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Advisors Raise No Stop Sign to Gene Therapy

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Rising to support Gelsinger, Rifkin called it "shameful" that the committee had waited until a patient death before finally coming to grips with key safety issues. "You're just getting around to asking questions you should have asked 10 years ago. You're just beginning to look at what ... appropriate protocols should be put in place to make sure there are no problems," said Rifkin.

On Wednesday, the RAC discussed the shutdown of another gene therapy experiment, this one involving Allovectin-7 -- a treatment for melanoma. Researchers at the University of South Florida suspended the study after a patient's death in 1999 was reclassified as "probably" caused by the treatment.

While the main focus for the meeting was safety, the committee also was pushed to endorse new treatments for desperately ill patients. On Thursday, Roger Karlin, MD, and his wife, Helene, who holds a doctorate in psychology, watched anxiously as the RAC debated a new type of gene therapy for Canavan's disease -- a devastating brain disorder affecting children. Their 5-year-old daughter Lyndsay has the disease and previously had been given a gene treatment called apaspartoacylase (ASPA).

However, researchers from Thomas Jefferson University in Philadelphia now believe that a new type of delivery system called an adeno-associated virus would produce better results, and they were asking the RAC for an endorsement of the approach.

Withholding the treatment would amount to giving her daughter a "death sentence," Helene Karlin told the committee. Roger Karlin, an internist practicing in New Fairfield, Conn., worked with the gene researchers as they developed their approach, and the couple even established a foundation to help the estimated one in 10,000 who contracts this deadly illness.

Still, after considerable discussion, RAC panelists insisted that the scientists provide more data about how this new treatment delivery system would work in animals and whether the replacement gene would really function correctly. The situation proved particularly frustrating for the Karlins, because they feel they know the risks and the benefits as well as anyone.

Ironically, the Karlins believe Jesse Gelsinger's death may have created an environment that is endangering their daughter. "Unfortunately, the bureaucratic element often interferes in these protocols, and what happens is that the ethical rights of these children ... [are] violated," says Helene Karlin.

Though the Karlins say they hope that their daughter will get started in a new gene trial in a couple of months, the process has made them angry. "Death doesn't wait for meetings, and that's what we object to," says Helene Karlin.

In spite of the pressure to come up with new procedures to publicize adverse events from gene trials, the committee couldn't reach a consensus on how much information researchers should report and when, even though it was a top priority of the meeting.

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