Since the first successful transplantation of umbilical cord blood in 1988, cord blood has become an important source of hematopoietic stem and progenitor cells for the treatment of blood and genetic disorders. Significant progress has been accompanied by challenges for scientists, ethicists, and health policy makers. With the recent recognition of the need for a national system for the collection, banking, distribution, and use of cord blood and the increasing focus on cord blood as an alternative to embryos as a source of tissue for regenerative medicine, cord blood has garnered significant attention. We review the development of cord blood banking and transplantation and then discuss the scientific and ethical issues influencing both established and investigational practices surrounding cord blood collection, banking, and use.In 1988, a 6-year-old boy from North Carolina with Fanconi anemia was transplanted in Paris with HLA-matched umbilical cord blood from his baby sister (1). Most scientists and physicians at the time were highly skeptical, doubting that a few ounces of cord blood contained sufficient stem and progenitor cells to rescue bone marrow after myeloablative therapy. However, this child engrafted without incident, fully reconstituting his blood, bone marrow, and immune system with donor cells. He remains well and durably engrafted with donor cells 17 years following the original transplant (J. Kurtzberg, personal communication).
From experimentation to practice: development of cord blood transplantationOver the 5-6 years following the first cord blood transplant, approximately 60 additional transplants between HLA-matched siblings were performed worldwide. Reports of results to a volunteer registry (2) demonstrated that cord blood contained sufficient numbers of stem and progenitor cells to reconstitute the entire hematopoietic system of a child after myeloablative therapy and that the incidence of graft-versus-host disease (GVHD) was 10-fold lower than that seen after transplantation with HLA-matched bone marrow obtained from a sibling.At this time it was becoming apparent that the diversity of HLA alleles and antigens was vast and that it was never going to be possible to find fully matched related and unrelated adult donors for all patients in need of allogeneic transplantation therapy from then-available sources. The National Marrow Donor Foundation (NMDP) and other international registries successfully recruited, typed, and listed millions of volunteer unrelated adult donors, but only 25-50% of patients in need could locate sufficiently matched donors in a timely fashion. Donors for patients of minority ethnic backgrounds were even scarcer and more difficult to locate. To provide donors for all patients in need, transplant physicians needed to find a way to transplant partially mismatched grafts.