Marianne S. Muhlebach, MD, associate professor of pediatrics in the University of North Carolina School of Medicine, is one of six researchers nationwide awarded grants to study pre-symptomatic lung disease in infants and young children with cystic fibrosis (CF), under a new grant program of the National Heart, Lung, and Blood Institute (NHLBI).
Charles Esther, MD, PhD is co-principal investigator at UNC with Dr. Muhlebach. They are collaborating with Dr. Stephen Stick at the University of Western Australia who is the principal investigator at that site.
Results of the research could reveal how CF develops, which in turn could lead to interventions that delay or prevent disease progression.
Results of the research could reveal how CF develops, which in turn could lead to interventions that delay or prevent disease progression. The studies also could provide critical information to help resolve competing theories on the origin and progression of CF-associated abnormalities.
Established by the NHLBI, part of the National Institutes of Health, the Early CF Lung Disease program will fund six institutions involving investigators in the United States, Canada, and Australia as part of a basic and clinical science program to characterize fully CF lung abnormalities in infants and children from birth to age 6.
“We still do not understand well the nature of cystic fibrosis during the first year of life, and this new program will stimulate the research needed to help us do so,” said James Kiley, PhD, director of the NHLBI Division of Lung Diseases. “Our long-term goal is to prevent or at least delay the onset of CF-related lung problems in the 1,000 children born with CF each year.”
CF is an inherited disorder that affects secretory glands, such as those that make mucus and sweat. It primarily affects the lungs. Microbial infections, lung damage, and respiratory failure are common CF complications and can lead to death.
“We still do not understand well the nature of cystic fibrosis during the first year of life, and this new program will stimulate the research needed to help us do so,” said James Kiley, PhD, director of the NHLBI Division of Lung Diseases.
Emerging evidence suggests that CF-associated lung disease begins in infancy, although its signs and symptoms may not appear for years. With the advent of genetic tests that allow physicians to screen all newborns, CF can be identified a few weeks after birth, before symptoms appear. As a result, researchers can now explore the earliest stages of CF lung disease, monitor disease progress over time, and test potential interventions before lung damage becomes irreversible.
The award recipients and corresponding grants are:
* John P. Clancy, M.D., Children’s Hospital Medical Center, Cincinnati (HL116226)
* Stephanie D. Davis, M.D., Indiana University-Purdue University at Indianapolis (HL116211)
* Barbara I. Kazmierczak, M.D., Ph.D., Yale University, New Haven, Connecticut (HL116235)
* Marianne S. Muhlebach, M.D., University of North Carolina at Chapel Hill (HL116228)
* Felix Ratjen, M.D., Hospital for Sick Children, Toronto, Ontario (HL116232)
* Steven M. Rowe, M.D., M.S.P.H., University of Alabama at Birmingham (HL116213)