Research Information

A number of experimental techniques that involve measurements in CF patients are crucial to advancing CF science – including our understanding of basic aspects of disease pathogenesis, characterization of novel therapeutics directed towards the CF ion transport defect, and the evaluation of CFTR modulators in human subjects. The Clinical and Translational Core, established at our Center in 1999, provides necessary resources and expertise to provide these capabilities for a wide variety of CF scientists, significantly improving the ability to identify and translate CF scientific advances to new therapeutics. There are three main functions of the Core.

Core Function # 1: Design and Conduct in vivo Measurements of CFTR Activity in Human Subjects.

Measures of ion transport in human subjects are diagnostic of the CF defect, and can also be used to monitor the response to agents that alter ion transport pathways, including pharmacologic agents intended to restore CFTR activity. The Core can conduct assays of CFTR activity in human subjects, including: -Nasal potential difference (NPD) -PD measurements of the lower airway and sinus tract (by the endoscopic approach) -Sweat chloride analysis -Sweat rate -Rectal intestinal current measurements The Core also provides data management including secure storage and backup, as well as biostatistical and regulatory expertise to assist in the design, conduct, and interpretation of studies intended to test modulation of the fundamental aspects of the CF ion transport defect in vivo.

Core Function # 2: Conduct Cardinal Measures of Airway Epithelial Cell Function in vivo.

Epithelial host defense relies on an intact mucosal barrier. In the CF lung and/or GI tract, abnormal mucus, alterations in airway surface liquid (ASL), and delayed mucocililary transport (MCT) compromise host defense, and are key targets for novel therapeutic interventions and the evaluation of emerging concepts in CF pathophysiology. To address this, the Core provides expertise in three high impact, well utilized, and complementary techniques for assessing key aspects of mucociliary clearance in vivo:

-Micro-Optical Coherence Tomography (μOCT) for in vivo use by endoscopic probes. μOCT is an innovative technique that provides real-time imaging of airway epithelia, and can quantitatively determine ASL/periciliary layer (PCL) depths, ciliary beat frequency (CBF), and MCT of living cells. It provides an ideal system for secondary characterization of agents thought to alter ion transport, ciliary beating, or physical properties of the mucus while also providing new insight towards CF pathophysiology in the airways.
-Whole lung MCC by Tc99 clearance
-Mucus rheology and solid content

Core Function # 3: Support The Execution of CF Clinical Studies

Our Center has been highly active in the design and conduct of CF and CFTR-related clinical trials, and the Core provides the following requisite expertise and support to continue this momentum:
-Provides clinical trial design and regulatory support (including CF-related IND and/or IDE applications, coordinated with the UAB Center for Clinical and Translational Science)
-Collects and stores biospecimens, including nasal cells, airway tissue, spontaneously expectorated and induced sputum (sputum rheology can be performed), and general biospecimens
-Supports key clinical outcome measures in infants, children, and adults with CF (i.e. nutritional outcomes, spirometry, lung clearance index (LCI), and infant PFTs)

Contact Information

Core users are associated both within and beyond the CF Research Center; there is no limitation on who may access the Core. For additional information regarding resources and services, please contact:

Amit Gaggar, M.D., PhD.
Co-Director, CF Clinical and Translational Core

Steven M. Rowe, M.D., M.S.P.H.
Co-Director, CF Clinical and Translational Core