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Gene Therapy Experts Seek Safer Studies

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March 8, 2000 (Bethesda, Md.) -- After a series of shock waves, including the first patient death from a gene therapy treatment last year, a federal review committee attempted to move on with its work of supervising these controversial experiments with a new emphasis on safety.

"There is a lot at stake here ... the people who are watching are the patients and families who are hoping in the long run that we develop new treatments," Lana Skirboll, PhD, told the Recombinant DNA Advisory Committee (RAC) at the beginning of a three-day meeting here Wednesday. Skirboll is director of the Office of Science Policy at the National Institutes of Health (NIH).

Last December, the RAC struggled to deal with the death of 18-year-old Jesse Gelsinger during a gene therapy study for an inherited liver disorder. Then in January, the FDA ended that study at the University of Pennsylvania amid charges the researchers withheld information about dangerous side effects linked to the procedure.

Last week, promising experiments about the possibility of using a gene to grow new blood vessels in patients with heart disease were halted, because the lead researcher apparently failed to report two patient deaths to the NIH.

In the wake of these irregularities, the FDA and the NIH, at the direction of President Clinton, announced new guidelines on Tuesday aimed at enhancing protections for patients in gene therapy studies. The effort, according to the release, comes in the wake of evidence that the "monitoring by study sponsors of several recent gene therapy trials has been less than adequate."

"The kind of event that happened with Jesse simply won't happen again. That doesn't mean there won't be deaths, but at least there won't be situation where people weren't aware of what was going on," W. French Anderson, MD, director of the gene therapy laboratories at the University of Southern California in Los Angeles tells WebMD. Anderson has been credited with performing the first gene treatment in 1990.

After a decade of rising expectations, but still no gene treatments on the market, Anderson says, "The growth spurt's about to begin." While he calls the Gelsinger episode painful, he says the changes it's bringing to the field are positive.

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