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    Wednesday, January 15, 2003

    Gene Therapy Set Back: Another bubble-boy treated with gene therapy has developed leukemia:

    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.


    At least it calls into question the use of gene therapy for this particular disease. Other gene therapies don't necessarily have the same risk:

    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.

    One reason that it might be a particular problem for SCID therapy is that the gene for the disease is very close to a gene that helps prevent cancer. In both cases the cancer gene was disrupted by the gene therapy.
     

    posted by Sydney on 1/15/2003 09:01:00 AM 0 comments

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