QUEEN’S INVOLVED IN GROUND-BREAKING CYSTIC FIBROSIS TRIAL

5 November 2019

Researchers at Queen’s University Belfast have been involved in ground-breaking research that holds promise for a vast majority of those with cystic fibrosis (CF).

The researchers identified how a combination of three drugs, ‘triple therapy’, can successfully treat the majority of people with CF by tackling the underlying cause of the disease.

The drug targets the root cause of cystic fibrosis, a genetic condition which clogs up the lungs and digestive system, making breathing difficult resulting in an early death for those affected.

Cystic fibrosis is a genetic condition affecting more than 10,400 people in the UK.  It is a progressive and life-threatening disease which results in the formation of thick mucus that builds up in the lungs and other parts of the body. It leads to severe respiratory infections that damage the lungs and cause them to fail.

Caused by a defective protein that results from mutations in the CFTR gene, the most common CF mutation is the F508del mutation. Although there is currently no cure for CF, it is possible to help control the symptoms and delay complications to make the condition easier to live with.

Dr Damian Downey, a Clinical Senior Lecturer at the Wellcome-Wolfson Institute for Experimental Medicine at Queen’s, was co-author on the worldwide trial to assess the safety and efficacy of a new triple drug combination (TRIKAFTATM) in patients with CF aged 12 years and older.

The study involved a four-week, randomized, double-blind, active-controlled trial in 107 patients who had two copies of the F508del mutation.

Dr Downey explained: “The trial was a success in demonstrating that this drug combination can potentially treat up to 90 per cent of people with CF by addressing the underlying cause of their disease.

“This new ‘triple therapy’ has the potential to transform the lives of people with CF. It results in a significant improvement in lung function and quality of life and also reduces the frequency of chest infections. This treatment will likely alter the future of CF care.”

The study was conducted with funding from Vertex Pharmaceuticals, which developed the triple-combination drug therapy, known as Trikafta.

Last week, the Food and Drug Administration approved Trikafta for use with about 90 percent of patients with cystic fibrosis — those who have the most common cystic fibrosis gene mutation and are 12 or older.

To support health-related research at Queen’s visit the Development and Alumni Relations Office website or contact Teresa Sloan, Head of Health Fundraising, telephone +44 (0)28 9097 5394. Medical queries arising out of this news story should be raised directly with a GP/consultant and not with DARO staff.

Media enquiries to Communications Officer or telephone +44 (0)28 9097 5292.

 

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