Queen’s University team in cystic fibrosis breakthrough
Researchers at Queen’s University Belfast have been involved in groundbreaking research which could help cystic fibrosis (CF) patients aged 12 and over.
The researchers identified how a combination of three drugs can successfully treat the majority of people with CF by tackling the underlying cause of the disease.
The drug targets the root cause of CF, a genetic condition which clogs up the lungs and digestive system, making breathing difficult, increasing the likelihood of infections and resulting in an early death for those affected.
CF is a genetic condition affecting more than 10,400 people in the UK.
It is a life-threatening disease which results in the formation of thick mucus that builds up in the lungs and other parts of the body.
CF is caused by a defective protein that results from mutations in a particular gene.
Although there is currently no cure for the condition, it is possible to help control the symptoms.
Dr Damian Downey – a clinical senior lecturer at Queen’s University’s Wellcome-Wolfson Institute for Experimental Medicine, who co-authored the worldwide trial on a new triple drug combination called TRIKAFTATM in patients aged 12 years and over with CF – said it was a “success”.
“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,” he explained.
Last week, the Food and Drug Administration in the United States approved Trikafta for use with about 90 percent of patients with cystic fibrosis – those who have the most common cystic fibrosis gene mutation and who are aged 12 or older.