The combination of three drugs “could be a game-changer” for patients with cystic fibrosis, which is still incurable today.
A combination of three drugs could open new perspectives for patients with cystic fibrosis, better known as cystic fibrosis. “It could be a game-changer,” enthuses Dr. Steven Rowe, director of the trial, published in the New England Journal of Medicine.
Cystic fibrosis is a genetic disease that primarily affects the lungs and digestive system. It is the most common fatal genetic disease in the Caucasian population. It is characterized by the accumulation of thick mucus in various organs, including the respiratory tract, sinuses, pancreas and intestines. The life expectancy of patients does not exceed 50 years.
Genetic mutation
Cystic fibrosis is caused by various mutations in a gene called CFTR. The most common mutation is called F508del. Some sufferers carry only one copy of the mutation, in addition to another defect known as the “minimal function” mutation, others carry two.
Rowe’s team first tested a combination of two available CFTR modulators – the tzakaftor and the ivakaftor – plus an experimental modulator, called VX-659. Another trial then used the same existing drugs, plus a similar new drug, named VX-445.
Improved lung function
Researchers randomly assigned 54 adults with cystic fibrosis to the triple drug treatment or control group. In the control group, patients with an F508del mutation took placebo tablets, while patients with two copies of the mutation took tezakaftor and ivakaftor alone.
After four weeks, the trial found that the triple therapy had “pronouncedly” improved lung function in patients with both types of mutations. “The ability of these potential drugs to treat people with a single F508del mutation means that more people could benefit from them,” said Dr Michael Boyle. In France, the number of patients with cystic fibrosis is estimated at 7,000. There are as many men as women.
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