WASHINGTON, Feb 02 (Reuters Health) -- At a congressional hearing
Wednesday looking into reporting irregularities by gene therapy researchers, a
National Institutes of Health (NIH) official said the agency had recently
received reports of 40 new adverse events that occurred in the 157 or so studies
supported by NIH in 1999.
These reports were made only after the NIH in November urged
federally-supported researchers to comply with rules requiring immediate
notification of adverse events, said Amy Patterson, director of the NIH's Office
of Biotechnology Activities.
Patterson testified at a hearing held by Sen. Bill Frist's (R-TN)
Subcommittee on Public Health of the Committee on Health, Education, Labor, and
Pensions.
The new reports were in addition to 100 the NIH already had for 1999,
Patterson said. And they come on top of 652 reports received as a result of a
letter sent in October to researchers working on adenovirus-related protocols.
Frist was dumbfounded. "The fact that NIH received 652 previously
unreported serious adverse events is inexcusable," he said. "Clearly, our
oversight system is failing."
The NIH is investigating why reports were not submitted on a timely basis.
The 652 events were not just for one year, but for 7 years, and encompassed
roughly 372 studies that had been funded by the NIH over the years, Patterson
said.
Frist asked why researchers would suddenly begin reporting.
"I think the death of Jesse Gelsinger galvanized the scientific
community," Patterson said. Gelsinger was the Arizona teenager who died
September 17, 1999, four days after receiving an infusion of corrective genes
introduced with an adenovirus in a University of Pennsylvania-led study.
The trial was halted by the Food and Drug Administration (FDA) in
mid-January, after an agency inspection found substantial lapses in reporting,
record-keeping, and informed consent.
Gelsinger's father, Paul, testified that after his son's death, he was
belatedly told of problems with the study, including adverse events and the
deaths of some monkeys in similar trials.
"I learned, after Jesse's death, that Penn had removed from the
information they gave Jesse and me any reference to deaths of monkeys, which had
previously appeared in their documents," said a tearful Gelsinger.
He called on the government to increase trial oversight, and to ensure
patients had enough data to make truly informed decisions about participating in
studies.
NIH's Patterson said the agency is building a website for the public and
scientists that will provide information on gene therapy studies, including
adverse events.
In addition, the FDA will soon propose that researchers disclose more to
the public about the dangers of gene therapy, said Jay Siegel, director of the
FDA's Office of Therapeutics Research and Review.
The FDA had been receiving adverse event reports as required, but its
rules are different than NIH's, Siegel said. And, the agency does not generally
release such reports to the public unless there is an imminent health threat.
