Pilot and Feasibility Program

Abstract P&F Overview: The UNC Cystic Fibrosis Research and Translation Core Center (CFRTCC) has the long-term goal to achieve a systemic cure for cystic fibrosis (CF) across the gastrointestinal (GI) tract, pancreas, and lungs and thus aims to synergize and accelerate CF therapeutic development. Our CF research base is extensive, with 120 faculty members specializing in CFTR biogenesis, ion transport, genetics, gene therapy, GI and airway epithelial cell biology, mucus/mucin biochemistry and biophysics, microbiology/immunology, inflammation/infection, and clinical research. Current funding by our research base for CF-related topics totals over $37 million direct costs. To advance therapeutics development, we require accurate and efficient in vitro and in vivo testing of drug candidates, access to diverse epithelial cell supplies, new mucus/mucin technologies, and a clinical research network for patient specimens, clinical testing, and advisory services. The CFRTCC will maintain its focus on systemic CF pathophysiology and a broad spectrum of therapies, supported by a strong Administrative Core of Multi-PIs (M. Gentzsch, W. O’Neal, R. Boucher; Core A) overseeing budgetary, communication, training, and program enrichment activities. We will resume and enhance capabilities of the following four highly successful Research Service Cores, which are designed to provide assays, technologies, and reagents for UNC, national, and international investigators that are unique and not readily available elsewhere: 1) The Molecular/Functional Measurement Core (Martina Gentzsch, PI; Wanda O’Neal, Co-PI; Core B) offers comprehensive in vitro models and in vivo mice and rabbit models for studying CF molecular pathogenesis, novel targets, and therapeutic interventions; 2) The Cell Models Core (Scott Randell, PI; Scott Magness, Co-PI; Phillip Clapp, Co-PI; Core C) has a long history of offering advanced human primary cell cultures and has expanded its capabilities to support growing research demands with advancements in GI tract models, small airway physiology, and primary cell genetic manipulation; 3) The Mucus Biochemistry and Biophysics Core (Brian Button, PI; Core D) provides access to specialized methodologies for evaluating biochemical and biophysical mucus abnormalities related to CF pathogenesis, therapeutics, and novel biomarker development; 4) The Clinical Translation Core (Scott Donaldson, PI; Shehzad Sheikh, Co-PI; Core E) bridges basic scientific research with human-based studies by providing GI and respiratory specimens, creating sputum repositories, introducing novel imaging technologies (e.g., F19 MRI ventilation), and offering expert advice for CF therapeutic development. Our program also provides three Pilot and Feasibility Awards aimed at supporting early-career investigators and recruiting new faculty members with novel, relevant expertise to enhance our CF research capabilities and treatment options. Thus, the UNC CFRTCC provides a comprehensive range of high-quality services essential for studying CF pathogenesis and driving therapeutic development, with a commitment to advancing strategies aimed at curing all individuals with CF.