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.