Overview
In 2020, Emory University, Children's Healthcare of Atlanta, Augusta University, and the Georgia Institute of Technology were awarded a P30 grant from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) to establish the Georgia Cystic Fibrosis Research and Translation Core Center (Georgia CF Core Center).
Dr. Nael McCarty, Marcus Professor of Cystic Fibrosis and Co-Director of The CF Center of Excellence, and Dr. Arlene Stecenko, Marcus Professor of Pulmonology and Co-Director of The CF Center of Excellence, are the Principal Investigators. This $5 million, five-year renewable grant will leverage the activities at these institutions to address critical issues associated with CF, particularly those within the NIDDK’s mission, and translate the findings more effectively to patient care.
The P30 mechanism will enable us to build on the investments already made by Emory and Children’s, creating a comprehensive CF research center.
To learn more about other aspects of CF and airways disease reseach, check out the CF-AIR center page.
The overarching goal of the Georgia CF Core Center is to improve the quality of life and overall healthy lifespan of children and adults with CF. Our strategy to reach this goal centers upon our ~900 pediatric and adult CF patients cared for within our clinical programs at the Atlanta and Augusta CF Centers. Our team of CF PhD-scientists, physician-scientists, engineers, and their trainees, use state-of-the-art approaches to study CF to identify underlying pathophysiology of non-pulmonary complications and co-morbidities, which in turn will inform novel primary and adjunctive therapies for this crippling disease. Because CF is a catabolic metabolic disorder, with complications such as CFRD and CFLD representing tipping points in that dysfunction can lead to rapid decline in lung function, we offer research resources to facilitate studies on the role of endocrine, GI, and nutritional status in the causes and consequences of CF pathophysiology that are sorely needed in CF research, along with studies to understand the impact that lifestyle and behavioral choices such as exercise, activity, diet, and sleep have on disease progression.
We address issues that are becoming ever more important to the health of CF patients, as successes with small-molecule therapeutics lead us to anticipate an “aging” CF population. The metabolic defects underlying CF, such as the development of CFRD and CFLD, are becoming the new factors that will limit life expectancy for PwCF. We integrate data from multiple platforms spanning from basic experimental approaches to electronic medical records to enable longitudinal study of our patients with the goal of identifying those changes that are associated with progression to co-morbidity. This will be accomplished by the coordinated activities of three biomedical research Cores supported by an Administrative Core that serves to ensure the efficient use of resources, to strengthen the intellectual environment for non-pulmonary research, and to gather investigators into the CF research community from all across Atlanta and Augusta. These activities will be invaluable to researchers as they work towards understanding complex conditions and comorbidities that impact the long-term outcomes in patients with CF and will enable translation of this new knowledge into the clinic.
Funded with partial support in September 2020, the Georgia CF Core Center leverages activities at these institutions to solve critical problems associated with CF. Center research focuses on non-pulmonary aspects of CF disease central to the mission of the NIDDK, including CF-related diabetes (CFRD), CF-related bone disease (CFBD), CF-related liver disease (CFLD), and the impact of CF on nutritional status and GI function. Unique to our Center compared to other CF P30 programs is the study of the relationship between these co-morbidities and lifestyle/behavioral choices such as diet, exercise/activity, and sleep. The Center takes advantage of established CF researchers in Atlanta who have attracted over $42M of institutional support over the last 12 years, and teams up with CF investigators in Augusta. These investigators, together with colleagues at other nearby institutions and accomplished scientists not previously focused on CF, comprise an investigative team with expertise in a broad range of relevant sciences. The proposed basic, clinical, and translational CF research supported via this application is centered around the ~900 people with CF (PwCF) followed by our CF clinical programs in Atlanta and Augusta. This large patient base enables multiple initiatives that will provide new knowledge critical to enhancing the quality and length of life of PwCF, including a Longitudinal Phenotyping Platform and the establishment of the Georgia CF Data Warehouse.
The 68 members of the Georgia CF Core Center are engaged in research focused on non-pulmonary aspects of CF and co-morbidities and their underlying mechanisms at multiple levels, from studies at the molecular level seeking to understand the link between mutant CFTR and the development of CFRD, to the nutritional, metabolic, and behavioral contributions to abnormal glucose metabolism and metabolic homeostasis. We address issues that are becoming ever more important to the health and wellness of people with CF, as successes with small-molecule therapeutics make it possible for us to anticipate an “aging” CF population. On the opposite end of the spectrum are studies in toddlers and young children on the initial stages of these co-morbidities which have the potential for defining early preventive interventions. We will integrate data from multiple platforms spanning from basic science to clinical research informatics to enable longitudinal study of patients with the goal of identifying factors associated with disease progression. We will identify new therapeutic targets and advance our understanding of current CF therapeutic and lifestyle approaches, ultimately leading to a more comprehensive understanding of CF disease. Specific Aims for the overall Georgia CF Core Center are:
Aim 1: To provide a robust, well-coordinated environment for biomedical research into the mechanisms and consequences of non-pulmonary complications of CF, including diabetes, gastrointestinal/hepatic disease, and nutrition, and to understand how lifestyle and behavioral choices could ameliorate or potentially exacerbate these comorbidities.
Aim 2: To facilitate and enable the expansion of Center Members’ funded research in non-pulmonary complications of CF, including the establishment of new collaborations.
Aim 3: To attract new investigators focused on non-pulmonary complications of CF who will bring new expertise that will advance new perspectives on pathophysiology and treatment.
We will accomplish these aims by the coordinated activities of three biomedical research Cores:
Diabetes, Endocrinology, and GI/Hepatology Core (DEG Core)
Nutrition, Lifestyle, and Behaviors Core (NLB Core)
Clinical Research and Informatics Core (CRI Core)
Overview of the P30 Phenotyping Platform utilizing the cores:
The phenotyping platform will be conducted at the CF Care centers at Augusta University and at Emory University in people with CF aged 4 years and older. The platform consists of an annual oral glucose tolerance test (OGTT) as well as measurements of body composition, systemic metabolomics/redox balance, physical activity, dietary intake, continuous glucose monitoring (CGM), and questionnaires on gastrointestinal health, overall symptomatology and quality of life, and quality of sleep. All of these data are available to Georgia CF Core Center investigators for future investigations as well as banked stool and blood (plasma and serum).