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Gene Therapy Shows Potential for Severe Immunodeficiency

Newer treatment seems to have less risk of leukemia, researchers say

Gene Therapy Shows Potential for Severe Immunodeficiency

THURSDAY, Oct. 9, 2014 (HealthDay News) -- A new form of gene therapy may offer a safe and effective way to treat severe combined immunodeficiency (SCID), according to research published in the Oct. 9 issue of the New England Journal of Medicine.

Over a decade ago, trials in Europe showed that gene therapy worked -- but five of the 20 children treated developed leukemia within two to five years, according to background information in the study. In the new study, researchers refined the gene therapy approach to hopefully negate the leukemia risk.

In the new trial, all of the boys had X-linked SCID, a form of the disease that only causes disease symptoms in boys. Eight of nine children who received the therapy are still alive one to three years later, the investigators report. And so far, none has developed leukemia.

It's too early to say the therapy carries no leukemia risk, cautioned researcher David Williams, M.D., a pediatric hematologist/oncologist at the Dana-Farber Cancer Institute and Boston Children's Hospital. "We'll continue to follow these children for 15 years," he told HealthDay. But based on the early results, he noted, the tweaked gene therapy appears as effective at generating a functional immune system as the earlier form of treatment.

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