Experts chosen for gene review

The six-erson panel will review the IHGT's research procedures. Following up on her promise to appoint an external committee of experts to review Penn's Institute for Human Gene Therapy, President Judith Rodin announced the members of the committee yesterday. The six-person committee -- which includes representatives from academic and private institutions -- will "conduct a comprehensive review of oversight and monitoring of clinical trials" at the IHGT to determine how the Institute can achieve compliance with government regulations, Rodin said in a written statement. Chairing the committee will be William Danforth, chancellor emeritus and vice chair of the Board of Trustees at Washington University in St. Louis. A professor of internal medicine, Danforth sits on the board of several charitable organizations as well as the Ralston Purina Company and the BJC Health System, both headquartered in Missouri. Danforth's assistant said yesterday that he is in Antarctica and could not be reached for comment. Rodin pledged to form the external committee following the U.S. Food and Drug Administration's decision to suspend all eight gene therapy clinical trials at the IHGT on January 21, citing multiple violations of federal research protocol that the FDA maintained put patients and test subjects at risk. The emphasis of the committee will be to examine the list of possible violations that the FDA alleged researchers committed during the clinical trial that left 18-year-old Jesse Gelsinger dead last September. Also on the committee are Joseph Martin, dean of the Faculty of Medicine at Harvard Medical School; Edward Benz, director of the Department of Medicine at the Johns Hopkins University School of Medicine; and Rochelle Hirschhorn, chief of the Division of Medical Genetics at the New York University School of Medicine. Representing private research organizations on the committee are Inder Verma, a professor of molecular biology at the Salk Institute in La Jolla, Calif., and Daniel Callahan, director of international relations at the Hastings Center in Garrison, N.Y. Rodin said she will give the committee "a broad mandate to conduct a comprehensive review of every aspect of IHGT's procedures for oversight and monitoring of clinical trials." Last week Rodin said she expects the external committee, which will report directly to her, to release its findings in six to eight weeks. After conducting its review, the committee will recommend any additional actions that may be necessary to "ensure that the highest standards are set and met" for future IHGT clinical trials. Also yesterday, Gelsinger's father testified before the U.S. Senate Subcommittee on Public Health at a hearing on government oversight of the 10-year-old gene therapy field. The hearing came days after The Washington Post reported that Gelsinger's death may not have been the first associated with gene therapy, which seeks to cure diseases by essentially changing the patient's DNA. Researchers at Harvard University did not report the deaths of three patients who were enrolled in a gene therapy experiment last summer, the paper reported, citing previously-unreleased FDA records. The report raises new questions about ineffective government oversight of gene therapy. Paul Gelsinger told the lawmakers yesterday that he and his son were largely uninformed about possible dangers associated with the clinical trial when Jesse agreed to take part. "[Jesse] believed? that the worst that could happen in the trial would be he would have flu-like symptoms for a week," Gelsinger testified. "With the knowledge I had at that time, I was comfortable enough to send my son? to Philadelphia alone." He explained that neither he nor his son were aware that lab monkeys died in similar clinical trials, or that patients prior to Jesse -- receiving the same treatment -- experienced severe liver injury. "Looking back, I can see that I was fairly naive to have been as trusting as I was," he said. Gelsinger also expressed his concern about current federal oversight of gene therapy, referring to recent reports that less than 6 percent of gene therapy adverse events were reported to the National Institutes of Health in a timely fashion. "I am all for the continued development of gene therapy, but it must be better regulated," he urged. "The concern should be not on getting to the finish line first, but on making sure no unnecessary risks are taken." The chairman of the subcommittee, Sen. Bill Frist (R-Tenn.), said that the death of Gelsinger has "sombered us all." "Clearly, our oversight system is failing," he noted. "These events trigger serious questions regarding federal oversight of gene therapy trials." The Associated Press contributed to this article.

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