A groundbreaking gene therapy has restored and maintained immune system function in 59 out of 62 children born with a rare and deadly genetic immune disorder called ADA-SCID. The experimental treatment, developed by researchers at UCLA, University College London, and Great Ormond Street Hospital, uses a modified lentivirus to deliver a healthy copy of the ADA gene, allowing corrected stem cells to produce healthy immune cells capable of fighting infections.
The therapy has shown remarkable durability and safety, with no treatment-limiting complications reported beyond the initial recovery period. The study, published in the New England Journal of Medicine, 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.
Researchers have made significant advancements in accessibility by developing a frozen preparation method for corrected stem cells, allowing patients to receive treatment locally without having to travel long distances. The team is now working towards FDA approval within two to three years, citing strong clinical data and the potential to manufacture the therapy under commercial pharmaceutical standards.
One remarkable success story is Eliana Nachem, an 11-year-old girl who received gene therapy at 10 months old after being diagnosed with ADA-SCID at birth. Despite initial complications, Eliana has thrived, attending public school, playing basketball, and living a life filled with possibility. Her family credits the gene therapy for giving them their daughter back, allowing her to lead an ordinary childhood that was once impossible.
Source: https://www.news-medical.net/news/20251015/Experimental-gene-therapy-restores-immune-system-function-in-children-with-genetic-immune-disorder.aspx