A decade after gene therapy, children born with deadly immune disorder remain healthy

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.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.ADA-SCID, oradenosine 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. 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.In the gene therapy approach detailed in the new paper, Dr. 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. Finally, they transplanted the cells back into the children’s bone marrow. The therapy, when successful, prompts the body to produce a continuo us supply of healthy immune cells capable of fighting infections. Because the transplanted stem cells are the baby’s own, there is no risk of rejection.Kohn and his team now report in the journal  Blood that of the 10 children who received the one-time treatment between 2009 and 2012 as part of a phase 2 clinical trial, nine have continued to remain stable. The studyfollows a 2017 pa...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news