Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke
The goal of this project is to produce a stem cell-based therapy for stroke (also known as an ischemic cerebral infarct). 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. 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 and rats) in preparation for a human clinical trial. We will collect a great deal of data on the mice and rats to determine if the stem cells indeed become new nerve to replace the damaged tissue and to assess if the behavior of the mice and rats has improved. If successfully developed and commercialized, this approach has the potential for revolutionizing stroke therapy.
Stroke (cerebral ischemia) is the third leading cause of death in California, and a leading cause of disability among adults. Currently, there are no effective treatments once a stroke has occurred. 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. 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 California is to couch the work in the business concept of “Return on Investment.” Not only 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, but also this program will generate potentially tangible monetary benefits to the citizens of California.