A new triple-drug therapy that tackles the genetic causes of cystic fibrosis has been shown to be highly effective in treating the rare life-threatening disorder, scientists reportedfollowing landmark clinical trials.<\/p>\n
The finding, 30 years after the gene that causes the disease was identified, was called a \"cause for celebration\" by Francis Collins, the director of the National Institutes of Health who was part of the team that made the original genetic discovery.<\/p>\n
Trikafta is a combination of three drugs that target the CFTR gene responsible for the disease, which affects over 1,000 people in Ireland.<\/p>\n
The gene results in the formation of thick mucus build-up in the lungs, digestive tract and other parts of the body.<\/p>\n
That in turn results in respiratory and digestive problems, and exposes patients to complications such as infections and diabetes.<\/p>\n
Average life expectancy is in the 30s and 40s and was historically even lower before advances in drugs that alleviate symptoms.<\/p>\n
The new therapy targets the most common mutation of the CFTR gene, the Phe508del mutation, which represents around 90%of cases.<\/p>\n
\"The results of a pair of phase 3 clinical trials in the Journal and in a simultaneous publication in the Lancet document impressive benefits,\" Mr Collins wrote in an editorial for the New England Journal of Medicine.<\/p>\n
The two trials examined how much air patients could expel in a forced breath, an established marker of the disease's progression.<\/p>\n
In the first trial, mean levels increased by 13.8%compared to a placebo, while in the second trial, mean levels rose 10% from baseline compared to an earlier drug combination.<\/p>\n
\"This should be a cause for major celebration,\" said Mr Collins, but he added that more work was needed on patients with other forms of mutations who would not respond to the new therapy.<\/p>\n
Beyond that, he added, the best outcome would come when the more than 70,000 people with the disease worldwide do not need drugs because of a permanent cure achieved through gene editing.<\/p>\n
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The rest is here: A new triple-drug therapy that tackles the genetic causes of cystic fibrosis has been shown to be highly effective in treating the rare life-threatening disorder, scientists reportedfollowing landmark clinical trials. The finding, 30 years after the gene that causes the disease was identified, was called a \"cause for celebration\" by Francis Collins, the director of the National Institutes of Health who was part of the team that made the original genetic discovery. Continue reading
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