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.
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