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.
The majority of neurological disorders still remain without effective treatments, cures, or preventions. One contributing factor may be the lack of individuals who have a deep understanding of both the clinical conditions and of how to conduct translational laboratory research to use evolving technologies to develop treatments and cures. This grant supports an innovative education program for physicians who want to develop laboratory research skills so that they can be a bridge between clinicians and scientists, helping to translate the results of basic scientific research into new, innovative and effective preventions, treatments, or cures for persons with neurological injuries and diseases. Neurology is undergoing a transformation, and increasingly foundational neuroscience is being applied to new treatments, diagnostics and devices for neurological disease. Our conventional views of neurology are being remapped by new understanding, and the appreciation that there are many cross-cutting themes related to disease mechanism between heretofore separable disease groups. The present program aims to harness the strong interdisciplinary environment at the University of Pennsylvania, in order to train the translationalists of the future. In addition to unique programs that span new domains of neurology, our training programs emphasize the competency of our trainees to interact with professionals and researchers in other fields.