Acute spinal cord injury treatment receives Orphan Drug Designation

Written by Alexandra Thompson

Asterias Biotherapeutics (CA, USA) has received Orphan Drug Designation from the US FDA for its product development candidate, AST-OPC1, for the treatment of acute spinal cord injury.

Asterias Biotherapeutics, Inc. (CA, USA), has received Orphan Drug Designation from the US FDA for AST-OPC1. The product development candidate is an ESC-derived oligodendrocyte progenitor population under investigation for the treatment acute spinal cord injuries.

In vitro and animal in vivo results indicated three potential mechanisms by which the cell therapy product could help repair spinal cord injuries: secretion of neurotrophic factors, stimulation of vascularization and induction of remyelination of denuded axons. Administration of the product in animals resulted in axon remyelination, some recovery of limb locomotor function, decreased injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

A Phase I clinical trial of patients with neurologically complete, thoracic spinal cord injury was found to have no evidence of adverse events or rejection. A total of five patients were administered 2 million AST-OPC1 cells at the injury site 7—14 days post-injury alongside low-level immunosuppression for the next 60 days; four of these patients were found to have reduced spinal cord cavitation upon MRI. These results meant Asterias received FDA approval to carry out a complete cervical spinal cord injury trial, which represents the first targeted population for registration trials.

This ‘SCiStar’ trial will test three escalating doses of AST-OPC1 administered at 14—30 days post-injury in patients with neurologically complete cervical spinal cord injuries, has concluded recruitment of the initial safety cohort of the trial. The results of this cohort, which recruited three patients, so far indicate safety, and at 3 months post-injection the first patient in this cohort had demonstrated neurological improvement progressing from a complete ASIA Impairment Scale (AIS) A injury to an incomplete AIS C injury.

The second cohort is currently being recruited for, where Asterias are looking to administer 10 million AST-OPC1 cells to five patients to asses optimal dose range of AST-OPC1.