Families affected by severe leukocyte adhesion deficiency-I (LAD-I) report significant improvements in their children’s health following treatment with a newly approved gene therapy, according to an April 2 announcement. The U.S. Food and Drug Administration recently granted accelerated approval to the therapy, which was tested in clinical trials at UCLA.
The news is important because LAD-I is a rare genetic disorder that impairs the body’s ability to fight infections and heal wounds, affecting about one in a million children worldwide. For families like the Langenhops from Canton, Ohio, and Marley Gaskins from Live Oak, Florida, this breakthrough has meant a dramatic change in daily life.
Ava, Olivia, and Landon Langenhop have enjoyed good health since receiving the gene therapy in 2020. Their mother Alicia said: “It’s like night and day. 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.”
Marley Gaskins also participated in the UCLA clinical trial. Her mother Tamara Hogue described her daughter’s transformation: “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 therapy works by inserting a healthy copy of the affected gene into a patient’s own blood stem cells so that new immune cells can be produced. Dr. Donald Kohn led clinical trials for this approach at UCLA with support from Rocket Pharmaceuticals and funding 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 that more children with severe LAD-I will soon have access to this treatment option if they do not have sibling donors for stem cell transplants. Alicia Langenhop added: “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, these children are planning activities typical of any childhood—cheerleading tryouts or driving lessons—that once seemed out of reach due to their illness.
