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    The Potential of Gene Therapy Threatened by Early Tragedies

    WebMD Health News

    Aug. 4, 2000 (Washington) -- Just like any other tourist in Washington, D.C., Paul Gelsinger seemed lost as he tried to navigate his family through the city's formidable subway system last week. But even though he was on vacation, Gelsinger eagerly shifted the focus of conversation from sightseeing in the nation's capitol to the fate of his son Jesse.

    Since last September, when 18-year-old Jesse Gelsinger became the first patient to die as a result of a gene therapy experiment, his father has emerged as a symbol of a tragedy that has shaken the scientific community, government regulators, and patients in need of a miracle treatment. "You can't imagine what it's like," Gelsinger tells WebMD of Jesse's death.

    The elder Gelsinger says that some sort of legal action against the University of Pennsylvania, which administered the treatment for Jesse's rare liver disorder, is imminent. However, it's not clear that the matter will wind up in court.

    "We're certainly not anywhere near settling the case," Alan Milstein, Paul Gelsinger's lawyer, tells WebMD. Milstein says that Paul Gelsinger will ultimately be asking the university for millions of dollars in damages for the "wrongful death" of his son.

    In the meantime, the FDA shut down all the University of Pennsylvania's gene therapy programs in January, and in May officials at the university said the institution would no longer conduct gene therapy experiments on people. The lead researcher at the university's Institute for Human Gene Therapy, James Wilson, MD, did not respond to requests for comments.

    Aside from one family's grief, the Jesse Gelsinger tragedy has triggered a number of federal investigations as well as a national rethinking of the risks and benefits of gene therapy.

    Other programs under scrutiny by the FDA included a tumor vaccine study in which young cancer patients might have been accidentally exposed to deadly viruses. The research was underway at St. Jude's Children's Research Hospital in Memphis and at Baylor College of Medicine in Houston.

    But pediatric oncologist Laura Bowman, MD, of St. Jude's, says the problem was only a "false positive" lab test, and there was no vaccine contamination. "Our vaccine program is up and running full stream," Bowman tells WebMD. But she also says coverage of the issue caused a "lot of pain for the families."

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