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

Abstract

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.