The advisory committee that monitors human genetic therapy experiments for the US government plans to tighten its guidelines early next month on the reporting of deaths and adverse outcomes of gene therapy trials.
The move by the Recombinant DNA Advisory Committee (RAC) of the National Institutes of Health (NIH) follows attempts made over several years by researchers and drug companies to keep details of about six deaths that occurred in gene therapy experiments from becoming public. The six deaths occurred in heart studies headed by two leading gene scientists - Ronald Crystal of the New York Hospital-Cornell Medical Center in Manhattan and Jeffrey Isner of Tufts University in Boston. They are racing to be the first to grow new blood vessels around blocked ones as an alternative to bypass surgery. Crystal and Isner said that unlike the widely reported death of a teenager at the University of Pennsylvania in September, they are certain that the deaths in their study were not directly caused by gene therapy but by problems associated with the underlying illnesses. Details of these deaths are emerging because federal officials appealed for researchers across the US to report any undisclosed deaths or illnesses after the Pennsylvania death. That fatality is believed to be the first to directly result from gene therapy.
Current RAC guidelines require researchers to report any serious adverse events to the NIH's Office of Recombinant DNA Activities (ORDA), but such reports can be marked confidential and therefore kept off the RAC's public agenda. Crystal says that he asked for secrecy in the reporting of a number of cases to protect patients' families and added that he was 'very concerned about releasing confidential patient information in the context of a group that has neither the appropriate secrecy nor the expertise and manpower to evaluate it. NIH officials say that these are not isolated cases. Gene therapy was once mainly funded by federal money but is now almost totally reliant on industry. This has led to an increase in researchers and their funders asking for protocols to be kept confidential. But the RAC says that secrecy surrounding deaths and side-effects go against the spirit of a young science that needs to gain public confidence through openness.