Death of Teen Causes Dismay, Finger-Pointing
Reuters Online Service
Donnerstag, 9. Dezember 1999 07:09:00
Copyright 1999 Reuters Ltd. All rights reserved.@bThe following news report may not be republished or redistributed, in whole or in part, without the prior written consent of Reuters Ltd.
By Maggie Fox, Health and Science Correspondent
WASHINGTON (Reuters) - The death of a teenager undergoing
gene therapy -- the first death blamed directly on the
experimental procedure -- has caused not only dismay, but
finger-pointing and huge debate over what, exactly, killed him.
As researchers calmly laid out all the possible reasons that
gene therapy may have caused the giant immune revolt that killed
18-year-old Jesse Gelsinger, government regulators, the
researchers who did the experiment and Gelsinger's father held
their own, sometimes emotional, news conferences to either
assess blame or defend themselves.
If nothing else, the first day of a three-day meeting
investigating the death illustrates how little is known about
gene therapy, which has yet to cure a single patient. It also
highlighted the confusion that surrounds who regulates what when
it comes to gene therapy, and how much and how soon researchers
have to spill their secrets to regulators.
Gelsinger's father, Paul, blames the Food and Drug
Administration (FDA), one of two agencies assigned to oversee
gene therapy experiments. He accuses the FDA of failing to set
up a system that would have allowed researchers to quickly share
information about "adverse events" like the one that killed
his son.
Jesse Gelsinger had volunteered to take part in an
experimental trial at the University of Pennsylvania using gene
therapy to treat ornithine transcarbamylase deficiency (OTC), a
genetic defect that causes ammonia to build up in the body.
The Penn researchers used a common strategy -- an
adenovirus, known for its ability to sneak into human cells,
genetically engineered so as to weaken it, with a replacement
OTC gene spliced in.
Such experiments are very closely controlled, because
scientists are not sure what effects deliberately introducing a
virus into the body might have. But over nearly a decade in
which thousands of patients have been treated, no deaths had
been blamed on the treatment, until Gelsinger's.
Once the Penn team suspended its trial and notified
authorities, the finger-pointing started. They and other
researchers were accused in media reports of covering up other
adverse events, and event deaths, in gene therapy patients.
The FDA Wednesday said the Penn team failed to give it
timely information about adverse events in the trial, including
evidence that two patients may have had temporary liver damage
from the treatment.
Dr. Kathryn Zoon, director of the FDA's Center for Biologics
Evaluation and Research (CBER), said Gelsinger himself should
not have been included in the trial. "His ammonia levels were
higher than what was in the protocol," she told one news
conference.
But Dr. James Wilson of the Institute of Gene Therapy at
Penn, one of the researchers who worked with 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.
Other researchers defended gene therapy, describing trial
after trial in which patients did become sick but recovered
quickly. Most patients show flulike symptoms right after they
get the genetically engineered virus including fever, chills and
nausea.
Dr. W. French Anderson of the University of Southern
California told the meeting at the National Institutes of Health
(NIH) that he did not believe gene therapy research should be
slowed.
"Cutting-edge medical research is risky," Anderson, who
performed the first gene therapy experiment in 1990, said.
"In trying to develop treatments for deadly diseases like
heart disease and cancer, bad things happen."
He praised Wilson for having "the highest standards" and
called Jesse Gelsinger a hero.
"We need to work together to determine how to develop gene
therapy as safely, as effectively and as rapidly as possible,"
he said.
Discussions later in the week will center on whether
researchers, who often work on behalf of companies jealously
guarding trade secrets, must release information about adverse
events to the NIH for immediate public airing.