says youth who died in gene therapy trial should not have
received the treatment September 14, 2003
Maryland (CNN) -- Food and Drug Administration officials
investigating the death of 18-year-old Jesse Gelsinger
in a gene therapy trial are accusing doctors at the
University of Pennsylvania of failing to halt the trial
and alert the FDA of serious side effects suffered by
two patients earlier in the trial.
Gelsinger, investigators say, should not have been
given the experimental treatment, not only because
of dangerously high levels of ammonia in his blood,
but also because of the adverse side effects suffered
by others in the trial.
"The entry levels for
ammonia (readings) were not met," said Kathryn
Zoon, an FDA investigator. Gelsinger is considered
the first person to die while undergoing gene therapy.
Zoon said the Pennsylvania researchers
failed to notify the agency of two other patients
who suffered liver damage severe enough to stop the
trial. But Rebecca Harmon, a University of Pennsylvania
spokeswoman, said the FDA was properly informed as
Doctor defends therapy trial
At a meeting reviewing this and
other gene therapy trials at the National Institutes
of Health, Dr. Jim Wilson, one of the doctors treating
Gelsinger, said the patient's ammonia levels were
being controlled medically.
CNN's Eileen O'Connor reports
on the FDA gene therapy finding.
Wilson also said the
FDA was kept informed. "Our interpretation of
the data made available to the FDA eight months prior
to Jesse's therapy and death did not suggest to us
that the trial should be modified at that point or
halted," Wilson said.
Researchers at the university, where
the experiment was part of a series of gene therapy
trials, are to present their findings when the NIH
hearing resumes Thursday.
Gelsinger, of Arizona, was a participant
in the clinical trial conducted to study gene therapy
for a rare disorder called ornithine transcarbamylase
deficiency (OTC), an inherited liver disease that
causes life-threatening levels of ammonia to build
up in the blood.
Virus carried corrective
As part of that trial, his liver
was injected with a virus carrying a corrective gene.
The virus used in the trial usually
causes flu-like symptoms, but in Gelsinger the symptoms
escalated into respiratory distress. He died of multiorgan
failure in September.
The FDA approved the trial with
certain parameters, including that the trial be stopped
if patients had serious side effects.
Harmon said the FDA was informed
about the two other patients, as was Gelsinger's father.
Paul Gelsinger said he does not
blame the doctors for his son's death, but the FDA,
for failing to act on the information of adverse side
effects from earlier trials.
The elder Gelsinger said his son's
doctors told him about the other two patients but
indicated one may have had complications which would
have caused the side effects.
The FDA says these are preliminary
results, and the investigation is not complete.
Medical Correspondent Eileen O'Connor
and The Associated Press contributed to this report.