Cystic Fibrosis Translational Research in the Post-CFTR Modulator Era

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This P30 Cystic Fibrosis (CF) Research and Translational Core Center (CFRTC) is located at the University of Washington School of Medicine (UWSOM) and its affiliated institution, Seattle Children’s Research Institute (SCRI). This proposed P30 center program enhances an existing robust basic and clinical research base at these institutions totaling over $37 million direct costs in current research funding ($23.7 in DDK related funding representing 64% of total) and an additional $29 million in CF related infrastructure funding and includes 58 UW/SCRI and affiliate investigators in 19 departments and/or divisions across 4 Schools (Medicine: 51, Public Health: 3, Pharmacy: 2, Dentistry: 1). The UWSOM has a long history of excellence in CF-related translational research and microbiology with particular focus on gastrointestinal microbiome as well as CF related Diabetes (CFRD) in the past 5 years. The research for this P30 will focus on four Specific Aims: 1) Enhance the research cores and other infrastructure for CF research in NIDDK interest areas that promotes creative, interdisciplinary and clinically-impactful CF research. 2) Provide expertise to researchers world-wide to design innovative clinical studies that determine best practice and develop new therapeutic approaches to correct dysfunctional CFTR and its secondary consequences. 3) Direct a successful pilot and feasibility grant program that attracts young and “new to CF” investigators, emphasizing support for investigators studying CF-related diabetes, GI disease, renal physiology, and other extra-pulmonary disease manifestations. 4) Train and mentor the next generation of laboratory, clinical, and biostatistical science leaders in CF locally and across the US. The center will be led by Co-PDs, Bonnie Ramsey, MD, Professor of Pediatrics and Pradeep Singh MD, Professor of Microbiology and Medicine, CF investigators both internationally recognized in their respective areas of clinical research and bacterial pathogenesis. They will be supported by other key leadership: Lucas Hoffman MD, PhD, Professor of Pediatrics and Microbiology, Charles Frevert, DVM, ScD, Professor of Department of Comparative Medicine, Christopher Goss MD, MPH, Professor of Medicine and Pediatrics, Stephen Salipante MD, PhD, Associate Professor of Laboratory Medicine, Colin Manoil PhD, Professor of Genome Sciences and David Nichols, Associate Professor of Pediatrics. The center will consist of an Administrative Core (Ramsey and Singh, Directors) and three biomedical cores, Host-Microbe (Hoffman and Frevert, Directors), Genomics (Salipante and Manoil, Directors) and Clinical Translational (Goss and Ramsey, Directors). The CFRTC will operate a Pilot and Feasibility (P&F) program led by Drs. Singh and Nichols. Three newly approved P&F projects are included in the current proposal chosen through a competitive process and approved by the Internal Advisory Board.