The Niche

Stem cell trials: balancing hope and risk

In Madison, Wisconsin, the former US secretary of health bellows: “Some inner hope!” Tommy Thompson yells at the crowd—a room full of stem cell research advocates—preaching to the converted that embryonic stem cells give disease sufferers a reason to believe in a better life.

This is an unusual conference: patients and patient representatives plus industry executives, politically active scientists, lobbyists, ethicists, policy experts and more are here.

Although the conference organizer stresses that the meeting is not partisan, the crowd and some speakers are vocally anti-Bush because of his refusal to fund human embryonic stem cell research. Thompson tells the crowd that the policies could easily have been more restrictive and describes how Bush called in pro-research Thompson to debate anti-research Karl Rove on the issue. (The president munched a peanut butter sandwich during the impromptu but lengthy discussion; a few days after that he announced his compromise position to fund lines created before August 2001.) After relating the story, Thompson warns them not to be too harsh on Sarah Palin; they might need to work with her.

Later that afternoon, Alta Charo, a professor of law and bioethics at the University of Wisconsin–Madison, says debates over the moral status of five-day-old embryos are simple compared to what will come when cell-based products enter full-fledged trials for spinal cord injury and the like. She delineates problems with cell therapies, going from the difficult animal studies (monitoring cell transplants for months in infection-prone rodents) to the “polarizing debate around class and access to health care” that will ensue if an expensive cell therapy hits the market.

In between there is the hurdle of conducting clinical trials. When that happens, she predicts, the number of patients hoping to participate in trials far exceeds the number that can be enrolled.

Indeed, one of the scientists here told me privately that the constant invocation of “the ‘C word’” (cure) made him uncomfortable. Even if cell-based therapies help, most are a long way from being tested, and they are more likely to improve a patient’s condition than to reverse it.

The exuberant attitude worries scientists, but it’s part of US culture, says Charo. The American mindset is optimistic and forward thinking. That means US patients often assume that ‘the new thing’ must be better than the current standard. “Without controlled trials, we can be sorely misled. People can undergo terrible ordeals for something that might be worthless.”

This is something that Wise Young thinks about nearly every waking moment. The neuroscientist from Rutgers University, in New Jersey, has courted controversy by reaching out to help organize stem cell networks in China and elsewhere, urging commercial practitioners to disclose their procedures (some refuse). Patients would go anyway, he says, despite the high cost and risk. “No matter what we do and what we say, medical tourism will occur until we start providing something that will satisfy the demand.”

Young calls for greater willingness to do clinical trials in the United States and for scientists to talk with and monitor patients before and after they go abroad for poorly documented procedures. Anecdotes are very hard to assess, particularly for spinal cord injury. Over time, patients’ conditions do tend to improve somewhat, and performance can vary significantly from month to month.

A coordinated group of scientists who would assess patients at multiple time points before and after they undergo procedures could provide invaluable information, even in the absence of a clinical trial, suggests Graham Creasy, chief of Spinal Cord Injury Service at the VA Palo Alto Health Care System in California. Indeed, this has been done on a smaller scale already, one for a clinic in Portugal, another for a clinic in China. An evaluation showed no significant improvement in patients’ condition. But there are problems with the approach. Medical experts in the United States worry that even this kind of inquiry could legitimize and thus encourage potentially harmful approaches that disqualify patients from future trials in the United States. Besides, what incentive would the medical tourists have to participate in these inquiries?

BTW: The International Campaign for Cures of Spinal Cord Injury Paralysis provides information for the general public on participation in clinical trials. (This is different from paying money to a far-off clinic for undocumented procedures, but similar questions apply.)

The International Society for Stem Cell Research has proposed draft guidelines for the clinical translation of stem cells and is seeking comments until the beginning of October.

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