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3 November, 2019 00:00 00 AM
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Drug trio could give patients with cystic fibrosis a new option

Drug trio could give 
patients with cystic 
fibrosis a new option

A three-drug combo that significantly improves lung function in cystic fibrosis patients could benefit 90% of people with the life-threatening disease, a new study suggests. It included patients with a single copy of the most common genetic mutation for the disease.  Results of the international phase 3 clinical trial led the US Food and Drug Administration to approve the therapy last month.  "This should be a major cause for celebration," Dr. Francis Collins, director of the US National Institutes of Health, wrote in an editorial accompanying the new study. Cystic fibrosis (CF) is a progressive genetic disease that causes thick mucus to build up in the lungs, digestive tract and other parts of the body, resulting in severe respiratory and digestive problems and other complications such as infections and diabetes.

"Although there are over a thousand different disease-causing mutations, nearly 90% of people with cystic fibrosis have at least one copy of the most common mutation, the Phe508del CFTR allele," said study corresponding author Dr. Raksha Jain. She's director of the Adult Cystic Fibrosis Centre at University of Texas Southwestern Medical Centre in Dallas.

About 80,000 people worldwide have mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, according to Jain. People inherit a gene from each parent that encodes the CFTR protein.

"This three-drug combination was highly effective in people with cystic fibrosis who inherited the Phe508del CFTR mutation, improving health outcomes and symptoms," Jain said in a university news release.

The study, conducted at 115 sites in 13 countries, included 403 patients with one mutated copy of the gene.  Patients were randomly selected to receive either the elexacaftor-tezacaftor-ivacaftor combo (Trikafta) or a placebo. The trial was co-sponsored by Vertex Pharmaceuticals, which makes the drug combo. Its list price is $311,500 a year. Patients' lung function was assessed at four and 24 weeks. Compared to those in the placebo group, patients who took Trikafta had significantly improved lung function at both checkups.

Symptom flare-ups were 63% lower in the Trikafta group, and these patients also had better questionnaire scores on quality of life and respiratory symptoms than the placebo group, according to the study.

Excessive amounts of salt in sweat is a hallmark of cystic fibrosis. People in the Trikafta group had a lower concentration of salt in their sweat than those in the placebo group, which suggests that the therapy targets the disease's underlying cause, Jain said.

For his part, Collins said that enthusiasm over the study's results should be tempered by the knowledge that 10% of patients won't be helped by this therapy.

"We must not abandon the patients with cystic fibrosis who … will not have a response to these drugs," he wrote. "The 'best day ever' for all of us traveling down this long road together will be the day when the more than 70,000 persons with cystic fibrosis worldwide do not need to take drug therapy at all and there finally is a permanent cure for cystic fibrosis that works for everyone." And 1% of the Trikafta group stopped taking the drugs due to adverse events, the study authors noted. Further research is needed to learn more about potential side effects, Jain said.

HealthDay

 

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Published by the Editor on behalf of Independent Publications Limited at Media Printers, 446/H, Tejgaon I/A, Dhaka-1215.
Editorial, News & Commercial Offices : Beximco Media Complex, 149-150 Tejgaon I/A, Dhaka-1208, Bangladesh. GPO Box No. 934, Dhaka-1000.

Editor : M. Shamsur Rahman
Published by the Editor on behalf of Independent Publications Limited at Media Printers, 446/H, Tejgaon I/A, Dhaka-1215.
Editorial, News & Commercial Offices : Beximco Media Complex, 149-150 Tejgaon I/A, Dhaka-1208, Bangladesh. GPO Box No. 934, Dhaka-1000.

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