NIH, FDA Pledge More Oversight after Death in Gene Therapy Experiment

Article excerpt

In the wake of the death of a young man enrolled in a gene therapy experiment, NIH and the Food and Drug Administration (FDA), both of which have authority to oversee gene therapy experiments, have launched two initiatives designed to strengthen safeguards for clinical trials while improving communication within the scientific community.

FDA will now require sponsors of gene therapy trials to routinely submit for agency review their monitoring plans, including a summary of the experience and training of their monitors. The new Gene Therapy Clinical Trial Monitoring Plan will also support the organization of conferences for investigators to discuss monitoring practices with their peers and other professionals in the field. The second initiative is a series of NIH/ FDA-organized Gene Transfer Safety Symposia designed to allow investigators to share and analyze medical and scientific data resulting from gene transfer research.

After 18-year-old Jesse Gelsinger died while undergoing a gene therapy treatment at the University of Pennsylvania, NIH acknowledged that it had failed to track adverse events during gene therapy trials. Members of Congress have strongly criticized NIH's lapses. In a hearing held by the Senate Health, Education, Labor, and Pensions subcommittee, Senator Bill Frist (R-Tenn.), the subcommittee chair and a heart transplant surgeon, said that there is "a need for vigilant oversight to ensure patient safety. If we expect patients to participate in moving science forward, then we must be assured that gene therapy clinical trials are safe."

Much of the hearing focused on why researchers had failed to notify NIH of adverse events during gene therapy trials as required by federal guidelines. But there was also extensive discussion of whether scientists are providing patients with enough information to understand the potential risks of participating in a clinical trial.

The most riveting testimony came from the father of Jesse Gelsinger. Paul Gelsinger testified that he wasn't given information regarding adverse events in prior experiments conducted by the university and in the private sector-- information that would probably have influenced their final decision to participate. …