GENE THERAPY: UPenn Responds; Father Speaks at Hearings
A federal investigation into the gene therapy-related death of Jesse Gelsinger prompted federal officials to seek tighter enforcement of result-reporting guidelines, the Los Angeles Times reports. In a meeting at the NIH yesterday, officials from the FDA and NIH emphasized that researchers working on federally funded studies must adhere to federal regulations -- which require reporting of clinical trial results to both agencies -- and said that scientists could not declare data "confidential" to prevent the public or other researchers from accessing the information. The statements came after months of controversy during which researchers on several studies, including the UPenn trial in which Gelsinger was enrolled, have been accused of withholding information about adverse events in gene therapy treatments. Previously, data from gene therapy research reported to the FDA could remain confidential, while the NIH had no privacy mandate. Thus, the NIH -- and the public -- has been left in the dark about some of the recent adverse effects occurring in gene therapy trials, including several deaths. But director of the NIH's office of science policy Lana Skirboll said that the agencies now plan to increase information-sharing, so that "if an investigator has not submitted something to the NIH, we will know from the FDA." She remarked that, until recently, "we didn't know what we didn't know." Under the revised regulation measure, researchers would have to report adverse events to both agencies within 15 days, even if the effects are not assumed to be related to the therapy. Moreover, reports should not contain any "proprietary or trade secret information" so as not to be declared confidential. The proposed guideline changes, coming on the first of three days of hearings by the NIH's Recombinant DNA Advisory Committee, must undergo a comment period of several months prior to finalization (Cimons, 12/9).
UPenn Comments, Father Speaks
Gelsinger's death during the UPenn liver study -- the impetus for the hearings -- remains embroiled in controversy. Yesterday, the FDA released information suggesting that teenager's death was caused directly by the UPenn trial. Moreover, FDA officials said that they were particularly concerned about two "protocol deviations" that took place in the experiment. FDA investigators stated that one "very preliminary" finding was that Gelsinger's ammonia-level exceeded the allowable limits of the study, indicating that Gelsinger should have never been infused with the therapy. Secondly, FDA officials discovered elevated liver- enzyme levels had occurred in two prior trial participants, but was never reported. Such information should have resulted in a halt on the UPenn study, the Wall Street Journal reports. James Wilson, head of the UPenn trial, told reporters that he is "fully comfortable" with his team's decision to infuse Gelsinger because he believed the teen's condition to have been stable (Adams/Langreth, 12/9). Denying wrongdoing, university researchers are scheduled to defend their work at today's hearing. After yesterday's session, Gelsinger's father absolved Wilson from any blame associated with his son's death, saying "I touched souls with this man. There is nothing wrong with Wilson." Rather, the elder Gelsinger blames the FDA for not releasing confidential information and stopping the experiment before Jesse's death (Nelson/Weiss, Washington Post, 12/9). Wilson, who owns a stake in the profits from his research, has received much criticism suggesting that financial conflicts of interest resulted in lapses of the study's safety. Dr. Arthur Caplan of the UPenn Bioethics program, commenting on yesterday's "Nightly News," seemed to suggest that no one is really at fault in Gelsinger's death. He said gene therapy "experimentation is inherently risky. There's no getting around it. We want to believe that we can experiment without having people die. But we can't" (12/8).