A clinical safety and efficacy trial of omidubicel, an investigational cell therapy for patients in need of allogeneic hematopoietic cell transplantation, achieved its primary endpoint of time to neutrophil engraftment or how quickly the transplanted stem cells begin to create healthy new cells. Omidubicel is a product composed of ex vivo expanded allogeneic cells from one entire unit of umbilical cord blood; it is intended to increase migration, bone marrow homing, and engraftment of progenitor cells.
The treatment was tested in a phase III study of 125 patients between ages 12 and 65 with acute lymphocytic leukemia, acute myeloid leukemia, chronic myeloid leukemia, myelodysplastic syndromes, or lymphoma. Patients were randomized to either omidubicel or a standard umbilical cord blood transplant.
Rapid neutrophil engraftment has been associated with fewer infections and shorter hospitalization times. Median time to engraftment was 12 days for patients who received omidubicel (range = 10-15 days) compared to 22 days for the comparator group (range = 19-25 days).
“Shortening the time to engraftment can reduce a patient’s time in the hospital and decrease likelihood of infection,” said Mitchell Horwitz, MD, principal investigator and Professor of Medicine at the Duke Cancer Institute.
Results are consistent with a phase I/II study of omidubicel in 36 patients with hematologic malignancies, in which patients treated with omidubicel demonstrated more rapid neutrophil engraftment than a cohort of 146 patients who received standard umbilical cord blood. The median time to engraftment in this study was 11.5 days for patients who received omidubicel (range = 9-14 days) compared to 21 days for the cohort that received umbilical cord blood (range = 20-23 days).