Just days after announcing $45 million in new funding, startup Nohla Therapeutics has released extended results from a study of dilanubicel — its flagship leukemia treatment that works alongside stem cell transplants using donated umbilical cord blood.
Five years after their transplants, 86 percent of patients (13 out of 15) in the study have survived the disease and are still cancer free. None of the patients experienced severe graft versus host disease, a major concern for stem cell transplant recipients.
The results show a higher survival rate than the average for leukemia patients, although those rates vary greatly depending on the patients’ age and the kind of leukemia they have.
It’s good news for Nohla, which is developing a variety of cancer treatments that augment cord blood transplants and make them more effective.
“These results demonstrate that infusion of dilanubicel given with a cord blood transplant was safe and led to faster neutrophil and platelet recovery, but more importantly showed excellent long-term survival with no transplant-related mortality or severe (grade 3-4) acute graft vs. host disease,” Nohla Co-founder and Chief Medical Officer Colleen Delaney said in a press release.
“We expect to have top-line results later this year from Nohla’s ongoing randomized Phase 2b trial coordinated by the Fred Hutch evaluating dilanubicel in 160 patients with hematologic malignancies receiving a cord blood transplant,” she said.
The study followed 15 patients who ranged in age from just 5 years old to 45. They had a variety of leukemia sub-types: Eight had acute lymphoblastic leukemia, six had acute myeloid leukemia and one had myelodysplastic syndrome, a catch-all for blood disorders that are often related to other cancers.
Nohla’s technology is based on research conducted by Delaney and her colleagues at Seattle’s Fred Hutchinson Cancer Research Center. It aims to make cord blood transplants — a cousin of the more common bone marrow transplants — a better and more universal treatment option for leukemia patients.
Following intense, cancer-killing radiation treatments, a stem cell transplant rebuilds a patient’s bone marrow and immune system and get them on the road to recovery. Bone marrow transplants require a very close match to a donor’s bone marrow, often a relative.
But umbilical cord blood doesn’t need to be matched and is readily available from blood donation banks. It could also be more effective as a transplant material, but it takes longer to work, opening the door for deadly short-term complications.
Nohla is working to mitigate that problem with dilanubicel and other products and aims to make cord blood transplants a more effective and accessible treatment for leukemia.