Gene therapy for cystic fibrosis OK'd

December 04, 1992|By Los Angeles Times

WASHINGTON -- A scientific panel gave its approva yesterday for experimental gene therapy for cystic fibrosis patients, marking the first time the revolutionary but still-developing technique will be tried against so prevalent and lethal a disease.

The unanimous approval by the federal Recombinant DNA Advisory Committee underscores the accelerating pace of gene therapy experiments.

Until now, they had been tried only against a handful of extremely rare genetic diseases and a few types of cancers for which there are no cures.

"This is a major, watershed event," said Dr. Nelson A. Wivel, executive secretary of the committee.

The decision yesterday comes only three years after scientists discovered the genetic defect responsible for cystic fibrosis, an inherited respiratory disease that currently afflicts about 30,000 Americans and kills most of its victims before age 30.

And it was only three months ago that scientists in North $H Carolina learned how to breed mice that develop cystic fibrosis -- a major breakthrough that also will expedite the search for a cure.

Although the experimental gene treatments may not begin until after final approval is received from the Food and Drug Administration and the National Institutes of Health, it is highly unlikely either organization will reject the panel's decision.

The treatments are tentatively scheduled to start in 1993 with an initial group of 10 patients. The procedure will involve replacing defective cystic fibrosis genes in lung cells with normal genes.

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