Gene Therapy Risks To Be Revealed

Friday, January 19th 2001, 12:00 am

By: News On 6

WASHINGTON (AP) â€” Americans considering participating in studies of gene therapy may soon get better information about the risks as the government announced plans Wednesday for the first public disclosure of side effects almost as soon as they happen.

The Food and Drug Administration's major policy shift was sparked by uproar last year when an Arizona teen-ager died in a gene therapy experiment.

Under the proposed change, scientists would have to post on the FDA's Web site most of the safety information about an experiment when it begins. Until now, that information largely was kept secret.

The proposal affects mostly the hot new field of gene therapy, but also controversial xenotransplantation, implanting animal tissue into people.

``Both of these technologies hold great promise, but they may also pose a remote but unique risk to the individuals who have volunteered to participate in these types of studies,'' FDA Commissioner Jane Henney said.

Consumer advocates praised the change as a long overdue way to help sick Americans make better decisions about joining certain medical experiments. But the biotechnology industry promised to fight the proposed rules, arguing that disclosing such information could confuse the public about whether a study was truly unsafe or whether a participant's side effect really was due to the underlying disease.

Prompting the change was Jesse Gelsinger's death last year from a botched University of Pennsylvania gene therapy study; it was the first known death from gene therapy.

Among other problems, federal investigators discovered scientists never had warned study participants that two monkeys died from the experiment, and failed to stop the trial after some patients developed worrisome liver abnormalities. Gelsinger died of a liver disorder four days after his own treatment.

The FDA decides whether scientists can try experimental therapies in people based on safety studies in animals. Patients volunteering for such experiments are supposed to receive consent forms detailing all the potential risks. But the FDA largely depends on local oversight boards to ensure that happens, and it too often does not.

Once the experiment begins, scientists must report all side effects to the FDA â€” but until now the agency has almost always kept that information secret, bowing to industry's insistence that what happens in a clinical trial is a commercially important trade secret.

Even when the FDA stops an experiment because people have died, it seldom tells the public. Study participants often are left to learn about safety problems from scientists with a vested interest in the experimental therapy.

The proposed change would require scientists to reveal large amounts of nonproprietary information about new studies, including what patients qualify, exactly what is being tested, and results of animal safety studies or any previous human studies. As the study continues, any serious side effects would be revealed, too.

``It's terrific. It's a very major step forward,'' said Dr. Sidney Wolfe of Public Citizen, predicting thousands could comb the Web site with their personal physicians for less biased information in choosing a clinical trial.

But, noting that many more people have been injured in studies of regular medications than by gene therapy experiments, Wolfe said the change should be expanded to cover all FDA-approved experiments.

That is just what the Biotechnology Industry Organization fears.

``It's a dramatic deviation from over 30 years of FDA regulations,'' said Michael Werner, the trade group's government relations chief.

He said the public will not understand that many reactions suffered in clinical trials are due to participants underlying disease, not the therapy. If a study really is risky, the FDA should shut it down â€” and if not, participants should get safety information from researchers who know the study best, he contended.

But FDA biotechnology chief Dr. Kathryn Zoon said the new openness could boost gene therapy by helping a now-suspicious public better understand the complex technology. ``The more transparent the process is, we believe the more public acceptance and public confidence'' there will be.

The FDA will begin finalizing the rules, a process that could take months, after a 90-day public comment period.

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On the Net: http://www.fda.gov