Researchers say a drug that is already available on the market can help those affected by cystic fibrosis.

An antifungal medication may help people with cystic fibrosis.

Amphotericin, which is an antifungal medication, might help people with cystic fibrosis fight the chronic bacterial lung infections that tend to occur with this disease, according to a recent study.  This is especially good news since amphotericin is already available for patients in generic form.  The study, was published in journal Nature.

What is Cystic fibrosis?

Cystic fibrosis is present at birth.  In a person without cystic fibrosis there are proteins which manage the way we produce mucous and sweat.  In people with cystic fibrosis these proteins are either faulty or absent.  The faulty or absent proteins changes the way cells, tissues, and glands make mucus and sweat.  With cystic fibrosis, thickened mucus can build up in affected organs (like the lungs). The mucus build up makes the organs prone to chronic infections causing organ damage which can be life-threatening.  People with cystic fibrosis are also more prone to inflammation that can cause problems in organs, such as the lungs or pancreas.

While cystic fibrosis can affect different organ systems, such as the digestive tract, cystic fibrosis in the lungs can cause the most severe complications.  Thick mucus can create severe problems in a person’s lungs, as it not only causes issues with breathing but can also lead to chronic bacterial infections.

 

Treatment is available, but it does not work for everyone. This is because people have different types of mutated proteins, and some people do not make any proteins at all, which can make it difficult or impossible for doctors to treat.  People with cystic fibrosis often use treatment techniques that involve loosening the mucus and may have learned different ways of breathing and coughing. Some people take prescribed medications, such as bronchodilators and mucus thinners.  The primary aim of treatment is to slow down the progression of lung disease as much as possible. This is where amphotericin may come in.

In addition, people with cystic fibrosis could administer amphoteracin directly into the lungs, which should reduce side effects.

Amphoteracin and Cystic Fibrosis

The research team used lung tissue from people who have cystic fibrosis, as well as animal models of cystic fibrosis, to see how amphoteracin would drive changes in lung tissue.  They discovered that amphotericin did spark changes in the lung tissue and that these changes should have an association with improved lung function.  Specifically the researchers found that amphoteracin restored the acid balance (pH level), increased antibacterial activity, and thinned mucous.

The research team suggested that amphotericin would work for all people with cystic fibrosis, no matter what type of protein mutation they have; it can work even if that protein is absent.  This is because the amphoteracin drug seems to replicate the function of a working protein.  Amphoteracin treatment would be especially valuable for the people with cystic fibrosis who do not make the protein at all — currently, these people do not respond to any available treatment.

Next Steps

Certainly scientists need to do more research to test amphoteracin humans before doctors can offer this treatment to people with cystic fibrosis.  However, this research should be able to be conducted rather quickly as amphoteracin is already approved by the FDA as a safe and effective agent for fungal infections.