![\"\"](\"https:\/\/www.europesays.com\/us\/wp-content\/uploads\/2025\/11\/download-1024x683.jpg\")
6 of the children treated for SCID living their best lives \u2013 credit, UCLA, supplied by the parents<\/p>\n 6 of the children treated for SCID living their best lives \u2013 credit, UCLA, supplied by the parents<\/p>\n
Between 2012 and 2017, 62 babies and toddlers were treated with a genetic therapy for severe combined immunodeficiency, known colloquially as the \u201cBubble Boy disease.\u201d<\/p>\n
In 2021, GNN reported<\/a> on the results of the trial\u2014that by 2019, 95%, or all but two of the young patients, showed complete immune system reconstruction. Now, the long-term follow-up results are in\u2014still 95% effective.<\/p>\n \u201cThe durability of immune function, the consistency over time and the continued safety profile are all incredibly encouraging,\u201d said<\/a> the study\u2019s senior author, Donald Kohn, MD, a pediatric transplant physician at Univ. of California LA, where the trial was conducted.<\/p>\n Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, is caused by mutations in the ADA gene, which creates an enzyme essential for immune function. For children with the condition, day-to-day activities like going to school or playing with friends can lead to dangerous, life-threatening infections. If untreated, ADA-SCID can be fatal within the first two years of life.<\/p>\n In 1984, SCID become suddenly well-known in America because of \u201cthe boy in the bubble,\u201d David Vetter, who received a special spacesuit from NASA to allow him to leave his total medical isolation. Despite this, Vetter passed away from an infection at age 12.<\/p>\n The gene therapy method involves first collecting some of the child\u2019s blood-forming stem cells in their bone marrow, which have the potential to create all types of blood and immune cells.<\/p>\n Next, using an approach developed by the research team at UCLA with help from the UK\u2019s Great Ormond Street Hospital and University College London, a new copy of the ADA gene is delivered into the stem cells by a modified lentivirus, or \u201cviral vector.\u201d The corrected cells are then returned to the child\u2019s body, where they are intended to produce a continual supply of healthy immune cells capable of fighting infection.<\/p>\n \u201cBetween all three clinical trials, 50 patients were treated, and the overall results were very encouraging,\u201d said Kohn back in 2021. \u201cAll the patients are alive and well, and in more than 95% of them, the therapy appears to have corrected their underlying immune system problems.\u201d<\/p>\n This brand new study, also published in the New England Journal of Medicine<\/a>, represents the largest and longest follow-up of a gene therapy of this kind to date, with 474 total patient-years of follow-up data\u2014including five patients who received the therapy a decade ago.<\/p>\n For the 59 patients successfully treated, immune function has remained stable beyond the initial recovery period, with no treatment-limiting complications reported.<\/p>\n With support from the California Institute for Regenerative Medicine, the UCLA team is now working to complete the steps necessary to apply for FDA approval.<\/p>\n MORE INFANTILE DISEASES CURED: <\/strong>CRISPR Used to Remove Extra Chromosomes in Lab Model of Down Syndrome and Restore Cell Function<\/a><\/p>\n \u201cOur goal is to have this therapy FDA-approved within two to three years,\u201d Kohn said. \u201cThe clinical data strongly supports approval\u2014now we need to demonstrate that we can manufacture the treatment under commercial pharmaceutical standards.\u201d<\/p>\n When GNN originally reported on the 2021 study, a slide of photos was released of 6 of the children treated, taken at various times between 2012 and 2017. Accompanying the new study are slides of the same children, not necessarily all grown up, but grown up, and living their best healthy lives.<\/p>\n 11-year-old Eliana Nachem of Fredericksburg, Virginia, is starting sixth grade with dreams of becoming an artist. It\u2019s a remarkably ordinary life that once seemed impossible.<\/p>\n![\"\"](\"https:\/\/www.europesays.com\/us\/wp-content\/uploads\/2025\/11\/Screenshot-2025-11-04-125140-1024x613.jpg\")
Screenshot, GNN<\/p>\n![\"\"](\"https:\/\/www.europesays.com\/us\/wp-content\/uploads\/2025\/11\/Eliana2-1024x683.jpg\")
Eliana Nachem received gene therapy at 10 months (left) and is 11 now (right), free from living in medical isolation \u2013 credit, supplied by Caroline and Jeff Nachem<\/p>\n