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. More than 2 million people suffer a stroke in the U.S., Europe and Japan in each year, and the World Health Organization estimates that more than 15 million individuals suffer a stroke each year globally, resulting in 5.5 million deaths, and 5 million individuals that are permanently disabled. Increasing age is one of the most significant risk factors for stroke, and with the aging baby boomer population, the AHA has projected that healthcare costs associated with stroke and cardiovascular disease will climb dramatically in the years ahead.
Recent progress toward the development of safer and more effective treatments for ischemic stroke has been disappointing. Despite the fact that stroke is a leading cause of serious 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 in the brain 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, as well as other neurological conditions. 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 the University of Minnesota, Medical College of Georgia, and University of Texas Houston Health Center (UTHealth) 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.
At the American Heart Association International Stroke meeting in February, 2011 medical researchers from the University of Texas Health Science Center at Houston (UTHealth) presented data that demonstrated how MultiStem provided multiple benefits when administered in preclinical models of ischemic stroke. The study, conducted by leading researchers from the Department of Neurology at the UTHealth Medical School working in collaboration with scientists at Athersys, illustrated the potential benefits of MultiStem therapy for treating stroke. Researchers observed that intravenous administration of MultiStem one day after a stroke reduced inflammatory damage in the brain and resulted in a significant improvement in motor skills.
In the rat model of stroke used in the study, animals that received treatment with MultiStem showed statistically significant improvement in motor skills relative to animals that received placebo, and also showed reduced cell death, reduction of inflammatory cytokines and an increase in anti-inflammatory cytokines. Interestingly, researchers found that animals treated with placebo showed a reduction in spleen size and an increase in inflammatory cytokines in the blood, whereas animals that were treated with MultiStem showed normal spleen size and increased levels of anti-inflammatory cytokines in the blood. The spleen is believed to play a significant role in promoting and sustaining the inflammation that can result in substantial long-term damage following brain injury.
In previously published work, 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 well after the initial stroke event.
MultiStem has 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 blood vessel formation
- Augment tissue repair and healing
We are currently running a double blind, placebo controlled Phase 2 clinical trial to evaluate the safety of MultiStem administered intravenously to ischemic stroke victims. In contrast to the current standard of care, MultiStem is administered to stroke patients within approximately 1 - 2 days following the stroke, which we believe is a clinically practical time frame for the majority of patients. The study is being conducted at leading stroke centers across the United States.
For more information regarding this study, please contact the company at 216-431-9900 or visit this ClinicalTrials.gov link.