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Leukemia case triggers tighter gene-therapy controls

Nature Medicine volume 8, page 1189 (2002)Cite this article

Trials of gene therapy for severe combined immune deficiency (SCID), a fatal disease caused by deficiencies in immune-cell function, were put on hold in the United States and France following the report that a three-year-old patient treated by Alain Fischer and colleagues at the Necker Hospital in Paris had developed leukemia after being treated with a retroviral vector. Advisors to the British government decided against halting two similar trials in the United Kingdom, but recommended closer monitoring.

In this type of gene therapy, retroviral vectors are used to treat ex vivo a patient's bone marrow stem cells, which give rise to functioning blood cells. The FDA committee met on October 10 to ask whether there were “additional data or measures that clinical investigators need to provide before future and present clinical trials in SCID patients should proceed in the US.” They agreed that monitoring of abnormal activity of certain cells and of the sites in the genome where the retroviral vector integrates should be conducted on an ongoing basis during SCID trials. Salomon dismissed suggestions that this type of monitoring is too cumbersome and expensive. “It needs to be done,” he said.

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    Laura Bonetta

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  1. Laura Bonetta
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Bonetta, L. Leukemia case triggers tighter gene-therapy controls. Nat Med 8, 1189 (2002). https://doi.org/10.1038/nm1102-1189

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