By Debbie Strickland

Special To BioWorld Today

WASHINGTON - Three and a half years after a National Institutes of Health (NIH) committee provided "a reality check on gene therapy," the field has responded with more basic research and a more tempered approach to sharing data, NIH Director Harold Varmus told the American Society of Gene Therapy in a keynote address over the weekend.

Varmus formed the ad hoc Orkin-Motulsky committee in the mid 1990s to assess the status of gene therapy in part out of concern about the "hyperbolic claims by overly enthusiastic scientists and the journalists who listen to them.

"It was important that we recalibrate expectations to avoid premature loss of public support," Varmus said.

The committee recommended stepping back to do more basic research on vectors, how gene transfer and expression works, and on disease pathophysiology - advice that researchers have followed.

"The trends visible suggest these recommendations have been taken very seriously," Varmus said. "There's been a significant readjustment in public expectations. Meanwhile, the funding for gene therapy has risen, with nearly a doubling of support - an 80 percent increase - since 1995. There has been no decline in clinical trials, but the rapid escalation has ceased. Moreoever, gene therapy is addressing a wider variety of diseases, as opposed to the multiplicity of trials addressed to cancer in recent years."

Varmus urged more sharing of early data on fundamental matters, to avoid costly and time-consuming, redundant studies. He noted, as a possible step in that direction, his proposal for E-biomed, an Internet site with a two-tier publishing process - one that uses editorial boards for more traditional peer review publication, and one that allows researchers to publish data through a less arduous screening process involving the approval of two credentialed scientists. The site would also include content from participating journals.

A less formal publishing route would prevent much valuable research from being "buried in the basement."

The society's president, James Wilson, echoed Varmus' interest in sharing data, especially in the commercial realm, where pharmaceutical companies often hoard data to maintain a competitive edge.

"We should not compete with one another on these labor-intensive, expensive, often not hugely important studies," he said. "We should compete on novel ideas."

Philip Noguchi, director for cellular and gene therapies at the FDA, added, "Companies ask, 'How can we cut down the time of product development? A lot of it comes from doing redundant work [on questions that are not] product specific."

The American Society for Gene Therapy's second annual meeting, which drew 2,000, ended Sunday.