Omidubicel, an investigational enriched umbilical cord blood product being developed by Gamida Cell for transplantation in patients with blood cancers, appears to have some advantages over same old umbilical cord blood.
The outcomes come from a world allotment 3 trial (NCT02730299) supplied on the annual meeting of the European Society for Blood and Bone Marrow Transplantation.
“Transplantation with omidubicel when put next to same old cord blood transplantation ends in sooner hematopoietic recovery, fewer infections, and fewer days in clinical institution,” talked about coinvestigator Guillermo F. Sanz, MD, PhD, from the Health center Universitari i Politècnic la Fe in Valencia, Spain.
“Omidubicel could presumably additionally serene be regarded as because the recent same old of fancy patients eligible for umbilical cord blood transplantation,” Sanz concluded.
Zachariah DeFilipp, MD, from the Massachusetts General Cancer Center in Boston, a hematopoietic stem cell transplantation specialist, became once no longer interested by the ogle and became once approached for comment. He told Medscape Scientific Files that “omidubicel tremendously improves the engraftment after transplant, as when put next to same old cord blood transplant. For patients that lack an HLA-matched donor, this approach can support overcome the prolonged cytopenias that happen with same old cord blood transplants in adults.”
Gamida Cell plans to put up these files for approval of omidubicel by the US Meals and Drug Administration in the fourth quarter of 2021.
Omidubicel will likely be being evaluated in a Piece 1/2 clinical ogle in patients with excessive aplastic anemia (NCT03173937).
Even when umbilical cord blood stem cell grafts come from a readily on hand source and show conceal bigger tolerance across HLA limitations than various sources (a lot like bone marrow), the relatively low dose of stem cells in every unit ends in delayed hematopoietic recovery, increased transplant-connected morbidity and mortality, and longer hospitalizations, Sanz talked about.
Omidubicel includes two cryopreserved fractions from a single cord blood unit. The product contains both noncultured CD133-negative cells, at the side of T cells, and CD133-obvious cells which can perchance presumably perchance be then expanded ex vivo for 21 days in the presence of nicotinamide.
“Nicotinamide increases stem and progenitor cells, inhibits differentiation and increases migration, bone marrow homing, and engraftment efficiency while preserving mobile efficiency and phenotype,” Sanz defined all the most practical arrangement thru his presentation.
In an earlier allotment 1/2 trial in 36 patients with excessive-possibility hematologic malignancies, omidubicel became once connected with hematopoietic engraftment lasting on the least 10 years.
Crucial functions of Piece 3 Trial Results
The global allotment 3 trial became once conducted in 125 patients (used 13-65) with excessive-possibility malignancies, at the side of acute myeloid and lymphoblastic leukemias, myelodysplastic syndrome, continual myeloid leukemia, lymphomas, and uncommon leukemias. These patients had been all eligible for allogeneic stem cell transplantation nonetheless did no longer have matched donors.
Patients had been randomly assigned to get hematopoietic reconstitution with either omidubicel (n = 52) or same old cord blood (58).
At 42 days of apply-up, the median time to neutrophil engraftment in the plan-to-address (ITT) population, the main endpoint, became once 12 days with omidubicel versus 22 days with same old cord blood (P < .001).
In the as-handled population — the 108 patients who indubitably obtained omidubicel or same old cord blood — median time to engraftment became once 10.0 versus 20.5 days, respectively (P < .001).
Charges of neutrophil engraftment at 42 days had been 96% with omidubicel versus 89% with same old cord blood.
The secondary endpoint of time-to-platelet engraftment in the ITT population also liked omidubicel, with a cumulative day 42 incidence charge of 55% when put next with 35% with same old cord blood (P = .028).
In the as-handled population, median times to platelet engraftment had been 37 days and 50 days, respectively (P = .023). The cumulative rates of platelet engraftment at 100 days of apply-up had been 83% and 73%, respectively.
The incidence of grade 2 or 3 bacterial or invasive fungal infections by day 100 in the ITT population became once 37% amongst patients who obtained omidubicel, when put next with 57% for patients who obtained same old cord blood (P = .027). Viral infections occurred in 10% versus 26% of patients, respectively.
The incidence of acute graft versus host illness at day 100 became once the same between remedy groups, and there became once no most important disagreement at 1 365 days.
Relapse and nonrelapse mortality rates, as neatly as illness-free and overall survival rates also did no longer vary between groups.
In the first 100 days put up-transplant, patients who obtained omidubicel had been alive and out of the clinical institution for a median of 60.5 days, when put next with 48 days for patients who obtained same old cord blood (P = .005).
The ogle became once funded by Gamida Cell. Sanz has reported receiving evaluation funding from the firm and a total lot of different others, and consulting charges, honoraria, speakers bureau activity and trip costs from various firms. DeFilipp has reported no relevant monetary relationships.
EBMT 2021. Summary GS2-7. Presented March 15, 2021.