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FDA Halts Gene Therapy Trials After Patient Develops Disease

By Aaron Zitner

A French boy who had been considered one of the few people cured by gene therapy has developed a leukemia-like disease, prompting the Food and Drug Administration to halt three gene therapy trials in the United States.

It is unclear whether the gene-replacement technique caused the boy’s new illness.

Still, the incident threatens to further dampen hopes for gene therapy, which has been under intense scrutiny since the 1999 death of a teenager in a University of Pennsylvania experiment.

In gene therapy, healthy genes are placed in a patient’s cells in an attempt to compensate for the faulty genes that cause disease. The technique is considered one of the most exciting ideas to emerge from the explosion of information about human genes, with prospects for treating cancer, hemophilia, AIDS and other ailments.

The French boy, now 3, had undergone the process as an infant to control “bubble boy disease,” a rare immune system disorder that is fatal if untreated.

The boy’s immune system rebounded, and he was able to leave a sterile hospital environment -- or “bubble” -- and live at home. He was one of four boys included in a French study, published this spring, that is considered the only definitive proof that gene therapy can work.

But in August, doctors found that the boy had developed a leukemia-like illness. It was the first time the illness appeared in any of the 32 or so patients treated worldwide by gene therapy for the immune system disease, including nine other children in the French study.

Still, FDA officials disclosed Thursday that they had halted three U.S. gene therapy trials earlier this week, days after learning that French authorities stopped trials run by Dr. Alain Fischer and colleagues at the Necker Hospital for Children in Paris, where the boy had been treated.