Cincinnati Children's Receives $5.5 Million Grant To Establish Rare Lung Disease Center
Cincinnati Children's Hospital Medical Center has received a five-year, $5.5 million grant from the National Institutes of Health (NIH) to establish the nation's first Rare Lung Disease Clinical Research Consortium.
The mission of the international consortium is to accelerate the development of diagnostics and treatments that will not only benefit individuals with rare lung diseases but also may be applicable to treating more common lung disorders.
The University of Cincinnati Medical Center will be a key strategic partner in the consortium, which is one of seven funded by the NIH under the Rare Diseases Act of 2002. Each consortium focuses on a unique cluster of rare diseases. Bruce Trapnell, MD, a pulmonologist within the Division of Pulmonary Biology at Cincinnati Children's, and Frank McCormack, MD, chief of the Division of Pulmonary and Critical Care Medicine at the University of Cincinnati, will lead the team.
The consortium will investigate such rare lung diseases as alpha-1 antitrypsin deficiency (Alpha-1), lymphangioleiomyomatosis (LAM), pulmonary alveolar proteinosis (PAP) and hereditary interstitial lung disease (hILD). The consortium will complement the Cystic Fibrosis Therapeutics Development Center, which Dr. Trapnell co-directs.
As part of the Consortium, Cincinnati Children's and the University of Cincinnati will establish a treatment center to which people from the around the world can come to receive specialized care for rare lung disorders. "This is part of our initiative to provide comprehensive lung care for patients, education for professionals, and research to improve our understanding of these diseases and to test and develop new therapies for them," says Dr. Trapnell.
Although the Consortium will focus on rare lung diseases, the knowledge gained can be applied to more common disorders, according to Dr. Trapnell. "Rare diseases can provide insight into the cause of disease from the vantage point of a definable molecular defect, which is tremendously powerful," he says. "PAP is an excellent example. A mouse genetically engineered to be deficient in for one of the body's natural growth-promoting molecules, called GM-CSF, unexpectedly developed PAP. Ultimately, this led to the discovery of the cause of the human disease: an antibody directed against GM-CSF. The field is now poised for critical translational research to bring these discoveries to the bedside."
Dr. Trapnell recently published a sentinel article on the topic in The New England Journal of Medicine.
"The study of rare disease requires very strong collaboration between patient communities and the medical investigators," adds Dr. McCormack, who is also scientific director for the Cincinnati-based LAM Foundation. "The patient support groups that are an integral part of the Consortium -- the Alpha-1 Foundation, LAM Foundation, and the Pulmonary Fibrosis Foundation -- will perform critical functions for the project, including recruitment of patients for clinical trials and assistance with patient education."
These patient advocacy groups have also formed the Rare Lung Disease Foundation Consortium, whose mission it is to facilitate the development of foundations for PAP and other diseases that do not yet have an organized patient research community. "The ability to move the field forward by addressing critical needs, whether they occur at the scientific, clinical or social level, is the unique strength of the Foundation Consortium," says Dr. McCormack.
Children's will partner with the Cleveland Clinic Foundation, Harvard / Brigham & Women's Hospital, Medical University of South Carolina, National Heart Lung & Blood Institute, National Jewish Medical & Research Center, Oregon Health & Sciences University, University of Texas Health Center, University of Florida Medical Center, and centers in Japan and Australia.
Contact Information
Jim Feuer, 513-636-4656,
jim.feuer@cchmc.org
