Cystic fibrosis (CF) research at the University of Washington spans from the bench to the bedside and beyond. Our adult CF research program collaborates closely with Seattle Children’s Hospital and the Seattle Children’s Research Institute. CF research is supported by the National Institutes of Health (NIH), Cystic Fibrosis Foundation (CFF), Food and Drug Administration, and other sources. The UW is a top-performing site in the Cystic Fibrosis Therapeutics Development Network (TDN), a network of more than 80 CFF-accredited Cystic Fibrosis Centers in the United States who collaborate to complete multicenter research. Our CF research team employs several research coordinators with experience in industry-sponsored clinical trials, investigator-initiated studies, observational research, and biospecimen collection.
Basic research
Fundamental research in CF at the UW spans investigations focused on microbiology and CF-affected organ systems. The research is supported by a CFF Research Development Program, which is led by Drs. Pradeep Singh and Ajai Dandekar and a NIH/NIDDK P30 award led by Drs. Christopher Goss and Lucas Hoffman. These research grants support investigation into immunology, cell biology, and microbiology. Basic science projects currently supported by these grants include studies of CF kidney organoids and the mechanism of aminoglycoside toxicity; Pseudomonas aeruginosa and Staphylococcus aureus infections of CF airways; approaches to rapidly ascertain antibiotic resistance profiles of bacteria without the need for culture; and inflammasome phenotypes in CF. The RDP and P30 together support the development of novel genetic tools, sequencing approaches, and the collection and analysis of clinical samples from people with CF. These programs also foster collaboration to enhance translational research through the sharing and expansion of repositories of human and bacterial specimens linked to clinical databases, enabling the development of improved assays and clinical outcome measures.
Clinical Outcomes/Epidemiology
Faculty in the CF research program have extensive experience in clinical outcomes research and epidemiology. Dr. Christopher Goss is co-Chair of the national CFF Patient Registry committee and has specific expertise in pulmonary exacerbations, international registry studies, and myriad other topics in CF. Dr. Kathleen Ramos’ research program is focused on clinical outcomes in CF, advanced CF lung disease, and lung transplantation. Trainees working with Drs. Goss and Ramos have access to large datasets for epidemiologic studies in CF. Drs. Siddhartha Kapnadak, Tijana Milinic, and Erika Lease are key collaborators on many of these epidemiologic projects.
Interventional Trials
Drs. Goss and Ramos lead the UW CF interventional trials program, typically participating in 20-30 active studies that include industry-sponsored interventional drug trials from Phase I-IV studies, multicenter investigator-initiated trials, and single-center pilot trials. Dr. Goss is the co-Executive Director of the CF TDN Coordinating Center, located in Seattle, with leadership of national CF clinical trials including ABATE (a study of IV gallium to treat nontuberculous mycobacterium), STOP360 (a study of IV antibiotic treatment of CF pulmonary exacerbations), and others. Dr. Ramos is the Interim Medical Director of the CF TDN Coordinating Center, the Director of the UW adult CF Therapeutics Development Center, and the national PI of an R01-funded clinical trial of a lung transplant education intervention at 11 CF Centers. Interventional clinical trials in CF at UW have included many of the CFTR modulators that have revolutionized the care of people with CF. The UW serves as a local, national, and international resource to collaborators for pre-clinical and clinical development of novel and repurposed therapeutics, advanced clinical trial design and conduct, and clinical and translational studies.
Mentorship in research
Dr. Goss leads a national career development program (Clinical Research Scholars Program) for junior to mid-career faculty involved in CF research. Drs. Goss, Singh, Ramos, Morrell, and Dandekar mentor UW Pulmonary and Critical Care Fellows in the Physician-Scientist pathway, postdoctoral fellows, and graduate students, with years of success obtaining mentored fellowship and career development awards for trainees, and subsequent independent research awards. There are opportunities for investigators new to CF research to leverage the Cystic Fibrosis RDP pilot awards and NIDDK P30 pilot awards to gain a foothold in the CF research space. The RDP and P30 have supported dozens of trainees who have gone on to careers in CF research.
