Training Program in Stem Cell Translational Medicine for Neurological Disorders

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PROJECT SUMMARY 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. Traveling a path from “bench to bedside” is still a relatively new opportunity for researchers and provides novel challenges for training graduate students. Historically, pre-doctoral neuroscience students were trained in understanding the basic biological mechanisms underlying how the brain functions and guides behavior, learning and memory, movement, and to identify the processes that become dysregulated and result in neurological disease. Indeed, the biological mechanisms and causes for many neurological diseases have been identified, paving the way for the generation of transgenic animals and model systems to study neurological disorders. Critically, these advances, combined with the advent of stem cell biology, open a path to treat neurologic diseases with transplantation of stem cell derived cell populations or to develop treatment strategies in stem-cell derived cell models. This is a path that requires training beyond basic science. Accordingly, the goal of our Training Program in Stem Cell Translational Medicine for Neurological Disorders is to provide opportunities for pre-doctoral trainees to perform translational "bench" research that could treat human neurological disease and injury, in the spirit of the NIH vision for translational medicine. Additionally however, to meet the challenges presented by the advent of this new frontier, we provide opportunities for trainees in the clinical aspects of the disease they are studying. These opportunities enable identification of therapeutic targets with the greatest relevance to human disease, inform preclinical safety and efficacy testing, and define preclinical outcome measures that parallel clinical metrics. Furthermore, this program familiarizes trainees with the complexities of the regulatory processes required for human clinical trials and the business of taking research products to patients. A novel, and highly successful, aspect of the training is coaching in communication, conflict resolution, interpersonal and group interactions skills. These skills are important both for success in working in interdisciplinary teams and for helping in the success and retention of underrepresented minority students. Taken together, these opportunities will prepare students for careers inside and outside academia and will advance the NIH goals of enhancing biomedical research in the context of health and human services as a whole. The program we have implemented thus fills a significant training gap in the translational application of stem cell biology to neurological disorders, not typically met by traditional neurobiology, stem cell or clinical graduate programs.