The goal of this project is to produce a stem cell-based therapy for stroke (also known as an ischemic cerebral infarct) that moves into human clinical trials within four years. Stroke is the third leading cause of death in the USA, and a leading cause of disability among adults. Currently, there are no effective treatments once a stroke has occurred (termed completed stroke). In this proposal we aim to develop human stem cells for therapeutic transplantation to treat stroke within the four-year period of the grant. Potential benefits will outweigh risks because only patients with severe strokes that have compromised activities of daily living to an extreme degree will initially be treated. Currently, there are no effective treatments once a stroke has occurred. In this proposal we will develop human stem cells for therapeutic transplantation to treat stroke. Using a novel approach, we will generate stem cells that do not form tumors, but instead only make new nerve cells. We will give drugs to avoid rejection of the transplanted cells. Thus, the treatment should be safe. We will first test the cells in stroke models in rodents (mice) in preparation for a human clinical trial. We will collect a great deal of data on the mice to determine if the stem cells indeed become new nerve to replace the damaged tissue and to assess if the behavior of the mice has improved. We will perform safety and toxicology testing of the cells in two animal species, including mice and a large animal model, as required by the FDA. A comprehensive, interdisciplinary team, comprised of stem cell biologists, neuropathologists, neurobehaviorists, neurologists and neurosurgeons with clinical trial experience, FDA regulatory experts, statisticians, and immunologists has been assembled from academia and the biotech community of different regions of California to fulfill the aims of this proposal. With this Disease Team participating in frequent meetings with the FDA, we intend to file an NID for a stem cell-based therapeutic for stroke within four years time.
Statement of Benefit to California:
Stroke (cerebral ischemia) is the third leading cause of death in California and in all states in the USA, and a leading cause of disability among adults. Currently, there are no effective treatments once a stroke has occurred (termed completed stroke). Hence, many Californians are affected by stroke and because of this can no longer work, socialize with their family or friends, or enjoy life. The profound effect on family members of a stroke victim is also not to be minimized, since they must change their own life in order to care for their loved one who has suffered a stroke. Moreover, even Californians in families who have not suffered from a stroke are affected because they are directly or indirectly paying for the care of stroke victims who end up on welfare. Hence, both the human and economic burden of stroke is tremendous. In this proposal we aim to develop human stem cells for therapeutic transplantation to treat stroke patients within the four-year period of the grant. With such therapy we feel that we can improve the plight of stroke victims.
We also believe that an effective, straightforward, and broadly understandable way to describe the benefits of this proposal to the citizens of the State of California is to couch the work in the business concept of “Return on Investment.” The novel therapy for stroke that will be developed as a result of our research program will provide direct benefits to the health of California citizens. In addition, this program and its many complementary programs will generate potentially very large, tangible monetary benefits to the citizens of California. These financial benefits will derive from two sources. The first source will be the sale and licensing of the intellectual property rights that will accrue to the state and its citizens from this research program if financed by CIRM. The second source will be the many different kinds of tax revenues that will be generated from the increased bioscience and biomanufacturing businesses that will be active in California because of the success of this CIRM proposal.