FDA Shuts Down Three Gene Therapy Experiments; Two In Los Angeles

The Food and Drug Administration shut down three gene therapy experiments, including two in Los Angeles, after health officials learned that a similar French study resulted in the death of a child and another developing leukemia, a newspaper reported Friday.An FDA panel is scheduled to meet Friday to determine whether the cases abroad are precursors of problems that could arise from current and future experiments, the Los Angeles Times said.The experiments under review involve gene therapy treatments for severe combined immune deficiency, a genetic disorder that leaves victims unable to fight off infections and germs.The disorder is often called the “bubble boy disease,” named after a Houston victim, David, who lived for years in a plastic bubble filled with filtered air.According to the Times, the FDA’s move came after Dr. Alain Fischer of the Necker Hospital in Paris recently revealed that his gene therapy experiments led to the deaths of one of two children who had developed leukemia two years ago. A third child treated in Fischer’s experiments has developed leukemia, the newspaper said.The FDA, which has not formally announced the move, temporarily shut down three gene therapy experiments for severe combined immune deficiency, including two separate experiments by Dr. Donald Kohn and Dr. Kenneth I. Weinberg of the Keck School of Medicine at the University of Southern California.Federal officials also halted joint experiments by Dr. Harry L. Malech and Dr. Jennifer Puck of the National Institute of Allergy and Infectious Diseases.Malech and Puck have treated two patients, and Kohn has treated four patients.

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