Antiviral Drug Could Offer New Treatment for Cystic Fibrosis

Cystic fibrosis is a genetic disorder in which the secretory glands in the lungs overproduce mucus, thereby impeding breathing and increasing the risk of bacterial infections. While this condition affects approximately 70,000 individuals around the world, there is currently no cure for cystic fibrosis.

Now, research led by an international team from George Washington (GW) University, the University of Perugia and the University of Rome, have determined that a drug used to treat viral infections may be effective at slowing cystic fibrosis disease progression. The researchers published their findings in the journal, Nature Medicine.

The drug, thymosin alpha 1 (Tα1), is a synthetic version of a naturally-occurring protein that plays a role in the immune system. As cystic fibrosis affects more than just the lungs – including the liver, pancreas and intestines – researchers are always on the lookout for a drug which would have multiple therapeutic effects.

A mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene is the cause of the inheritable disease. While the protein is responsible for regulating the chloride channel, which balances salt and water levels in the lungs, the cystic fibrosis mutation causes the protein to be degraded.

The prematurely degraded protein does a poor job of regulating the flow of salts into, and out of the cell, leading to inflammation in the lungs. According to the researchers, Tα1 can correct the malfunctions in sodium and water transport in tissue taken from mice with cystic fibrosis.

What’s more, the drug was also effective in human tissue taken from patients with the specific cystic fibrosis-causing mutation. Not only was the drug found to reduce the inflammation associated with cystic fibrosis, but the stability of the CFTR protein was improved.

Tα1 has been approved by various regulators and is marketed as Zadaxin in 35 countries around the world.  It’s primarily used to treat viral infections, including HIV. Currently, the drug is not approved by the FDA in the US, however the researchers believe that Zadaxin has an excellent safety profile in the clinic.

“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,” said Dr. Allan L. Goldstein, study co-author and Professor Emeritus in Residence of biochemistry and molecular medicine at the GW School of Medicine and Health Sciences. “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.”