known
I talked with leaders of business and industry; I asked them what they had learned from the patients they're trying to help.
(dead silence)
That was their answer.
We need to hear you, advocates, patients, caregivers.
What a rabble-rouser! :)
Bill Linton of Promega
Information here.
Last night I mentioned a quote from Einstein about the importance of different persepectives . . here are a couple.
In 1961, Jack Kennedy asked for 1.7B for the moon program. It was a lot of money then. On July 21st, 1969, the first human being to walk on the moon was Neil Armstrong. Kennedy was determined to pass the Russians; he understood the importance of message and of setting a national goal.
many technologies and information came out of that effort--including the rapid development of computers.
2 years later we said, if we can put a man on the moon, let's cure cancer. The dream was to wipe out cancer in the same way we had wiped out polio. Nixon made a request for money in his 1971 state of the union address.
nearly 40 years later, how close are we? clearly not done, but since the mid-90's the death rate has been steadily decreasing. What used to be a quick death sentence has changed.
We've gained so much, though, from taking that journey.
International missions arise from capturing the imagination of an entire population. Our message must be compelling and inspiring. The "side benefits" must be just as important as the final goal.
This will be not just knowledge for the sake of knowledge, but change in the world.
Last night I mentioned a quote from Einstein about the importance of different persepectives . . here are a couple.
In 1961, Jack Kennedy asked for 1.7B for the moon program. It was a lot of money then. On July 21st, 1969, the first human being to walk on the moon was Neil Armstrong. Kennedy was determined to pass the Russians; he understood the importance of message and of setting a national goal.
many technologies and information came out of that effort--including the rapid development of computers.
2 years later we said, if we can put a man on the moon, let's cure cancer. The dream was to wipe out cancer in the same way we had wiped out polio. Nixon made a request for money in his 1971 state of the union address.
nearly 40 years later, how close are we? clearly not done, but since the mid-90's the death rate has been steadily decreasing. What used to be a quick death sentence has changed.
We've gained so much, though, from taking that journey.
International missions arise from capturing the imagination of an entire population. Our message must be compelling and inspiring. The "side benefits" must be just as important as the final goal.
This will be not just knowledge for the sake of knowledge, but change in the world.
Wise Young
In 1998, a young woman broke her neck in the Goodwill Games. She did her rehab in Mt Sinai . . . we had become good friends.
At her going away party, she was crying and when I asked her why she said that if we found the cure in the USA, she wouldn't be able to get it.
I promised her I would make sure that China would get its own program into place.
I went to China in 1998 and while I was there I met Suzanne Poon, whose son had been injured . . .
In 2004, there were 3 kinds of cells available
embryonic fetal cells
umbilical cord blood cells
bone marrow or peripheral blood stem cells
We didn't have the money to work with bone marrow . . . we decided to work on UCB cells, which has been shown repeatedly to be safe and beneficial in treating neurological conditions.
There are groups around the world working on transplanting UBC into spinal cord injured patients (mentions Mexico . . . says that people are promising cures when they're really just experimenting)
We isolated a cell line that we called NO1.1 . . . when we transplanted them they stayed where we wanted them.
Lithium has been shown to have regenerative effects on neurons. It acts on a single enzyme, which inhibits nuclear factors. The factors control growth and differentiation; lithium applied to stem cells stimulates growth, is the point. We tried it on every kind of stem cell we could think of.
We were concerned that lithium might cause differentiation in cells, but we found that it does not. We transplanted neonatal cells into spinal cords and added lithium and got robust axon growth.
Showing a slide with the cells in bright green migrating across the site of a dark blue injury.
Lithium also boosts growth factors . . .
We've asked the 25 members of the SCI network to let us do clinical trials in chronic spinal cord injury patients . . . these are very well-run hospitals. We propose 5 trials; the first one was an intradural decompression of the cord within a few weeks after injury-- the benefits of this have been very exciting.
They cut into the cord and remove the necrotic tissue . . this has allowed us to understand much better how to operate safely on the cord. Remarkably, the removal of necrotic tissue (macrophages) in itself resulted in functional recovery for many of the patients.
WE've already given a 6-week course of lithium to sci patients to test for tolerance. We've applied for permission to do a cord blood mononuclear cell transplant with 40 patients . . . aiming for a trial with 400 patients. Looking at cord blood cells and then cord blood cells plus lithium.
In order to prepare for the final trial . . . we needed to get the Chinese to start doing follow up. The model had been that sci people in China just left the hospital right after their rehab and never were heard from again. They've now got 100 patients who were treated, re-habbed, and are registered for the trials.
The great advantage of doing trials in China is the number of patients; we can easily get 6,000 patients to randomize. The standards are very high, and we've just spent 4 years teaching them how to conduct rigorous trials. We're preparing to do parallel trials in the USA.
Thousands of Americans have come to me and asked how they can go to China and be part of these clinical trials? This is shameful. This is shameful that Americans are begging to go take part in these trials, and we're committed to conducting them here.
Wow.
At her going away party, she was crying and when I asked her why she said that if we found the cure in the USA, she wouldn't be able to get it.
I promised her I would make sure that China would get its own program into place.
I went to China in 1998 and while I was there I met Suzanne Poon, whose son had been injured . . .
In 2004, there were 3 kinds of cells available
embryonic fetal cells
umbilical cord blood cells
bone marrow or peripheral blood stem cells
We didn't have the money to work with bone marrow . . . we decided to work on UCB cells, which has been shown repeatedly to be safe and beneficial in treating neurological conditions.
There are groups around the world working on transplanting UBC into spinal cord injured patients (mentions Mexico . . . says that people are promising cures when they're really just experimenting)
We isolated a cell line that we called NO1.1 . . . when we transplanted them they stayed where we wanted them.
Lithium has been shown to have regenerative effects on neurons. It acts on a single enzyme, which inhibits nuclear factors. The factors control growth and differentiation; lithium applied to stem cells stimulates growth, is the point. We tried it on every kind of stem cell we could think of.
We were concerned that lithium might cause differentiation in cells, but we found that it does not. We transplanted neonatal cells into spinal cords and added lithium and got robust axon growth.
Showing a slide with the cells in bright green migrating across the site of a dark blue injury.
Lithium also boosts growth factors . . .
We've asked the 25 members of the SCI network to let us do clinical trials in chronic spinal cord injury patients . . . these are very well-run hospitals. We propose 5 trials; the first one was an intradural decompression of the cord within a few weeks after injury-- the benefits of this have been very exciting.
They cut into the cord and remove the necrotic tissue . . this has allowed us to understand much better how to operate safely on the cord. Remarkably, the removal of necrotic tissue (macrophages) in itself resulted in functional recovery for many of the patients.
WE've already given a 6-week course of lithium to sci patients to test for tolerance. We've applied for permission to do a cord blood mononuclear cell transplant with 40 patients . . . aiming for a trial with 400 patients. Looking at cord blood cells and then cord blood cells plus lithium.
In order to prepare for the final trial . . . we needed to get the Chinese to start doing follow up. The model had been that sci people in China just left the hospital right after their rehab and never were heard from again. They've now got 100 patients who were treated, re-habbed, and are registered for the trials.
The great advantage of doing trials in China is the number of patients; we can easily get 6,000 patients to randomize. The standards are very high, and we've just spent 4 years teaching them how to conduct rigorous trials. We're preparing to do parallel trials in the USA.
Thousands of Americans have come to me and asked how they can go to China and be part of these clinical trials? This is shameful. This is shameful that Americans are begging to go take part in these trials, and we're committed to conducting them here.
Wow.
Hans Keirstead, UC Irvine
Thrilled to be here, my first visit to wisconsin
Hans has up his slide of a human egg sitting inside a fallopian tube with a speck of blue heading towards it . . . we don't have this much fun making stem cells, we do it by a different means
we've just started a scnt program after 2 years of working with the irb to get a source of eggs
the point is that we have a variety of sources for stem cells; research on all of them is very important
what ive been trying to do in my lab is generate high-purity human cell populations
we're going to need them
we could:
get cellular replacement strategies
do drug screening
do predictive toxicology -- which means use them to do tests in vitro to lower costs and not have to test in hman beings
we've got a couple of principles
we make fda compliant cells, we have 3 people making sure our cells are made with gmp
we aim for commercial viability because without the m we can't get to the clinic
we sa started with hesc-derived lo oligodendrocytes in high purity
my lab has done a great deal of work validating demylenation as a target for spinal cord injuries
we owe a debt to geron for pushing this through to the clinic . . . probably in 09 . . .
we then moved on to making high purity human motor neurons, certified as 99.6% pure . . . we're making 1-10 billion cells per week.
our animal models (unpublished data) show tremendous recovery
california stem cell is producing these plates that allow scientists to use these cells -- 5 days to use them --
this program is marching along toward the clinic.
both studies have a great deal of efficacy data generated with millions and millions of dollars on safety studies
the first study for the motor neurons will be a disease that attacks infants and kills them . . . characterized by highly specific motor neuron loss.
challenges?
repetition of experimental findings--takes a long time
safety studies--it took us 8 months and 1.5 million bucks to get done
large animal studies--must develop
manufacture and qualify the cells before you get to the FDA
medium in which the cells are made
clinical synopsis must be developed
outcome measures must be developed
financing the whole shebang.
one of the greatest challenges boils down to the isolation of academics
the change from basic research and discovery requires that we scientists engage with government,
Hans has up his slide of a human egg sitting inside a fallopian tube with a speck of blue heading towards it . . . we don't have this much fun making stem cells, we do it by a different means
we've just started a scnt program after 2 years of working with the irb to get a source of eggs
the point is that we have a variety of sources for stem cells; research on all of them is very important
what ive been trying to do in my lab is generate high-purity human cell populations
we're going to need them
we could:
get cellular replacement strategies
do drug screening
do predictive toxicology -- which means use them to do tests in vitro to lower costs and not have to test in hman beings
we've got a couple of principles
we make fda compliant cells, we have 3 people making sure our cells are made with gmp
we aim for commercial viability because without the m we can't get to the clinic
we sa started with hesc-derived lo oligodendrocytes in high purity
my lab has done a great deal of work validating demylenation as a target for spinal cord injuries
we owe a debt to geron for pushing this through to the clinic . . . probably in 09 . . .
we then moved on to making high purity human motor neurons, certified as 99.6% pure . . . we're making 1-10 billion cells per week.
our animal models (unpublished data) show tremendous recovery
california stem cell is producing these plates that allow scientists to use these cells -- 5 days to use them --
this program is marching along toward the clinic.
both studies have a great deal of efficacy data generated with millions and millions of dollars on safety studies
the first study for the motor neurons will be a disease that attacks infants and kills them . . . characterized by highly specific motor neuron loss.
challenges?
repetition of experimental findings--takes a long time
safety studies--it took us 8 months and 1.5 million bucks to get done
large animal studies--must develop
manufacture and qualify the cells before you get to the FDA
medium in which the cells are made
clinical synopsis must be developed
outcome measures must be developed
financing the whole shebang.
one of the greatest challenges boils down to the isolation of academics
the change from basic research and discovery requires that we scientists engage with government,
Ian Duncan, University of Wisconsin
Delighted to be here . . .
Want to show one slide that shows everything we're involved in.
Left side is 2 images of cells, right side is a woman standing with laufband crutches
The target for MS repair is myelin . . . sheaths that cover millions of axons in our central nervous systems. Without it, we get dysfunction.
Myelin is made by cells called oligodendrocytes.
We want to restore the myelin, not just to restore function but to protect axons from degeneration.
Oligodendrocytes are very complex; we know from years of research in labs around the world that the precursor stage of development holds the most promise for repair.
He's talking about the process in the lab that leads to very pure populations of the exact kind of early-stage oligodendrocyte that you need to make myelin . . . can't write down the details of it here . . .
Cool!! Just saw a time-lapse video of a cell dividing and migrating . .. the migrating action is very important because you need cells to find their way to axons and remyelinate them.
Showing slides of sections of a myelin-mutant rat who got a transplant . . . the little guy got a lot of myelin out of his transplant
Slide showing that it works in humans too . . . (he describes it as beautiful, which it is--the slide he's got up is art, stunning in composition and color) the cells we need are at the 5th iteration of development away from a stem cell.
So, do the cells survive? They checked the rats after 3 months, and the answer was yes.
To finish . . . we're going to transplant our cells right into the area where they need to go and abra cadabra, restoration of myelin and return of function.
Want to show one slide that shows everything we're involved in.
Left side is 2 images of cells, right side is a woman standing with laufband crutches
The target for MS repair is myelin . . . sheaths that cover millions of axons in our central nervous systems. Without it, we get dysfunction.
Myelin is made by cells called oligodendrocytes.
We want to restore the myelin, not just to restore function but to protect axons from degeneration.
Oligodendrocytes are very complex; we know from years of research in labs around the world that the precursor stage of development holds the most promise for repair.
He's talking about the process in the lab that leads to very pure populations of the exact kind of early-stage oligodendrocyte that you need to make myelin . . . can't write down the details of it here . . .
Cool!! Just saw a time-lapse video of a cell dividing and migrating . .. the migrating action is very important because you need cells to find their way to axons and remyelinate them.
Showing slides of sections of a myelin-mutant rat who got a transplant . . . the little guy got a lot of myelin out of his transplant
Slide showing that it works in humans too . . . (he describes it as beautiful, which it is--the slide he's got up is art, stunning in composition and color) the cells we need are at the 5th iteration of development away from a stem cell.
So, do the cells survive? They checked the rats after 3 months, and the answer was yes.
To finish . . . we're going to transplant our cells right into the area where they need to go and abra cadabra, restoration of myelin and return of function.
Executive Director of Fast Forward
What are the opportunities for stem cell research in MS?
MS is chronic disease that attacks the central nervous system
You lose myelin, which paralyzes you
Slide of an image showing active lesions in the brain of a person with MS
You get sensory problems, vision problems, vertigo, spasticity, fatigue, termor, balance problems, cognitive issues . . . most frustrating
There are 4 known types
relapsing, remitting 85 %
primary progressive 12%
secondary progressive 50%
and the worst, which i missed the name of but is the worst and affects 5% of people who have MS
So what causes it? Don't know. A genetic predisposition combined with an environmental trigger
Image of a nerve cell with its axon all bare
T cell cross the blood/brain barrier, which attack myelin and results in symptoms
current treatments
corticosteroids, disease modifying treatments, symptom management
one of the things we're beginning to see is that even with all these disease modifying treatments, there is still an increase in disability . . . a progression in a downward direction
so if we can control the disease, can we reverse it?
how do stem cells fit in?
stimulate repair by transplantation
use stem cells as platforms for neuroprotection
figure out what causes the loss of axons
use stem cells as a platform for discovery
these 4 areas are exciting opportunities for us in terms of funding and moving forward
MS is chronic disease that attacks the central nervous system
You lose myelin, which paralyzes you
Slide of an image showing active lesions in the brain of a person with MS
You get sensory problems, vision problems, vertigo, spasticity, fatigue, termor, balance problems, cognitive issues . . . most frustrating
There are 4 known types
relapsing, remitting 85 %
primary progressive 12%
secondary progressive 50%
and the worst, which i missed the name of but is the worst and affects 5% of people who have MS
So what causes it? Don't know. A genetic predisposition combined with an environmental trigger
Image of a nerve cell with its axon all bare
T cell cross the blood/brain barrier, which attack myelin and results in symptoms
current treatments
corticosteroids, disease modifying treatments, symptom management
one of the things we're beginning to see is that even with all these disease modifying treatments, there is still an increase in disability . . . a progression in a downward direction
so if we can control the disease, can we reverse it?
how do stem cells fit in?
stimulate repair by transplantation
use stem cells as platforms for neuroprotection
figure out what causes the loss of axons
use stem cells as a platform for discovery
these 4 areas are exciting opportunities for us in terms of funding and moving forward
Sabrina Cohen
Waiting for the morning session to begin . . . a sad moment for me when they turn out the lights. This room goes completely dark, and the panel sits way up on a stage far from where I am, next to the camera crew that's making the webcast.
CC readers will know a few of these panelists: Sabrina Cohen, Wise Young, Hans Keirstead; I'll get to the others was they show up.
Sabrina is first:
I personally have had a remarkable couple of days and am delighted to be here
1992, halloween night, i was a sophomore in high school. my best friend and i jumped into a car with 3 guys and another girl, i was a freespirited teenager. by 9:30 that night i was a c3-c4 quadriplegic. we had split up into two cars i sat in the back seat without a seatblet. the cars got into a drag race going 90 mph n a 30 mph zone. the other car lost control and hit us . . . we hit a tree. all the other kids were fine.
i woke up a couple of days later not knowing where i was, surrounded by friends and family. 2 months in intensive care, then wheeled into a cafeteria . . .i saw all these other people in chairs and realized i had become one of them
pt for many months . . . my mom insisted that i get back to school . . completely paralyzed from the neck down, a nurse pushing me through the halls. . . finished on time with my class.
became a public speaker, though i didn't want to. i was always talking about driving to young kids and teenagers . .. peer pressure, drag racing, seatbelts . . . went to the university of miami, psychology, advertising . . . started my own company in 2003
a year into my business, in april of 2004 on a jewish holiday i went to jackson memorial hospital for pt . . . saw a poster advertising danny neumann . . . waited for 3 hours to see him. he was phenomenal
next up was bernie siegel, who lit something up in me within 2 minutes of opening his mouth. he invited me to work with him, which i did for 2 years
in 2006 i decided to channel some of my frustration with the lack of progress . .. started the sabrina cohen foundation
i travel and speak to other organizations . .. now have a documentary in progress . . . this year will announce a grant raised for furthering science
i need to do a quick exercise.
everybody put your pens down, put your hands in your laps . . . when i say go, sit up straight, stay still, and don't move a muscle . . . ready?
go
no cheating
if you have an itch, forget it
we're halfway through
almost there
5 4 3 2 1
okay. raise your hand if that felt like forever . .. that was one minute.
for us in chairs, it always feels like forever, no matter how long we've been diagnosed
i wanted you to do that exercise to get us out of this condition
today when the conference is over it's not an end, it's a beginning.
spread the education as much as you can -- i know i will.
love you, sabrina!
she's just perfectly composed, very clear and of course beautiful.
CC readers will know a few of these panelists: Sabrina Cohen, Wise Young, Hans Keirstead; I'll get to the others was they show up.
Sabrina is first:
I personally have had a remarkable couple of days and am delighted to be here
1992, halloween night, i was a sophomore in high school. my best friend and i jumped into a car with 3 guys and another girl, i was a freespirited teenager. by 9:30 that night i was a c3-c4 quadriplegic. we had split up into two cars i sat in the back seat without a seatblet. the cars got into a drag race going 90 mph n a 30 mph zone. the other car lost control and hit us . . . we hit a tree. all the other kids were fine.
i woke up a couple of days later not knowing where i was, surrounded by friends and family. 2 months in intensive care, then wheeled into a cafeteria . . .i saw all these other people in chairs and realized i had become one of them
pt for many months . . . my mom insisted that i get back to school . . completely paralyzed from the neck down, a nurse pushing me through the halls. . . finished on time with my class.
became a public speaker, though i didn't want to. i was always talking about driving to young kids and teenagers . .. peer pressure, drag racing, seatbelts . . . went to the university of miami, psychology, advertising . . . started my own company in 2003
a year into my business, in april of 2004 on a jewish holiday i went to jackson memorial hospital for pt . . . saw a poster advertising danny neumann . . . waited for 3 hours to see him. he was phenomenal
next up was bernie siegel, who lit something up in me within 2 minutes of opening his mouth. he invited me to work with him, which i did for 2 years
in 2006 i decided to channel some of my frustration with the lack of progress . .. started the sabrina cohen foundation
i travel and speak to other organizations . .. now have a documentary in progress . . . this year will announce a grant raised for furthering science
i need to do a quick exercise.
everybody put your pens down, put your hands in your laps . . . when i say go, sit up straight, stay still, and don't move a muscle . . . ready?
go
no cheating
if you have an itch, forget it
we're halfway through
almost there
5 4 3 2 1
okay. raise your hand if that felt like forever . .. that was one minute.
for us in chairs, it always feels like forever, no matter how long we've been diagnosed
i wanted you to do that exercise to get us out of this condition
today when the conference is over it's not an end, it's a beginning.
spread the education as much as you can -- i know i will.
love you, sabrina!
she's just perfectly composed, very clear and of course beautiful.
Monday, September 22, 2008
Governor Jim Doyle
Welcomes us to Wisconsin, where sun and 70 degrees are normative.
It was right here that Jamie Thomson and a few other brilliant researchers made the discoveries that changed the way we think about medicine and health.
Here's a quick story. I was in NYC with Michael J Fox, and we had met with some of the researchers there . . . he told me that when he got on the plane to Madison he felt like he was making a pilgrimage--going to the place where incredible breakthroughs have been made.
We feel very honored to have played such an important role in this accomplishment. I get to brag. We're not just home to Jamie Thomson; we have 40 stem cell researchers, and they're cited more frequently than any other team in the literature.
We've got the institute for medical research associated with the school of public health that will make it possible to move therapies faster from concept to patients.
The Milwaukee medical college has also created a program in stem cell research, so it's not just happening in Madison.
When Forbes named the 12 revolutionaries in stem cell science, 2 of them came from Wisconsin.
The private Morbridge institute will allow our scientists to work directly with the private sector . . . we'll have collaboration.
We now have 34,000 people working in bio-technology, contributing $9B to our economy.
Like lots of you, we've fought through some very significant hostility in our legislature. I'm proud to say that in this state the voices of our families were heard, and many of them are here in this room. We had help from other states; but stem cell research was never slowed down in Wisconsin.
The battle here is over. In Michigan there is a battle going on right now; voters will decide in six weeks whether or not they want to allow hESc research in their state.
Federal funding has been flat since 2002; we need to give our researchers the resources they need to do their work.
The political battle in Wisconsin over stem cell research ended with the election of a governor who supported it. Nationally the same thing can happen. I want to make sure that we have a president who rescinds the restriction on NIH funding as one of his first acts.
We have to decide whether personal ideologies must triumph over science. One of the most harmful things to come out of the conversation in the last few years is the perception that scientists operate outside of moral boundaries.
Your work has the capacity to heal and to alleviate human suffering. Please let the public know that we're not talking about mad, evil guys in a lab . . . that these are well-considered and balanced decisions being made.
We in Wisconsin are prepared to make significant investments into companies that are making practical applications out of stem cells. How does it get better than saving human lives while growing your economy? That's what we've worked hard to build here in Wisconsin.
I'm often asked if Wisconsin is going to "lose" in the competition to create therapies. This isn't football. This isn't a competition. The breakthroughs that happened here led to more of the same elsewhere; breakthroughs elsewhere will lead to more of the same here.
Wisconsin is going to be a major factor in this research for many years to come.
It was right here that Jamie Thomson and a few other brilliant researchers made the discoveries that changed the way we think about medicine and health.
Here's a quick story. I was in NYC with Michael J Fox, and we had met with some of the researchers there . . . he told me that when he got on the plane to Madison he felt like he was making a pilgrimage--going to the place where incredible breakthroughs have been made.
We feel very honored to have played such an important role in this accomplishment. I get to brag. We're not just home to Jamie Thomson; we have 40 stem cell researchers, and they're cited more frequently than any other team in the literature.
We've got the institute for medical research associated with the school of public health that will make it possible to move therapies faster from concept to patients.
The Milwaukee medical college has also created a program in stem cell research, so it's not just happening in Madison.
When Forbes named the 12 revolutionaries in stem cell science, 2 of them came from Wisconsin.
The private Morbridge institute will allow our scientists to work directly with the private sector . . . we'll have collaboration.
We now have 34,000 people working in bio-technology, contributing $9B to our economy.
Like lots of you, we've fought through some very significant hostility in our legislature. I'm proud to say that in this state the voices of our families were heard, and many of them are here in this room. We had help from other states; but stem cell research was never slowed down in Wisconsin.
The battle here is over. In Michigan there is a battle going on right now; voters will decide in six weeks whether or not they want to allow hESc research in their state.
Federal funding has been flat since 2002; we need to give our researchers the resources they need to do their work.
The political battle in Wisconsin over stem cell research ended with the election of a governor who supported it. Nationally the same thing can happen. I want to make sure that we have a president who rescinds the restriction on NIH funding as one of his first acts.
We have to decide whether personal ideologies must triumph over science. One of the most harmful things to come out of the conversation in the last few years is the perception that scientists operate outside of moral boundaries.
Your work has the capacity to heal and to alleviate human suffering. Please let the public know that we're not talking about mad, evil guys in a lab . . . that these are well-considered and balanced decisions being made.
We in Wisconsin are prepared to make significant investments into companies that are making practical applications out of stem cells. How does it get better than saving human lives while growing your economy? That's what we've worked hard to build here in Wisconsin.
I'm often asked if Wisconsin is going to "lose" in the competition to create therapies. This isn't football. This isn't a competition. The breakthroughs that happened here led to more of the same elsewhere; breakthroughs elsewhere will lead to more of the same here.
Wisconsin is going to be a major factor in this research for many years to come.
Panel: Stem Cell Nations
Bernie Siegel is introducing the new panel. These are superstars . . .
Linda Powers of Toucan Capital, which is a VC that funds many, many enterprises in stem cell research.
Bob Klein of the California Institute for Regenerative Medicine, rock star in the hESc world
Chris Mason from the University of London
Fanyi Zeng of the Shanghai Stem Cell Institute
Moderator is Tom Still of the Wisconsin Technology Council, who is now saying that he's convinced Chris Mason that Wisconsin weather is always like this (70's and sunny, sweet, sweet air . . .)
Here's Bob Klein, author of Prop 71.
I'd like to make it clear that I understand that funding of this research is a support function. Quotes Schwarzenegger: The scientists and doctors who make it happen are the action heroes of the 21st century.
Thanks Governor Doyle of Wisconsin, who has been another strong leader, along with Schwarzenegger.
Wants to focus on two governing principles w/respect to CIRM.
What have we done to advance research outside the federal restrictions?
Ahem. Prop 71, which was approved by 59% of CA voters in November 2004, established the CIRM and authorized $3B in principal and $3B in interest to fund stem cell research inCA. This is not operating capital taken out of the budget, and it was important to do it that way.
We don't fund operating costs. We fund intellectual capital to carry us into the 21st century.
Prop 71 also wrote hESc into the CA constitution and banned human cloning.
The independent citiens oversight committee is made up of the governing board of the CIRM and 14 other members. There is a grants working group made up of scientists from outside the state of CA; it's their job to review grants.
By June of 2009, they will have committed $1B $19M in funds.
A lot of this money is in the form of high risk loans; if we put $500M into loans, when that money cycles we have $600M . . .and when that money cycles we have $700M.
There will be much more than $3billion in the end, is the point. He's going through his spreadsheet--not line by line but hitting highlights to show that one effect of Prop 71 has been to expand donor participation far above any levels ever seen before.
Next will be private industry and the contribution they can make. The goal is to get to a Phase 1 trial with human beings within 48 months.
Fine, all this was approved in 2004, but the US just spent many hundreds of billions of dollars on financial rescue. 2004 was the worst financial year since the 50's for CA -- and they still managed to get the signatures to get it on the ballot.
On a national basis, if the program is not disease-specific you will get the money you need. The states have an advantage because if they use the bonds method, it's obvious you're financing capital--and you're not stealing from existing hospitals and public programs to do it.
In the last century, we invested in physical capital--roads and bridges and infrastructure. This century the capital will be intellectual.
We're talking about interventionist therapies, like the Salk vaccine. The cost per year to have juvenile diabetes is $15,000/year for the life of the child. It's worth an investment to save that money, when you multiply that child by how many of him there are, and how many other conditions there are that eat up dollars and productivity.
In CA our perspective is that stem cell research will not advance as a competition; it will advance as a global war on diseases and conditions.
Next is Linda Powers, who has been an investor, a professor at Georgetown, a leader of seminars for scientists on how to be entreprenurial.
So, how are we doing?
What's the progress in research and translation to the clinic?
What's the progress in economic development?
I spent 7 years building a family of 16 stem cell companies. We're in the trenches every day. A lot of states have been glavanized by CA's leadership. NY has started with $600M over 11 years. MD has made 3 kinds of grants. CT has created investigaor grants. NJ has invested $279M into 5 facilities. One of them puts stem cell treatment into the same facility as a hospital.
In MD, there are 3 separate grants, all coming from general budget funds, not bonds. We've created a level playing field for all cell types. "let the best cells win"
To get grants, researchers must spell out specifically what the translation plans are . . . they have physicians on their review board to make sure these plans are robust.
On the scientific front, there has been an explosion of papers in the last few years from around the world.
There are right now nearly 200 clinical trials underway today--all of them using adult stem cells--and aimed at a wide range of conditions.
Shows a Forbes Cover that says "Stem Cells Get Real"
Both the military and medical tourism are helping to accelerate regenerative medicine. It's very important to be cautious about how balance here . . . we need a middle ground where offshore approaches that are different from ours are not just dismissed because we don't understand them. Transparency must be the goal.
Economically, there have been lots of new stem cell companies formed in just the last year . . . there are lots of business models and a full pipeline of products.
Next will be Dr. Chris Mason, who "symbolizes the strength of what's going on in the UK"
(Read this with a very elegant British accent, please) In the UK, there has always been strong public and political support.
We have established centers of excellence, willingness to collaborate, and infrastructure.
The UK stem cell initiative was a 10 year plan beginning in december 2005. funding was 1,300M per year
We created both national and local networks.
Martin Evans got a Nobel Prize in 2007. They have a stem cell bank under construction. They've enjoyed strong UK Government support for translation. They need to translate both into the clinic and into successful commercial practice.
Their clinical trials will be easier to implement than in some places becaue they have a single healthcare service.
They've had partnerships with private companies, including big pharma, have already spent about $100M on regenerative medicine.
They've made themselves a leader in creating global standards for quality,safety, and performance.
They've funded international missions, especially in the far east, and they're keen to collaborate.
Regenerative Medicine is a new journal they established in 2006.
To sum up: UK is as small island with lots of enthusiasm!
Last speaker is Fanyi Zeng from the Shanghai Stem Cell Institute
In 1978 the society of medical genetics was created; the focuse was disease screening and early diagnosis. In 1982 they won a NIH developing grant, which funded them for 10 years.
Mmmmm. Full brain syndrome attack here. She's giving us the history of what they've done in China in the last 30 years, carried out in the context of the giant economic progress there.
They hope to have a trillion dollars invested by 2020, which, she points out, would put them ahead of the USA in terms of who leads. They have very robust funding resources. They could not have done this without the opening of the door in 1978 or without funding from the NIH, or without great planning and support from their government.
They have a national strategy. What a concept.
They have 50 research groups, 30 of which are working on iPS technology. They're constantly looking for investment partners. I bet.
So, now the panel is asked to evaluate who the leaders are right now and who they will be in ten years.
Powers says the people here obviously represent countries that are leaders, along with Australia . . . India not so much. Klein says that Canada is emerging as a player, because they have an economic surplus. Adds that we in the USA have a limited window of time to get to that therapeutic breakthrough.
How does the UK go about collaboration so successfully? His microphone is not close enough to his face, unfortunately . . .
What will change on January 20th, 2009?
Klein says that the states must continue to step up. Stanford was offered money to build a center, which they rejected because they could not depend on the stability of the federal government. States can provide longterm stability because they don't have to depend on year to year budgeting processes; they can use bonds. One of the UK's centers of excellence came from CA; he left the USA because of funding restrictions.
Powers says that there are lots of things holding hESc back in the USA and Bush is only one of them. She names the piddling amount spent at NIH on adult stem cells and on medical research in general. There are also regulatory hurdles,the specific regulatory requirements to simply get into the clinic.
In the usA we draw no distinctions in regulation between implanting a patient's own cells and those of someone else--which seems wrong.
Linda Powers of Toucan Capital, which is a VC that funds many, many enterprises in stem cell research.
Bob Klein of the California Institute for Regenerative Medicine, rock star in the hESc world
Chris Mason from the University of London
Fanyi Zeng of the Shanghai Stem Cell Institute
Moderator is Tom Still of the Wisconsin Technology Council, who is now saying that he's convinced Chris Mason that Wisconsin weather is always like this (70's and sunny, sweet, sweet air . . .)
Here's Bob Klein, author of Prop 71.
I'd like to make it clear that I understand that funding of this research is a support function. Quotes Schwarzenegger: The scientists and doctors who make it happen are the action heroes of the 21st century.
Thanks Governor Doyle of Wisconsin, who has been another strong leader, along with Schwarzenegger.
Wants to focus on two governing principles w/respect to CIRM.
What have we done to advance research outside the federal restrictions?
Ahem. Prop 71, which was approved by 59% of CA voters in November 2004, established the CIRM and authorized $3B in principal and $3B in interest to fund stem cell research inCA. This is not operating capital taken out of the budget, and it was important to do it that way.
We don't fund operating costs. We fund intellectual capital to carry us into the 21st century.
Prop 71 also wrote hESc into the CA constitution and banned human cloning.
The independent citiens oversight committee is made up of the governing board of the CIRM and 14 other members. There is a grants working group made up of scientists from outside the state of CA; it's their job to review grants.
By June of 2009, they will have committed $1B $19M in funds.
A lot of this money is in the form of high risk loans; if we put $500M into loans, when that money cycles we have $600M . . .and when that money cycles we have $700M.
There will be much more than $3billion in the end, is the point. He's going through his spreadsheet--not line by line but hitting highlights to show that one effect of Prop 71 has been to expand donor participation far above any levels ever seen before.
Next will be private industry and the contribution they can make. The goal is to get to a Phase 1 trial with human beings within 48 months.
Fine, all this was approved in 2004, but the US just spent many hundreds of billions of dollars on financial rescue. 2004 was the worst financial year since the 50's for CA -- and they still managed to get the signatures to get it on the ballot.
On a national basis, if the program is not disease-specific you will get the money you need. The states have an advantage because if they use the bonds method, it's obvious you're financing capital--and you're not stealing from existing hospitals and public programs to do it.
In the last century, we invested in physical capital--roads and bridges and infrastructure. This century the capital will be intellectual.
We're talking about interventionist therapies, like the Salk vaccine. The cost per year to have juvenile diabetes is $15,000/year for the life of the child. It's worth an investment to save that money, when you multiply that child by how many of him there are, and how many other conditions there are that eat up dollars and productivity.
In CA our perspective is that stem cell research will not advance as a competition; it will advance as a global war on diseases and conditions.
Next is Linda Powers, who has been an investor, a professor at Georgetown, a leader of seminars for scientists on how to be entreprenurial.
So, how are we doing?
What's the progress in research and translation to the clinic?
What's the progress in economic development?
I spent 7 years building a family of 16 stem cell companies. We're in the trenches every day. A lot of states have been glavanized by CA's leadership. NY has started with $600M over 11 years. MD has made 3 kinds of grants. CT has created investigaor grants. NJ has invested $279M into 5 facilities. One of them puts stem cell treatment into the same facility as a hospital.
In MD, there are 3 separate grants, all coming from general budget funds, not bonds. We've created a level playing field for all cell types. "let the best cells win"
To get grants, researchers must spell out specifically what the translation plans are . . . they have physicians on their review board to make sure these plans are robust.
On the scientific front, there has been an explosion of papers in the last few years from around the world.
There are right now nearly 200 clinical trials underway today--all of them using adult stem cells--and aimed at a wide range of conditions.
Shows a Forbes Cover that says "Stem Cells Get Real"
Both the military and medical tourism are helping to accelerate regenerative medicine. It's very important to be cautious about how balance here . . . we need a middle ground where offshore approaches that are different from ours are not just dismissed because we don't understand them. Transparency must be the goal.
Economically, there have been lots of new stem cell companies formed in just the last year . . . there are lots of business models and a full pipeline of products.
Next will be Dr. Chris Mason, who "symbolizes the strength of what's going on in the UK"
(Read this with a very elegant British accent, please) In the UK, there has always been strong public and political support.
We have established centers of excellence, willingness to collaborate, and infrastructure.
The UK stem cell initiative was a 10 year plan beginning in december 2005. funding was 1,300M per year
We created both national and local networks.
Martin Evans got a Nobel Prize in 2007. They have a stem cell bank under construction. They've enjoyed strong UK Government support for translation. They need to translate both into the clinic and into successful commercial practice.
Their clinical trials will be easier to implement than in some places becaue they have a single healthcare service.
They've had partnerships with private companies, including big pharma, have already spent about $100M on regenerative medicine.
They've made themselves a leader in creating global standards for quality,safety, and performance.
They've funded international missions, especially in the far east, and they're keen to collaborate.
Regenerative Medicine is a new journal they established in 2006.
To sum up: UK is as small island with lots of enthusiasm!
Last speaker is Fanyi Zeng from the Shanghai Stem Cell Institute
In 1978 the society of medical genetics was created; the focuse was disease screening and early diagnosis. In 1982 they won a NIH developing grant, which funded them for 10 years.
Mmmmm. Full brain syndrome attack here. She's giving us the history of what they've done in China in the last 30 years, carried out in the context of the giant economic progress there.
They hope to have a trillion dollars invested by 2020, which, she points out, would put them ahead of the USA in terms of who leads. They have very robust funding resources. They could not have done this without the opening of the door in 1978 or without funding from the NIH, or without great planning and support from their government.
They have a national strategy. What a concept.
They have 50 research groups, 30 of which are working on iPS technology. They're constantly looking for investment partners. I bet.
So, now the panel is asked to evaluate who the leaders are right now and who they will be in ten years.
Powers says the people here obviously represent countries that are leaders, along with Australia . . . India not so much. Klein says that Canada is emerging as a player, because they have an economic surplus. Adds that we in the USA have a limited window of time to get to that therapeutic breakthrough.
How does the UK go about collaboration so successfully? His microphone is not close enough to his face, unfortunately . . .
What will change on January 20th, 2009?
Klein says that the states must continue to step up. Stanford was offered money to build a center, which they rejected because they could not depend on the stability of the federal government. States can provide longterm stability because they don't have to depend on year to year budgeting processes; they can use bonds. One of the UK's centers of excellence came from CA; he left the USA because of funding restrictions.
Powers says that there are lots of things holding hESc back in the USA and Bush is only one of them. She names the piddling amount spent at NIH on adult stem cells and on medical research in general. There are also regulatory hurdles,the specific regulatory requirements to simply get into the clinic.
In the usA we draw no distinctions in regulation between implanting a patient's own cells and those of someone else--which seems wrong.
Panel: Parkinson's, ALS, Neurological disorders
After introductions, Stephen Byer gets up to speak as patient advocate. He lost his son to ALS just weeks ago.
He's saying (with great dignity) that in 2004 he and Ben found themselves in China, hoping desperately that the promises they'd been given would pan out.
Speaking very eloquently about the hazards of medical tourism, when it comes to spending tens of thousands of dollars for unproved and untested and potentially unsafe treatments.
Offshore facilities treating ALS patients:
Carlos Lima Portugal, Israel, Beijing, Tel Aviv, Rio de Janeiro, Germany, Ukraine, El Salvador, Jerusalem, London -- all of them, he says, ought to come with caveat emptor (buyer beware)
Now gives the really bad list, beginning with Dr. Kumar in India. Advanced Cell Therapeutics is next, which has operated in lots of countries and which is wanted for fraud. A Ukraine company headed by a former psychiatrist who claims to fix everything with a single injection of fetal cells. Finally Eden Laboratories. This time he says the model ought to be buyer run away.
This is bloody depressing.
He says that anyone thinking of traveling to another country for treatment must do exhaustive research and see substantive data. He knows a lot about this because he's spent the last 7 years (of the Bush administration) focused on it.
Next is Lucie Bruijn of the ALS association; she says that she's spoken about medical tourism with a great many families of patients, but it's very difficult -- people must make their own choices.
She has a slide up about translational research, and is talking about how they've focused on two stem cell treatments.
ALS is likely to be caused by many different things; their effort has been aimed at getting motor neurons to grow from human ES cells; California Stem Cell has been able to make them very successfully . . . a Hans Keirstead lab effort.
Okay, unlike many others here today, she's presenting science; it's a bit past the scope of what can be captured in print. There's a webcast of this up and those reading here who are up to it will need to go and see it.
The basic message is very positive and detailed.
Mark Noble, from the University of Rochester: let's expand the conversation.
The tool kit must be built. We work on progenitor cells, which lie between stem cells and differentiated cell types. We don't work on stem cells.
We work on Glial restricted precursor cells
The o2a progenitor cell grows best in the presence of 2 growth factors and astrocytes. When we transplanted these into demylenated lesions in rats they were indistinguishable in terms of function from healthy rats.
The move now underway to use oligodendrocyte precursors extends the work we did 15 years ago; it would benefit from more understanding of what we're doing today.
Mark works with Stephen and Jeanette Davies on this project. He's talking about their astrocytes, and the big message is that if you put the wrong kind of astrocyte into a damaged cord you get neuropathic pain. If you put the right kind in you get return of function and no pain.
The challenge of toxicology -- the WHO estimates that there are 80-150 unregulated chemicals in our bodies, and we have not a clue how they react with one another.
Here's bad news: progenitor cells are primary targets of environmental toxicants -- not stem cells or astrocytes, though. Just progenitors.
Sorry, again this is science I'm not familiar with; the tape will show you the details. The basic message is that it's very tricky to manage stem cells into becoming the exact cells you want.
Next is a bit about adult adult stem cells being used in neurological disease or injury . . . they've got "very interesting results with collaborators" in head injury and spinal cord injury.
We have approval from the FDA; we can take cells from one donor and expand them multiple times; we process them in a closed system; we get to a master bank level, we check the cells according to multiple different products; and finally the get to patients.
(How do I find out what he's talking about w/respect to sci?)
People think of stem cells as going in and replacing cell by cell the ones that were damaged or lost. Right now that's not the model we have in mind . . . sometimes we just think of going in and "calming things down" instead.
We have to be very careful with trial designs because we won't get second chances with the FDA.
Ischemic stroke: this is their target right now. The market for this in the USA is $8 billion. They've worked with scientists in Houston and Georgia. Will the patients need immunosuppression? No.
They've filed the IND for a study on this in humans already. A lot of different clinical centers came to them to ask if they could be included in the trial.
CEO of Neuralstem, Richard Garr: one problem is that people use the same words to describe different things. Our technology is regionally specific; they don't push their cells to a different fate, the cells are already what they're supposed to be.
We've already created a GMP cell bank for our spinal cord cells.
The reason I'm here today is to talk about our first trial, which will be for ALS. The data supporting it will be published soon. There will be 15 patients, who will get multiple injections to preserve ambulation and to preserve and enhance the breathing function. The cells don't migrate and don't need to be turned into something else; the issue will be making sure they put them into the right places . . . the safety of the surgical technique will be the big focus . . .putting a needle into the spinal cord 30 times . . . we've done a tremendous number of animals.
We expect to file the IND this fall, probably within the next few weeks. Our hope is to begin the trial early next year.
Next is Teepu Siddique of Northwestern University, a physician. ALS, he says, is a very frustrating proposition.
The art of medicine requires that we do no harm, but we also must keep hope alive . . . we're in a difficult time where the streets are full of sharks wanting to prey on patients -- who do not have a choice but to take the journey.
The end point of course is to find a cure for ALS and other diseases. Before that there is a set of information that hard-nosed scientists must focus on. ALS is not a disease of the petri dish . . . it lives in an organic human being.
People have been doing silly injections for a long time, because there is a big gap in our knowledge about what actually takes place inside a living spinal cord or brain. We have to address that gap. He thinks we've rushed ahead of ourselves and ought to slow down and follow a rational plan. Here's his:
1. cure a mouse
2. understand the progenitor bioology and cell fate commitment steps
3. do a thorough CNS development project
That would be a beginning. This is followed by a conversation amonst the panelists that unforunately goes mostly over the heads of us out here. It's interesting to watch stakeholders challenge each other . . . the gist I'm getting is that the last speaker feels that many in the research community have pushed away too many important questions, and they are responding --variously--that he has a point but not when it comes to their particular approach. Or that he's even more right than he knows.
It ends with the father of the ALS victim saying very thoughtfully that his own response is probably not what it might have been a year ago . . . he does think safety matters a lot . . . and he says that we can learn from all those offshore efforts, if only what not to do.
He's saying (with great dignity) that in 2004 he and Ben found themselves in China, hoping desperately that the promises they'd been given would pan out.
Speaking very eloquently about the hazards of medical tourism, when it comes to spending tens of thousands of dollars for unproved and untested and potentially unsafe treatments.
Offshore facilities treating ALS patients:
Carlos Lima Portugal, Israel, Beijing, Tel Aviv, Rio de Janeiro, Germany, Ukraine, El Salvador, Jerusalem, London -- all of them, he says, ought to come with caveat emptor (buyer beware)
Now gives the really bad list, beginning with Dr. Kumar in India. Advanced Cell Therapeutics is next, which has operated in lots of countries and which is wanted for fraud. A Ukraine company headed by a former psychiatrist who claims to fix everything with a single injection of fetal cells. Finally Eden Laboratories. This time he says the model ought to be buyer run away.
This is bloody depressing.
He says that anyone thinking of traveling to another country for treatment must do exhaustive research and see substantive data. He knows a lot about this because he's spent the last 7 years (of the Bush administration) focused on it.
Next is Lucie Bruijn of the ALS association; she says that she's spoken about medical tourism with a great many families of patients, but it's very difficult -- people must make their own choices.
She has a slide up about translational research, and is talking about how they've focused on two stem cell treatments.
ALS is likely to be caused by many different things; their effort has been aimed at getting motor neurons to grow from human ES cells; California Stem Cell has been able to make them very successfully . . . a Hans Keirstead lab effort.
Okay, unlike many others here today, she's presenting science; it's a bit past the scope of what can be captured in print. There's a webcast of this up and those reading here who are up to it will need to go and see it.
The basic message is very positive and detailed.
Mark Noble, from the University of Rochester: let's expand the conversation.
The tool kit must be built. We work on progenitor cells, which lie between stem cells and differentiated cell types. We don't work on stem cells.
We work on Glial restricted precursor cells
The o2a progenitor cell grows best in the presence of 2 growth factors and astrocytes. When we transplanted these into demylenated lesions in rats they were indistinguishable in terms of function from healthy rats.
The move now underway to use oligodendrocyte precursors extends the work we did 15 years ago; it would benefit from more understanding of what we're doing today.
Mark works with Stephen and Jeanette Davies on this project. He's talking about their astrocytes, and the big message is that if you put the wrong kind of astrocyte into a damaged cord you get neuropathic pain. If you put the right kind in you get return of function and no pain.
The challenge of toxicology -- the WHO estimates that there are 80-150 unregulated chemicals in our bodies, and we have not a clue how they react with one another.
Here's bad news: progenitor cells are primary targets of environmental toxicants -- not stem cells or astrocytes, though. Just progenitors.
Sorry, again this is science I'm not familiar with; the tape will show you the details. The basic message is that it's very tricky to manage stem cells into becoming the exact cells you want.
Next is a bit about adult adult stem cells being used in neurological disease or injury . . . they've got "very interesting results with collaborators" in head injury and spinal cord injury.
We have approval from the FDA; we can take cells from one donor and expand them multiple times; we process them in a closed system; we get to a master bank level, we check the cells according to multiple different products; and finally the get to patients.
(How do I find out what he's talking about w/respect to sci?)
People think of stem cells as going in and replacing cell by cell the ones that were damaged or lost. Right now that's not the model we have in mind . . . sometimes we just think of going in and "calming things down" instead.
We have to be very careful with trial designs because we won't get second chances with the FDA.
Ischemic stroke: this is their target right now. The market for this in the USA is $8 billion. They've worked with scientists in Houston and Georgia. Will the patients need immunosuppression? No.
They've filed the IND for a study on this in humans already. A lot of different clinical centers came to them to ask if they could be included in the trial.
CEO of Neuralstem, Richard Garr: one problem is that people use the same words to describe different things. Our technology is regionally specific; they don't push their cells to a different fate, the cells are already what they're supposed to be.
We've already created a GMP cell bank for our spinal cord cells.
The reason I'm here today is to talk about our first trial, which will be for ALS. The data supporting it will be published soon. There will be 15 patients, who will get multiple injections to preserve ambulation and to preserve and enhance the breathing function. The cells don't migrate and don't need to be turned into something else; the issue will be making sure they put them into the right places . . . the safety of the surgical technique will be the big focus . . .putting a needle into the spinal cord 30 times . . . we've done a tremendous number of animals.
We expect to file the IND this fall, probably within the next few weeks. Our hope is to begin the trial early next year.
Next is Teepu Siddique of Northwestern University, a physician. ALS, he says, is a very frustrating proposition.
The art of medicine requires that we do no harm, but we also must keep hope alive . . . we're in a difficult time where the streets are full of sharks wanting to prey on patients -- who do not have a choice but to take the journey.
The end point of course is to find a cure for ALS and other diseases. Before that there is a set of information that hard-nosed scientists must focus on. ALS is not a disease of the petri dish . . . it lives in an organic human being.
People have been doing silly injections for a long time, because there is a big gap in our knowledge about what actually takes place inside a living spinal cord or brain. We have to address that gap. He thinks we've rushed ahead of ourselves and ought to slow down and follow a rational plan. Here's his:
1. cure a mouse
2. understand the progenitor bioology and cell fate commitment steps
3. do a thorough CNS development project
That would be a beginning. This is followed by a conversation amonst the panelists that unforunately goes mostly over the heads of us out here. It's interesting to watch stakeholders challenge each other . . . the gist I'm getting is that the last speaker feels that many in the research community have pushed away too many important questions, and they are responding --variously--that he has a point but not when it comes to their particular approach. Or that he's even more right than he knows.
It ends with the father of the ALS victim saying very thoughtfully that his own response is probably not what it might have been a year ago . . . he does think safety matters a lot . . . and he says that we can learn from all those offshore efforts, if only what not to do.
Why the CDRF is investing in stem cell research
Bernie Siegel is introducing Danny Heumann, mega-rock star from Ann Arbor. Michigan apparently has a ballot initiative up this year that will support hESc research in Michigan.
He says that his family and he were able to raise $5 million dollars to fund research to cure spinal cord injury. (Danny is speaking from his wheelchair). He decided recently that he wanted to juice it up a bit, and so joined forces with CDRF. Now he's giving a fine introduction to Peter Kiernan, whom he describes as superhuman.
Keirnan . . . how I got here. I worked on wall street. I was a mercenary. So what does wall street know about nonprofits. Actually, a lot, if you read the papers. ha ha ha
So, he got a phone call from a dear friend who asked him to
We used to have 2 living breathing iconoclasts who were the DNA of our organization. Their lasting legacy is that we're now raising more money today than we ever did when they were alive.
"Join with us."
Here's the top 10 things we're fighting for, Letterman style
10. Get the politics out of stem cell research & let the scientists do their work. I always thought scientists were objective, cool, dispassionate . . . NO. They're driven. Let them work.
9. Fund the young scientists. We're making them into politicians, always going around with their hands out. The only get 5% of the dough the NIH has handed out, and we need them in the world of hESc research. It's time to invest in the human infrastructure.
8. Get more dough into translational researchers. There's a valley of death between basic science and clinical work. We're pretty good at picking the right idea. The way venture capital works is, you pick 10 things and hope that 2 or 3 of them work out. We need to become a magnet for money.
7. Create the global world network. What we know from the human genome project isthat it can be done. When it comes to sci--that complex of a problem-- will need lots and lots of colaborators
6. Change the subject. Dolly the sheep hi-jacked the conversation and we need to take it back. We're
5. Educate the press. They're very good at distilling complicated issues into simple things. They don't know where to focus, and that is our fault. We have to direct them, or else they turn everything into a competition. (Adult stem cells are the best!)
4. Embrace business more effectively than we have. Pharma knows that they need to figure this out -- we've spent more time with them in the last year than we have in all the last 10 put together. We're on the tipping point for human clinical trials . . . there will be some victories and some pain. We have to be collaborating with the business community or we'll get set back.
3. Create that national stem cell bank. Like a federal reserve bank . . . we need to be looking at how this will evolve and be prepared to organize so that there are standards and procedures everyone can depend on. In the USA we're in a fits and starts mode . . but we should follow the UK/Canada model.
2. Police ourselves. We live in an impoverished (but blissful) world of zero regulation together with very little money. What we don't want is to get the big regulation and the same crumbs of cash. It's our job to be vigilant about bad science and to call out the snake oil sellers. Makes the comparison to wall street: police yourself or you're going to get policed.
1. Achieve a clinical breakthrough. In the 70's IVF was the work of the devil. Joy Louise Brown changed that conversation. The world's best researchers are in this room. Amazing things are about to happen and everybody here knows it; we're going to bend the river.
MMMM, Danny was right. he's a good speaker.
He says that his family and he were able to raise $5 million dollars to fund research to cure spinal cord injury. (Danny is speaking from his wheelchair). He decided recently that he wanted to juice it up a bit, and so joined forces with CDRF. Now he's giving a fine introduction to Peter Kiernan, whom he describes as superhuman.
Keirnan . . . how I got here. I worked on wall street. I was a mercenary. So what does wall street know about nonprofits. Actually, a lot, if you read the papers. ha ha ha
So, he got a phone call from a dear friend who asked him to
We used to have 2 living breathing iconoclasts who were the DNA of our organization. Their lasting legacy is that we're now raising more money today than we ever did when they were alive.
"Join with us."
Here's the top 10 things we're fighting for, Letterman style
10. Get the politics out of stem cell research & let the scientists do their work. I always thought scientists were objective, cool, dispassionate . . . NO. They're driven. Let them work.
9. Fund the young scientists. We're making them into politicians, always going around with their hands out. The only get 5% of the dough the NIH has handed out, and we need them in the world of hESc research. It's time to invest in the human infrastructure.
8. Get more dough into translational researchers. There's a valley of death between basic science and clinical work. We're pretty good at picking the right idea. The way venture capital works is, you pick 10 things and hope that 2 or 3 of them work out. We need to become a magnet for money.
7. Create the global world network. What we know from the human genome project isthat it can be done. When it comes to sci--that complex of a problem-- will need lots and lots of colaborators
6. Change the subject. Dolly the sheep hi-jacked the conversation and we need to take it back. We're
5. Educate the press. They're very good at distilling complicated issues into simple things. They don't know where to focus, and that is our fault. We have to direct them, or else they turn everything into a competition. (Adult stem cells are the best!)
4. Embrace business more effectively than we have. Pharma knows that they need to figure this out -- we've spent more time with them in the last year than we have in all the last 10 put together. We're on the tipping point for human clinical trials . . . there will be some victories and some pain. We have to be collaborating with the business community or we'll get set back.
3. Create that national stem cell bank. Like a federal reserve bank . . . we need to be looking at how this will evolve and be prepared to organize so that there are standards and procedures everyone can depend on. In the USA we're in a fits and starts mode . . but we should follow the UK/Canada model.
2. Police ourselves. We live in an impoverished (but blissful) world of zero regulation together with very little money. What we don't want is to get the big regulation and the same crumbs of cash. It's our job to be vigilant about bad science and to call out the snake oil sellers. Makes the comparison to wall street: police yourself or you're going to get policed.
1. Achieve a clinical breakthrough. In the 70's IVF was the work of the devil. Joy Louise Brown changed that conversation. The world's best researchers are in this room. Amazing things are about to happen and everybody here knows it; we're going to bend the river.
MMMM, Danny was right. he's a good speaker.
Panel: International perspectives: Collaboration Opportunities
Moderator is Doug Sipp of the RIKEN center for developmental biology . . . talking about the difficulty of regulatory issues from country to country.
Northern Europe does not like primate research; there's a saying that in Scandinavia it's easier to do human trials than primate trials.
The US has famously lost ground due to the president's position.
Globally there are 3 large organizations: ISCF, ISSCR, and, oy . . . lost it, sorry
In the USA there's the Alliance for Stem Cell Research
In Europe, a similar organization
In Asia, another one whose acronym is SNAP
It's not a competitive effort, it's a collaborative one.
Introduces Dr. Stephen Minger from King's College, London. He's not a Brit, though, but an American transplant. There's a strong collaborative effort there, existing in a very tight-knit community of people working with stem cells.
He sits on a board that will be the regulatory body that makes policy for how stem cells will be used. Also has a role in creating collaboration between UK scientists and Chinese scientists.
Says that in China stem cell biology is surging ahead very quickly; have the best equipped labs in the world at this moment because the gov't has funded them very well. Chinese scientists who were trained at US universities are heading back in droves because they can work in a much more friendly environment. I tell my students, "Don't look west, look east."
Next is Marilyn Robertson of the Scottish Stem Cell Network.
Wants to talk about the role of networks . . the stakeholder group is quite diverse, including all the networks mentioned just above . . . there are
scientists, clinicians and health professionals
patients and caregivers, patient charities and advocacy groups
lawyers and ethicists, policy makers and government
industry, subject matter experts, biotech and big pharma, suppliers and maunfacturers
investors, public and private, banks and insurance
the general public
The job of networks is to bring all these groups together.
They do a lot of work:
Provide links between stakeholder groups
Run training events for members
Support enterprise development
Fund research
Lobby policy makers
Provide information to inform the public debate
Support teachers in schools and colleges
Act as the contact for international researchers
She's talking about the Scottish Stem Cell Network
You can check out their agenda at the link, but one important thing she just said is that in a couple of weeks they'll be hosting a group of immunologists to get them involved now in figuring out how to make therapies that don't require immunosuppression to keep transplants from being rejected.
Glyn Stacey, director of the UK Stem Cell Bank
"I'm one of those scientists who feels very scared if I don't have a powerpoint presentation . . . apologize for that . . . "
So, what is a stem cell bank? It's a collection of stem cells housed in tubes and kept in a state of suspended animation. It allows scientists to strictly keep and control quality.
You'd want to know as a patient that the cells you're going to get are exactly the same as the ones the last guy got . . . that's what the banks are about.
People have to understand that this is a new and dynamic field with techniques yet to be optimised . . . it's going to take steady nerves to persevere.
So, he's describing the process they followed to get the banking underway. Started with forming a steering committee that forbade them from creating a conflict of interest by doing research on their cells or from forming any commercial relationships. Next was to create what he calls a Human Tissues Authority to control the supply for research and clinic, maintain a quality forum, establish a a patient depositary, and give advice to big pharma and others.
Challenges
1. the cells they're working with were altruistically donated, which will eventually be turned to commercial products . . . not clear how this should be handled
2. there are complex ethical issues which are changing as time passes as well as across international boundaries
3. the scientific development is very fast and requires heavy investment to keep up and assure quality
4. there is a broadening user community which means we're looking at new disciplines, and that implies a need for training
5. progress to therapy will involve problems and failures, must plan ahead for how to deal with that
He has a slide up that shows the extent of international coordination, talking fast now, but I think he just said that they jointly published a very rich data set available to all the members . . .
Last speaker is Derek Hei, who is a prof at UW Madison and a bioengineer.
Talking about how to evolve the stem cell banks; he's leading the effort in the US
they want divers banks of human cell lines, they want genetically modified cell lines, they want disease mdels developed from iPS cells
they want to do technology transfer . . cell banking practices including culture freezing and testing methods
(editorial comment . . . may just be me, but I'm getting a very strong feeling that many of the presenters here are aware that they're climbing on top of a very big wave and are working very hard to keep their balance and ride it safely to shore.)
We have 21 cell lines available through the NIH registry. One of the challenges faced as the cells are shared worldwide with other investigators is how to share the information about them.
So they made a website (the national stem cell bank) that gives every detail of what has happened w/respect to those lines, giving researchers clear history about what they're getting.
Support for translational researchers is a major goal of the national stem cell bank . . . will want to create a GMP grade collection of stem cells so that when investigators want to take their therapies to trial they have a ready source of cells to do so.
Questions?
Why is there not more interest from the insurance industry in supporting hESc?
Robertson says that we don't really know, but of course we'd welcome it . . . possibly they don't understand what the benefits might be.
Hei: Wants to clarify that the cell banks will be undifferentiated cells . . . there's a lot of work to be done to nudge them into specific cell types, and it's likely that insurance companies won't get on board until after a lot more work has been done.
Question: What about contamination? What about manufacturing to most common HLA lines?
Hei: We've been concerned all along about our lines picking up pathogens that we've been unable to detect, which is why we continue to test them as time passes. From our point of view it's important to get cells into researchers' hands right now.
Stacey: We're going to have to move forward . . . basically he's saying that even though it will be very expensive and issues will continue to be complex, we have to make choices.
Question: The british are the only ones that now allow putting a human nucleus into an animal egg . . . where is that going?
Minger: In our lab we're going to go forward with it, because there is still so much to be learned . . .makes a comparison between this and not giving up on hESc just because we have iPS.
Northern Europe does not like primate research; there's a saying that in Scandinavia it's easier to do human trials than primate trials.
The US has famously lost ground due to the president's position.
Globally there are 3 large organizations: ISCF, ISSCR, and, oy . . . lost it, sorry
In the USA there's the Alliance for Stem Cell Research
In Europe, a similar organization
In Asia, another one whose acronym is SNAP
It's not a competitive effort, it's a collaborative one.
Introduces Dr. Stephen Minger from King's College, London. He's not a Brit, though, but an American transplant. There's a strong collaborative effort there, existing in a very tight-knit community of people working with stem cells.
He sits on a board that will be the regulatory body that makes policy for how stem cells will be used. Also has a role in creating collaboration between UK scientists and Chinese scientists.
Says that in China stem cell biology is surging ahead very quickly; have the best equipped labs in the world at this moment because the gov't has funded them very well. Chinese scientists who were trained at US universities are heading back in droves because they can work in a much more friendly environment. I tell my students, "Don't look west, look east."
Next is Marilyn Robertson of the Scottish Stem Cell Network.
Wants to talk about the role of networks . . the stakeholder group is quite diverse, including all the networks mentioned just above . . . there are
scientists, clinicians and health professionals
patients and caregivers, patient charities and advocacy groups
lawyers and ethicists, policy makers and government
industry, subject matter experts, biotech and big pharma, suppliers and maunfacturers
investors, public and private, banks and insurance
the general public
The job of networks is to bring all these groups together.
They do a lot of work:
Provide links between stakeholder groups
Run training events for members
Support enterprise development
Fund research
Lobby policy makers
Provide information to inform the public debate
Support teachers in schools and colleges
Act as the contact for international researchers
She's talking about the Scottish Stem Cell Network
You can check out their agenda at the link, but one important thing she just said is that in a couple of weeks they'll be hosting a group of immunologists to get them involved now in figuring out how to make therapies that don't require immunosuppression to keep transplants from being rejected.
Glyn Stacey, director of the UK Stem Cell Bank
"I'm one of those scientists who feels very scared if I don't have a powerpoint presentation . . . apologize for that . . . "
So, what is a stem cell bank? It's a collection of stem cells housed in tubes and kept in a state of suspended animation. It allows scientists to strictly keep and control quality.
You'd want to know as a patient that the cells you're going to get are exactly the same as the ones the last guy got . . . that's what the banks are about.
People have to understand that this is a new and dynamic field with techniques yet to be optimised . . . it's going to take steady nerves to persevere.
So, he's describing the process they followed to get the banking underway. Started with forming a steering committee that forbade them from creating a conflict of interest by doing research on their cells or from forming any commercial relationships. Next was to create what he calls a Human Tissues Authority to control the supply for research and clinic, maintain a quality forum, establish a a patient depositary, and give advice to big pharma and others.
Challenges
1. the cells they're working with were altruistically donated, which will eventually be turned to commercial products . . . not clear how this should be handled
2. there are complex ethical issues which are changing as time passes as well as across international boundaries
3. the scientific development is very fast and requires heavy investment to keep up and assure quality
4. there is a broadening user community which means we're looking at new disciplines, and that implies a need for training
5. progress to therapy will involve problems and failures, must plan ahead for how to deal with that
He has a slide up that shows the extent of international coordination, talking fast now, but I think he just said that they jointly published a very rich data set available to all the members . . .
Last speaker is Derek Hei, who is a prof at UW Madison and a bioengineer.
Talking about how to evolve the stem cell banks; he's leading the effort in the US
they want divers banks of human cell lines, they want genetically modified cell lines, they want disease mdels developed from iPS cells
they want to do technology transfer . . cell banking practices including culture freezing and testing methods
(editorial comment . . . may just be me, but I'm getting a very strong feeling that many of the presenters here are aware that they're climbing on top of a very big wave and are working very hard to keep their balance and ride it safely to shore.)
We have 21 cell lines available through the NIH registry. One of the challenges faced as the cells are shared worldwide with other investigators is how to share the information about them.
So they made a website (the national stem cell bank) that gives every detail of what has happened w/respect to those lines, giving researchers clear history about what they're getting.
Support for translational researchers is a major goal of the national stem cell bank . . . will want to create a GMP grade collection of stem cells so that when investigators want to take their therapies to trial they have a ready source of cells to do so.
Questions?
Why is there not more interest from the insurance industry in supporting hESc?
Robertson says that we don't really know, but of course we'd welcome it . . . possibly they don't understand what the benefits might be.
Hei: Wants to clarify that the cell banks will be undifferentiated cells . . . there's a lot of work to be done to nudge them into specific cell types, and it's likely that insurance companies won't get on board until after a lot more work has been done.
Question: What about contamination? What about manufacturing to most common HLA lines?
Hei: We've been concerned all along about our lines picking up pathogens that we've been unable to detect, which is why we continue to test them as time passes. From our point of view it's important to get cells into researchers' hands right now.
Stacey: We're going to have to move forward . . . basically he's saying that even though it will be very expensive and issues will continue to be complex, we have to make choices.
Question: The british are the only ones that now allow putting a human nucleus into an animal egg . . . where is that going?
Minger: In our lab we're going to go forward with it, because there is still so much to be learned . . .makes a comparison between this and not giving up on hESc just because we have iPS.
Jamie Thomson and Panel: iPS Cells: How they change everything, and nothing
Clive Svendsen is moderating; he introduces Jamie Thomson.
I'm wowed.
Thomson: in the early embryo there are cells that don't know what they want to be . . . if you take these cells out of an intact embryo, they will not become anything but more of themselves.
I got into this deal in the early 90's. I was trained as a mouse embryologist; he shows a picture of a mouse embryo next to a human embryo--the point being that they're very different.
So, moved to primates, including our close relative the rhesus monkey.
If you take a skin cell and put it into a growth culture, it will divide a few times and then stop. A cancer cell will just keep going. An embryonic stem cell will also just keep going.
The iPS cells would NOT have been derived successfully if we had not had 10 years of working with embryonic stem cells.
We know now how to take a cell from your body and reprogram it to become like an embryonic stem cell . . . it will have that magic property of being able to keep reproducing itself forever, and of being able to turn into any other kind of cell.
Describing how they pulled this off in the lab, giving credit to his post-docs, talking too fast (and kind of mumbling) for me to write down how exactly it worked. Check the webcast, because the audio there may be better than what we're getting in this room.
Okay, he just said something important--there's less variation in the reprogammed cells than there was in the cells they originally made back in 1998.
3 things that make iPS matter:
1. They provide a tissue culture model for understanding processes at the cellular level
2. They will provide a purified population of specific differentiated cells for drug discovery and testing
3. Could provide a source for therapies eventually
He believes that the central nervous system will not be repaired by cell transplantation-- but that the cells will make it possible to understand how to make therapies that don't depend on transplantation.
He thinks it may be possible to make blood cells cheaply enough to use them for transfusion. He thinks that they'll be able to make cells that will help leukemia patients within the next 5 years.
So, what's the agenda?
Make the cell types that interest us most
Address safety concerns, including resolving question of cancer
Address immune rejection -- using iPS would work, but it's expensive
Preventing the process that killed the cells in the first place
Integration in a physiologically useful form
How to end this little talk on a positive note? I realize that I sound pessimistic here, but I'm really not. It's just that we have so much work to do and we need to roll up our sleeves.
Ends by saying that it would be a very bad idea to stop working with embryonic stem cells while gearing up to work iPS cells.
Question: From the university of Sydney . . .
How long until broad therapeutic application might be possible? We're not good at predicing timelines. Sometimes we get things done in 5 years that we thought would take 25. In 1998, I said that it would take 10 years to get to therapeutic applications . . .that was obviously wrong. My expectation is that there will be setbacks and challenges. We're a very risk-averse culture, which makes things go more slowly.
Up comes Hideyuki Okano from Keio University in Japan
Talking about iPS historical development; they did it with mice in Japan in 2006, and then along with Thomson in 2007
Japan established a system of comprehensive strategies to speed this research in their country. They want to be the world center . . . they're mostly interested in regeneration of the brain and central nervous system.
Kyoto University owns the iPS cell patent to make the cells and
Regeneration of the injured spinal cord; here's a little news about a new study.
if 5 - 10% of axons can be regenerated, there will be a lot of functional return-- new that
Take skin cells, reprogram them to become iPS cells, differentiate them into nerve cells, and then put the nerve cels back into the patient. That's the agenda and the goal.
Talking now about differentiating cells into neural cells, including
They did a mouse 9 days post and transplanted neural stem ccells andtested for functional recovery.
Which they got, though the slide is too dim to see the actual numbers.
Tested for teratomas 6 months post transplant, and found no evidence of them at all.
Agenda:
Make iPS cells (3 months) Induce to grow into neural progenitors, (4 months) Do a safety check (at lest a year)
They will do a primate study.
New speaker: Amy Comstock Rock, from CAMR, the coalition of advancement for medical research
iPS cells . . . how they change everything and nothing
She's got a picture of George Bush up with the countdown clock under his face. 120 days to go. People clap and laugh.
She says that one side benefit of Bush's policy has been huge growth in individual states.
When the iPS breakthroughs were announced in November, CAMR was very fearful that we would lose congressional support for hESc-- not because the members individually felt troubled about hESc, but because they represent districts where constituents are vocal and passionate opponents.
During the August political conventions, the case was made to leave hESc behind and move on "along with science" -- Jamie Thomson is quoted out of context to make that case.
Picture up of McCain and Obama . . . McCain has voted twice for funding of hESc research, but has been making noise about how "one day" there won't be a need for it. The problem is that iPS research will be misused for political purposes. No, really??!!
Obama has promised to issue an immediate executive order changing the no funding policy upon taking office.
She encourges all of us to be very active in advocating for continuing support for hESc research. We can't afford to be quiet about this; CAMR is doing background work drafting white papers for congressmembers, and is depending on us out in the world to carry the message.
Svendsen asks Jamie Thomson to comment on the patent issue; Japan has filed for iPS patents in the USA . . . Thomson says that he thinks it's not going to slow anything down.
Break time!
I'm wowed.
Thomson: in the early embryo there are cells that don't know what they want to be . . . if you take these cells out of an intact embryo, they will not become anything but more of themselves.
I got into this deal in the early 90's. I was trained as a mouse embryologist; he shows a picture of a mouse embryo next to a human embryo--the point being that they're very different.
So, moved to primates, including our close relative the rhesus monkey.
If you take a skin cell and put it into a growth culture, it will divide a few times and then stop. A cancer cell will just keep going. An embryonic stem cell will also just keep going.
The iPS cells would NOT have been derived successfully if we had not had 10 years of working with embryonic stem cells.
We know now how to take a cell from your body and reprogram it to become like an embryonic stem cell . . . it will have that magic property of being able to keep reproducing itself forever, and of being able to turn into any other kind of cell.
Describing how they pulled this off in the lab, giving credit to his post-docs, talking too fast (and kind of mumbling) for me to write down how exactly it worked. Check the webcast, because the audio there may be better than what we're getting in this room.
Okay, he just said something important--there's less variation in the reprogammed cells than there was in the cells they originally made back in 1998.
3 things that make iPS matter:
1. They provide a tissue culture model for understanding processes at the cellular level
2. They will provide a purified population of specific differentiated cells for drug discovery and testing
3. Could provide a source for therapies eventually
He believes that the central nervous system will not be repaired by cell transplantation-- but that the cells will make it possible to understand how to make therapies that don't depend on transplantation.
He thinks it may be possible to make blood cells cheaply enough to use them for transfusion. He thinks that they'll be able to make cells that will help leukemia patients within the next 5 years.
So, what's the agenda?
Make the cell types that interest us most
Address safety concerns, including resolving question of cancer
Address immune rejection -- using iPS would work, but it's expensive
Preventing the process that killed the cells in the first place
Integration in a physiologically useful form
How to end this little talk on a positive note? I realize that I sound pessimistic here, but I'm really not. It's just that we have so much work to do and we need to roll up our sleeves.
Ends by saying that it would be a very bad idea to stop working with embryonic stem cells while gearing up to work iPS cells.
Question: From the university of Sydney . . .
How long until broad therapeutic application might be possible? We're not good at predicing timelines. Sometimes we get things done in 5 years that we thought would take 25. In 1998, I said that it would take 10 years to get to therapeutic applications . . .that was obviously wrong. My expectation is that there will be setbacks and challenges. We're a very risk-averse culture, which makes things go more slowly.
Up comes Hideyuki Okano from Keio University in Japan
Talking about iPS historical development; they did it with mice in Japan in 2006, and then along with Thomson in 2007
Japan established a system of comprehensive strategies to speed this research in their country. They want to be the world center . . . they're mostly interested in regeneration of the brain and central nervous system.
Kyoto University owns the iPS cell patent to make the cells and
Regeneration of the injured spinal cord; here's a little news about a new study.
if 5 - 10% of axons can be regenerated, there will be a lot of functional return-- new that
Take skin cells, reprogram them to become iPS cells, differentiate them into nerve cells, and then put the nerve cels back into the patient. That's the agenda and the goal.
Talking now about differentiating cells into neural cells, including
They did a mouse 9 days post and transplanted neural stem ccells andtested for functional recovery.
Which they got, though the slide is too dim to see the actual numbers.
Tested for teratomas 6 months post transplant, and found no evidence of them at all.
Agenda:
Make iPS cells (3 months) Induce to grow into neural progenitors, (4 months) Do a safety check (at lest a year)
They will do a primate study.
New speaker: Amy Comstock Rock, from CAMR, the coalition of advancement for medical research
iPS cells . . . how they change everything and nothing
She's got a picture of George Bush up with the countdown clock under his face. 120 days to go. People clap and laugh.
She says that one side benefit of Bush's policy has been huge growth in individual states.
When the iPS breakthroughs were announced in November, CAMR was very fearful that we would lose congressional support for hESc-- not because the members individually felt troubled about hESc, but because they represent districts where constituents are vocal and passionate opponents.
During the August political conventions, the case was made to leave hESc behind and move on "along with science" -- Jamie Thomson is quoted out of context to make that case.
Picture up of McCain and Obama . . . McCain has voted twice for funding of hESc research, but has been making noise about how "one day" there won't be a need for it. The problem is that iPS research will be misused for political purposes. No, really??!!
Obama has promised to issue an immediate executive order changing the no funding policy upon taking office.
She encourges all of us to be very active in advocating for continuing support for hESc research. We can't afford to be quiet about this; CAMR is doing background work drafting white papers for congressmembers, and is depending on us out in the world to carry the message.
Svendsen asks Jamie Thomson to comment on the patent issue; Japan has filed for iPS patents in the USA . . . Thomson says that he thinks it's not going to slow anything down.
Break time!
Panel 1: How stem cell research is transforming health care
Timothy Kamp is moderating, introduces the panel:
Lawrence Goldstein UC San Diego
Alan Trounson, president of the almighty CIRM -- California Institute of Regenerative Medicine
John Wagner, University of Minnesota
All strong advocates and pros in the field.
First up: Alan Trounson, to give a brief description of what embryonic stem cells are; Trounson is another Brit . . . he thinks everybody here knows that a stem cell can reproduce itself; that's what makes it special. Some of them are also pluripotent, meaning they can become any other kind--those are embryonic stem cells.
He says that community education and especially managing expectation are very important . . . we recognize that there are certain psychological and religious barriers that have to be addressed.
Patient advocates have an important role -- able to provide an immediate realtime case . . . partnerships with businesses are where the "rubber meets the road"--without business we're not going to get to the clinic, ever.
Clinicians must be brought into the process very early.
In California we're building capacity. The first 20 months in the life of the institute has been about that. We've funded 12 new institutes,
Have spent $383M in institutions, and an additional $300M in support . . . building the 12 new institutes must be done within 2 years.
We're building a pipeline.
We've started making grants to scientists with the very best ideas; the process is that 15 scientists from outside CA review and recommend, after which there's a 29-member public committee.
We're building new stem cell lines, both embryonic and iPS, have many new faculty, are buying equipment and technology.
Puts up a pie chart showing how the money from the first 68 grants was spent . . . neurology looks like it got about a third of them. They're also working on cancer.
World wide funding for regenerative medicine: over the next ten years will be around $14 billion world wide. (Amazing that CA will be spending more than 20% of that all by itself.)
His point is that meetings like this are hugely important because collaboration now sets things up for speed and efficiency as time passes. CIRM has produced a "memo of understanding"
Next is Lawrence Goldstein from UC San Diego. Says that cells are something like the interstate highway system. Some of the nerve cells in your body are over a yard long . . .
The basic biology of how materials are moved inside cells for those long distances has led to new ideas about what might be going on in things like alzheimers.
We've almost worn out what we can learn from animals . . that end of the road has led us to bring pluripotent cells into our research to try to understand how ALS and alzheimer's disease work.
In his lab, it looks like this:
for ALS, what happens is that the neurons in the peripheral nervous system that make your muscles contract -- the motor neurons-- die. So you'd think that the top priority would be to replace those dying motor neurons . . .however, in practice that's "devilishly difficult" to pull off. It's like trying to re-wire a hugely complicated system.
so, another idea . . . from a mouse model (mice get a version of ALS) they've learned that there are neighboring cells that can rescue the motor neurons from dying. In labs you often see cells living in dishes in little clumps; but this is not how cells live really.
They live surrounded by other kinds of cells, and the effort now is to use embryonic stem cells to sort of create a friendly neighborhood . . . they're trying this now in rats.
What about alzheimers . . . the hellish condition that so many of us are slated to get. There are basically no treatments, because we fundamentally do not understand what's going wrong in those nerve cells, in spite of animal studies.
Humans are not just big mice, eh?
His lab is now trying to use human embryonic stem cells to create "platforms" that can cause rare forms of inherited alzheimers. Then they take skin cells from humans who have alzheimers. tehy want to capture the basic architecture of the disease inside cells. Then they look at those cells and try to nail down the points of difference between the normal ones, the genetic- alzheimers, and the random alzheimers.
The point of this is that they're not trying to take stem cells and use them to make therapies. They're using them to look very finely at what's going on.
John Wagner speaks. I'm here as a clinician, an advocate for my patients. There are hundreds of thousands of people waiting for the therapies you're working on.
Clinic and lab must be inter-related, must be learning from one another. What is a realistic expectation? The first clinical trials must be about safety. (Phase 1) When they first did bone marrow transplants, they were surprised by results, but did not give up.
We have to figure out how to modulate the immune response so that people getting transplants don't reject the treatment.
Need to minimize the possibility of genetic disease transmission, something not much discussed.
Have to be concerned with the risk of disease transmission.
None of this should stop us from moving forward. What we do today will be different from what we do in two years, and that's good.
As scientists, we're always thinking about the next step, the next evolutionary step in designing trials . . . but what happens when we find a cure?
How do we prepare for a cure for, say, diabetes or sci? Are we prepared for the economic impact of that discovery? How will patients pay for it? Who will have access?
This meeting is important because it gets everybody to engage in the longterm thinking.
Kamp is back up, asking each panelist to name one major roadblock in moving forward.
Trounson: The delivery of cells to patients that are not their own, immune response is a potentially huge problem. We need to engage the immunologists and come up with strategies.
Goldstein: The answer to ALL questions is money--it's just the amount that varies, depending on the question. Funding of the actual work is what matters. In the past few years we've experienced a disastrous decline in funding for research in the USA. We invest a tiny fraction of our national resources and get giant returns 5, 10, 20 years out. The rate of return on public funding for research has historically been 25%.
Not bad. I'd make that spend.
Wagner: Being able to track the cells; once we inject the cells we have to be able to know where they go. Also animal models need to get better.
Goldstein again: Public understanding of science and of what we're trying to do. It's so important that we all learn to distinguish good science form bad, reputable research from the other kind. We need to stop this silly proliferation of stories that other countries are delivering cures. People should not be allowed to deliver snake oil without challenge.
Questions?
There's going to be a press conference today at 1 pm. Patient advocate speaking, says that we should show up.
Question for Larry Goldstein: if we're studying a disease in humans that takes 50 or 60 years to develop, how can we possibly figure it out in a lab? He says it turns out that when we've examined the genetic changes that cause alzheimers in people, we see short term changes that we believe are on the pathway to developing the disease. In fruit flys, we can see changes in a few days. Some of the changes we can see in a few weeks or months are part of what develops over time. Also, by the time adults show symptoms of the disease, they've had it for a very long time--because the brain has redundancy and covers for itself as long as it can. So it may not be that we need 50-60 year models.
Question: About that 25% return on investment . . . what's that based on? Goldstein answers: that number comes from a bi-partisan, bi-cameral commission report and it's based on the aggregate return. You're not looking at any single company or attempt . . . it's more broad than that; he can point us at the report if people want to read it.
Trounson: Our ROI calculations at CIRM are based on success in a certain group of specific diseases and conditions.
Question: For Trounson, we in New York state are in awe of what's happened in CA . . our governor has just set aside $600 million to be spent on stem cell research. What's the most productive way to move forward?
Trounson: The paradigm of the NIH model is probably wrong . . how can we help one another? We're going to be learning from one another. The intellectual property program needs to be standardized, because it's often there that people have differences of opinion.
Goldstein: Nationwide there is a very diverse legislative and regulatory framework, which makes it difficult to collaborate.
Phew. Next panel time.
Lawrence Goldstein UC San Diego
Alan Trounson, president of the almighty CIRM -- California Institute of Regenerative Medicine
John Wagner, University of Minnesota
All strong advocates and pros in the field.
First up: Alan Trounson, to give a brief description of what embryonic stem cells are; Trounson is another Brit . . . he thinks everybody here knows that a stem cell can reproduce itself; that's what makes it special. Some of them are also pluripotent, meaning they can become any other kind--those are embryonic stem cells.
He says that community education and especially managing expectation are very important . . . we recognize that there are certain psychological and religious barriers that have to be addressed.
Patient advocates have an important role -- able to provide an immediate realtime case . . . partnerships with businesses are where the "rubber meets the road"--without business we're not going to get to the clinic, ever.
Clinicians must be brought into the process very early.
In California we're building capacity. The first 20 months in the life of the institute has been about that. We've funded 12 new institutes,
Have spent $383M in institutions, and an additional $300M in support . . . building the 12 new institutes must be done within 2 years.
We're building a pipeline.
We've started making grants to scientists with the very best ideas; the process is that 15 scientists from outside CA review and recommend, after which there's a 29-member public committee.
We're building new stem cell lines, both embryonic and iPS, have many new faculty, are buying equipment and technology.
Puts up a pie chart showing how the money from the first 68 grants was spent . . . neurology looks like it got about a third of them. They're also working on cancer.
World wide funding for regenerative medicine: over the next ten years will be around $14 billion world wide. (Amazing that CA will be spending more than 20% of that all by itself.)
His point is that meetings like this are hugely important because collaboration now sets things up for speed and efficiency as time passes. CIRM has produced a "memo of understanding"
Next is Lawrence Goldstein from UC San Diego. Says that cells are something like the interstate highway system. Some of the nerve cells in your body are over a yard long . . .
The basic biology of how materials are moved inside cells for those long distances has led to new ideas about what might be going on in things like alzheimers.
We've almost worn out what we can learn from animals . . that end of the road has led us to bring pluripotent cells into our research to try to understand how ALS and alzheimer's disease work.
In his lab, it looks like this:
for ALS, what happens is that the neurons in the peripheral nervous system that make your muscles contract -- the motor neurons-- die. So you'd think that the top priority would be to replace those dying motor neurons . . .however, in practice that's "devilishly difficult" to pull off. It's like trying to re-wire a hugely complicated system.
so, another idea . . . from a mouse model (mice get a version of ALS) they've learned that there are neighboring cells that can rescue the motor neurons from dying. In labs you often see cells living in dishes in little clumps; but this is not how cells live really.
They live surrounded by other kinds of cells, and the effort now is to use embryonic stem cells to sort of create a friendly neighborhood . . . they're trying this now in rats.
What about alzheimers . . . the hellish condition that so many of us are slated to get. There are basically no treatments, because we fundamentally do not understand what's going wrong in those nerve cells, in spite of animal studies.
Humans are not just big mice, eh?
His lab is now trying to use human embryonic stem cells to create "platforms" that can cause rare forms of inherited alzheimers. Then they take skin cells from humans who have alzheimers. tehy want to capture the basic architecture of the disease inside cells. Then they look at those cells and try to nail down the points of difference between the normal ones, the genetic- alzheimers, and the random alzheimers.
The point of this is that they're not trying to take stem cells and use them to make therapies. They're using them to look very finely at what's going on.
John Wagner speaks. I'm here as a clinician, an advocate for my patients. There are hundreds of thousands of people waiting for the therapies you're working on.
Clinic and lab must be inter-related, must be learning from one another. What is a realistic expectation? The first clinical trials must be about safety. (Phase 1) When they first did bone marrow transplants, they were surprised by results, but did not give up.
We have to figure out how to modulate the immune response so that people getting transplants don't reject the treatment.
Need to minimize the possibility of genetic disease transmission, something not much discussed.
Have to be concerned with the risk of disease transmission.
None of this should stop us from moving forward. What we do today will be different from what we do in two years, and that's good.
As scientists, we're always thinking about the next step, the next evolutionary step in designing trials . . . but what happens when we find a cure?
How do we prepare for a cure for, say, diabetes or sci? Are we prepared for the economic impact of that discovery? How will patients pay for it? Who will have access?
This meeting is important because it gets everybody to engage in the longterm thinking.
Kamp is back up, asking each panelist to name one major roadblock in moving forward.
Trounson: The delivery of cells to patients that are not their own, immune response is a potentially huge problem. We need to engage the immunologists and come up with strategies.
Goldstein: The answer to ALL questions is money--it's just the amount that varies, depending on the question. Funding of the actual work is what matters. In the past few years we've experienced a disastrous decline in funding for research in the USA. We invest a tiny fraction of our national resources and get giant returns 5, 10, 20 years out. The rate of return on public funding for research has historically been 25%.
Not bad. I'd make that spend.
Wagner: Being able to track the cells; once we inject the cells we have to be able to know where they go. Also animal models need to get better.
Goldstein again: Public understanding of science and of what we're trying to do. It's so important that we all learn to distinguish good science form bad, reputable research from the other kind. We need to stop this silly proliferation of stories that other countries are delivering cures. People should not be allowed to deliver snake oil without challenge.
Questions?
There's going to be a press conference today at 1 pm. Patient advocate speaking, says that we should show up.
Question for Larry Goldstein: if we're studying a disease in humans that takes 50 or 60 years to develop, how can we possibly figure it out in a lab? He says it turns out that when we've examined the genetic changes that cause alzheimers in people, we see short term changes that we believe are on the pathway to developing the disease. In fruit flys, we can see changes in a few days. Some of the changes we can see in a few weeks or months are part of what develops over time. Also, by the time adults show symptoms of the disease, they've had it for a very long time--because the brain has redundancy and covers for itself as long as it can. So it may not be that we need 50-60 year models.
Question: About that 25% return on investment . . . what's that based on? Goldstein answers: that number comes from a bi-partisan, bi-cameral commission report and it's based on the aggregate return. You're not looking at any single company or attempt . . . it's more broad than that; he can point us at the report if people want to read it.
Trounson: Our ROI calculations at CIRM are based on success in a certain group of specific diseases and conditions.
Question: For Trounson, we in New York state are in awe of what's happened in CA . . our governor has just set aside $600 million to be spent on stem cell research. What's the most productive way to move forward?
Trounson: The paradigm of the NIH model is probably wrong . . how can we help one another? We're going to be learning from one another. The intellectual property program needs to be standardized, because it's often there that people have differences of opinion.
Goldstein: Nationwide there is a very diverse legislative and regulatory framework, which makes it difficult to collaborate.
Phew. Next panel time.
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