Man Blames Government in Son's Gene Therapy Death
Reuters Online Service
Mittwoch, 8. Dezember 1999 23:40:00
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By Maggie Fox, Health and Science Correspondent
BETHESDA, Md. (Reuters) - The father of a teenager who died
during an experimental gene therapy trial accused the government
on Wednesday of failing to make public information that might
have saved his son.
The government regulators he accused, meantime, said they
had found serious problems with the experiment itself, and said
the researchers who treated the youth withheld what could have
been critical information from them.
The National Institutes of Health (NIH) and the Food and
Drug Administration (FDA) are investigating the death of Jesse
Gelsinger, 18, in September, and considering whether to tighten
government oversight over gene therapy researchers.
Gelsinger had volunteered to take part in an experimental
trial at the University of Pennsylvania using gene therapy to
treat ornithine transcarbamylase deficiency (OTC), an inherited
genetic defect that causes ammonia to build up in the body.
The idea behind this treatment, and other gene therapy
experiments, is to replace a faulty gene with a healthy one.
Often a virus is used to carry the new gene into cells.
Gelsinger became severely ill within hours of his treatment,
and died from acute respiratory syndrome caused by massive organ
failure -- an apparent result of a huge over-reaction by his
immune system.
Researchers are checking now to see if the virus used in
Gelsinger, called an adenovirus, may have somehow caused his
death, even though it had been weakened.
The FDA said it had already found several problems with the
University of Pennsylvania experiment.
Dr. Kathryn Zoon, director of the FDA's Center for Biologics
Evaluation and Research (CBER), which oversees gene therapy
trials, said Gelsinger himself should not have been included in
the trial. "His ammonia levels were higher than what was in the
protocol," she told a news conference, referring to rules that
govern clinical trials.
She said two other patients also had "grade 3 toxicity" --
meaning suggestions of liver damage -- which was not reported
"in a timely manner".
It is not yet clear whether Gelsinger should have been kept
out of the trial, she said. "At this point in time, our results
are very preliminary," she said. "We are doing a complete
investigation."
Dr. James Wilson of the Institute of Gene Therapy at the
University of Pennsylvania, one of the researchers who worked on
the experiment that included Gelsinger, denied he withheld any
information from the FDA. "We have coordinated with the FDA on
all aspects of analysis of the data," he told a separate news
conference.
"Our interpretation of data that was made available to the
FDA 8 months prior to Jesse's ... death did not suggest to us
that the trial should be modified at that point or halted."
He said Gelsinger did have high ammonia levels, but his team
chose to use drug treatment to lower them and that this
treatment made the teenager eligible for the trial.
Gelsinger's father Paul, a 47-year-old handyman, said he
fully trusted his son's doctors. "I have touched souls with
this man and there nothing wrong with Jim Wilson," Gelsinger
said at his own news conference.
He blames the FDA and wants tighter government oversight of
all gene therapy trials.
Gelsinger has pored over records of meetings of the
committees at FDA and NIH, which oversee gene therapy trials. He
said promised cooperation between the two agencies never
occurred and accused the FDA of letting industry pressure it
into speeding up gene therapy trials without properly checking
the danger. "Why are we allowing industry to influence our
government?" he asked.
He said problems had earlier been unearthed in a gene
therapy trial being conducted by Schering-Plough (SGP.N) -- one
that was stopped after his son's death -- which may have halted
the Pennsylvania trial. The problems included one suspected
stroke, later diagnosed as a serious migraine, and two other
strokes.
He said he did not know whether he would have asked his son
to leave the trial had he known of these "adverse events."
"It's not up to Dr. Wilson to stop his protocol based on
that. It is up to the FDA or RAC to recommend that the protocol
be stopped. If they don't have the information, they can't do
anything about it," Gelsinger said.