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


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.


CONCLUSIONS
These results supported initiation of a Phase I clinical trial in patients with sensorimotor complete thoracic SCI.
Geron Corporation, 149 Commonwealth St., Menlo Park, CA 94025, USA.|California Institute of Regenerative Medicine, 210 King St., San Francisco, CA 94107, USA.
Catherine A
CA
Priest
Asterias Biotherapeutics Inc., 230 Constitution Drive, Menlo Park, CA 94025, USA.
Nathan C
NC
Manley
Geron Corporation, 149 Commonwealth St., Menlo Park, CA 94025, USA.|Dark Horse Consulting, 1999 South Bascom Ave Suite 700, Campbell, CA 95008, USA.
Jerrod
J
Denham
Geron Corporation, 149 Commonwealth St., Menlo Park, CA 94025, USA.|Asterias Biotherapeutics Inc., 230 Constitution Drive, Menlo Park, CA 94025, USA.
Edward D
ED
Wirth
Geron Corporation, 149 Commonwealth St., Menlo Park, CA 94025, USA.|Asterias Biotherapeutics Inc., 230 Constitution Drive, Menlo Park, CA 94025, USA.
Jane S
JS
Lebkowski
D000818:Animals; D064593:Heterografts; D000066449:Human Embryonic Stem Cells / Q000378:metabolism / Q000637:transplantation; D006801:Humans; D051379:Mice; D008819:Mice, Nude; D009836:Oligodendroglia / Q000378:metabolism / Q000637:transplantation; D013119:Spinal Cord Injuries / Q000378:metabolism / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*
biodistribution; clinical trial; human embryonic stem cells; oligodendrocyte progenitors; preclinical safety; thoracic spinal cord injury; toxicology; tumorigenicity