Can Amphotericin Aid in Treatment of Cystic Fibrosis?

cystic fibrosis
Researchers see promise in NIH study

Researchers say an 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 animal 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.”

In their studies, the researchers used lung tissue from patients with cystic fibrosis, as well as pig models of cystic fibrosis, and found that amphotericin spurred a host of changes associated with improved lung function — restoration of pH levels, improved viscosity, and increased antibacterial activity, among others. 

Cystic fibrosis is caused by a defect in a gene called CFTR (cystic fibrosis transmembrane conductance regulator). This gene normally makes a protein that controls or channels the movement in and out of cells of such materials as salt, bicarbonate, and water—all of which are important to normal lung function.

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