Gene therapy oversold, panel says.

Gene therapy holds great promise, but it won't be realized until the field gets back to good basic science, a special advisory panel told Harold E. Varmus, director of the National Institutes of Health in Bethesda, Md., at a meeting Dec. 7. The panel, convened by Varmus earlier this year, noted that scientists and journalists have "oversold" the concept to the public and raised false hopes. Thus far, no human gene therapy has proven effective.

"The public simply doesn't understand that gene therapy is not around the corner," says Stuart H. Orkin of Harvard University Medical School in Boston, who cochaired the panel.

NIH spends approximately $200 million of its $11 billion annual budget on developing techniques to treat disease by replacing defective genes. While Orkin maintains that this investment in the therapy is "about right," he and the panel recommend that more of the money be directed toward laboratory efforts than long-shot studies of patients.

Currently, gene therapy is hamstrung by difficulties in getting the intended gene into a patient's cells (SN: 10/28/95, p.284). Scientists often use viruses in their attempts to transfer a gene, but most of these virus vectors don't insert the gene effectively, and some of them cause side effects such as serious inflammation. Orkin points out that if a researcher can't get the gene into a patient's cell, "you get no information."

The panel also noted that in the rush to try gene therapy, scientists are overlooking opportunities to study disease physiology. Such haste may delay drug development. Orkin noted that over a decade ago, scientists discovered a genetic defect that causes high cholesterol. By studying how this gene regulates cholesterol, they developed a new class of cholesterol-lowering drugs. Had the researchers rushed to gene therapy, those drugs might not exist today.

The current climate of excitement that surrounds gene therapy left the panel concerned that people with genetic diseases would come to expect quick cures for their problems. Pediatrician David Valle of Johns Hopkins Medical Institutions in Baltimore, who serves on the NIH director's standing advisory committee, relates the story of one of his patients who stopped a restricted diet that could save his eyesight because "gene therapy is right around the corner."

The panel also recommended that NIH sponsor interdisciplinary workshops to focus the research on basic science and urged scientists and journalists to inform the public about not only the promise of gene therapy but also its limitations.