F.D.A. Clears Study to Inject Gene in Parkinson's Patients

By THE ASSOCIATED PRESS

ASHINGTON, Oct. 10 — Genes that make a vital brain chemical will be injected into the brains of 12 patients with Parkinson's disease in a first effort to assess the safety of a treatment intended to slow the progression of the disease and ease its symptoms.

The Food and Drug Administration this week approved a Phase 1 clinical trial of the gene therapy, basing its decision in part on the results of a study in laboratory rats, scheduled to be published on Friday in the journal Science.

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The experiment, to begin this year, will involve patients who have exhausted standard therapy and are in a late stage of the disease, said an author of the study, Dr. Michael G. Kaplitt, a Parkinson's researcher at the Weill Medical College of Cornell University in New York.

Other brain researchers said the gene transfer technique was a promising therapy, but extensive clinical trials would be needed.

"You've got to be cautious with gene therapy," said Dr. Christopher A. Ross, a professor of psychiatry and neuroscience at the Johns Hopkins University School of Medicine. "If they see the same results in humans, it will be an important new approach to therapy."

Other gene therapies that have worked in animals, including fetal cell implants, have failed when tested in humans. Gene therapy in humans has had setbacks, including the death of a teenager at the University of Pennsylvania three years ago and the halting of a trial in France this month when a 3-year-old patient contracted a leukemialike disease.

In the Parkinson's study, the researchers placed into a benign virus a gene that makes GABA, a brain chemical that is deficient in Parkinson's patients. Lack of the chemical causes tremors, a halting walk and a tendency to freeze while in motion.

Using rats with chemically induced Parkinson's, the researchers injected the manipulated virus into the animals' brains.

A co-author of the study, Dr. Matthew J. During of the University of Auckland in New Zealand, said that movement in all the rats improved, "but for 50 percent of them there was a remarkable improvement."

Both authors are paid consultants for Neurologix, a private company that owns the patent rights to the techniques used in the experimental gene therapy. The researchers said their connection with Neurologix complied with their universities' conflict-of-interest policies.