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A cocktail of three drugs could change the lives of patients with cystic fibrosis, researchers said Thursday.
They found that adding a third drug to an existing combination of two drugs could improve the symptoms of cystic fibrosis patients and could even help some of them to live longer.
The big problem: current drugs already cost around $ 300,000 a year and the addition of a third drug would greatly increase that cost.
Nevertheless, the results are interesting for researchers who treat cystic fibrosis, a rare genetic disease that causes lung and digestive problems and shortens the lives of patients.
"These results represent a major breakthrough in the treatment of cystic fibrosis, with potential for improving the health and possibly survival of all patients with the most common CFTR mutation," said Dr. Fernando Holguin from the University of Colorado, involved in studies. , said in a commentary published in the New England Journal of Medicine.
The journal publishes two articles on two experimental cystic fibrosis drugs, both developed by Vertex Pharmaceuticals. The results are also presented at the North American Conference on Cystic Fibrosis in Denver.
"Within 24 hours of taking the combination of pills, patients suffered less. Their sinuses were clearer. They had more energy, "told NBC News, Dr. Jennifer Taylor-Cousar of National Jewish Health in Denver, who was leading one of the study teams.
Cystic fibrosis is rare and affects only 80,000 people worldwide. More than 1,000 mutations in the CFTR gene may be causing this condition by affecting specific cell functions in the lungs, digestive tract and elsewhere. People of North European ancestry are the most likely to carry these mutations, and if two carriers have a child, that child has a 25% chance of developing the disease.
Most often, patients suffer from frequent lung infections and an accumulation of mucus that prevents them from breathing. Patients usually die before the middle age.
New drugs may help correct some cell dysfunctions, but their effectiveness depends on the particular genetic mutation causing the patient's disease.
A combination of two drugs was approved by the FDA earlier this year for patients with specific mutations. Patient groups complained about the price of $ 292,000 a year.
The researchers tested two experimental Vertex drugs in patients taking another double combination of lumacaftor and tezacaftor drugs. In both trials, patients from around the world either received the combination of one of two new drugs, or a placebo.
The interest of the third drug is that it helps correct a genetic dysfunction by adding a synergistic effect to the double combinations, said the researchers.
"This approach has the potential to treat the underlying cause of cystic fibrosis in approximately 90% of patients," wrote Taylor-Cousar and colleagues.
In both trials of the two experimental Vertex drugs, lung function improved from 11% to 13%, reported both teams.
This might not seem like a lot, but that adds up, say the cystic fibrosis experts.
"We learned from every cystic fibrosis trial, if you could slightly improve lung function, the other clinical outcomes observed," said Dr. Christopher Richards, a specialist in cystic fibrosis at Massachusetts General Hospital, who did not participated in the tests.
"These improvements will be huge for patients. What we do not know about these studies are the clinical results – exacerbations, hospitalizations. But thanks to other trials, we know that if you are able to improve lung function at the scale of what they did here, it should be correlated with improved clinical outcomes. "
"We anticipate that people who will start this medication sooner, children, will lead a normal life, except for having to take these medications every day."
In 2012, Vertex obtained FDA approval for Kalydeco, known as the generic name of ivacaftor. It was the first drug to tackle the underlying cause of cystic fibrosis symptoms. The company is developing drugs that treat other mutations and testing them with different groups of patients for permission to use these drugs to treat more people.
"Kalydeco alone has improved lung function by 10%," Taylor-Cousar told NBC News.
"It was a game changer. This reduced the time needed to need antibiotics, hospitalizations and significantly reduced the exacerbation rate by 50 to 60%. "
The addition of new drugs should improve these effects, she said. "In the end, we hope to see a longer survival," she said.
And early treatment can be better. "It is lung damage that causes early death in patients with cystic fibrosis. Therefore, if we can prevent early-stage lesions, we expect that these patients will be able to live a normal life, "said Taylor-Cousar.
The results are still at an early stage. Medications must be tested on larger groups of people over longer periods of time to determine if they have ongoing benefits. And, warn researchers, it would be years before the long-term effects are known.
But Vertex hopes to get FDA approval for at least one of the drugs by next year.
"We anticipate that people who will start this medication sooner, children, will lead a normal life, with the exception of having to take these pills every day," said Taylor-Cousar.
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