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Businesses, Patients Share Excitement at World Stem Cell Summit

A cluster of neural cells were derived from human embryonic stem cells SOURCE: Su-Chun Zhang, University of Wisconsin-Madison The face of stem cell research is changing as research moves towards the clinic and commercialization, and as patients demand access to experimental treatments. Above: a cluster of neural cells derived from human embryonic stem cells.

Weiss’s Notebook

CAP Senior Fellow Rick Weiss

CAP Senior Fellow Rick Weiss covered science and medicine for The Washington Post for 15 years, and now he brings his investigative eye to science policy. From cloning and stem cells to agricultural biotechnology and nanotechnology, Weiss examines the issues at the intersection of cutting edge research and public policy.

I was in Madison, Wisconsin, last week at the 2008 “World Stem Cell Summit,” a gathering of hundreds of scientists, pharmaceutical company reps, patient advocates and policy folks united in their evidence-based faith that stem cells are going to revolutionize biology and medicine.

I’ve been to a fair number of stem cell meetings over the years, but this one was remarkable in several respects. For one, it was held in Madison, where Jamie Thomson and his colleagues first discovered and isolated human embryonic stem cells ten years ago this fall. So it was something of a decadal celebration and homecoming for the Wisconsin team. Go badgers!

For another, there was a palpable sense that the United States is fast approaching the end of the Bush administration, reviled by advocates for the restrictions it has maintained on embryonic stem cell research funding. In short, there was a lot of “good riddance” buzz in the air and equal measures of conjecture and concern about what stem cell policies will emerge in the next administration.

Third, I have never seen so many people in wheel chairs at a stem cell meeting, an indication, it seemed, that patients are getting more uppity about gaining access to the first experimental therapies that may soon be tested in clinical trials.

And finally, most striking and no doubt related to that third point, there was an extraordinary and, in my experience, unprecedented focus on the commercialization of stem cells for research and therapy—a sign that stem cell science has achieved a certain level of maturity despite the endless ethical and political debates that have hobbled progress since the field’s inception.

Jamie Thomson was the star attraction, closely watched not only because of his stature as the founder of the field but because his lab was one of two that recently transformed adult human skin cells directly into cells that seem virtually identical to human embryonic stem cells—so-called iPS cells. That has suggested the scientifically and politically tantalizing possibility that embryonic stem cells themselves—controversial in some circles because their derivation requires that five-day-old embryos be destroyed—may before long become irrelevant.

But Thomson said it makes no sense to drop studies on embryonic stem cells on the presumption that iPS cells will satisfy all of scientists’ and patients’ needs. For now, he said, both approaches deserve full-bore exploration.

Their “Where do I sign up?” spirit was reminiscent of the early days of the AIDS epidemic, when angry patients ultimately forced the FDA to give them access to AZT and other experimental drugs.

“I personally believe the future is in these iPS cells, but the future isn’t quite here today,” Thomson said. “If they’re identical to embryonic stem cells, then everything we’re doing with embryonic stem cells today will apply directly to these new stem cell lines. If they’re fatally flawed in some way, not clinically relevant, then everything we’re doing with embryonic stem cells will apply to embryonic stem cells and we won’t waste any time.”

His comments were in sync with the conclusions of a National Academy of Sciences panel that earlier this month declared: “It is far from clear at this point which types cell types will prove to be the most useful for regenerative medicine, and it is likely that each will have some utility.”

Former Wisconsin governor Tommy Thompson, who served as secretary of Health and Human Services in the early years of the Bush administration, was there as well, telling a never-told-before and perhaps only partially believable story that it was he who singlehandedly convinced Bush to allow at least some funding for the controversial work.

But to my ears, the most interesting talks—whether inside the meeting rooms or around the coffee urns and cupcakes during breaks—were about how and when the long-suffering research specialty will at last lose its virginity and get Food and Drug Administration approval for testing in people. And how and when, in the aftermath of those clinical trials, the field will graduate to a full-blown, commercially viable, and medically useful subspecialty of medicine.

None of this is going to happen tomorrow. But the seriousness of the prospect was ineluctable.

Among the strongest evidence for real pending change was a talk by John McNeish of Pfizer, the pharmaceutical giant, who revealed details about that company’s newly founded regenerative medicine unit, which will focus largely on stem cells. Although the company plans to use stem cells initially as laboratory tools to test the safety and efficacy of experimental drugs, it and a smattering of other big players, including GlaxoSmithKline, are starting to see an economic future in stem cell-based therapies. Many at the meeting spoke unabashedly about the need to move on to human testing.

“We’ve almost worn out what we can do with animals in disease biology such as fruit flies and mice,” said Lawrence Goldstein, a stem cell researcher at the University of California, San Diego. “The problem at the end of the day is that humans are not just big mice.”

Geron Corp. of Menlo Park, California, has been saying for years that it will soon start what would be the first human studies of an embryonic stem cell therapy—one for people partially paralyzed because of spinal cord injuries. Approval has been put off repeatedly by the FDA for reasons that have not been released. Several experts at the meeting said they suspect two main issues are in play: A desire by regulators to be as certain as possible that Geron’s cells will not, even in rare instances, cause tumor formation (a fear that can never be 100 percent allayed but which has led to demands that the company test the treatment in an ever-growing number of rodents); and perhaps a preference among regulators to launch the first stem cell clinical trial not in patients who, despite being paralyzed, can be expected to live long and reasonably productive lives, but in patients with terminal conditions—just in case the treatment proves worse than the disease.

Comments from some of the attendees in wheelchairs made plain that they had a different perspective. Their “Where do I sign up?” spirit was reminiscent of the early days of the AIDS epidemic, when angry patients ultimately forced the FDA to give them access to AZT and other experimental drugs. That chapter of medical history tested the U.S. regulatory system—and the nation’s conscience—as officials struggled to find a balance between their conventional, paternalistic protectionism and the patients’ hyper-libertarian view that people have the right to risk their lives as guinea pigs no matter how slim the chances of success or how high the chances they will be harmed.

Already, a growing number of patients suffering from a wide range of ailments are traveling to India, China and other less-regulated countries to throw the dice with experimental stem cell therapies. As evidence of potential efficacy amasses here at home, it will be a challenge to thread that therapeutic and regulatory needle and ethically speed access to those patients most likely to benefit—or at least to those most willing to take the gamble.

Rick Weiss is a Senior Fellow at the Center for American Progress and Science Progress.

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