OPC1 exhibits multiple reparative properties that have broad applicability to neurological injury and disease, particularly as a treatment for demyelination. Current research efforts are focused on the development of OPC1 as a candidate treatment for ischemic stroke and multiple sclerosis (“MS”), two severely debilitating conditions that afflict a large number of individuals (approximately 692,000 stroke cases and 10,000 new MS cases per year in the U.S. (www.cdc.gov/stroke, www.nationalmssociety.org)), lack any form of reparative treatment options, and for which demyelination is a central component to their pathology.
To develop OPC1 as a treatment for multiple sclerosis (MS), initial proof-of-concept efficacy data has been demonstrated in collaboration with Yale University using a non-human primate model of MS. Results of this study showed robust OPC1 engraftment that was associated with substantial remyelination of the lesioned primate spinal cord up to 5 months post-treatment. These data are in preparation for scientific publication. Subsequently, we initiated a collaboration with UC Irvine to assess OPC1 efficacy in additional mouse models of MS that better recapitulate the autoimmune components of the disease. Preliminary results indicate that in addition to OPC1’s capacity to remyelinate the lesioned spinal cord, the cells may also help stimulate proliferation of a distinct class of immune cells known as regulatory T cells that can help reduce or eliminate autoimmunity. Using the anticipated in vivo efficacy data that will be obtained from the collaboration, we will seek additional non-dilutive funding for further development of OPC1 as a treatment for MS.
For ischemic stroke, initial proof-of-concept efficacy data for OPC1 has been demonstrated in a collaborative study with the University of California Los Angeles using a mouse model of white matter ischemic stroke. Results of this study demonstrated that within the stroke injury site, OPC1 cells engrafted, reduced lesion formation and inflammation, and increased myelination, culminating in improved functional recovery. We have since initiated a second preclinical study in collaboration with the University of South Florida to test two different doses of OPC1 in a rat model of ischemic subcortical and white matter stroke. Results of these combined studies will be used to seek additional funding and guide further preclinical development of OPC1 as a treatment for ischemic stroke.