Analysis of Umbilical Cord Blood Stem Cell Therapy for Regenerative Medicine Applications

Over the past 20 years umbilical cord blood (UCB) has become valuable source of hematopoietic stem cells for use in transplantation to treat malignancies, blood disorders and genetic diseases. Cord blood stem cells have shown promising results in animal regenerative medicine studies for the repair of damaged or diseased tissues outside of the hematopoietic lineage. Family UCB banks provide collection and storage services for families to store their child’s UCB for clinical use. This study compared the release of UCB units from one family bank (Cord Blood Registry, CBR) for regenerative medicine versus traditional transplantation since the release of its first UCB unit for regenerative medicine in 2005. UCB was collected from the placenta of consenting mothers and transported to the processing facility from all 50 states and over 80 countries. The UCB was separated into a mononuclear cell fraction from the red blood cells and other non-engrafting cells. The processed UCB is stored in vapor-phase liquid nitrogen at −195° C until requested for use. To date, 230,000 units have been stored. Prior to release, viability and percent CD34+ (or CFU) content were assessed by the treating institution. Through July 2008, a total of 82 UCB units were released for clinical use, 47 of these units (57%) have been used for traditional transplantation and 35 (43%) have been used for regenerative medicine. From January 2005 through July 2008, 47 UCB units were released, 12 (26%) were used in traditional transplantation and 35 (74%) were used in regenerative medicine applications. Over this period the bank experienced a mean of 338% percent growth per year in the release of UCB units for regenerative medicine. The indications for regenerative medicine autologous infusions were cerebral palsy (24 units), brain injury (4 units), type 1 diabetes (6 units), and a rare immune disorder (1 unit). All patients in the regenerative medicine category did not receive any myeloablative chemotherapy or radiation. Released UCB units were stored a mean of 37 months prior to use, the mean CD34+ percent of TNC was 0.53% and mean viability was 95.9%. These data indicate that regenerative medicine applications utilizing autologous cord blood stem cells represent a significant and growing percentage of all treatments facilitated by a family cord blood bank. Regenerative medicine treatments for cerebral palsy and type 1 diabetes have the potential to impact many lives given that 10,000 cases of cerebral palsy and 13,000 cases of type 1 diabetes are diagnosed each year which compares to 2,500 cases of childhood leukemia and 650 cases of neuroblastoma, two common indications for pediatric transplantation. Further analysis is needed to assess the clinical outcomes which will provide background for larger clinical trials in the future.