THE TRAGIC death of 18-year-old Jesse Gelsinger has focused intense attention and concern on the field of gene therapy. But a three-day conference last week at the National Institutes of Health, which aired abundant and sobering information about apparent shortcomings in the University of Pennsylvania experiment in which Mr. Gelsinger died, showed how far short that concern falls of a consensus on the best ways to monitor this volatile area of research.
The conference showcased a long-running conflict. On one side are those seeking more oversight and public disclosure--including disclosure of the kind of "serious adverse events" that might, if revealed early enough, have led to the halting of the Penn experiment before Mr. Gelsinger died. On the other side are those in the biotech industry who think the current level of disclosure is already too high and a burden on a burgeoning field.
Biotech industry advocates argued at the conference that mandatory detailed reporting of all "adverse events"--deaths or injuries during an experiment, whether obviously related to it or not--runs the risk of revealing proprietary information or compromising patient privacy. Companies also worry that public disclosure of problems during an experiment will scare off sponsors or drive down stock prices, choking off investment in new therapies.
The Food and Drug Administration, which oversees all drugs, including gene therapies, requires comprehensive reporting on drug trials but keeps the information confidential to protect manufacturers' interests. The case for disclosing more information about gene therapy experiments goes back to the field's early days, when the NIH created an advisory committee to provide a second, more public level of scrutiny and to help information about possible problems travel faster among researchers. (Such public disclosure is the norm in NIH-funded medical research, most of which takes place in academia.) In the wake of the Gelsinger death, and of reports that companies had failed to report as many as four deaths during other gene experiments, the NIH has sought to make these public reporting requirements more explicit. The biotech companies have redoubled their efforts to shield some information.
The conference's examination of the Penn experiment suggested that mistakes went beyond failures to make public disclosure--that investigators' reports to the FDA were incomplete as well. Genetic research may not be intrinsically more dangerous than other drug trials. But any regulatory scheme must take account of precisely the kinds of worst-case scenarios the Gelsinger case represents.
Though it's important to stay alert to the needs of the private sponsors who mostly fuel the biotech field, those needs are outweighed by the pressing public need to know as much as possible about gene therapy experiments as they unfold. In the long run, more disclosure will bolster confidence in the field, if in fact such confidence is warranted.