Remapping neurology through translation and innovation

 描述(由申请人提供)：临床医生-科学家在提出新的和有洞察力的科学问题方面处于独特的地位，这些问题受到患者观察的启发，但他们往往缺乏能够将他们的观察转化为精心设计的基本科学和翻译实验的专业知识。造成这种情况的原因可能有很多，但被引用最多的障碍是缺乏具体的培训、指导、资金和时间。如果这些障碍能够消除，更有积极性的临床医生和科学家可以从事以实验室为基础的转化研究，从而帮助扭转许多神经疾病的现状，在这些疾病中，基础研究正在以越来越快的速度进行，但转化研究滞后，大多数神经疾病患者得不到有效的治疗或治愈。在这里，我们提出了一个研究培训计划的医学博士或医学博士，谁已经完成了他们的神经科学相关专业的临床培训，并高度有动力追求职业生涯的医生-科学家在创新的实验室为基础的神经疾病的翻译研究。该项目包括在两名教师导师的密切指导下进行高强度的研究培训，他们选择提供互补的经验。受训者设计和实施独立的多学科研究项目，当他们过渡到独立支持时，他们可以随身携带这些项目，并以此为基础申请NIH K奖。研究项目旨在将神经疾病的遗传、分子和细胞病理生理学转化为预防、治疗或治愈的策略。学员将被鼓励从事协作和跨学科的项目，因为协作促进了他们的研究发展，而将学科联系起来有助于产生新颖和创新的想法。受训者将可以进入核心教职员工的22个实验室，但也可以与其他小组合作。实习生将与博士研究人员合作，参加期刊俱乐部、实验室会议和基础科学研讨会。课程包括一门关于疾病的神经生物学的课程，这是一项以患者为中心的研究证书计划，其中还包括实验设计和生物统计学，以及关于翻译研究中关键主题的结构化讨论。受训者还将接触到将治疗从实验室带到床边所需的过程，包括对Clinica试验方法和相关监管问题的指导。受训者参加关于制定K奖提案、赠款和科学写作、实验室和项目管理以及负责任的研究行为的强制性培训的讲习班。该计划的一个独特之处在于，未来的受训者可以在他们的临床培训结束之前知道他们被接受了，从而允许他们在剩余的临床时间内安排研究，从而在撰写K奖提案之前扩大他们将拥有的研究经验总量。该计划的主要预期短期结果是申请NIH K奖。

 DESCRIPTION (provided by applicant): Clinician-scientists are uniquely positioned to ask new and insightful scientific questions inspired by patient observations, yet, they often lack the expertise to be able to translate their observations into carefully designed basic scientific and translational experiments. There are likely many reasons for this, but the most cited barriers are lack of specific training, mentoring, funding, and time. If these barriers could be removed, more highly motivated clinician-scientists could pursue careers in laboratory-based translational research, thereby helping to reverse the current state of affairs in many neurological disorders, in which basic research is proceeding at an increasingly rapid pace but translational research is lagging, and most patients with neurological disorders are left without effective treatments or cures. Here we propose a research training program for MD-PhDs or MDs who have finished their clinical training in a neuroscience-related specialty and are highly motivated to pursue careers as physician-scientists in innovative laboratory-based translational research in neurological diseases. The program consists of intense research training under the close mentoring of two faculty mentors, chosen to offer complementary experience. Trainees design and conduct independent multidisciplinary research projects that they can take with them when they transition to independent support, and upon which they will base their NIH K-award application. Research projects are directed toward the translation of the genetic, molecular and cellular pathophysiology of neurological diseases into strategies for prevention, treatment or cure. Trainees will be encouraged to pursue projects that are collaborative and cross-disciplinary, as collaboration fosters their research development, and linking disciplines helps generate ideas that are novel and innovative. Trainees will have access to 22 laboratories within the core faculty, but could collaborate with other groups as well. Trainees will work with PhD researchers; participate in journal clubs, lab meeting and basic science seminars. The curriculum includes a course on Neurobiology of Disease, a certificate program in patient-oriented research, which also covers experimental design and biostatistics, and structured discussions on critical topics in translational research. Trainees will also be exposed to the processes required to bring a therapy from the lab to the bedside, including instruction in clinica trial methodology and relevant regulatory issues. Trainees participate in workshops on developing a K- award proposal, grant and scientific writing, and laboratory and project management, plus mandatory training in responsible conduct of research. A unique feature of this Program is that prospective trainees can know of their acceptance before their clinical training ends, allowing them to schedule research into their remaining clinical time, thereby expanding the total amount of research experience they will have before writing a K- award proposal. The main expected short-term outcome for this program is application for an NIH K award.