Cystic fibrosis is an inherited disease that affects tens of thousands of people in the United States and worldwide. There is currently no cure for the condition, but new research proposes a novel therapeutic approach that may soon stop the disease from progressing.
Our secretory glands are responsible for producing mucus and sweat, but in CF, these secretions build up in the lungs, blocking the airways and providing a fertile ground for bacteria. As a result, the condition causes severe and recurring lung infections.
Apart from the lungs, CF also affects other vital organs such as the liver and the pancreas. Additionally, it can affect the intestines, the sinuses, and the body’s reproductive organs.
At the moment, CF is incurable. However, an international team of researchers from the George Washington (GW) University in Washington, D.C., in collaboration with the University of Perugia and the University of Rome (both in Italy), may have discovered a new drug that can treat and halt the progression of CF.
Tα1 is a synthetic version of a natural polypeptide that was first isolated from the
CF occurs as a result of a mutation in the genetic code of a protein called cystic fibrosis transmembrane conductance regulator (CFTR). This protein normally regulates the activity of the chloride channel, which is responsible for the balance between salt and water in the lungs.
In CF, the mutation – called “p.Phe508del” – makes the CFTR protein degrade prematurely, which causes poor chloride permeability and can lead to lung inflammation.
In the new study, the researchers point out that the complex pathogenic mechanisms behind CF call for a therapeutic approach that combines several drugs. The authors add that until now, however, no single drug with multiple beneficial effects has been discovered for CF.
However, the researchers report that their study shows that Tα1 can correct the defects in the tissue of mice with CF, as well as the defects of cells from human patients that have the p.Phe508del mutation.
The study found that Tα1’s beneficial action is twofold: the drug was able to simultaneously reduce the inflammation typical of CF and improve the activity, maturation, and stability of CFTR.
Tα1 – known as “Zadaxin” on the market – has been clinically approved for more than 15 years in 35 countries, and it is widely used for treating viral infections, immunodeficiency disorders, and HIV.
Zadaxin is not currently available in the U.S., but the researchers say that the drug has “an excellent safety profile in the clinic when used as an adjuvant or an immunotherapeutic agent.”
Allan L. Goldstein, Ph.D., study co-author and Professor Emeritus in Residence of biochemistry and molecular medicine at the GW School of Medicine and Health Sciences, comments on the significance of the study:
“Right now there are multiple treatments for cystic fibrosis, and while these have improved life expectancy dramatically, there is still only a lifespan of about 40 years for patients. No one treatment can stand alone. We developed a single treatment that can potentially correct the genetic defect that causes cystic fibrosis and decrease the inflammation that happens as a result.”