Operation Milestone OM: #D
The sponsor submitted the one-year clinical study report to FDA. This was an important achievement documenting the safety and preliminary activity of OPC1 for spinal cord injuries.
The safety data indicates that AST-OPC1 can be safely administered to subjects in the subacute period after cervical SCI. The injection procedure and the low-dose temporary immunosuppression regimen were well tolerated. The 25 subjects who received AST-OPC1 completed 1 year of follow-up and showed no evidence of neurological deterioration or adverse findings on MRI scans.
Serial MRI scans of the cervical spinal cord obtained on days 7-, 30-, 180-, and 365-days post-injection have demonstrated engraftment and long-term cell survival of transplanted cells in 95% of the subjects.
The data from this study support that optimal dosing for future studies of safety and efficacy is 1 x 107 cells dosed 21-42 days after SCI.
We believe that this trial sufficiently demonstrated the safety of transplantation of AST-OPC1 in humans. Regarding efficacy, at one year in this cervical study 32% of the subjects recovered 2 or more levels of neurologic function on at least one side of their body.