[REDACTED] Treatment for Patients Suffering an Acute Ischemic Stroke: A Phase II Clinical Study
Stroke is the leading cause of disability and the third leading cause of death in the United States. Recent estimates by the American Heart Association suggest that in 2011, ~800,000 people in the US suffered a stroke, with 80-90% of those strokes being ischemic strokes involving blockage of a blood vessel in the brain, resulting in a lack of oxygen and nutrients to the tissue and subsequent cell death. Calculated costs of stroke on our health care system point to total combined direct and indirect costs of $73.7 Billion dollars annually, with lifetime costs associated with caring for stroke victims approaching $200,000 per survivor.
Despite these statistics, there are few treatment options for people suffering strokes. Drug development efforts have largely focused on stroke prevention, "clot-buster" drugs, or neuroprotectants and have been almost universal failures. Only tPA, a thrombolytic or clot-buster, has received FDA approval for treatment of ischemic stroke, and unfortunately, must be administered within 4.5 hours of the onset of the stroke. As such, only 3-8% of all Americans suffering an acute ischemic stroke who could benefit receive tPA due to the delayed recognition of the symptoms associated with stroke, coupled with the limited window for receiving tPA treatment. This equates to ~650,000 Americans in 2011 who could have benefited from an alternative effective stroke therapy. Other than tPA, the primary standard of care and treatment for stroke victims is hospitalization and rehabilitation, which are clearly not intended to target the primary tissue injury and the associated lasting neurological deficits. The numbers of affected individuals, and costs to facilitate their care and rehabilitation, combined with the lack of current therapies reiterates stroke represents a current unmet medical need of significance.
Our company has been developing an adult stem cell product for treatment of stroke. We have completed extensive preclinical safety and efficacy studies with our stem cell product and have developed an FDA reviewed manufacturing plan for producing the cells for intravenous administration following stroke. We have received FDA authorization of an IND for using the stem cell product to treat patients suffering from an acute stroke in the first 1-2 days after onset, increasing the window of therapeutic intervention for stroke patients. We are currently enrolling patients in a Phase I clinical study evaluating the safety of the cells in ischemic stroke patients. We are submitting this application requesting funds to do process development to improve the formulation of the cell product to allow for administration of the product at all hospitals, as well as funding to test the stem cell product in a larger Phase II study, evaluating both the efficacy and safety of the cells. Funding this study could accelerate a novel cell based therapy for treating stroke patients who would otherwise have no therapeutic options.
The proposed project could benefit the citizens of California in at least four important ways, including: improving clinical outcomes and enhancing patient quality of life for patients that have suffered a stroke; reducing overall healthcare costs, especially those covered by the taxpayers of California; reducing the loss of economic productivity for working age individuals that suffer long term disability after a stroke, and; by creating high quality jobs in the state.
Stroke is a leading cause of death and serious long term disability, and represents a major area of healthcare costs. In 2011 the American Heart Association estimated that approximately 800,000 people suffered a stroke in the U.S. (85 – 90% are ischemic strokes). Roughly half of stroke survivors are disabled or experience permanent weakness on one side of their body. Approximately three-quarters of all stroke victims are over the age of 65, and of these, ~26% require full time institutional care following the stroke, while many others require home care or assistance from family members. According to U.S. Census data, California represents approximately 12% of the national population, which suggests more than 82,000 victims of ischemic stroke annually in California alone (although a 2006 study by the Milken Institute estimated the number of California residents suffering a stroke was more than 240,000 annually). Given the aging demographic profile, and that the risk of stroke increases substantially with age, the number of Californians affected by stroke is expected to increase significantly in the years ahead.
The economic burden of stroke is enormous. The national impact of stroke was recently estimated at more than $73 billion annually. Although there have been few efforts to precisely measure the economic impact within the state of California alone, the national data suggests that this exceeds $8.7 billion annually. This includes the cost of hospitalization, extended physical therapy and rehabilitation for patients with permanent weakness on one side of their body or more serious disability, long term institutional care or home care, and the loss in economic productivity. By improving clinical outcomes, and achieving a better quality of life for stroke patients, much of the downstream costs that are currently incurred could be minimized or avoided entirely. Given that most strokes occur in the elderly population, much of this economic impact occurs through the Medicare and Medicaid systems, and is born by the taxpayers of California. Over time, the economic benefits of reducing direct costs and improving productivity could be many billions of dollars for the state.
Finally, if CIRM makes the award, it would enable our company to establish a more meaningful commercial presence in the state of California, which could lead to the creation of many high quality jobs over time, such as in research and development, bio-manufacturing, and commercialization.