GENE THERAPY: Senate Hearing Investigates Violations
Declaring the protection system for gene therapy patients a "multisystem failure," Sen. Bill Frist (R-Tenn.), chair of the Health, Education, Labor and Pensions public health subcommittee, yesterday called for corrective action during a three-and-a-half hour subcommittee hearing (Stolberg, New York Times, 2/3). Lamenting the recent death of 18-year-old Jesse Gelsinger, a gene therapy trial participant at the University of Pennsylvania, Frist said, "Jesse's death is not merely a pothole that we can simply ride over on the road to find genetic cures. We must take a new path that ensures that no family will be forced to endure what the Gelsinger family has experienced." At the hearing, Gelsinger's father Paul testified that he was "misled" about the experiment's risks, noting, "It was presented as safe" (Recer, AP/Contra Costa Times, 2/3). But, he added, "I wasn't given all the information. And some of the information I was given was not true" (Weiss/Nelson, Washington Post, 2/3). Gelsinger urged the government to require an independent patients' advocate to be present at all sessions involving risk explanation in order to ensure that patient consent is informed (New York Times, 2/3). He also chastised the biotechnology industry for "putting money and fame ahead of patient safety." He said, "The concern should not be on getting to the finish line first, but on making sure no unnecessary risks are taken." For their part, FDA officials, charged with monitoring gene therapy trials, said they failed to uncover numerous protocol violations in the UPenn experiment earlier because of staffing shortages, which forced them to forgo on-site inspections in favor of written reports from scientists. Both the FDA and the NIH, however, say they now plan to devote more money and time to on-site reviews. As for recent discoveries that only 39 of 691 gene therapy- related deaths and illnesses were reported to the NIH, officials said they have "no explanation," but claimed they are trying to devise new policies that will increase researchers' compliance with federal reporting rules (Collins, Philadelphia Inquirer, 2/3).
Stemming the Tide
Dr. LeRoy Walters, a bioethics professor at Georgetown University, told the committee that there is a simple reason for researchers' failure to report adverse effects: they do not take the NIH's Recombinant DNA Advisory Committee, which oversees gene therapy, seriously because it lost its authority to approve research protocols after a 1996 reorganization. (New York Times, 2/3). Walters suggested the creation of an independent "data monitoring and safety board" to closely watch experiments and identify potential safety concerns. H. Stewart Parker of the Biotechnology Industry Organization also proposed a measure that would urge companies to report "some adverse events" to the NIH, where, unlike admissions to the FDA, reports can be made public" (Washington Post, 2/3). Not all people support assuming a cautionary research pace, however. Cystic fibrosis patient Eric Kast said that "if the science slowed down too much, a cure would come too late for him" and others. "My battle with C.F. is a race. Don't let me lose that race when the finish line might be just around the corner," he said (New York Times, 2/3).