The first and only modified allogeneic umbilical cord blood-based cellular therapy — omidubicel (Omisirge®, Boston MA) — is being offered at Duke to treat patients 12 years and up with high-risk blood cancers, including acute lymphoblastic leukemia, acute myeloid leukemia, chronic myelogenous leukemia, lymphoma, and myelodysplastic syndromes.
The Duke Blood Cancer Center was the first center to perform stem cell transplantation with omidubicel and completed more than any center worldwide by the time the new stem cell product received Breakthrough Therapy Designation from the FDA in April. Five of the patients treated by Duke received the therapy as a result of the FDA’s compassionate use exception.
“One of the most important advantages of cord blood is that it doesn’t need to be matched to the recipient,” says Mitchell E. Horwitz, MD, director of the Adult Blood and Marrow Transplant Program at Duke Blood Cancer Center and PI of the multi-center international phase 3 clinical trial that led to FDA approval. “Whereas the process of locating and confirming a donor can take months, donated cord blood is prescreened, tested, frozen, and ready to use immediately.”
He says omidubicel could be a lifesaver for patients of African, Hispanic, Middle Eastern, southern European, Asian, or mixed ethnic backgrounds, for whom finding a matched or even partially matched donor in their family or in the national bone marrow donor registry is more difficult.
Cord blood-based stem cell transplantation works best in children
Cord blood-based stem cell transplantation has typically worked best in young children, but the therapy is not considered optimal for older children or adults because it doesn’t contain enough stem cells, which are dosed according to body weight. Low doses can lengthen or compromise the chances of engraftment.
Omidubicel, however, boosts the number of stem cells in cord blood so that one unit of blood is more than sufficient for transplant. The time required for engraftment is nearly half that of a conventional cord blood-based stem cell transplant (an average of 12 days vs. 22 days). This results in a faster recovery, a lower risk for infections, and less time spent in the hospital.
Duke treats nearly 1,000 patients with blood cancers each year, more than any other facility in North Carolina. There are 15 cell therapy/stem cell transplant specialists at Duke Health, all of whom can offer patients access to stem cell transplants at Duke using either donated bone marrow, blood, or umbilical cord blood, as part of their treatment arsenal.
“It’s rare these days that we turn somebody away because we do not have a suitable stem cell donor,” says Horwitz.