The U.S. Food and Drug Administration (FDA) has begun proceedings that could disqualify gene therapy researcher James Wilson of the University of Pennsylvania in Philadelphia from conducting any future clinical trials. Wilson oversaw the trial in which 18-year-old Jesse Gelsinger died after a genetically altered virus was injected into his liver (Science, 17 December 1999, p. 2244).

Disqualification is the harshest penalty the FDA can impose on an investigator. It bars a researcher from receiving drugs for use in clinical trials--in effect, preventing that investigator from administering experimental drugs to patients. In a 30 November letter to Wilson, the FDA stated that Wilson had "repeatedly or deliberately violated regulations governing the proper conduct of clinical studies." The agency wrote that Wilson and his colleagues enrolled patients who were ineligible for the trial, did not monitor patients properly, did not halt the trial when patients experienced serious side effects, and failed to inform patients that a trial of a similar drug had severely sickened monkeys.

"This is obviously a very serious matter," the university said in a brief statement. "We know that Dr. Wilson understands its importance, is reviewing the letter carefully and intends to respond in a timely way." Wilson has 30 days to reply to the FDA's letter. After reviewing Wilson's response, FDA administrators will make a final decision.

The FDA has also issued warning letters--a less severe sanction--to two of Wilson's collaborators in the study--Steven Raper of the Institute for Human Gene Therapy and Mark Batshaw of Children's National Medical Center in Washington, D.C.

This is a "drastic" step, says Inder Verma of the Salk Institute for Biological Studies in La Jolla, California, who headed a special working group at the National Institutes of Health that investigated the Gelsinger trial. But Savio Woo, a gene therapy researcher at Mount Sinai School of Medicine in New York City and past president of the American Society of Gene Therapy, says that vigorous FDA oversight will strengthen gene therapy research.

--GRETCHEN VOGEL

Related sites

Text of FDA letter to Wilson
Text of warning letter to Batshaw
Text of warning letter to Raper
American Society of Gene Therapy