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Gene Therapy on Trial
The recent death of Jesse Gelsinger has served as a wake-up call for researchers, research institutions, government regulators and the public about how research is carried out in the United States.
Gelsinger, 18, had a genetic disorder that prevented his body from properly metabolizing ammonia. A resident of Arizona, he enrolled in a clinical trial at the University of Pennsylvania that was attempting to use gene therapy to correct the disorder. But rather than curing him the technique caused his death.
The chain of events that followed -- including the shutdown of all research at Penn's Institute for Human Gene Therapy -- call into question not only the safety of the techniques of gene therapy, but the conditions and protections under which all clinical research is conducted.
High stake cures come with high stake risks
Gene therapy and other cutting edge research are attempting to cure diseases in a new way. They focus on the root of illness by making repairs at the genetic level. This has allowed treatment for diseases whose symptoms haven't emerged or chronic illnesses that don't necessarily cause death -- such as some forms of arthritis. But these repairs are not risk-free, as the death of Gelsinger reminds us. And so we need to ask what levels of risk are acceptable for the gains of potential cures for maladies that may not even kill us anytime soon.
As in all clinical research, informed decision-making by potential subjects is a critical but elusive goal. The investigation following Jesse Gelsinger's death uncovered evidence of incomplete and inaccurate disclosure of the risks. Even when information is adequate, studies show that subjects tend to overestimate potential benefits of research while underestimating its risks. This is a dangerous combination when we are asking patients to put themselves in harm's way primarily for the good of patients who will come after them, with only a limited chance that they will benefit.
A growing complication in these types of high tech clinical research is the blurring of lines between researchers and the pharmaceutical or biotechnology companies who fund the projects. There are increasing numbers of outright conflicts of interest when researchers have a financial stake in biotechnology start-ups that are created to develop and market their discoveries. This can create incentives for recruitment of patients as much in the interest of researchers' financial gain as for the interests of subjects.
Adequate oversight or looking the other way?
A key concern of regulators is what looks like a widespread and alarming failure to report harmful side effects to subjects in gene therapy trials at Penn and elsewhere. While such reporting is a required part of federal oversight, there are grumblings from inside the beltway that policies may inadequate. The question then is whether we need greater adherence to existing rules, additional new tighter regulation and oversight, or both. Following existing rules would certainly be a good start.
Never before have issues of protecting research subjects been so carefully scrutinized or taken so seriously, and the ongoing gene therapy case at the University of Pennsylvania has created a drama whose cast includes medical researchers, Congress, disease group advocates and federal bureaucrats. Each has a somewhat different stake in the outcome. But whatever the outcome, all must keep their eyes on the ultimate goal -- high quality, careful research conducted in ways that respects the rights and interests of subjects. Otherwise we face the prospect of a research enterprise that subjects us to science rather than carrying out science in service to us.
"Ethics Matters" Archive
where you'll find other columns from Jeffrey Kahn
on a wide range of bioethics topics.
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