FDA proposes stringent new gene therapy rules

The death of a teenager in a Penn gene therapy clinical trial a year and a half ago continues to haunt the national medical community as the U.S. Food & Drug Administration has proposed a rule that would significantly increase the disclosure of information of such trials. The proposal calls for the release of confidential details of the design and safety of gene therapy clinical trials -- the 10-year-old experimental field that tries to inject sick patients with functioning genes. The policy would also apply to xenotransplantation, also an experimental field in which tissue or organs from animals are transplanted into people. The FDA believes that both procedures pose a unique risk to participants. "The goal is to provide consistent information that would allow the FDA to fully participate in public discussion," FDA spokeswoman Lenore Gelb said. "Ultimately, the FDA and the institutional review boards cannot be everywhere. There is real responsibility by the investigators and the sponsor." Under the new rule, results from preliminary safety tests, informed consent forms that patients must sign, any disciplinary actions by the FDA pertaining to each study, and monitoring procedures for the participants of each study would be released to ensure that patients are completely informed before they volunteer for a trial. Since the death of the Tucson, Ariz., teenager, Jesse Gelsinger, in September 1999 at Penn's Institute for Human Gene Therapy, the University's program in particular has fallen from its prominent perch atop the field to being restricted to research on animals and cell cultures. James M. Wilson, IHGT director and coordinator of the study, also was impacted by the tragedy. In November, the FDA sent Wilson a letter starting procedures to bar him from any human study. Yesterday, the FDA would not comment on whether it had received a response from Wilson, which it told him to send by last week. Wilson could not be reached for comment yesterday. "I realize that the investors were not reporting properly to the FDA," said Paul Gelsinger, Jesse's father, who recently settled a wrongful death and negligence suit he brought against Penn. "But the FDA had enough information to warn everyone that there were dangerous side effects." "We did not have the information we needed," Paul Gelsinger, said. "I don't know if this is the fix, but it is a step in the right direction. I think the FDA is seeing the light." "There is a sense that people do not trust the process," Jeffrey Kahn, director of the University of Minnesota Center for Bioethics, said. "This will help restore public confidentiality." But Dusty Miller, a researcher and professor at the Fred Hutchinson Cancer Research Center in Seattle warned that the disclose-all policy will backfire. "People have difficulty reading medical reports," he said. "If they do not know what is said, they will get scared off." And also under the new rule, the FDA can ask companies that sponsor the research to release information that they normally do not divulge until the study is finished. Kahn says that companies might fear that their competitors will use the released information. "Companies may be hesitant to allow proprietary information to go public," he said. But Gelsinger sees the patient's side. "People are far more important than investments and stock options," he said. "I have a responsibility as a citizen to make sure the government is doing the right thing," Gelsinger said. "So I can sleep at night." Gelsinger asked, "If we are saving people's lives, shouldn't we be working together?"

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