Asterias Biotherapeutics Begins Next Phase of Cervical SCI Trial

Alameda, Calif.-based BioTime Inc.’s subsidiary Asterias Biotherapeutics Inc. has received U.S. Food and Drug Administration (FDA) clearance to initiate a phase I/IIa clinical trial of its product, AST-OPC1, in patients with complete cervical spinal cord injury. The approved trial follows the successful completion of a phase 1 clinical study of the product, and is designed to assess safety and activity of escalating doses of AST-OPC1 in patients with complete cervical spinal cord injuries, the first targeted indication for AST-OPC1 and the first of future product registration clinical trials.

“The FDA clearance provides Asterias with imminent access to the previously announced $14.3 million California Institute for Regenerative Medicine grant, which provides non-dilutive funding to support both the clinical trial and other product development activities for AST-OPC1,” said Pedro Lichtinger, president and CEO of Asterias.

AST-OPC1 is a population of cells derived from human embryonic stem cells that contains oligodendrocyte progenitor cells (OPCs). OPCs and oligodendrocytes perform supportive functions for nerve cells in the central nervous system. The foundation for the clinical trial comes from results from the phase I clinical trial of AST-OPC1, which met its primary endpoints of safety and feasibility when administered to five patients with neurologically complete, thoracic spinal cord injury. These five patients were administered 2 million AST-OPC1 cells and have been followed to date for two to three years. No serious adverse events were observed associated with the delivery of the cells, the cells themselves, or the short-course immunosuppression regimen used. According to trial data there was no evidence of expanding masses, expanding cysts, infections, cerebrospinal fluid leaks, increased inflammation, neural tissue deterioration or immune responses targeting AST-OPC1 in these patients. In four of the five subjects, serial magnetic resonance imaging scans performed throughout the two- to three-year follow-up period indicate that reduced spinal cord cavitation may have occurred and that AST-OPC1 may have had some positive effects in reducing spinal cord tissue deterioration.

There currently are no approved therapies to treat spinal cord injury (SCI), and the complex pathology of the injury is unlikely to be addressed by a traditional small-molecule or protein therapeutic. AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown to have three potentially reparative functions that address the complex pathologies observed at the SCI injury site. The activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site.

BioTime is a biotechnology company engaged in research and product development in the field of regenerative medicine.