Family of three Ohio children finds hope in experimental gene-addition therapy for rare immune disorder
Three siblings with LAD-1 participated in a small clinical trial that introduced a corrected gene into their own stem cells, and they have remained infection-free for five years.
Ava, Olivia and Landon Langenhop were diagnosed with LAD-1, a condition that prevents white blood cells from reaching infection sites. Their parents entered a Rocket Pharmaceuticals study at UCLA that modifies each patient’s own stem cells with a virus carrying the absent gene. The protocol required an initial chemotherapy to mobilize stem cells, blood collection, laboratory gene addition, a second high-dose chemotherapy to clear existing marrow, and infusion of the corrected cells.
Over nine months the siblings underwent treatment sequentially while the family lived in Los Angeles. Five years later the children are active in school sports and have not needed hospital care beyond routine follow-ups, and all nine trial participants continue to generate healthy immune cells. Researchers caution that long-term cure cannot yet be claimed, but the results remain stable. The therapy is currently being reviewed by the Food and Drug Administration.
