NIH Plans More Oversight of Gene Therapy Trials
A thorough new look at what role the National Institutes of Health should be playing in gene therapy clinical trials will be submitted next week to the NIH director, an agency official said here Friday.
Dr. Amy Patterson, director of the NIH’s Office of Biotechnology Activities, said a special committee set up in the wake of a gene therapy patient’s death is expected to submit its report Thursday. Its task was to reassess what kind of oversight the federal health research agency should exert.
“As a result of recent events,” Patterson said during a news conference, “the public has been seeing all of clinical research through the lens of gene therapy.” As a result, the NIH is reexamining its rules and practices governing all clinical trials. It already has decided to take three steps quickly.
First, all researchers engaged in gene therapy trials will be asked to attend a symposium meant to alert scientists to the hazards inherent in experimental treatments.
Second, telephone calls are being made by NIH staff members to the lead scientists and doctors running the 500 or so gene therapy clinical trials already underway. The discussions will cover safety issues and help resolve any questions about NIH rules.
Third, NIH officials are beginning a site-visit program in which agency experts will visit ongoing gene therapy trials. The goal is to help the scientists understand and comply with NIH requirements.
The recent events that Patterson discussed at the third annual meeting of the American Society for Gene Therapy included the death last fall of 18-year-old Jesse Gelsinger of Arizona in a gene therapy trial at the University of Pennsylvania and the discovery that hundreds of “adverse events” in other clinical trials had not been reported to the NIH. Most of these adverse events did not involve the gene treatments that were being given, however, but instead were linked to the patients’ original diseases, a Food and Drug Administration investigation found.
Dr. Inder Verma, incoming president of the Gene Therapy Society, said the young organization “is delighted with the close collaboration” that is emerging between the NIH, the FDA and the society. He noted that “clinical trials all have a certain degree of risk” but emphasized that “safety is paramount.”
“We hope that the guidelines will be practical and doable,” said Verma, a molecular geneticist from the Salk Institute in La Jolla.
Dr. Philip Noguchi, director of Cell and Gene Therapy at the FDA, said the agency has a tendency toward secrecy--it can’t divulge confidential commercial information about drugs under development--but said new efforts are being made to be “more transparent” in its oversight of gene therapy.
