FDA Panel Suggests Limits on Gene Therapy for Kids

Federal health advisors recommended Friday that gene therapy treatments for children with a severe immune deficiency be restricted to those who had 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 therapy for treatment for X-linked severe combined immunodeficiency disease, or X-linked SCID. The FDA had asked U.S. researchers doing similar work to put it on hold.

Doctors and scientists on the advisory panel said they didn’t want to hold up the therapy for children who had failed to respond to bone marrow transplants, an alternative treatment. But they noted that the number of such children -- who under Friday’s recommendation would still be able to have experimental gene therapy -- was small.

“What is happening here today -- the big picture -- is that it shows the difficulty in developing any new class of therapy,” said Dr. Daniel Salomon, a professor at 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 could go forward for a similar ailment called ADA-linked SCID, finding it was a different issue and that research need not be limited.