Cystic Fibrosis Research

CF is the most common life-shortening genetic disorder in the Caucasian population, and Children’s Healthcare of Atlanta and Emory University share the second largest pediatric and adult CF clinic in the nation, in terms of numbers of patients. This CF program within center focuses research efforts on developing new therapies, drugs and new devices that improve the quality and longevity of the lives of CF patients. The goal is to foster the growth of the premier internationally-recognized interdisciplinary CF basic and translational research program that leads to new breakthroughs which will advance a deeper understanding of the mechanism of CF disease and thus identify completely new areas for clinical intervention.

Emory University, Children’s, and the CF Foundation have made large investments in CF clinical care and clinical research at the Emory-Children’s CF care center over the past two decades, including support for the clinical operations, the hiring of new clinician-scientists and basic scientists dedicated to CF research, and the recent establishment of an independent adult CF program. The Emory+Children’s program serves more pediatric and adult CF patients than almost any other CF care center in the country. With support from Children’s, we established the Center for CF and Airways Disease Research (CF-AIR): a collaborative research team centered around translational and basic research in CF that takes advantage of the availability of CF patients at our CF care center and other assets at Children’s, Emory, Georgia Tech, and elsewhere in Atlanta. This will be achieved by supporting research projects and research cores that will draw together investigators with various expertise to solve critical problems associated with CF disease.

The scientific theme for the CF program, therefore, is “The Systems Ecology of the CF Lung”.

Within this theme, three disease foci have been defined as areas where our team will directly impact patients’ lives:

1)  Cystic Fibrosis-Related Diabetes

Developing new approaches to understand and control CFRD, especially the pulmonary consequences thereof.

2)  Acute Pulmonary Exacerbations

Identifying biomarkers of disease progression and developing innovative devices to monitor CF patients and enable rapid and proactive treatment of lung disease; and development of therapeutic approaches targeting the protein defective in CF (called “CFTR”), to better understand how drugs can impact the function of this protein.

3)  Early CF Lung Disease

Tracing the fundamental mechanisms underlying the early origin of pulmonary disease in CF patients from birth to 6 years of age.

Programs range from basic research on the underlying pathogenesis of CF to outcomes research defining the best treatment and prevention regimens for this group of children and young adults. Specific programs in the role of innate immunity in CF, the role of redox stress on pulmonary pathology in the CF lung, and systems biology approaches to understanding the airway ecology of the CF lung are in place.

In addition, a new focus area has developed around CF drug discovery and development. This builds upon the work of existing Center members, and others, which has now shown that the protein defective in CF, CFTR, is druggable, and that this can have great impact on patient outcomes. This new effort takes advantage of strengths at Emory and Georgia Tech including the Emory Institute for Drug Development and the Emory Chemical Biology Discovery Center.

Recruitment of investigators to the CF research program is underway, including both PhD scientists and physician-scientists. For more information, please contact Dr. McCarty.

For more information on Cystic Fibrosis, click here.