GENE THERAPY: Committee Wants More Say in Studies
In the wake of recent concerns over patient safety and failure to report some deaths in gene therapy studies, the Recombinant DNA Advisory Committee (RAC) has proposed two measures that would expand its role in the oversight of those studies, the Wall Street Journal reports. In an effort to clear up confusion surrounding what information should be submitted to the committee, the first proposal, published in the Federal Register this week, would require researchers to report any deaths or serious illnesses within 15 days to RAC, regardless of whether the researchers believe the complication is related to the actual study. The second proposal would require researchers to submit their study protocol to the committee at the same time they submit it to their institutions' internal review board. This proposal would allow the RAC to review the study design well before the FDA, and eliminate some potential problems. Dr. Claudia Mickelson, chair of the RAC, said, "We want our discussion to be early enough that it's valuable." Some health officials contend that these proposals would restore the committee's credibility. The RAC was stripped of some of its authority over gene therapy research three years ago. Former RAC Chair Dr. LeRoy Walters said, "The only way the RAC will be taken seriously is if researchers know they have to come to it for approval." Philip Noguchi, the FDA official who oversees gene therapy studies, said, "I do think they are moving more and more toward a role that is critical." The FDA and the RAC have concentrated on different areas of gene therapy studies. Traditionally, the FDA analyzes the science while the RAC focuses on the ethics of proposed clinical trials. However, many in the biotechnology field are opposed to expanding RAC's role. Michael Werner of the Biotechnology Industry Organization said, "We flatly reject that there needs to be another regulatory agency here." In light of the recent misreporting of deaths in gene therapy trials, some believe that the RAC has more momentum behind its push for greater authority. Dr. French Anderson, one of the pioneers in gene therapy, said, "The RAC is there to provide public confidence, and things are a little shaky right now" (Adams, 11/26).
Up to the Task
Further bolstering the committee's position, the RAC will hold one of its most significant meetings ever this month when scientists convene at NIH to debate the merits of gene therapy. At the three day meeting, scientists and researchers will pour over information regarding the death of Jesse Gelsinger, an 18- year-old gene therapy patient who died in September after receiving the experimental therapy. At issue is the use of adenovirus to insert new genes into a patient's cells. The committee will review the results of every adenovirus study currently underway to analyze its safety (Langreth/Adams, Wall Street Journal, 11/26).