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The Potential of Gene Therapy Threatened by Early Tragedies

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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."

The vaccine program at Baylor remains on hold, and patients who participated are being carefully followed, says a spokeswoman.

In March, the FDA shut down a gene therapy study aimed at growing new blood vessels in patients with severe heart disease at Boston's St. Elizabeth's Hospital. The issue still being negotiated is whether the researchers properly reported two patient deaths to the FDA, according to a hospital spokeswoman there.

And in July, the newly created Office for Human Research Protections, established to help the government oversee all human clinical trials, stopped federally funded medical research at the University of Oklahoma College of Medicine in Tulsa over concerns about the safety of a vaccine to treat melanoma. An audit revealed a number of manufacturing flaws in the product, raising questions about whether patients with the deadly skin cancer understood the risks involved in the trial.

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