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Advisors Raise No Stop Sign to Gene Therapy

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"I had no idea there were 38 trillion viral particles being put into my son. All I knew is it was the maximum dose allowed by the FDA," said Gelsinger. He also complained he should have seen a detailed description of the experimental protocol, and that he should have known more about the lead researcher's financial interest in the treatment.

"It is a national issue, and I think it's something that should be done by the National Institutes of Health. It is that important," said Gelsinger. A source, who did not want to be identified, indicated Gelsinger is likely to take some kind of legal action against the University of Pennsylvania.

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.

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