Transplantation of umbilical cord blood-derived cells for novel indications in regenerative therapy or immune modulation: a scoping review of clinical studies

Biol Blood Marrow Transplant. 2014 Jan;20(1):20-5. doi: 10.1016/j.bbmt.2013.09.010. Epub 2013 Sep 22.


Although used mainly for transplantation of hematopoietic stem cells in the treatment of blood disorders, umbilical cord blood (UCB)-based therapies are now being used increasingly for novel applications in nonhematopoietic diseases and as a form of cellular regenerative therapy or immune modulation. We performed a systematic scoping review by searching Medline, EMBASE, and the Cochrane Library for published articles, and we searched and the World Health Organization International Clinical Trials Registry Platform to describe the breadth of published studies and ongoing clinical activity in umbilical cord-based cellular therapy for regenerative therapy and immune modulation. The most commonly published area of expertise in the use of UCB-derived cellular transplantation for novel indications is for neurological disorders and this remains the most active area of study in ongoing registered trials. An increasingly broad range of disorders, however, are reflected in ongoing registered trials, which suggests greater activity, interest, and investment in UCB-derived cellular therapy. Interestingly, adult patients compose the majority of patients reported in published reports and registered ongoing clinical studies continue to enroll predominantly adult subjects. Geographically, Asian countries appear most active in UCB-derived cellular therapy and our analysis of ongoing studies suggests this trend will likely continue. Regular assessment of published and ongoing activity in UCB transplantation for emerging novel indications will be critical for informing UCB banking establishments and funding agencies to guide changes in banking practices related to emerging trends in cell therapy.

Keywords: Clinical studies; Immune modulation; Regenerative therapy; Transplantation; Umbilical cord blood.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review
  • Systematic Review

MeSH terms

  • Adult
  • Blood Banks
  • Cardiovascular Diseases / immunology
  • Cardiovascular Diseases / pathology
  • Cardiovascular Diseases / therapy*
  • Cell- and Tissue-Based Therapy
  • Clinical Trials as Topic
  • Cord Blood Stem Cell Transplantation / statistics & numerical data*
  • Databases, Bibliographic
  • Diabetes Mellitus / immunology
  • Diabetes Mellitus / pathology
  • Diabetes Mellitus / therapy*
  • Gastrointestinal Diseases / immunology
  • Gastrointestinal Diseases / pathology
  • Gastrointestinal Diseases / therapy*
  • Humans
  • Immunomodulation
  • Lupus Erythematosus, Systemic / immunology
  • Lupus Erythematosus, Systemic / pathology
  • Lupus Erythematosus, Systemic / therapy*
  • Nervous System Diseases / immunology
  • Nervous System Diseases / pathology
  • Nervous System Diseases / therapy*
  • Regenerative Medicine