Placental Cord Blood as a Biological Product: Challenges and Opportunities

Since the first successful umbilical cord blood transplantation was reported in 1989, more than 513,000 donated cord blood units have been stored worldwide in public cord blood banks for unrelated allogeneic hematopoietic stem cell transplantation. In addition, more than 900,000 cord blood units have been stored in private cord blood banks for potential autologous or allogeneic family use. In November 2011, the United States Food and Drug Administration (FDA) approved the first biologics license for hematopoietic progenitor cells from cord blood (Hemacord) submitted by the New York Blood Center, the first and largest public cord blood bank. Hemacord is intended for use in unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, are acquired, or result from myeloablative treatment. Currently, US public cord blood banks are required to submit to the FDA a Biologics License Application (BLA) or an Investigational New Drug (IND) application for unlicensed cord blood products. Private cord blood banks in the United States are not required to file an IND or BLA with the FDA. Instead, US private cord blood banks register with the FDA and follow the US federal regulations for human cells, tissues, and cellular and tissue-based products (tissue rules). The regulatory requirements for the FDA tissue rules and IND applications allow cord blood banks to store cord blood units that may not qualify for FDA BLA approval because of positive infectious disease markers or low cell yield collections. This article reviews the current US FDA regulatory requirements for cord blood banks and some challenges and opportunities in working with a diverse cord blood unit inventory.