Seven years ago, Jon and Alicia Langenhop learned that all three of their children suffered from the same devastating, one-in-a-million genetic disorder. The constant, life-threatening infections and rashes that began weeks after each child’s birth, emergency room visits and hospital stays — accompanied by exorbitant bills — now had an explanation: severe leukocyte adhesion deficiency-I. But it wasn’t until they found an investigational gene therapy trial at UCLA that they had any hope their young family could beat the deadly odds of the disease.
Ava, 10, Olivia, 8, and Landon, 6, have been in good health since receiving the therapy in 2020. They adventure outdoors, play softball and dream of jobs as veterinarians and magicians.
“It’s like night and day,” Alicia said from the family’s 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.”
The Langenhops aren’t the only family who spent years grappling with the consequences of this rare disease. Marley Gaskins, now 16, spent so much of her early life in and out of doctors’ offices that her mom, Tamara Hogue, quit her job to provide her daughter with round-the-clock care. Marley, who has grown up in Live Oak, Florida, battled near-constant infections and followed a strict regimen of medicines at precise hours to stave off the effects of her condition.
Marley is now an active member of the Girl Scouts, a lover of all things arts and crafts, and as Tamara said with a grin, a typical teenager who sleeps in late and spends too much time on her phone. These activities — even the wonderfully mundane — are possible because she received treatment in the same UCLA clinical trial. Marley’s trial participation, along with Ava, Olivia and Landon’s, helped enable the U.S. Food and Drug Administration to grant accelerated approval for the therapy that changed their lives.
Commercially known as Kresladi, the therapy targets the defective gene that causes LAD-l, which affects approximately one in one million children worldwide. The genetic defect prevents white blood cells from functioning normally, impairing the body’s ability to fight infections and heal wounds.
The one-time therapy inserts a healthy copy of the affected gene into a patient’s own blood stem cells, which are then returned to the child’s body. These blood stem cells can produce the functional immune cells necessary to fight infection and keep the body healthy.
The therapy represents a different treatment approach from stem cell transplants, which have historically been the definitive treatment option for some patients with severe LAD-I.
Clinical trials for the therapy were led by Dr. Donald Kohn, a physician-scientist and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and sponsored by Rocket Pharmaceuticals, which licensed and developed the therapy, as well as the California Institute for Regenerative Medicine, a California state agency that funds stem cell and gene therapy research.
“Clinical trial participation is absolutely critical to making medical advances — it’s how we learn what works and doesn’t work,” said Kohn, who is also a distinguished professor of microbiology, immunology and molecular genetics in the UCLA College and of pediatrics and molecular and medical pharmacology at the David Geffen School of Medicine at UCLA. “For these patients to be some of the very first to receive this treatment, it’s very courageous.”
Ava and Olivia Langenhop
When the Langenhop siblings came back to UCLA for a follow-up appointment in April 2025, Kohn marveled at how the gene therapy has transformed their lives. “It’s wonderful to see them — they’re just normal kids, running around and chasing each other outside the hospital cafeteria,” he said.
Both families said they hope that the gene therapy can become more widely available to other children affected by severe LAD-l without an available stem cell transplant from a sibling donor. They’ve each been contacted by strangers wanting to know how their children were able to receive the therapy — conversations that serve as keen reminders of the hopelessness they once felt when facing their own children’s diagnoses. Marley’s immunologist would also inquire during checkups if Tamara had heard any news of when the therapy might be available to other patients she was seeing.
“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.”
After the hardships and heartache caused by the disease, the families reflected that things seem to come more easily to them now. Noting the timing and availability of the trial to all three of her children, Alicia, who once said they felt like the “unluckiest family in the whole entire world,” said they now feel like the luckiest.
“It was the best thing for our family,” Alicia 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.”
The kids are planning for a future that otherwise might’ve been taken away from them. They’re looking forward to cheerleading tryouts, driving lessons and in the case of Landon, continuing to put his new immune system to the test through a plethora of cuts and bruises earned playing.
