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Health Agencies Update
April 27, 2005

Gene Therapy Trials on Hold

JAMA. 2005;293(16):1966. doi:10.1001/jama.293.16.1966-d

In a meeting held last month, a US Food and Drug Administration committee put a clinical hold on three experimental trials that use retrovirus-mediated gene transfer to treat X-linked severe combined immunodeficiency disease (X-SCID), which is caused by a defect in the GammaC gene.

The committee convened after a French boy recently became the third child to develop leukemia after receiving gene therapy for treatment of the condition. The child was one of a number of children with X-SCID treated by French researchers, who put a healthy copy of GammaC into a modified mouse leukemia virus. The virus is capable of inserting the gene into embryonic blood cells that are then infused into the patient. Two of the group’s patients, including the child who died recently, had developed leukemia several years ago, and another patient has apparently developed the cancer as well.

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