8-Humans: Second boy receiving gene therapy develops cancer
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TITLE: Second Boy Receiving Gene Therapy Develops Cancer
FDA Responds to Event in France by Suspending 27 U.S. Studies
Involving Similar Techniques
SOURCE: The Washington Post, USA, by Rick Weiss
DATE: Jan 15, 2003
------------------ archive: http://www.gene.ch/genet.html ------------------
Second Boy Receiving Gene Therapy Develops Cancer
FDA Responds to Event in France by Suspending 27 U.S. Studies Involving
For the second time in four months, a child treated with an experimental
gene therapy in France has developed a form of leukemia apparently caused
by the treatment.
The new cancer case, in a boy who was given new genes to cure a severe
immune system deficiency, undercuts scientists' initial hopes that the
first case was a fluke, and calls into question the value of the radical
treatment, which had been promoted as the first successful use of DNA to
cure a disease.
In response, the Food and Drug Administration yesterday suspended as "a
precautionary measure" more than two dozen U.S. gene therapy studies that
involve techniques similar to those in the French experiment. Three U.S.
gene therapy studies that even more closely resemble the French experiment
had already been on hold since the first leukemia case came to light in
The new cancer case is a serious blow for an experimental field that has
struggled for a dozen years to produce its first cure and which suffered a
terrible setback in 1999 with the death of an American patient, Arizona
teenager Jesse Gelsinger. The approach involves the delivery of new genes
to take over for missing or broken ones.
Gelsinger died in a gene therapy experiment at the University of
Pennsylvania that was later heavily criticized by the FDA for violating
basic safety rules. But until the recent leukemia cases, gene therapy had
at least seemed safe when used in accordance with approved protocols. Now,
scientists said, that sense of safety has been undermined.
"When the first leukemia showed up, we as a community were certainly upset
and concerned about the patient," said Joe Glorioso, president of the
American Society of Gene Therapy and chairman of molecular genetics and
biochemistry at the University of Pittsburgh Medical Center. "But when the
second event happened, that really is a red flag."
Nonetheless, Glorioso and others noted, no cases of leukemia have been
documented in any of the thousands of other people who have received some
form of gene therapy, which suggests that the risk may be specific to this
particular disease or treatment plan. Researchers said they held out hope
that they will learn how to modify the treatment so it can still be used in
children born with the boys' life-threatening disorder -- severe combined
immunodeficiency, or SCID. Affected children can die from even minor
infections, and the only cure -- a bone marrow transplant from a well-
matched donor -- is unavailable for many.
"We continue to see gene therapy as a promising therapy for all those who
have not benefited from current technologies," said Philip Noguchi, acting
director of the FDA's office of cellular, tissue and gene therapies, which
regulates gene therapy experiments in this country.
Noguchi praised the leader of the French study, Alain Fischer of the Necker
Hospital in Paris, for promptly informing the FDA about the new leukemia
case last month. The setback became public yesterday when the FDA placed a
"clinical hold" on all U.S. gene therapy experiments that, like the French
experiment, use retroviruses to deliver new genes into blood stem cells.
The FDA hold demands that studies already underway be stopped and
enrollment of new patients be suspended.
Noguchi estimated that 27 such trials have been approved in this country
and are at various stages of patient enrollment or testing. The agency will
assess the latest data at a Feb. 28 meeting, he said, but it may lift the
hold on some studies before then if it believes patients are more likely to
be harmed by the shutdown of a study.
Few details about the new case were available yesterday. The boy was one of
about 11 children treated by Fischer in the past several years, nine of
whom Fischer has said appear to be cured of their immune system disease.
The treatment uses retroviruses to deliver a crucial immune system gene to
Glorioso said yesterday he had been told that the latest leukemia case
involved a boy who was admitted to a hospital in Louisiana, suggesting he
may be an American who was treated in Paris.
In both leukemia cases, tests showed that the cancer was apparently
triggered when the newly delivered gene disrupted a nearby gene whose job
is to help prevent cancer.
Both boys have been treated for their leukemia and are "stable," Noguchi
Staff researcher Lucy Shackelford contributed to this report.
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