The Empire Strikes Back
One would hope and expect that Jolee Mohr's recent death during a gene therapy clinical trial would give pause to the field's researchers as well as the multi-billion dollar clinical trial industry. After all, almost two decades of gene therapy research have produced numerous serious adverse events without one product safe or effective enough for FDA approval. But the response has been surprisingly defiant; everyone acknowledges that Mohr's death is tragic, yet a notable number of gene therapy insiders are defending the conduct of the trial and framing Mohr's death as an unavoidable consequence of scientific progress.
Theodore Friedmann, UCSD medical professor and past president of the American Society of Gene Therapy, notes in Bioethics Forum that modern medical advances such as bone marrow transplants and chemotherapy "rel[y] on many forms of therapy that, during their development, went through long periods of setbacks and failure before the underlying conceptual and technical unknowns were solved." Remarkably, Friedmann fails to acknowledge his own role in the most recent gene therapy mishap: when the NIH's advisory committee on human gene transfer discussed the study's protocol and raised several concerns about its design, he was the committee's chair.
Northwestern Professor Eric M. Ruderman argued in the Chronicle of Higher Education that too much was being made of the ethical concerns surrounding Mohr's death. Ruderman believes the fact that (a) Mohr's personal physician proposed her participation after his clinic was paid to recruit subjects and (b) Mohr signed the consent form immediately rather than taking it home for closer consideration were perhaps not best practices, but did not violate any real "rules." And in response to my op-ed in the Seattle Post-Intelligencer, David Miller - CEO of Biotech Stock Research - wrote a passionate (though remarkably off-point) defense of using gene therapy for patients suffering from conditions that aren't life-threatening.
Taken together, these arguments reflect a Rumsfeldian logic that "stuff happens" in biomedical research and we should just accept it and get over it. Which is simply astonishing.
Gene therapy has been hyped more than the iPhone; it has been proposed as a cure for everything from diabetes to erectile dysfunction. And contrary to Friedman's argument, it's not like most other experimental treatments: its properties are less predictable and scientists have yet to figure out how to halt its activities when things appear to go wrong. Yet even as the gene therapy industry raises public expectations (and investment dollars), it wants to keep public oversight low by framing any critical questions as uninformed or simply anti-science.
While most biomedical researchers are doing their best to improve the public's health, what gets lost is how commercial interests drive research agendas and clinical practice. Far more than during the times when bone marrow transplantation and chemotherapy were developed, today's scientists and doctors all too often switch seamlessly from clinic to boardroom and from researcher to profiteer. And what Mohr's case so chillingly shows is that procedures that are supposed to protect patients have become more effective in protecting profitability and normalizing blatant conflicts of interest. Why are companies allowed to hire the IRBs that oversee their studies? Why are doctors receiving payments for enrolling their own patients?
In the wake of Jesse Gelsinger's 1999 death during a gene therapy experiment, Stuart Newman of New York Medical College told the BBC that "because of the commercialization of [gene therapy] research there really is an incentive to keep secret anything that reflects badly on the progress of the work." Are the same commercial interests spinning Mohr's death to keep stronger oversight at bay?