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On January 21, 2000, the agency ordered a temporary stop to all gene-therapy trials at Wilson’s institute. In 2005, Wilson settled with the U.S. Department of Justice: he was not to lead any clinical trials regulated by the FDA for five years.

Hope for cures based on gene therapy, it appeared, had all but died with Jesse Gelsinger. But in February 2000, Friedmann gave the opening talk at a Monday-morning session of an annual meeting of the American Association for the Advancement of Science, in Washington, DC. He reviewed the fundamental difficulties of gene therapy, spoke of the many hundreds of protocols approved but so far not productive. He reminded his audience of Varmus’s impatient charge in 1995 that the field had been wildly oversold. Then–with a marked change in tone–he said, “We are on the verge of therapeutic efficacy.”

Two lines of work seemed to him to “have the feel of being correct.” A pair of American laboratories were beginning clinical trials of gene therapy for hemophilia. Proper blood clotting requires a cascade of responses, controlled by a series of proteins. Hemophilia A, the most common form of the disease, is caused by a defect in the gene for one of those proteins, factor 8; hemophilia B is caused by a defect in the gene for another, factor 9. The study ­Friedmann thought had that “sense of correctness” came from work with hemophilia B by Katherine High, a hematologist at the Children’s Hospital of Philadelphia. At Stanford, the gene therapist and virologist Mark Kay was also working with hemophilia B. Kay and High had combined their efforts. Their methods worked with animal models of the disease. They were ready to start human trials.

But the most convincing results, Friedmann said, were just then coming from a group of pediatricians in Paris. Their leader was a man named Alain Fischer, a clinician working with small boys who had a form of SCID. Like the girls whom NIH’s Anderson had treated for ADA deficiency, these children produced no T lymphocytes, the white blood cells that fight infection. But their disorder was caused by a different gene. The children had been sick; they were not thriving. Then Fischer and his colleagues tried gene therapy. “These kids are now to all appearances immunologically reconstituted entirely,” ­Friedmann said. “All their immune properties seem to be optimized.” He went on, “And the thing that’s so impressive about it is, first of all, that it came from nowhere. It came from left field.” Experts on immune-system disorders “certainly must have known of Alain Fischer and his group,” Friedmann said, but the gene-therapy community was not as familiar with his work. “And it also is presented in meetings in a very low-key, very modest sort of way,” Friedmann said. “They say straight out there’s nothing new in method–they’ve done just a combination of a fortuitously good disease model [with] a lot of standard retrovirology that’s been developed over many years.”

Fischer and a dozen colleagues reported their method, and their success with their first two patients, in Science on April 28, 2000. They followed up with a report in the New England Journal of Medicine for April 18, 2002.

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Credit: Illustration by Chris Buzelli

Tagged: Biomedicine

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