In cystic fibrosis (CF), a defective gene makes a defective protein that causes acidic and sticky mucus that clogs the lungs and puts patients at risk for bacterial infections. Because different people have different protein mutations, and 10% of patients with CF make no protein at all, treatments are limited. But amphotericin apparently has the potential to work regardless of the mutation and even when the protein is missing. The researchers liken it to a “molecular prosthetic,” because it restores function much as a prosthetic device replaces a limb.
In the study, supported in part by the National Heart, Lung, and Blood Institute, researchers used lung tissue from patients with CF as well as animal models. Instead of trying to correct the protein or do gene therapy, which the researchers say is not yet effective in the lung, they used a small molecule protein that can perform the channel function of the missing or defective protein.
They found that amphotericin restored pH levels, improved viscosity, and increased antibacterial activity.
Amphotericin also can be delivered directly to the lungs to avoid common adverse effects, the researchers say. Although more studies are needed, according to the National Institute of Health, “experts are hopeful.”