Cystic Fibrosis Research Development Program

The CFF RDP has been in existence at the University of Washington and the Seattle Children’s Research Institute (SCRI) since 1989. Our center’s three-decade history of promoting basic, clinical and translational CF research originated from the RDP, and the RDP remains its foundation. The scientific and human resources of the RDP have given rise to the Therapeutics Development Network (TDN) Coordinating Center (Goss/Hamblett PI’s), TDN pediatric and adult clinical sites (Ong and Ramos PI’s), and two CF NIH P30 grants including the current Translational Research Center to Expedite Novel Therapies in Cystic Fibrosis (Goss and Singh PIs). Our RDP has generated new knowledge about CF infections, lung and intestinal disease, and the basic biology of CF pathogens. The RDP has developed new therapeutics in use world wide, and has fostered collaboration within our institution, and internationally. The RDP has trained several generations of CF researchers and clinicians, including innovative physician-scientists committed to CF research over the duration of their careers, and leaders in CF research and care at other institutions. The RDP also developed internationally-valued resources such as isolate collections, mutant libraries, genomics resources, and research tools utilized by hundreds of investigators worldwide.

Cystic Fibrosis Research Translation Center

The University of Washington’s CF research program has a distinguished history of translational research, starting with CF pathogen biology and CF lung infections, with results including the development of CF therapeutics in use worldwide such as inhaled tobramycin; defining best practices for Pseudomonas aeruginosa (Pa) eradication; exacerbation treatment; and anti-inflammatory therapies. We also identified the first cases of nontuberculous mycobacteria (NTM) transmission in people with CF and performed important work on S. aureus small colony variants. We developed resources that are important for the national and international scientific community, such as the first whole-genome sequence of Pa, transposon mutant libraries, clinical isolate collections, new bacterial genomic methods, clinical outcome measures, and other tools. Simultaneously, we expanded our studies in NIDDK emphasis areas, refining our understanding of CF-related diabetes (CFRD) with novel studies of the CF pancreas, new biomarkers of kidney injury, making instrumental discoveries regarding the CF gastrointestinal (GI) microbiome with implications for growth in children with CF8, and leading key studies to move novel CF Transmembrane Conductance Regulator (CFTR) modulators into the clinic and to study their myriad non-pulmonary effects. Our center has also trained generations of CF researchers and clinicians and created the Therapeutics Development Network (TDN), the Clinical Research Scholars Program (CRSP), and the Cystic Fibrosis Statistical Expertise and Network (STATnet).

The UW CFRTC strategic vision is:

Our vision leverages Highly Effective Molulator Therepy (HEMT) initiation, particularly in infants and children, to discover novel aspects of CF pathophysiology, define the clinical manifestations and best treatment paradigms, and perform basic and translational research on disease aspects that persist despite HEMT