Hoping to quell controversy, NIH panel votes to ease access to gene therapy

January 15, 1993|By Liz Bowie | Liz Bowie,Staff Writer

BETHESDA -- Looking to end a controversy over access to promising experimental treatments, the National Institutes of Health advisory committee adopted a policy yesterday to allow terminally ill patients quick access to unproven gene therapy.

But it cautioned that such a policy should be used only in rare circumstances.

The Recombinant DNA Advisory Committee (RAC) met yesterday in special meeting after NIH director Bernadine Healy bypassed the normal review process to allow the use of an experimental gene therapy on a 51-year-old brain cancer patient.

Her decision last month to personally intervene and approve use of the experimental treatment touched off a controversy in the scientific community over who should be permitted to receive the treatment and when the treatment should be given.

The case involved the first time that a gene therapy experiment had been tried on a patient without prior approval of the RAC.

RAC members were highly critical of Dr. Healy's decision in the specific case, saying in written and public statements that they were skeptical about the scientific basis for the experiment. They also expressed concern that political influence by Sen. Tom Harkin might have played a role in getting the woman the therapy. The Iowa Democrat chairs the subcommittee that oversees the NIH budget. A number of RAC members said they would not have approved the experiment.

"This is an unproven procedure that stands almost no chance of working," wrote RAC member Moselio Schaechter, chairman of the Tufts University Department of Molecular Biology and Microbiology. He said he was concerned about public safety, though not in this case.

"There is a remote possibility that viruses used to engineer the cells injected into the patient may cause damage, notably cancers, in members of the community," he wrote. Dr. Schaechter said at the meeting that his wife of 34 years had died of brain cancer four years ago.

RAC members said at yesterday's meeting at the NIH in Bethesda that they did not want to create false expectations in patients and treat them as guinea pigs. There is no documented case in which gene therapy has cured a patient of cancer or any other illness.

"We are dealing with something still in its infancy," said 'u Alexander Capron, a professor at the University of Southern California Law Center in Los Angeles. "There are considerations about not leading to false expections and having patients

become human subjects in something that is too preliminary."

Dr. Healy firmly defended her stand, saying that people could take issue with whatever decision she had made in the case.

"Today, I stand by that decision. First and foremost, the decision was a compassionate response to the request of a dying patient," Dr. Healy said. But she urged the committee to come up with a policy to expedite such cases in the future.

The San Diego doctors treating the patient chastised RAC members yesterday for some of their positions and said they found the comments "abhorrent."

The brain cancer patient's husband, who came from California to be at the meeting, made a passionate request that the committee set up a mechanism that allowed it to act quickly on applications for treatment by dying patients.

BHe detailed his wife's year-long struggle with brain cancer, which included radiation, chemotherapy and an experimental antibody treatment as well as two surgeries, one which removed a large portion of her brain.

He said he had talked to doctors in four other countries, contacted 30 cancer centers in the United States, studied brain cancer and gene therapy for more than a thousand hours and had spent $18,000 of his own money on treatment.

But he had difficulty getting access to the NIH, he said. "I had spent years working for my government but I am going to be damned if I am going let my government kill my wife," he said, his voice cracking with emotion. The RAC did not disclose the man's identity.

After Dr. Healy's approval, the woman was treated Jan. 4 at a San Diego hospital with genetically engineered cells that are aimed at getting her immune system to produce cells that combat cancer. Her doctors say it is too early to tell if the treatment is working.

The policy statement approved by a vote of 9-to-3 by the RAC is only a recommendation to Dr. Healy.

It calls for her to strongly encourage researchers who apply to treat even one patient to go through the RAC just as if they were applying to treat a dozen.

But because the committee meets only four or five times a year, there might be cases when the patient could die waiting for RAC approval. In that case, the RAC said, Dr. Healy should make the decision herself.

But they urged her to supply RAC members with all scientific information to support the application, to seek their advice before making a decision and to report back to them. In this case, the RAC members felt they had not been given enough information about the treatment.

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