According to a team of researchers, a new treatment for cystic fibrosis could be the best treatment and even the cure for this deadly disease. The new treatment could help this born with the genetic condition to live longer, said the scientists.
Although there are several treatments on the market designed to alleviate the cystic fibrosis symptoms, like reducing the clogging in the lungs and that of the digestive system that make it very hard for the patients to breathe.
However, the new treatment is “groundbreaking” because it can modify the genetic problem that leads to the cystic fibrosis, a condition that affects more than 30,000 people in the United States, both children and adults.
The researchers said that the new treatment consists of two drugs: one is called ivacaftor and the other one is lumacaftor.
In the trial, these two drugs combined improved the symptoms of cystic fibrosis, improving the function of the lungs of young patients. The drugs helped the young patients reduce the dose of antibiotics they usually take in order to fight infections, which are very common in their condition.
Also, the new treatment helped the patients by improving their breathing, helped them put on weight and overall, improved their life quality.
Ivacaftor, one of the two drugs, has already been used separate to help children who suffer from a genetic mutation responsible for 5% of the disease.
The ivacaftor drug proved to be successful even used on its own, said the researchers.
The new trial wanted to find a cure for the genetic defect that causes more than half of the cystic fibrosis cases.
The researchers said that they didn’t even expect to get such positive results especially because the young patients did not have the protein they were after. This protein was supposed to sit on the cell membrane but instead it was hidden, scientists explained.
Professor Stuart Elborn, a researcher at the School of Medicine, Dentistry and Biomedical Sciences at Queen’s University Belfast and one of the scientists who conducted the new trial, explained that although the results were not “spectacular”, they are still a “breakthrough”.
According to Professor Elborn, the new trial confirmed that the genetic defects responsible for cystic fibrosis respond to treatment and they can be fixed.
Elborn said that although the new treatment is not a cure, it’s the most effective and remarkable one he has ever seen.
The scientists published their findings in the New England Journal of Medicine.
Image Source: thetimesgazette