The Niche

Stanford conference: patient-heroes of clinical research

“When you think you have a policy, you’re too late.” That’s the opinion of Pearl O’Rourke, who directs human research affairs at Partner Healthcare Systems She was referring to the pace of research in the stem cell field and the need to fashion policies to protect subjects. Embryonic stem cell research oversight committees are becoming too much of a catchall, she worries.

Insoo Hyun, of Case Western Reserve University, said that the ethical considerations for stem cell–based clinical trials were similar to those for other experimental procedures but with the “heat” turned up — patients are more desperate, procedures are riskier, snake-oil salesmen more of a factor and slots in research trials are fewer. Outside clinical trials, perhaps some of the thinking developed for assessing new surgical techniques could be useful. Organ transplants, after all, did not go through clinical trials. Thomas Okarma, president and CEO of Geron, which is developing an embryonic stem cell product for spinal cord injury, described how he thought the cells might work (one mechanism is the secretion of various health factors, which could have implications for stroke and Alzheimer’s disease). At the same time, he said his company is committed to “maintain[ing] a conservative risk-benefit calculus”, which basically means only trying the riskiest therapies in very ill patients with very few other options, thus reducing the potential to harm patients.

Stanford University’s Hank Greely shared a few thoughts to guide this kind of analysis. Regulators demand that research be safe, but the very uncertainty makes this impossible, he said. That, in a sense, makes those who participate in Phase I trials heroes. No one can be certain what will happen when some new therapy or procedure is tried in humans. And consent is particularly important when the risk is high, he said, indicating that risky procedures should be tested in adult patients before children, even if a procedure is more likely to work in a younger population.

Finally, he lamented the fact that because conversations between regulatory authorities and clinical trial sponsors are confidential, crucial risk-reducing, therapy-speeding knowledge does not spread freely. He called on those involved to reveal the thinking behind regulatory decisions.

Editor’s note: Students from the law and medical schools at Stanford University brought together an impressive group of world-class experts last week to discuss stem cell policy. I’ll describe some (very select) highlights over three blogs. Check the site for the Stanford Journal of Law Science & Policy over the next few weeks for powerpoints presentations and audiorecordings.


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