Experimental gene therapy for patient raises questions of influence, access NIH chief, senator interceded in case

January 14, 1993|By Liz Bowie | Liz Bowie,Staff Writer

An unprecedented decision by the director of the National Institutes of Health to personally intercede in allowing gene therapy for a dying brain cancer patient has raised a controversy over access to promising experimental treatments.

The decision by NIH Director Bernadine Healy will be the subject of a special meeting today of a scientific review panel at NIH headquarters inBethesda.

The panel of outside experts, the Recombinant DNA Advisory Committee (RAC), has reviewed all similar gene therapy experiments but was bypassed in this case, and some critics are questioning whether the experiment underwent the rigorous review it should have.

In addition, some believe that the 51-year-old San Diego woman who received the treatment last week might not have gotten it without help from Sen. Tom Harkin, D-Iowa. Mr. Harkin chairs the subcommittee that oversees the NIH budget and wrote a letter to Dr. Healy on the woman's behalf.

The case illustrates the wrenching issues that will confront doctors as the science of gene therapy progresses and they are deluged by terminally ill patients seeking any chance for a cure. Gene therapy has been used in only a handful of patients to treat deadly or untreatable diseases such as cancer and a rare genetic disease that leaves children without a functioning immune system.

A spokeswoman for Dr. Healy said her decision was not politically motivated, but rather an attempt to confront the policy issues immediately. To have denied the woman the treatment would have been "a very bureaucratic response," said Johanna Schneider, the NIH spokeswoman.

But one member of the RAC, Robert Haselkorn at the University of Chicago, complained about Mr. Harkin's involvement in a letter to fellow committee members last fall: "This is monstrous. . . . The manipulation of a U.S. senator coupled with the threat of further congressional action is unconscionable."

Attorney Andrew Kimbrell said, "There is a fine line between compassionate use [of a treatment] and treating a terminally ill -- patient as a guinea pig." Mr. Kimbrell is with the Foundation on Economic Trends, a Washington group that monitors the impact of genetic engineering, and he plans to testify at the meeting.

The woman's story began more than a year ago, when researchers at the San Diego Regional Cancer Center developed an experiment to treat brain cancer patients with gene therapy. The idea was to inject genetically altered cells into a person's body to combat cancer.

The researchers, who are also physicians, did experiments on laboratory animals and, in November 1991, they asked the RAC for permission to conduct the experiment on humans, said Dr. Robert E. Sobol, one of the San Diego researchers. The committee turned them down because it believed they did not have enough proof that the treatment would work. They told the researchers to do more animal studies.

During this time, the San Diego woman developed brain cancer and, like other cancer patients, allowed a portion of the tumor that was removed from her brain to be given to the researchers for their work.

After surgery, chemotherapy, two types of radiation and an experimental antibody treatment had all failed, the woman asked the researchers if they would try the experiment on her.

Because they believed that the woman would die before they could go through the usual application process with the RAC, Dr. Healy and the Food and Drug Administration, the doctors decided to try an expedited process that had never been tried before. They applied to the agencies for a "compassionate use exception" and made changes in their proposed experiment to match the technology of one that had been previously approved.

"We felt the time to treat her with the therapy for some benefit was now and not later," Dr. Sobol said.

At about the same time, the woman's sister, who lived in Iowa and had worked on Mr. Harkin's campaign, asked the senator's staff for help. In October Mr. Harkin wrote to Dr. Healy expressing concern that the NIH did not have a process for giving patients a timely response to such requests, and he detailed the plight of the woman as an example.

Mr. Harkin denied any attempt to use political influence.

"He wasn't singling anyone out; he was raising a policy issue with them," said Peter Reinecke, a Harkin staff member.

But Mr. Kimbrell said his group, which has generally criticized the use of gene therapy, believes the case is "nothing but political power used in an unethical fashion."

In an Oct. 22 response to Mr. Harkin, Dr. Healy underscored the difficulty in granting exceptions. "It is not possible to make decisions as to which case is more worthy than others when one is dealing with what are uniformly fatal diseases: Attempting such kind of decisions would compromise the review process and not be in the best interest of such patients."

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