THE DISCLOSURE of worse-than-shoddy research practices at the University of Pennsylvania's Institute for Human Gene Therapy sends a cold shiver through the field. Even ardent supporters of gene therapy--the promising but as yet unrealized hope of curing people's inherited disorders by giving them new genes--are hard-pressed to explain why a well-regarded research institute would cut corners, ignore safety checks, fail to file required documents with the government and otherwise appear to disregard established procedures for the protection of human subjects in experiments.
Critics have long expressed concern (which we share) that gene therapy research is vulnerable to abuse because it is mostly backed by venture capital. Researchers who are also investors, such as Penn principal investigator James Wilson, may be tempted to oversell the promise of experiments and keep "adverse events" quiet lest they depress stock prices. Dr. Wilson has said financial interest plays no role in his research decisions. And no federal conflict-of-interest rule prevents public research money from being used together with private funding; such mingling is common.
The Food and Drug Administration, investigating after 18-year-old Jesse Gelsinger died in a Penn experiment, has now shut down all seven pending trials in the institute, including attempts to cure cystic fibrosis and breast cancer. The FDA cites 18 violations that include failure to inform patients of risks, failure to follow the trial's own rules as to which patients (including Mr. Gelsinger) were healthy enough to qualify as subjects, failure to notify the FDA in timely fashion of serious side effects and failure to halt the experiment as required when four patients suffered those side effects.
This is all the more shocking when you consider that these researchers were working in an area where even the most meticulous and conscientious experiments carry risk: Experiments with gene therapy frequently involve people already suffering life-threatening conditions, who may be more vulnerable to unexpected side effects. All this calls, at the very least, for scrupulous reporting of risks to the patients concerned, backed up by strong oversight.
Further investigation--Penn has said it will form an outside committee, while Sen. Bill Frist intends to hold hearings--will likely probe the degree to which the FDA missed danger signs in confidential reports, waking up only when public reports emerged through NIH. Biotech companies have been calling for less disclosure to NIH, but this disaster argues for more.