B ETHESDA , M ARYLAND - Doctors and scientists from University of Pennsylvania defended their clinical judgment today in the case of Jesse Gelsinger, 18, who died September 17 while receiving experimental gene therapy. At a meeting here, the National Institutes of Health, a team leader and gene therapy pioneer James Wilson said he "felt comfortable" with the decision to treat the teenager with a strong dose of virus GM, though it is clear in retrospect that the viral concoction has killed Gelsinger.
Wilson and his co-researchers, Mark Batshaw and Steven Raper, presented data suggesting that unexplained anomaly is Gelsinger in metabolism, genetic makeup, or immune status triggered a toxic reaction to the therapy --a response that could not have been expected, they said, on the basis of earlier data.
Gelsinger had a rare genetic disorder, ornithine transcarbamylase (OTC) deficiency, which can cause a lethal buildup of ammonia in the body. Although he kept his symptoms under control through diet and the ammonia-drugs lowering, he volunteered to join the trial for gene therapy for risk, hoping it would lead to a cure. Gelsinger, 18 patient to be treated in the study died of respiratory failure four days after receiving a high dose of adenovirus genetically crippled (a common cause of human infections) which was equipped with the human gene little by little.
defense team came after gene therapy authorities to the Food and Drug Administration (FDA) yesterday blamed the Penn trial. Kathryn Zoon, director of FDA's Center for Biological evaluation products and research, said a preliminary investigation had found "gaps" to a protocol approved by the FDA agreed. An official at the FDA found that when Gelsinger underwent therapy, the ammonia level in the blood was more than 50% above the maximum permissible level specified in the memorandum, a deviation from the plan that the FDA n has not been notified in advance. Batshaw acknowledged that "we would have called FDA" about the change.
Other researchers say the team took a risky approach by administering high doses of the modified virus. "They pushed the edge of the envelope, "says Thomas Caskey, director of the therapy program and Merck Genome Gene West Point, Pennsylvania.
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