Federal health advisers recommended yesterday that gene therapy for children with a severe immune deficiency be restricted to those who have no alternative.
The Food and Drug Administration convened the panel after a 3-year-old French boy became the third child to develop cancer after receiving gene treatment for X-linked severe combined immunodeficiency, or X-SCID. The FDA has asked U.S. researchers doing similar work to put it on hold.
Doctors and scientists on the advisory panel said they do not want to hold up the therapy for children who have already failed to respond to bone-marrow transplants, an alternative treatment. But they noted that the number of such children is very small.
"What is happening here today -- the big picture -- is that it shows the difficulty in developing any new class of therapy," said Daniel R. Salomon, a professor in the Scripps Research Institute and a member of the FDA panel.
"There was a period of time that there was a tendency to say gene therapy . . . had been safe. What's clear now is that [problems can develop in] some gene therapy for some diseases," Salomon said.
The panel recommended that gene therapy trials for a similar ailment called ADA-SCID should go forward.
