A small clinical trial using gene therapy to improve immune system function in infants diagnosed with a rare and life-threatening immunodeficiency disease saw effective and promising results, according to the National Institutes of Health.
The study was developed by scientists at the NIH's National Institute of Allergy and Infectious Diseases and the Memphis, Tenn.-based St. Jude Children's Research Hospital to treat infants with X-linked severe combined immunodeficiency (X-SCID), an inherited disorder caused by mutations in the IL2RG gene.
Eight infants at St. Jude and the Benioff Children's Hospital of the University of California, San Francisco, ranging in age from 2 months to 14 months and lacking optimal genetically matched donors, had normal copies of the IL2RG gene inserted into their stem cells. Up to two years after the treatment, the infants' immune system function had reportedly improved substantially and they were growing normally, though one of the patients required a second transfusion of the genetically altered stem cells to achieve these results.
According to the NIH, this form of gene therapy is a safer and more effective approach than previous X-SCID treatments. Scientists have also seen similarly promising results from an ongoing companion study of the treatment in older children and young adults.
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