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. Last Updated: 07/27/2016

Gene Therapy Deaths Kept Hidden From Public

WASHINGTON -- Scientists and drug companies have failed to notify the National Institutes of Health about six deaths that occurred in gene therapy experiments in the past 19 months, keeping details of the deaths from becoming public, according to interviews with researchers and federal officials.

The deaths are the first in gene therapy to come to light that were purposely withheld from the NIH, one of two federal agencies charged with overseeing the safety of the controversial field of medical research, which seeks to cure diseases by giving patients new genes.

The six deaths occurred in heart studies headed by two leading gene researchers, Ronald Crystal of the New York Hospital-Cornell Medical Center in Manhattan and Jeffrey Isner of Tufts University in Boston. The two are racing to be the first to grow new blood vessels around blocked ones as an alternative to bypass surgery.

Crystal and Isner said they believe the fatalities were not caused by the gene therapy but by complications stemming from the patients' illnesses. Therefore, they argued, federal regulations don't require them to notify the NIH.

The researchers said they reported the deaths to the Food and Drug Administration.

But NIH officials in the federal office overseeing gene therapy are adamant that even deaths not believed to have been caused by the therapy must be reported to the NIH and made public.

The FDA can suspend a study if it determines the therapy being tested is dangerous, but the agency only makes such information public if the therapy is approved or with the permission of the study's sponsor. Most of the new deaths are coming to light because federal officials put out a plea for gene researchers across the country to report any undisclosed deaths or illnesses, after the death of a teenager at the University of Pennsylvania. His death is thought to be the first directly caused by gene therapy.

Federal regulations require researchers to report deaths and serious illnesses of patients enrolled in gene therapy experiments not only to the FDA, but also to the NIH for public review.

But drug companies and scientists with a stake in their research are challenging the interpretation of that rule. They are filing reports with demands for confidentiality or maintaining they don't have to file them with the NIH at all.

Asked why he hadn't reported his deaths to the NIH, Isner said it was an oversight and it wasn't clear he had to.

Crystal said Parke-Davis has assumed responsibility for reporting deaths to the appropriate agencies. However, he added that while he supported the reporting of deaths and illnesses to the NIH, he did not consider it a legal requirement.

At a meeting of the Recombinant DNA Advisory Committee (RAC), the NIH group that reviews gene therapy experiments and collects reports of serious side-effects and deaths, RAC members learned that Schering-Plough had demanded confidentiality for three recently filed reports of serious patient illness during gene therapy trials.