Date: 2015-09-09
Type of information: Publication of results in a medical journal
phase: preclinical
Announcement: publication of results in Regenerative Medicine
Company: Asterias Biotherapeutics (USA - CA)
Product: AST-OPC1 (oligodendrocyte progenitor cells)
Action
mechanism: cell therapy. AST-OPC1 are oligodendrocyte progenitor cells derived from human embryonic stem cells. The cells have been shown to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These 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.
Disease: spinal cord injury
Therapeutic area: Rare diseases - Regenerative medicine
Country:
Trial details:
Latest
news: * On September 9, 2015, Asterias Biotherapeutics, a leading biotechnology company in the emerging field of regenerative medicine, announced the publication of a manuscript in Regenerative Medicine relating to AST-OPC1 (oligodendrocyte progenitor cells). The publication describes the results from preclinical safety studies that were submitted to the FDA as part of an Investigational New Drug Application. AST-OPC1 is currently in a Phase 1/2a dose-escalation clinical trial for complete cervical spinal cord injury (SCI). The preclinical results showed that AST-OPC1 cells did not cause any adverse clinical observations, toxicities, allodynia or tumors. AST-OPC1 exhibited robust persistence and limited migration within the thoracic and cervical spinal cord. In addition, AST-OPC1 demonstrated nerve growth stimulating properties and remyelinating properties that supported restoration of function in animal models. The publication, titled \"Preclinical Safety of hESC-Derived Oligodendrocyte Progenitors Supporting Clinical Trials in Spinal Cord Injury,\" appeared online ahead of the print edition of Regenerative Medicine. The majority of safety testing was conducted in nude rats subjected to thoracic SCI, providing a well-established model of the target clinical population, and additional tumorigenicity studies were conducted in uninjured SCID/bg mice. Importantly, these rodent models enabled testing of AST-OPC1 in a large number of subjects and in an immunocompromised environment that was permissive to human cell survival. Both of these attributes facilitated assessment of key safety concerns associated with AST-OPC1 administration, including the resultant biodistribution, toxicity, and tumorigenic potential of the transplanted cells.
