Cystic fibrosis may have new medication

People with cystic fibrosis could have hope thanks to a new medication that has proven to drastically improve the pulmonary function of those who suffer it.

The pill is the first drug that attacks the cause of the problem and not the symptoms of this hereditary disease; However, only 5% of people who suffer from the condition have the mutation necessary for the medicine to work. A second drug is under investigation so that with the combination of the two, they can correct the disease more effectively.

Peter Mueller , scientific director of Vertex Pharmaceuticals in Cambridge, Massachusetts, and his team, tested the medication VX-770 and a placebo to a group of 161 people with cystic fibrosis over a year.

The results showed that most participants recovered up to 60% of regular lung function. Those who received VX-770 they had an improvement of about 20% in their lung function compared to those who received a placebo.

"The results exceeded our expectations," Mueller said after publicizing the discovery.

Cystic fibrosis is a genetic disease , presented more frequently in children, which attacks the whole body, progressively causing disability and possibly death. It affects the lungs, digestive system, sweat glands and other areas.

The cause of the cystic fibrosis it is due to a defect in a protein of the lung cells, called the transmembrane conductance regulator of cystic fibrosis (CFTR, for its acronym in English). The drug VX-770 attacks and corrects the damage caused by this protein.

The CFTR proteins form channels on the surface of the membrane of the lungs and other cells that carry chloride ions outside and inside the cell. When they fail, the lungs begin to fill with mucus , making breathing difficult and, to the most vulnerable patients, infections.

The VX-770 corrects the defect and "opens the channels", says Stuart Elborn , from Queen's University in England and principal investigator of the test.

Vertex announced that it expects that in 2 or 3 months the medicine will be approved by the United States and Europe.

Source: Cystic Fibrosis Foundation, News Scientist and the Mexican Association of Cystic Fibrosis A.C.

Video Medicine: New Therapies to Treat Cystic Fibrosis (August 2022).