Project Summary/Abstract Leukodystrophies are a collection of rare genetic disorders that affect cerebral myelin development. Exciting recent therapeutic advances, including gene therapy development for adrenoleukodystrophy and metachromatic leukodystrophy, underscore the critical unmet need for therapeutic development more broadly across the leukodystrophies. The future of innovation, however, is limited by the pipeline of future translational researchers interested in rare diseases. The Rare Diseases Clinical Research Network (RDCRN) Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN; U54TR002823) is an NIH-funded research consortium for leukodystrophy collaboration and innovation across a network of 8 large US-based academic institutions. This infrastructure provides a unique opportunity to recruit and support the next generation of rare disease research leaders. We hypothesize that a formal education program in rare disease research targeting junior research coordinators who are early in their career will facilitate long-term commitment and passion for rare disease research. This is a population that is enriched with young scholars interested in science who are often undecided about career paths or have had insufficient exposure to translational research. The proposed program represents a novel collaboration between the Leukodystrophy Center of Excellence (LCE) at the Children’s Hospital of Philadelphia (CHOP), a GLIA-CTN site, and the Institute for Translational Medicine and Therapeutics Education (ITMAT Ed) programs at the Perelman School of Medicine, University of Pennsylvania. In 2021-2022, the LCE and ITMAT Ed piloted the Predoctoral Preparatory Program (P3) with 8 junior research coordinators, known as P3 scholars, based exclusively within the Children’s Hospital of Philadelphia (CHOP). In 2022-2023, the program expanded to include 14 coordinators working at sites across the GLIA-CTN. Each year we aim to enroll 12-15 scholars who are junior research coordinators working within the GLIA-CTN. We recognize the importance of diversity among future translational research leaders and will continue to recruit from populations underrepresented in medicine (UIM). In this application, we propose an education program, the Predoctoral Preparatory Program (P3), which will (1) provide a curriculum related to responsible conduct of research and (2) prepare the next generation of physician scientists in translational research through professional development workshops and mentorship. This proposal is to further the development and implementation of a one-year program focused on research and professional skills development, community building, and mentorship which is fully integrated into a hands-on research experience through the GLIA-CTN research network.

Project Narrative Leukodystrophies are a collection of rare genetic disorders that are predominantly fatal, progressive disorders of childhood with few targeted therapeutic options. Exciting recent advances, including gene therapy development for adrenoleukodystrophy and metachromatic leukodystrophy, underscore the critical need for continued innovation and a pipeline of future translational researchers interested in rare diseases. Leveraging resources within the Rare Diseases Clinical Research Network Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN) and educational expertise of the Institute for Translational Medicine and Therapeutics Education (ITMAT Ed) program, we propose an education program, the Predoctoral Preparatory Program (P3), targeting junior research coordinators within the GLIA-CTN which will (1) provide a curriculum related to responsible conduct of research and (2) prepare the next generation of physician scientists in translational research through professional development workshops and mentorship.