![]() “We hope someday we’ll be able to say that these results last for 80 years.” ![]() “What we saw in the first few years was that this therapy worked, and now we’re able to say that it not only works, but it works for more than 10 years,” said Kohn, senior author of the study and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. The study follows a 2017 paper, also published in Blood, on the initial success of the treatment in those nine children. Kohn and his team report in the journal Blood that of the 10 children who received the one-time treatment between 20 as part of a phase 2 clinical trial, nine have continued to remain stable. Because the transplanted stem cells are the baby’s own, there is no risk of rejection. The therapy, when successful, prompts the body to produce a continuous supply of healthy immune cells capable of fighting infections. Finally, they transplanted the cells back into the children’s bone marrow. Donald Kohn of UCLA and his colleagues removed blood-forming stem cells from each child’s bone marrow, then used a specially modified virus, originally isolated from mice, to guide healthy copies of the ADA gene into the stem cells’ DNA. In the gene therapy approach detailed in the new paper, Dr. For babies with the disease, exposure to everyday germs can be fatal, and if untreated, most will die within the first two years of life. They now report that the effects of the therapy appear to be long-lasting, with 90% of patients who received the treatment eight to 11 years ago still disease-free.ĪDA-SCID, or adenosine deaminase–deficient severe combined immunodeficiency, is caused by mutations in the gene that creates the ADA enzyme, which is essential to a functioning immune system. Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder.
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