BDCRC - Career Enhancement Core

CAREER ENHANCEMENT CORE – PROJECT SUMMARY/ABSTRACT The neuronal ceroid lipofuscinoses (NCLs, Batten disease) are collectively the most prevalent childhood neurodegenerative diseases. Despite a growing understanding of the molecular and cellular pathophysiology, too few clinician investigators are conducting NCL translational research. This hinders development of the next wave of diagnostic and therapeutic approaches for these fatal disorders. The Batten Disease Clinical Research Consortium (BDCRC) Career Enhancement Core aims to prepare the next generation of clinician investigators to lead multidisciplinary, translational research in the NCLs. Specifically, we aim to (1) train diverse post-graduate clinician investigators with interests across the translational science continuum, through a structured one-year fellowship program in NCL research, and (2) expose diverse clinical professionals and rare-disease community audiences to multi-modal, short-term research training opportunities focused on state-of-the-art diagnostics, management, and research advances in the NCLs. In Aim 1, the most promising, diverse early-career clinician investigators will be recruited and selected from fields critical to innovative translational research in NCLs and related disorders. Funding will support career development activities and a research project for one fellow each year. Each fellow will be paired with a BDCRC mentor to complement the existing mentoring team at their home institution. In Aim 2, three NCL-focused educational initiatives will be launched for audiences ranging from doctoral students to faculty: (a) clinical research immersion sprints for up to four weeks of intensive clinical research training experience at an established BDCRC site, (b) translational research journal club to provide an open forum for critical review of recently published manuscripts, and (c) webinars covering key challenges and potential solutions in rare neurodegenerative disorders research, focusing on the NCLs and small-sample data analysis. These efforts leverage resources and expertise from across BDCRC sites, existing relationships with patient advocacy organizations, and the recent collaborative framework established by the Centers of Excellence Program of the Batten Disease Support, Research, & Advocacy Foundation. The Career Enhancement Core's commitment to diversity, equity, and inclusion is reflected in its approach to seek applications from individuals of underrepresented racial/ethnic groups, individuals with disabilities, and individuals from disadvantaged backgrounds. The Core will also prioritize gender equity as well as diversity of clinical disciplines and institutions during the selection of trainees and speakers. The efforts of the BDCRC Career Enhancement Core align with the Rare Disease Clinical Research Network's goal to establish and maintain an environment that fosters collaborative, patient-oriented, multi-site, multi-disciplinary research collaborations and career enhancement. The proposed work is directly in line with the commitment by the National Institute of Neurological Disorders and Stroke to develop researchers that are diverse, are trained in the rigorous conduct of science, and possess the skills needed to thrive in an ever-changing research landscape. Project Number: 1U54HD122210-01 | Fiscal Year: 2025 | NIH Institute/Center: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Principal Investigator: Elizabeth Berry-Kravis | Institution: HUGO W. MOSER RES INST KENNEDY KRIEGER, BALTIMORE, MD | Award Amount: $169,901 | Activity Code: U54 | Study Section: Special Emphasis Panel[ZTR1 RD-4 (02)] View on NIH RePORTER: https://reporter.nih.gov/project-details/1U54HD12221001