Overview
According to the American Heart Association (AHA), approximately 800,000 individuals in the United States suffered a stroke in 2008 - a number that is expected to increase over time with the aging of the "baby boomer" generation. Neurological injury as a result of a stroke represents one of the leading causes of death and disability in the U.S. and the rest of the world. Ischemic stroke, (caused by a blockage in blood flow to the brain) accounts for more than 85% of all strokes according to AHA estimates.
Recent progress toward the development of safer and more effective treatments for ischemic stroke has been disappointing. Despite the fact that stroke is the leading cause of disability and third leading cause of death, 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 four hours of the onset of the stroke. Administration of tPA beyond 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. MultiStem is a patented and proprietary biologic therapy 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 frozen until needed. Material from a single qualified donor may be used to produce hundreds of thousands or even millions of clinical doses of MultiStem, which are extensively characterized to ensure product consistency and safety. MultiStem is administered without tissue matching or immune suppression, analogous to type O blood.
In preclinical studies we have evaluated the safety and effectiveness of administering MultiStem intravenously 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.
MultiStem has been recognized by independent analysts as having 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 consistent safety profile. MultiStem appears capable of delivering a therapeutic benefit in multiple ways, such as through the production of factors that:
- Protect damaged or injured neurons
- Reduce inflammation common in ischemic injury
- Promote new bood vessel formation
- Augment tissue repair and healing
In October 2008, MultiStem was selected by an independent panel of analysts at Frost & Sullivan as the North American Product Innovation of the year.
On December 11th, 2008 we announced authorization by the FDA of an IND for the initiation of a Phase I clinical study involving administration of MultStem to patients suffering an ischemic stroke. The study will be a double-blind, placebo controlled dose escalation trial evaluating the safety and maximum tolerated dose of single-dose administration of MultiStem following an ischemic stroke, and patients will receive treatment two days after the intial stroke event. The trial is designed to include up to 48 patients and will be conducted at multiple clinical centers in the United States. This authorization will allow commencement of the third clinical study for MultiStem.
For more information regarding this study, please contact the company.