Training in the Development of Novel Interventions for the Treatment of Neurological and Neurobehavioral Disorders

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The mission of the NIH is to “…seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability.” Achieving this end requires scientists who are not only capable of generating that fundamental knowledge, but also driving the successful translation of that knowledge into therapeutic interventions to reduce illness and disability. The objective of the proposed two-year training program is to provide one pre-doctoral and one post- doctoral/clinical fellow per year with formal education in the translational processes needed for moving basic science discoveries to clinical practice. The program will capitalize on strong translational training opportunities already available at the University of Utah and will complement those opportunities with new programming. Specifically, trainees will benefit from: 1) a series of monthly workshops delivering formal education in the process of moving pharmaceutics, devices, and apps from discovery to clinical use; 2) substantive experiences in clinics, in which patients with disorders of relevance to the trainee’s research are seen and with whom the trainee will engage in the clinic and in patient support groups to gain greater insight into the needs of patients; and 3) an internship to gain first-hand knowledge of the regulatory requirements and practices required to move products through to clinical use. Trainees will conduct their primary research under the direction of a translational supervisory team consisting of a basic scientist, a clinician/clinical scientist, and a researcher with experience in translating basic research discoveries into the clinic. In consultation with this mentoring team, the trainee will design and conduct preclinical research using best practices for enhancing the translational potential of their work. At the end of this training, participants will be able to: 1) Describe the issues to be considered in designing basic, preclinical studies and making decisions about whether to move a preclinical study forward with respect to development of a therapeutic agent, device, or app; 2) Apply knowledge of these considerations to the development of their research proposal; 3) Evaluate the research proposals of others with respect to translational considerations; and 4) Create new knowledge through research and publications that incorporate appropriate translational principles. Together, these experiences will develop burgeoning “professional translators” poised to promote the translation of basic research findings to improved therapeutic outcomes in patients.