Each year, approximately 60,000 patients with leukemia or lymphoma need a bone marrow transplantation. Of these, only approximately 25,000 are transplanted, primarily because the other 35,000 are unable to find a fully matched bone marrow donor. Cord blood transplantation (CBT) does not require full tissue matching and is considered a good alternative for transplantation. However, CBT is limited by a low number of TNC and CD34+ cells, influencing the incidence and rate of hematopoietic recovery and risk of early transplant-related mortality. Ex-vivo expansion is a strategy employed to increase the number of stem/progenitor cells and improve clinical outcomes.
StemEx is a graft of stem/progenitor cells isolated and expanded from a portion of a single unit of umbilical cord blood (CBU) and transplanted in combination with non-expanded cells from the same unit. The portion of umbilical stem cells is expanded using Gamida Cell’s proprietary copper chelator technology.
StemEx is poised to answer the dire unmet clinical need in the field of bone marrow transplantation by supplying alternative cell grafts to the 50,000 patients with hematological malignancies, who are indicated for transplantation but do not have a matched family related bone marrow donor. The market potential of StemEx is estimated at more than one billion USD.
StemEx is now being studied as an alternative therapeutic treatment for hematological malignancies in an international, Phase III, pivotal registration clinical trial. This is an open study with a historical control cohort. The pivotal study is now completing recruitment. Safety and efficacy clinical outcomes of the study will be available in Q4/2012.