The Challenge

Bone marrow transplants (also known as hematopoietic stem cell transplantation, or HSCT) have been a life-saving therapy for patients with severe blood cancers and other blood disorders for several decades. Worldwide, more than 70,000 transplants are performed each year for illnesses such as leukemia, lymphoma and anemia. But it remains a high-risk and sometimes fatal procedure, and the majority of patients do not respond as hoped.

There are three possible sources of stem cells for HSCT: the patient, a donor, or umbilical cord blood. When possible, the patient’s own stem cells are used (an autologous transplant) as there is less risk of transplant rejection or development of graft-versus-host-disease. However, this is not always ideal or safe for the patient and as a result nearly half of all HSCTs involve cells taken from a sibling or unrelated donor (allogeneic) sources. These allogeneic transplants present several challenges, including a painful and risky donor process, long wait times to find a fully tissue-matched donor, and the difficulty finding matches for ethnic minorities. Nearly 30% of patients will be excluded from treatment because a compatible donor cannot be found.

Cord blood is an underused source of life-saving blood stem cells for transplant, due to the relatively small number of usable cells in a single cord blood unit. ExCellThera’s cell expansion solution is well positioned to solve this problem.

Despite being less prone to graft-versus-host disease complications and relapse, and being more readily available, grafts from cord blood are currently not preferred — the low number of stem cells in cord blood results in slower engraftment, prolonged hospitalization and risk of infections. Increasing the number of stem cells in transplant units remains an important unmet medical need for blood cancers and other blood or autoimmune/inflammatory diseases (such as sickle cell anemia or Crohn’s disease, among others), for which this procedure would be curative.