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Preclinical Assessment for hESC-derived Oligodendrocyte Progenitor Cells for Cervical Spinal Cord Injury



eview of “Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitor Cells: Preclinical Efficacy and Safety in Cervical Spinal Cord Injury” from STEM CELLS Translational Medicine by Stuart P. Atkinson

Given the complex nature of the cellular events that underlie the clinical outcomes of spinal cord injury (SCI) [1], stem cell-based treatment strategies may represent our best therapeutic option. Preclinical testing has proven the safety of human embryonic stem cell-derived oligodendrocyte progenitor cells (AST-OPC1) for the treatment of thoracic SCI [2], and this has led to the initiation of a Phase I clinical trial.

Now, in a new STEM CELLS Translational Medicine article, researchers led by Nathan C. Manley (Asterias Biotherapeutics Inc., California USA) have assessed the efficacy and safety of AST-OPC1 transplantation in a nude rat model of cervical SCI in the hope of taking this SCI treatment strategy forward to clinical trials [3].

One week after injury, a unilateral cervical spinal cord contusion injury at level C5, the direct administration of OPCs (~2.4 × 105 cells per nude rat) into the cervical spinal cord led to robust OPC engraftment, OPC migration into the lesion site, reduced parenchymal cavitation, and increased sparing of myelinated axons within the injury site. Encouragingly, these improvements combined to mediate an overall enhancement in locomotor performance up to four months post-treatment, as measured employing an automated TreadScan system.

Additional large-scale biodistribution and toxicology studies 9 months after cell transplantation demonstrated a lack of OPC migration out with the spinal cord and brainstem (minimal or absent from the cerebral spinal fluid and blood) and no unwanted side effects, toxicities, allodynia (central pain sensitization), or formation of teratomas/tumors.

This preclinical animal study has demonstrated efficacy and safety of AST-OPC1 for cervical SCI, and these results have already served to enter AST-OPC1 into Phase I/IIa clinical testing in the USA (SCiStar clinical trial). Furthermore, the authors hope that this detailed report will stand as a prime example of the level of preclinical testing apt for cell therapy products in development for central nervous system injury and disease.

Fantastic news for stem cell therapy for spinal cord injury; stay tuned to the Stem Cells Portal to keep yourself up to date!


  1. Anderson, K.D., J. Friden, and R.L. Lieber, Acceptable benefits and risks associated with surgically improving arm function in individuals living with cervical spinal cord injury. Spinal Cord 2009;47:334-8.
  2. Priest, C.A., et al., Preclinical safety of human embryonic stem cell-derived oligodendrocyte progenitors supporting clinical trials in spinal cord injury. Regen Med 2015;10:939-58.
  3. Manley, N.C., et al., Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitor Cells: Preclinical Efficacy and Safety in Cervical Spinal Cord Injury. Stem Cells Transl Med 2017;6:1917-1929.