MultiStem is a biologic product that consists of undifferentiated human stem cells obtained from adult bone marrow or other non-embryonic tissue sources. The cells may be produced on a large scale for future clinical use and stored in frozen form until needed. Material from a single qualified donor may be used to produce hundreds of thousand or even millions of clinical doses of MultiStem, which are extensively characterized to ensure product consistency and safety.
MultiStem has great potential as a “best-in-class” cell therapy based on its ability to deliver therapeutic benefit through multiple mechanisms of action, its ability to be delivered “off-the-shelf” like a pharmaceutical product and its safety profile.
Neurological injury as a result of ischemic stroke accounts for 80% of all strokes. Recent progress toward the development of safer and more effective treatments for ischemic stroke has been disappointing. Despite the fact that stroke is one of the leading causes of death and disability in the United States, affecting more than 700,000 new patients annually according to the CDC, there has been little progress toward the development of treatments that improve the prognosis for stroke victims. The only FDA-approved drug currently available for ischemic stroke is the anti-clotting factor, tPA, which must be administered to the patient within three to six hours of the onset of the stroke. Administration of tPA after this time frame is not recommended, since it can cause bleeding or even death. Given this limited therapeutic window, it is estimated that less than 5% of ischemic stroke victims currently receive treatment with tPA.
We are developing MultiStem as a novel approach for the treatment of damage caused by ischemic stroke. In preclinical studies we have evaluated multiple delivery strategies, including intravenous delivery, performed at various times following a surgically induced ischemic stroke in well validated animal models.
In preclinical studies conducted by independent investigators at both the University of Minnesota and the Medical College of Georgia, significant functional improvements have been observed in rodents that have undergone an experimentally induced stroke, or that have incurred significant neurological damage as a result of neonatal hypoxic ischemia, and then received treatment with a single dose of MultiStem. Through research conducted by collaborators at the Medical College of Georgia and presented at the annual American Academy of Neurology meeting in April 2006, we observed that administration of MultiStem even one week after a surgically induced stroke results in substantial and long-term therapeutic benefit, as evidenced by the improvement of treated animals compared with controls in a battery of tests examining mobility, strength, fine motor skills, and other aspects of neurological functional improvement. These results have been confirmed in subsequent studies that demonstrate MultiStem treatment is well tolerated, does not require immunosuppression, and results in a robust and durable therapeutic benefit even when administered one week after the initial stroke event.
Upon completion of remaining preclinical safety studies, we intend to submit an IND for this application. The initiation of an initial clinical study will depend on the availability of capital resources.
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