‘Compassionate Approvals’ for Unproven Gene Therapies OKd

Prompted by a case involving a San Diego woman dying of brain cancer, a government advisory committee ruled Wednesday that the director of the National Institutes of Health could issue “compassionate approvals” for the use of unproven gene therapies in medical emergencies. The decision is expected to result in a flood of hundreds, perhaps thousands, of requests for such waivers for terminally ill patients.

The decision grew out of a request by physicians at the San Diego Regional Cancer Center for approval to treat the 51-year-old woman with a form of gene therapy that has not yet been proved successful in animal trials. Dr. Bernadine Healey, NIH director, issued the approval because of the possibility that the woman might die before the advisory committee could meet. The treatments began Jan. 4.

Critics have charged undue political influence in the San Diego case because the request for compassionate approval was forwarded by Sen. Tom Harkin (D-Iowa), who chairs the subcommittee that funds NIH. The San Diego woman’s sister-in-law was a campaign worker for Harkin and her husband has held posts in four U.S. administrations.

Harkin has said that he merely asked the NIH to set up guidelines for handling such requests--which the committee did Wednesday. Healey said political considerations played no role in her approval of the San Diego request.

Although some committee members feared that unproven gene therapies might prove hazardous to patients and health care workers, the committee voted 9 to 3 to permit the compassionate approvals. But it also required that the NIH director return to the panel and justify each approval.