UCLA Gene Therapy Shows 95% Success Rate in ADA-SCID Treatment
An experimental gene therapy from UCLA has successfully restored immune function in 59 of 62 children with ADA-SCID, demonstrating long-term efficacy and safety over 474 patient-years of follow-up. The therapy addresses a rare genetic disorder that severely impairs immune function.
Researchers from UCLA, University College London, and Great Ormond Street Hospital have reported significant success with an experimental gene therapy that restored immune function in 59 out of 62 children. These patients were born with ADA-SCID, a rare genetic disorder caused by mutations in the ADA gene. Without a functional immune system, these children face life-threatening infections, but follow-up data spanning more than 10 years suggests the therapy offers a stable, long-term solution.
Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, is often fatal within the first two years of life if it remains untreated. While bone marrow transplants and enzyme replacement therapies exist, they carry substantial risks and limitations. This gene therapy takes a different approach by addressing the genetic defect directly. Doctors collect a child’s blood stem cells, use a lentivirus to insert a healthy copy of the ADA gene, and reinfuse the modified cells back into the patient.
The results of this work, published in the New England Journal of Medicine, indicate that the restored immune function remains stable over time. Across 474 patient-years of data, researchers found that most side effects were mild and related to the preparation process rather than the therapy itself. The success rate was high, and in the three cases where the treatment did not achieve the desired results, the children were able to return to standard care options.
Beyond its effectiveness, the therapy may also be easier to distribute than previous methods. More than half of the children in the study received stem cells that had been frozen after modification, a process that proved just as effective as using fresh cells. This allows stem cells to be collected locally, sent to a specialized facility for processing, and then shipped back to local hospitals. Such a system could make treatment more accessible by reducing the need for families to travel long distances.
The UCLA team is currently working to secure FDA approval for the therapy, with support from organizations including the California Institute for Regenerative Medicine. They hope to complete the regulatory process within the next two to three years. If successful, this approach could provide a new model for treating ADA-SCID and other genetic conditions, potentially improving long-term outcomes for many children and their families.
Enjoyed this story?
Show the newsroom a little love — one tap per reader.
Priya reports on breakthroughs that change lives, big and small.
Be part of the good
Stories like this start with people who care. Share it, or submit your own uplifting story to inspire millions today.
