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Gene Tx May Benefit Children, Teens With Wiskott-Aldrich Sx

Patient's own blood stem cells are removed to correct the WAS gene, then injected back into the patient

THURSDAY, April 23, 2015 (HealthDay News) -- Gene therapy may benefit children and teens with Wiskott-Aldrich syndrome, a rare immunodeficiency caused by mutations in the WAS gene, according to a small new study published in the April 21 issue of the Journal of the American Medical Association, a theme issue on child health.

Researchers looked at a therapy in which the patient's own blood stem cells are removed to correct the WAS gene. The blood stem cells are then injected back into the patient. The gene therapy was performed in seven children and teens with Wiskott-Aldrich syndrome. They were then followed for nine to 42 months. One patient died of a preexisting infectious disease. The six surviving patients had fewer infections, less severe eczema, and a decrease in autoimmune symptoms. No severe bleeding episodes occurred after the therapy, and hospitalizations were reduced from roughly 25 days in the two years before treatment to zero in most cases in the two years after treatment, according to the researchers.

Because this was such a small study, the researchers said they "cannot draw conclusions on long-term outcomes and safety." Additional trials are needed, along with further follow-up of these patients and participants in a similar study reported last year, they noted. However, the study provides evidence that this type of gene therapy benefits patients with severe Wiskott-Aldrich syndrome, said the authors of an accompanying editorial in the journal.

"At a time when many are championing personalized medicine, no advance is as representative of that fundamental biological approach as gene therapy," Harry Malech, M.D., of the U.S. National Institutes of Health in Bethesda, Md., and Hans Ochs, M.D., of the University of Washington in Seattle, write in the accompanying editorial.

Research funded by Genethon, a French company that develops gene therapies.

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