Families affected by severe leukocyte adhesion deficiency-I, a rare genetic disorder, say their children are now healthy and able to enjoy normal activities following treatment with a newly approved gene therapy. The U.S. Food and Drug Administration granted accelerated approval for the therapy in 2026, according to an April 2 announcement.
The approval is significant for families whose children have struggled with frequent infections and hospitalizations due to the disease. The condition affects about one in a million children worldwide and severely impairs the immune system’s ability to fight infections.
Jon and Alicia Langenhop discovered seven years ago that all three of their children had the disorder. After joining an investigational gene therapy trial at UCLA in 2020, Ava, Olivia, and Landon have experienced good health. “It’s like night and day,” Alicia said from their home in Canton, Ohio. “Before they were treated, it was just doctor visit after doctor visit, hospital stays nonstop. Now they’re normal, happy kids who were even able to start going to public school on time.”
Another family described similar experiences. Marley Gaskins spent much of her early life managing near-constant infections until she also participated in the UCLA clinical trial led by Dr. Donald Kohn. Marley is now active in Girl Scouts and enjoys arts and crafts activities that were once impossible due to her illness.
The gene therapy works by inserting a healthy copy of the defective gene into a patient’s own blood stem cells before returning them to the body so they can produce functional immune cells needed for fighting infection. This approach differs from traditional stem cell transplants that require donor matches.
Dr. Donald Kohn said clinical trial participation is crucial for medical advances: “Clinical trial participation is absolutely critical to making medical advances — it’s how we learn what works and doesn’t work… For these patients to be some of the very first to receive this treatment, it’s very courageous.” He added his observations during follow-up visits: “It’s wonderful to see them — they’re just normal kids, running around and chasing each other outside the hospital cafeteria.”
Both families hope wider access will become available for other children affected by severe LAD-I who do not have sibling donors for stem cell transplants.
Reflecting on their experience with the new treatment option at UCLA, Alicia Langenhop said: “With this entire situation, we feel like we just owe our lives to the doctors and nurses at UCLA. They gave our kids their lives.”
Looking ahead, both families expressed optimism as their children plan typical childhood milestones such as cheerleading tryouts or driving lessons—opportunities previously out of reach due to illness.
