Clinical and Specimen Core

Seattle Children'S Hospital, Seattle WA

Investigators

Linked publications, trials & patents

Abstract

PROJECT SUMMARY/ABSTRACT The Clinical and Specimen Core (CSC) provides services, expertise, and specialized training for cystic fibrosis (CF) research. The services focus on regulatory support, biospecimen acquisition and processing, and assistance with study design and conduct. We serve 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. Among the key studies currently supported by the CSC are those characterizing the physiologic and clinical impacts of highly effective modulator therapy (HEMT) on the systemic, digestive, renal, endocrine, and hepatic complications of CF in adults with established disease and among children with milder, and potentially reversible, comorbidities at initiation of HEMT. The Core supports the central aims of the University of Washington Cystic Fibrosis Research and Translation Center (UW CFRTC): to use HEMT initiation to discover novel aspects of CF pathophysiology, define the clinical manifestations and best treatment approaches for the “new” disease that CF will become, and perform basic and translational research on disease aspects that persist despite HEMT. The CSC will advance our understanding of the long-term consequences of CF across the entire age span following initiation of HEMT, including the burden of care, complications of CF-related diabetes, chronic GI symptoms, risk of GI cancers, risk of liver disease, and risk of kidney insufficiency secondary to nephrotoxic drugs and systemic inflammation in an aging CF population. Further, the CSC fosters collaboration across the CFRTC biomedical cores 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. We are well-positioned to continue to advance novel therapeutics through the drug development pipeline, and to enhance our major focus on improving the understanding of CF disease with the initiation of advanced therapies – specifically HEMT, which will forever change the clinical landscape in CF.