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Cystic Fibrosis Patients Offered Hope with New Drug Combination

21/05/2015

Cystic Fibrosis Patients Offered Hope with New Drug CombinationDoctors say that a “ground-breaking” cystic fibrosis therapy could dramatically improve the quality of life for sufferers of the condition.

Patients usually die before they reach the age of 40 as they’re left prone to infection from the mucus that clogs and damages their lungs.

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But now, a major clinical trial on some 1,108 patients, the results of which were published in the New England Journal of Medicine, revealed that a combination of drugs had the ability to bypass the genetic errors that caused the condition and increase life expectancy as a result.

In the UK alone, one in every 2,500 babies are born with cystic fibrosis and the Cystic Fibrosis Trust believes the new findings could “improve the lives of many”.

A genetic condition, the DNA of cystic fibrosis sufferers contains an error which means the individual is unable to control salt and water levels in their lungs. A thick mucus forms and inexorably damages the lungs.

Antibiotics have been used historically to prevent infection, but nothing has been developed to address the underlying problem for most sufferers.

Lumacaftor and ivacaftor are the two drugs which when combined, improved the lung function of those patients that received them over the course of a 24-week trial.

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It was also reported that patients gained weight during the trial, something which was attributed to the mucus lining in the gut being affected too.

Professor Stuart Elborn, who headed up the Queen’s University, Belfast part of the trial, said: “It is not a cure, but it is as remarkable and effective a drug as I have seen in my lifetime.”

 

Photo credits: Discover magazine, The New York Time Magazine

 
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