written by Holly Alyssa MacCormick
Alicia Langenhop was seven months pregnant with her third child when she and her husband, Jon, learned that their two toddlers had a life-threatening immune disease called leukocyte adhesion deficiency-1 (LAD-1).
One in a million
LAD-1 is extremely rare—about one in a million births—but because it is genetic, the Langenhops’ unborn child had roughly a 25 percent chance of inheriting the disease.
Gene mutations cause LAD-1 by limiting, or in severe cases blocking, production of the CD18 protein. White blood cells rely on this protein to reach and fight infections. Without it, the immune system cannot function properly, and few children with severe, untreated LAD-1 survive to adulthood.
The clock was ticking for the Langenhop family, but they caught a break. Soon after the diagnosis, researchers invited their children to join a CIRM-funded clinical trial of a gene therapy for LAD-1 developed by Rocket Pharmaceuticals.
“We feel very fortunate to live in a time and place with access to modern medicine,” Alicia said.
The therapy worked in all nine clinical trial participants and restored immune function. Researchers published a summary of the results in the New England Journal of Medicine in 2025.
Life with LAD-1
When Ava and Olivia—just 3 and 2 years old—received an LAD-1 diagnosis, both had already been hospitalized twice for severe infections.
“Ava was sick constantly from the time she was two weeks old,” Alicia said. “When Olivia was born a year and a half later, she followed the same pattern—constant ear infections, and she caught every respiratory virus. I worked at a daycare, and the girls came with me. We thought we were just unlucky and that they picked up every germ there.”
After doctors diagnosed Ava and Olivia and later confirmed that their third child, Landon, also had LAD-1, life changed for the Langenhop family. Alicia left her job at the daycare because the infection risk was too high.
Constant worry
Lost income and mounting medical bills strained the family’s finances. After the diagnosis, even minor cuts or fevers required urgent care.
“Before the diagnosis, deciding whether to go to the doctor was a toss-up,” Alicia said. “Are we overreacting? Are they actually sick? Do they need to be seen, or can we stay home? We were never right. We either took them in for nothing or waited too long. Once we knew they had LAD-1, it became automatic. Any fever meant a hospital visit, but we never knew how serious it would be.”
Doctors suggested bone marrow transplants as a possible treatment. Neither girl had a match in the bone marrow registry. Landon had a donor, but even with a perfect match, the transplant had only about a 75 percent chance of success.
“Ava would ask me, ‘Mommy, why don’t we go to school anymore? Why do we just take medicine and go to the doctor all the time?’” Alicia said.
The Langenhops searched for a miracle. Then one found them.
Life after LAD-1
Jon’s brother shared the family’s story on social media to encourage more people to join the registry as potential bone marrow transplant donors. Their videos inspired hundreds of people to register and caught the attention of Donald Kohn, distinguished professor of microbiology, immunology, and molecular genetics at the University of California, Los Angeles (UCLA). He served as one of three principal investigators on the LAD-1 gene therapy clinical trial and invited the Langenhop children to enroll.
“Enrolling in the trial was a big decision, but we’ve always trusted science and medicine, and the gene therapy didn’t carry the same risks as a bone marrow transplant,” Alicia said. “It felt like fate. This trial was happening just as we learned that all three of our children needed this intervention.”
“The worst outcome seemed like we’d end up back at square one, still needing a traditional bone marrow transplant,” Jon said. “In our minds, it was a no-brainer to move forward with the clinical trial and see it through.”
In December 2019, just three months later, the Langenhops flew to UCLA to collect Ava’s stem cells. By April 2020, they moved from Ohio to California and lived there for eight months while their children underwent treatment in the clinical trial.
The treatment included gene therapy for LAD-1, chemotherapy, and several other medications. Ava, Olivia, and Landon had especially vulnerable immune systems at that time and needed to quarantine. In a second twist of fate, nearly everyone stayed home then because of the COVID-19 pandemic.
A second shot at a normal life
In May 2021, the family received the all-clear for Ava, Olivia, and Landon to return to daycare. Landon got sick first after they went back, but his immune system fought it off, and he recovered like any other child with a cold.
“They were so excited [to go back to school],” said Alicia. “Olivia wanted to get her ears pierced, but we had been holding off on that. Just getting to play with other kids and going back to school, they couldn’t wait.”
Now the kids are in school, playing sports, and joining as many extracurricular activities as they can.
“They are just normal kids,” said Jon. “They’re like, ‘let’s go!’” ‘let’s go!’”
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