Adult Cystic Fibrosis Program

Cystic Fibrosis (CF) is the most common life-shortening genetic disease among Caucasians in the United States.  While substantial improvements in quality and longevity of life for Cystic Fibrosis patients have been achieved over the past four decades, the median survival age remains less than half of that for the general U.S. population.  Hence, aggressive care, new therapies, and new therapeutic targets are still in need of identification. The Emory Adult Cystic Fibrosis Program, as part of the Emory and Children’s Cystic Fibrosis Center, is poised to contribute to the health and well-being of our patients with CF through establishing the highest standards of clinical care as well as achievements in both basic and clinical research.

Cystic Fibrosis is a multi-organ system disease, with a plethora of functional defects.  However, most of the morbidity and mortality associated with CF results from the progressive loss of lung function, due to chronic inflammation and infection, with resultant oxidative stress, that arises from the loss of the functional protein encoded by the CFTR (cystic fibrosis transmembrane conductance regulator) gene.  The airway is a complex environment, with multiple mesenchymal and epithelial cell types working to generate and regulate the composition of the airway surface fluid, in concert with components of the innate immune system - primarily macrophages and neutrophils - that are responsible for clearing pathogens.  In the CF airway, the delicate balance between these cell types, and the signals that are passed between them, are disrupted by the loss of CFTR. 

CF scientists at Emory have formed a team to study the complex environment of the CF lung by considering each component as a member of a community, and applying the scientific principles of both systems biology and community ecology.  The scientific theme for this effort, therefore, is "The Systems Ecology of the CF Lung".  The goal is to create a CF Research Development Program, taking advantage of a variety of unique strengths and opportunities that exist in Atlanta in order to advance life expectancy for patients with this disease.  This initiative is spearheaded by Nael McCarty, PhD.

There are many diverse clinical studies within the Emory Cystic Fibrosis clinical research program.   First, we are a member site of the CF Foundation Therapeutic Development Network and participate in many multi-center studies of new therapeutic medicines and devices through this network, including recent and ongoing studies of CFTR potentiators and correctors, as well as observational studies that provide new information on the course and nature of CF disease. 

We are also involved in investigator-initiated research. Vin Tangpricha, MD has examined the role of Vitamin D in inflammation in CF lung disease, as well as its effects on outcomes during exacerbations of lung disease. On a similar theme, other investigators such as Jessica Alvarez, RD, PhD, are looking at nutrient content in meals, lean body mass, and outcomes.  Seth Walker, MD is starting projects looking at chronic respiratory failure screening and treatment, and antibiotic options during acute exacerbations of CF lung disease. Arlene Stecenko, MD, the director of the Emory+Children’s Pediatric CF Program, is performing clinical and translational studies looking at the oxidative stress seen in Cystic Fibrosis-related diabetes.  Lastly, several members of the adult team participate in clinical research committees for the CFF.