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Hope or hype for spinal injuries?
MENLO PARK, Calif.—Asterias Biotherapeutics Inc., a biotechnology company specializing in the emerging field of regenerative medicine, has 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 U.S. Food and Drug Administration 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 positive preclinical results support the general safety of AST-OPC1 and indicate minimal risk of the transplanted cells reaching unintended locations,” said Dr. Edward Wirth, chief medical officer. “In addition, the results indicate that AST-OPC1, when administered during the subacute phase of SCI, can act through multiple repair pathways that are relevant to SCI, including trophic factor signaling, cavity reduction and stimulation of axon outgrowth and myelination. We believe that the results summarized in this manuscript support the continued clinical development of AST-OPC1 as a subacute treatment for SCI.”
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.
“The breadth and depth of the preclinical studies now published provided the basis for a successful first-in-man Phase 1 study with AST-OPC1, and set up the foundation for overall safety in support of the use of AST-OPC1 in other neurological diseases, including multiple sclerosis and stroke,” said Jane Lebkowski, president of research and development and chief scientific officer at Asterias.
Not everyone agrees, though. In a somewhat scathing analysis, Seeking Alpha goes to some length to develop and underscore its criticism. Asterias is repackaging old clinical data as new, the analysts claim, writing in part that “it is worth pointing out that Asterias has not presented any new data or shown further development of Geron’s stem cell portfolio. [Geron was the original owner of the intellectual property]. The recent investor meeting that Asterias held on May 8, 2015, was more or less a review of Geron’s previously presented data on AST-OPC1. Asterias did review the trial design of their current Phase 1/2a clinical trial of AST-OPC1, which is underway. However, this clinical trial is nearly identical to Geron’s initial Phase 1 conducted in 2010, which Geron terminated midway through because the compound failed to show any signs of efficacy.”