Multi-center program focusing on infants and young children could provide a roadmap for prevention
Researchers will 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). Results 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, Ph.D., 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.
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)
For more information or to schedule an interview, contact the NHLBI Communications Office at 301-496-4236 or firstname.lastname@example.org.