The families of several children diagnosed with severe leukocyte adhesion deficiency-I (LAD-I) said on Apr. 2 that their children have experienced significant improvements in health following participation in a gene therapy trial at UCLA, which recently received accelerated approval from the U.S. Food and Drug Administration.
The news is important because LAD-I is a rare genetic disorder affecting about one in a million children worldwide, often leading to life-threatening infections and frequent hospitalizations. Traditional treatments have included stem cell transplants, but not all patients are eligible for this option.
Jon and Alicia Langenhop discovered that all three of their children had LAD-I, resulting in repeated hospital stays and medical bills before they joined the UCLA trial. Since receiving the investigational gene therapy in 2020, Ava, Olivia, and Landon Langenhop have returned to normal childhood activities. “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 participant was Marley Gaskins from Live Oak, Florida. Her mother Tamara Hogue described years spent managing Marley’s near-constant infections before treatment through the same clinical trial: “It’s a blessing to hear at each annual checkup that Marley’s still doing great and holding steady,” Tamara said. “Aside from the test results, I can see it in the way she quickly recovers from colds, and even in the way she’s able to wear costume jewelry now without risking an infection.”
The gene therapy works by inserting a healthy copy of the affected gene into a patient’s own blood stem cells so that functional immune cells can be produced to fight infection. Dr. Donald Kohn led clinical trials at UCLA with sponsorship by Rocket Pharmaceuticals and support from the California Institute for Regenerative Medicine.
“Clinical trial participation is absolutely critical to making medical advances — it’s how we learn what works and doesn’t work,” said Kohn.
Both families hope more patients will gain access to this treatment as awareness grows among clinicians treating other children with severe LAD-I who lack sibling donors for stem cell transplants.
Looking ahead, Alicia reflected on her family’s journey: “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.” The participating children are now planning for typical milestones such as cheerleading tryouts or driving lessons.
