Bone marrow transplant, also called hematopoietic stem cell transplantation (HSCT), is used to treat a variety of hematologic malignancies and other serious bone marrow disorders. In this procedure, patients are treated with chemotherapy, radiation or a combination of both to destroy residual cancerous or defective cells in the bone marrow. Patients then receive new bone marrow stem cells, often from a healthy donor (allogeneic HSCT). The donor cells are intended to fully reconstitute the patient’s bone marrow and immune system, potentially curing the disease.
Despite the curative potential of a bone marrow transplant, many eligible patients do not receive one or have a significant wait to get a match. Even for patients who do receive transplant, treatment is not always effective and can lead to serious complications that can dramatically affect quality of life, such as infections and graft-versus-host-disease (GVHD). Complications can also lead to lengthy hospitalizations.
Omidubicel1 is an advanced cell therapy in Phase 3 development as a rapid, potentially life-saving solution for patients in need of allogeneic HSCT. Omidubicel has two main components: umbilical cord blood that has been expanded through our proprietary nicotinamide-based, or NAM, technology, which allows the cells to grow while maintaining stem-like functionality; and immune cells capable of providing an immediate immune benefit. Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration, and has also received Orphan Drug Designation in the U.S. and EU.
In a Phase 1/2 study, patients receiving omidubicel experienced rapid and durable engraftment of neutrophils, platelets and other blood cells, compared to a historical evaluation of patients who received cord blood alone. Engraftment is a crucial treatment milestone in which donor cells begin producing healthy cells. Additionally, treatment with omidubicel was associated with rapid and robust immune reconstitution, as well as fewer infections, shorter hospital stays and a low incidence of GVHD.2
We are conducting a multi-center, international, randomized Phase 3 study to evaluate the safety and efficacy of omidubicel compared to standard umbilical cord blood transplant in patients with hematological malignancies and no available matched donor. We are also conducting a Phase 1/2 study of omidubicel in patients with severe aplastic anemia, a rare, life-threatening bone marrow failure disease. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.
1 Gamida Cell’s lead development candidate consists of omidubicel (expanded hematopoietic stem cells) and differentiated immune cells, including T cells. Gamida Cell refers to the two components collectively as “omidubicel.”
2 Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo with Nicotinamide. Horwitz M.E., Wease S., Blackwell B., et al. Journal of Clinical Oncology. 2018 DOI: 10.1200/JCO.18.00053.