Stem cell medicine promises to revolutionize the treatment of human diseases and injuries, and has captured the hopes of the scientific community and the public alike. Perhaps nowhere is the potential of stem cells to treat human disease and injury more promising than for neurologic disorders. For this promise to become a reality, not only must basic research in this rapidly evolving field advance, but these advances must be translated through pre-clinical and clinical development into clinical practice. This bench-to-bedside pathway represents an enormous multidisciplinary effort, and many hurdles, both scientific and technical, must be overcome to bring stem cell therapies safely and effectively to reality. Traveling a path from bench to bedside is a relatively new opportunity fo researchers and provides novel challenges for training graduate students. The advent of this new frontier also means that students need additional training in the clinical aspects of the disease they are studying to identify therapeutic targets with the greatest relevance to human disease, inform preclinical safety and efficacy testing, and define preclinical outcome measures that parallel clinical metrics. In addition, the complexities of the regulatory processes required for human clinical trials and the business of taking research products to patients require a working knowledge of bedside in translational applications of laboratory research. Finally, students need preparation for alternative careers outside academia that will advance the NIH goals of enhancing biomedical research in the context of health and human services as a whole. This proposal seeks to fill this training need in the translational application of stem cell biolog to neurological disorders, a need that is not met by traditional neurobiology, stem cell or clinical graduate programs. The goal of the proposed Training Program in Stem Cell Translational Medicine for Neurological Disorders is to train a new generation of scientists in the translational application of stem cell biology to neuroscience. This proposed training program will be the first training grant on campus that is specifically focused on three integrated areas: stem cells, neuroscience and translation to the clinic. To train the next generation of stem cell translational scientists we will provide a Regenerative Neuroscience Boot Camp, clinical experience to understand research in the context of clinical translation, industry Internships to learn commercial and practical aspects of moving discoveries to clinical trials, coursework focused on providing the specialized scientific background required, a Stem Cell Translational Medicine in Neuroscience Retreat to provide an annual education in the latest cutting-edge science, and Workshops to focus on specific technologies. Especially important will be real and unique engagement with clinical faculty, the inclusion of internships with successful pharmaceutical and biotech companies and support from the FDA who oversee all stem-cell based trials in the US. UCI is in a strong position to provide the highest quality training to fellows preparing for a carer in the new field of stem cell translational medicine in neurological disorders.
Stem cell based treatments offer unprecedented hope to treat human neurological disorders. Training of new scientists having expertise in stem cell biology and neuroscience, together with industry, regulatory and clinical approaches, is essential to this goal. UCI provides an outstanding environment in which to establish a new NIH Training Program in Stem Cell Translational Medicine for Neurological Disorders to train predoctoral students in integrated and translational approaches to stem cell biology, neuroscience and human neurological disease.
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