Training Program in Stem Cell Translational Medicine for Neurological Disorders

描述（由申请人提供）：干细胞医学有望彻底改变人类疾病和损伤的治疗，并抓住了科学界和公众的希望。也许干细胞在治疗人类疾病和损伤方面的潜力没有比在神经系统疾病方面更有希望。为了使这一承诺成为现实，不仅必须在这一快速发展的领域进行基础研究，而且必须通过临床前和临床开发将这些进展转化为临床实践。这条从实验室到床边的途径代表了一项巨大的多学科努力，必须克服许多科学和技术障碍，才能安全有效地实现干细胞治疗。从“实验台到床边”的路径对研究人员来说是一个相对较新的机会，也为培养研究生提供了新的挑战。这一新领域的出现也意味着学生需要在他们正在研究的疾病的临床方面进行额外的培训，以确定与人类疾病最相关的治疗靶点，为临床前安全性和有效性测试提供信息，并定义与临床指标平行的临床前结果指标。此外，人体临床试验所需的监管程序的复杂性和将研究产品带给患者的业务需要在实验室研究的转化应用中具有“床边”工作知识。最后，学生需要为学术界以外的其他职业做好准备，这将推动NIH在整个健康和人类服务背景下加强生物医学研究的目标。该提案旨在填补干细胞生物学转化应用于神经系统疾病的培训需求，这是传统神经生物学，干细胞或临床研究生课程无法满足的需求。拟议的神经疾病干细胞转化医学培训计划的目标是培养新一代的科学家， 干细胞生物学在神经科学中的应用。这项拟议的培训计划将是校园内第一个专门针对三个综合领域的培训补助金：干细胞，神经科学和翻译到诊所。为了培养下一代干细胞翻译 科学家，我们将提供一个再生神经科学靴子营，临床经验，以了解研究的背景下，临床翻译，行业实习，以学习商业和实践方面的移动发现临床试验，课程集中在提供所需的专业科学背景，干细胞转化医学神经科学务虚会提供年度教育的最新前沿科学，和研讨会，重点放在具体的技术。尤其重要的是与临床教师的真实的和独特的接触，包括与成功的制药和生物技术公司的实习，以及监督美国所有干细胞试验的FDA的支持。UCI处于有利地位，为准备在神经系统疾病干细胞转化医学新领域担任护理人员的研究员提供最高质量的培训。

DESCRIPTION (provided by applicant): 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.