Cord blood as a potential therapeutic for amyotrophic lateral sclerosis

Expert Opin Biol Ther. 2017 Jul;17(7):837-851. doi: 10.1080/14712598.2017.1323862. Epub 2017 May 8.


Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive motor neuron degeneration in the brain and spinal cord. Treatment options are limited due to the complexity of underlying disease factors. Cell therapy, using human umbilical cord blood (hUCB) cells may be a promising new treatment for ALS, mainly by providing a protective microenvironment for motor neuron survival. Areas covered: Composition, in vitro and in vivo differentiation of hUCB cells, and the advantages of cord blood as a source of transplant cells are discussed. A brief history of hUCB in treatment of an ALS animal model and the feasibility of these cells in therapy for ALS patients is provided. Current ALS clinical trials are also deliberated. Expert opinion: Among multiple advantages, hUCB cells' production of various anti-inflammatory/growth/trophic factors makes them an attractive cell source for ALS therapy. Biodistribution and optimal hUCB cell dose for transplantation have been determined in preclinical studies. Repeated intravenous cell doses during disease progression may be the best approach for cell-based ALS treatment. Accumulated evidence shows the efficacy of naïve or genetically modified MNC hUCB cells in the treatment of ALS and provide a superior basis for the development of clinical trials in the near future.

Keywords: ALS; Human umbilical cord blood; multipotency; neuroprotection; treatment.

Publication types

  • Review

MeSH terms

  • Amyotrophic Lateral Sclerosis / metabolism
  • Amyotrophic Lateral Sclerosis / pathology
  • Amyotrophic Lateral Sclerosis / therapy*
  • Animals
  • Cell Differentiation
  • Cell- and Tissue-Based Therapy
  • Clinical Trials as Topic
  • Disease Models, Animal
  • Fetal Blood / cytology
  • Fetal Blood / metabolism
  • Fetal Blood / transplantation*
  • Humans
  • Neural Cell Adhesion Molecule L1 / genetics
  • Neural Cell Adhesion Molecule L1 / metabolism
  • Tissue Distribution
  • Vascular Endothelial Growth Factor A / genetics
  • Vascular Endothelial Growth Factor A / metabolism


  • Neural Cell Adhesion Molecule L1
  • Vascular Endothelial Growth Factor A