Researchers say a widely-used antifungal drug may hold promise for treating people with cystic fibrosis, a life-threatening genetic disorder that causes serious damage to the lungs.
Researchers say a widely-used antifungal drug may hold promise for treating people with cystic fibrosis, a life-threatening genetic disorder that causes serious damage to the lungs. In studies using human cells and animals models, the researchers found that the medication, called amphotericin, helps lung cells function in a way that could make it easier for patients to fight chronic bacterial lung infections that are a hallmark of the disease. The findings from the study, which was supported in part by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health, will appear in the journal Nature.
If human studies validate the findings, the use of the drug could be good news to the more than 30,000 people in the United States and 70,000 worldwide who live with cystic fibrosis, a disease with no cure and few treatment options. It holds special promise for a subset of patients, about 10 percent of the people with cystic fibrosis, who do not respond to any treatment.
“The really exciting news is that amphotericin is a medicine that’s already approved and available on the market,” said Martin D. Burke, M.D., Ph.D., leader of the study and a professor of chemistry at the University of Illinois in Champaign. “We think it’s a good candidate.”
Read more at NIH/National Heart, Lung, and Blood Institute
Image: This illustration depicts a microscopic view of the lining of the lung surface of a person with cystic fibrosis (CF). In people with CF, a protein that releases bicarbonate, a key infection-fighting agent (represented by red spheres), is missing or defective (brown ribbon structure). The drug amphotericin (white structure) can form channels to release bicarbonate in lung tissue, restoring the antibiotic properties of the airway surface liquid, the thin layer of fluid covering the surface of the lungs that plays a key role in maintaining lung health. (Credit: Rebecca Schultz, Carle Illinois College of Medicine)
