Preclinical safety of human embryonic stem cell-derived oligodendrocyte progenitors supporting clinical trials in spinal cord injury

Regen Med. 2015 Nov;10(8):939-58. doi: 10.2217/rme.15.57. Epub 2015 Sep 8.


Aim: To characterize the preclinical safety profile of a human embryonic stem cell-derived oligodendrocyte progenitor cell therapy product (AST-OPC1) in support of its use as a treatment for spinal cord injury (SCI).

Materials & methods: The phenotype and functional capacity of AST-OPC1 was characterized in vitro and in vivo. Safety and toxicology of AST-OPC1 administration was assessed in rodent models of thoracic SCI.

Results: These results identify AST-OPC1 as an early-stage oligodendrocyte progenitor population capable of promoting neurite outgrowth in vitro and myelination in vivo. AST-OPC1 administration did not cause any adverse clinical observations, toxicities, allodynia or tumors.

Conclusion: These results supported initiation of a Phase I clinical trial in patients with sensorimotor complete thoracic SCI.

Keywords: biodistribution; clinical trial; human embryonic stem cells; oligodendrocyte progenitors; preclinical safety; thoracic spinal cord injury; toxicology; tumorigenicity.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Heterografts
  • Human Embryonic Stem Cells* / metabolism
  • Human Embryonic Stem Cells* / transplantation
  • Humans
  • Mice
  • Mice, Nude
  • Oligodendroglia* / metabolism
  • Oligodendroglia* / transplantation
  • Spinal Cord Injuries / metabolism
  • Spinal Cord Injuries / therapy*
  • Stem Cell Transplantation / adverse effects
  • Stem Cell Transplantation / methods*