GENET archive


8-Humans: Second boy receiving gene therapy develops cancer

genet-news mailing list

-------------------------------- GENET-news --------------------------------

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: ------------------

Second Boy Receiving Gene Therapy Develops Cancer
FDA Responds to Event in France by Suspending 27 U.S. Studies Involving 
Similar Techniques

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 
blood cells.

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.



|                   GENET                     |
| European NGO Network on Genetic Engineering |
|                                             |
|             Hartmut MEYER (Mr)              |
|               Kleine Wiese 6                |
|           D - 38116 Braunschweig            |
|                 Germany                     |
|                                             |
| phone:  +49-531-5168746                     |
| fax:    +49-531-5168747                     |                      
| mobile: +49-162-1054755                     |
| email:                   |