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Biotech — Jury Still Out on the Promises of Gene Therapy

Biotech: Consistent

Successes Continue

To Elude Scientists

Experts in the field of gene therapy at a recent meeting sponsored by the local chapter of the American Heart Association said while the technology holds great promise, many hurdles remain.

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Gene therapy, a field widely heralded as the key to new medicines, has yet to fully deliver on its promise.

Experts cited news of an 18-year-old patient who died last September after being injected with genes designed to correct an inherent liver disease during an experimental study at the University of Pennsylvania. That led lawmakers and federal regulators to investigate lapses in gene therapy studies.

Dr. Philip Noguchi, director of the Food and Drug Administration’s division of Cellular and Gene Therapies, addressed issues of safety in gene therapy trials at the June 16 roundtable at the La Jolla Marriott.

Noguchi compared clinical trials to a “marriage license,” where the doctor and sponsor are the parents, and patients are like their children. The responsibility of the doctor is to protect the well-being of the patients. The sponsor checks that trials are run properly.

Clinical trials become especially tricky when the investigator and the sponsor are the same person, he admitted.

Aside from the heavy load bestowed upon a single individual in monitoring patients and filing endless reports, the question whether it is ethical for academic scientists to wear both hats remains.

Typically, a clinical trial is sponsored by a large drug company, which checks that research on benefits and side effects of a therapy are properly disclosed.

Checks And Balances

When a company and the researcher are the same, the impartial analysis is absent.

This absence could potentially lift the safety net of “checks and balances,” Noguchi said.

In the case of 18-year old Jesse Gelsinger, it was reported in the New York Times that researchers had failed to tell the National Institutes of Health about some serious health conditions experienced by patients enrolled in the study.

It was also reported the investigator of the study founded a biotechnology firm in which both he and the university hold stock.

The company reportedly didn’t pay for the study; the National Institutes of Health paid for the study, the New York Times reported.

Still, a newspaper article stated the company had an “indirect interest” in the investigator’s work.

Some critics have long charged that companies involved in gene therapy exaggerated data in hopes of raising money.

After 390 initiated gene therapy studies involving more than 4,000 patients, according to the National Institutes of Health. Researchers achieved their first victory in February.

Finding Success

Scientists in Paris had successfully used gene therapy to replace a defective gene, which saved the lives of several infants.

Gene therapy experts were thrilled , among them Eugene Levin, associate professor at the Scripps Research Institute in San Diego.

“Gene therapy is not easy (and it) is not guaranteed , but very promising,” said Levin, who attended the association’s fourth annual research roundtable.

Gene therapy enthusiasts say the technology , which involves introducing a normal gene into the body to correct a genetic abnormality , could change the way medicine is practiced, opening the door to more specific therapies.

Medicines that could correct genetic defects, prevent heart disease, interfere with the progression of such neurological diseases as Parkinson’s and Alzheimer’s disease and kill cancer cells.

But success won’t come immediately, Levin said.

“People have to understand what may look promising in animals or early clinical trials may turn out to not be as promising,” he said.

Bradford J. Duft, a partner at the law firm of Brobeck, Phleger & Harrison LLP, has found that on Wall Street, patents don’t carry as much weight as some people may have thought.

Duft, who gave a presentation on patents at the event, finds investors pay more attention to products, their success in clinical trials and the product market size than the number of patents companies claim.

He reached the conclusion after comparing the value of gene therapy firms with large portfolios for nine days during early and mid-March.

In early March, biotech stocks were hot.

By mid-March the stock of many biotechs, especially that of gene therapy firms, fell sharply after President Clinton said data in the mapping of human genes should be made public.

The next day, the White House said Clinton’s statement wouldn’t represent a change in government policy.

“I wanted to point out that folks who indicate they have broad patent portfolios didn’t fare so well in the market collapse,” Duft said. “It didn’t protect them from a sustained market cap loss.”

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