Researchers say a common drug Amphotericin may successfully treat patients with cystic fibrosis. Cystic fibrosis is a dangerous disorder affecting the lungs. National Institute of Health supported this research which appears in the journal Nature.
National Heart, Lung and Blood Institute and the National Institute of General Medical Sciences conducted this retrospective study. Both are a part of the National Institute of Health, USA. The study revealed that Amphotericin forms pathways to release bicarbonates. These bicarbonates restore the antibiotic property of the airway surface liquid. As a result, the cells present in the liquid fights against infectious agents, treating the disease.
What did they do in the research?
Researchers collected lung tissues from two sources: Human patients and pig models. Both were affected by Cystic Fibrosis. The scientists treated the infected tissue with the drug. They used a small molecule surrogate that acted as a pathway for the missing protein in the surface liquid.
They found that Amphotericin induced changes that were beneficial to lung function. The medication restored the disturbed pH levels and viscosity of the respiratory system. It also increased anti-bacterial activity.
Furthermore, they noted that it is better to inject Amphotericin directly to the lungs. This is to avoid any adverse side effects from the drug. However, they do not find it wise to rely on one study. They think there is a need to conduct repeated trials to ensure the safety of the drug. But experts are hopeful for the future.
Why current treatments are a failure?
A defect in the gene CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) causes cystic fibrosis. This gene makes a protein that regulates the movement of important substances through the cell surface. These substances include water, bicarbonate, and salt which are important for lung function.
In cystic fibrosis, the defective gene does not make the normal protein. As a result, there is little movement and mucus begins to clog the lungs. Sadly, treatment is rare for this condition.
Different people have different type of mutations, so the current treatment is not effective. In addition, genes of about 10% of people do not respond to any kind of treatment.
How this research will be helpful?
Amphotericin does not work by specifying a target. So, it has the potential to work no matter what the mutated gene might be. Moreover, it can work even when the whole protein is missing. It plays the role of a molecular prosthetic as it restores the lung function as a prosthetic device does.
On validation, this drug can save the lives of 30,000 and 70,000 patients in the U.S and around the globe respectively. Pharmaceutical industries already produce this drug on a large scale. Furthermore, it is labeled as a common fungicidal drug. So, there is no question about its availability.
Researchers are hopeful to see it as a potential treatment for the disease.