Advances in Umbilical Cord Blood Therapy: Hematopoietic Stem Cell Transplantation and Beyond

Abstract

Hematopoietic stem cells (HSCs) have been used for bone marrow transplantations for over four decades. Traditionally, HSCs for bone marrow transplants are retrieved from bone marrow aspirates, but other sources such as peripheral blood and umbilical cord blood (CB) are now being used. Notably, CB has become the leading source of HSCs for many transplant centers due to its rich source of HSCs and ease of collection, storage, and shipment. Also, the development of HSC expansion methods and the use of double-cord transplantations have expanded the use of CB beyond just small children. Moreover, existing banks of CB can provide immediate matched allogeneic and autologous sources of HSCs for transplantation. In addition to hematological diseases, CB is being applied to the field of regenerative medicine and is currently being tested in clinical trials predominantly in the field of neurological disorders, including cerebral palsy, autism, hypoxic-ischemic encephalopathy, stroke, and hearing loss. While still early, published results from clinical trials using allogeneic CB suggest a benefit to children with cerebral palsy. Results from clinical trials using autologous CB for children with cerebral palsy are anticipated in 2016. A benefit of autologous CB has been reported for infants suffering from hypoxic-ischemic encephalopathy. Other ongoing clinical trials are not expected to be completed until 2016 and beyond. Complementary to the application of CB to regenerative medicine is the production of induced pluripotent stem cells (iPSCs) from CB. CB is an attractive source of young cells for iPSC generation, and the production of umbilical cord-blood-derived iPSCs renders the prospective applications of umbilical cord blood endless.