FDA Panel Calls for Resumption of Suspended Gene Therapy Trials

FDA advisers have called for gene therapy trials for severe combined immunodeficiency to resume despite the recent revelation that a French child contracted a "leukemia-like illness" from the experimental treatment for SCID, also known as "bubble boy disease." The panel determined that the "risk [is] too low to deprive desperate children of treatment," the AP/Las Vegas Sun reports (Neergaard, AP/Las Vegas Sun, 10/11). The FDA on Oct. 3 announced that three gene therapy trials were suspended in early September after the agency learned that a child who underwent treatment in France -- one of 14 enrollees in gene therapy trials in the United States and France for SCID -- developed the leukemia-like disease (California Healthline, 10/4). Although the link between the gene therapy and the cancerous side effects was unclear at first, Dr. Philip Noguchi, who oversees gene therapy research for the FDA, said yesterday that the evidence of the causal relationship "is pretty convincing." The National Cancer Institute's Dr. Crystal Mackall, an FDA adviser, noted that the French boy is the only case of a gene-therapy-related cancer-like side effect. Another adviser, Dr. Joanne Kurtzberg of Duke University, said that "harm" can be done by "withhold[ing] a promising treatment." The advisers recommended that gene therapy trials be reopened to SCID patients who do not have the option of bone marrow transplants from a "well-matched donor." In addition, they recommended that future trial participants be warned of the risk of cancer, including "specific discussion" of the French case. The FDA usually follows the recommendations of its advisers (AP/Las Vegas Sun, 10/11).

In other genetics news, the FDA has approved the first human trial to test a gene therapy technique to treat Parkinson's disease, Knight Ridder/Indianapolis Star reports. The experiment will begin later this year and will involve 12 patients who have had "severe" Parkinson's for at least five years and have not responded to existing treatments. The therapy involves injecting a "foreign gene" directly into a patient's brain. According to the Knight Ridder/Star, the treatment could provide a new option for people with Parkinson's who have not responded to drugs. At least one million Americans have Parkinson's (Flam, Knight Ridder/Indianapolis Star, 10/11).