Irish researchers are leading a major new study, which aims to investigate the real-world effects of the newest cystic fibrosis (CF) medications as they become available for people with the condition.
CF is an inherited chronic disease that primarily affects the lungs and the digestive system. A defective gene causes the body to produce unusually thick sticky mucus that clogs the lungs and obstructs the pancreas, stopping natural enzymes from enabling the body to break down and absorb food.
Ireland has the highest incidence of CF in the world, with almost 1,400 people diagnosed here. Ireland also has some of the more severe forms of the disease.
Those affected experience a range of symptoms including frequent lung infections, persistent coughing, wheezing and shortness of breath.
The new study, known as RECOVER, will be led by a team from the Royal College of Surgeons in Ireland (RCSI) University of Medicine and Health Sciences. They will be collaborating with researchers in 16 clinical and university sites in Ireland, the UK, Europe and the US.
RECOVER will investigate how a new triple combination CF drug, Kaftrio, will affect people in their day-to-day lives, rather than in controlled clinical trial conditions.
Kaftrio is a successor to Orkambi. It has demonstrated striking improvements in clinical trials of people with the most common form of CF.
The study is due to begin in August. Over a two-year period, it will recruit patients and begin to examine clinical outcomes across CF centres in Ireland and the UK. In addition to collecting routine health measurements, the researchers will study detailed imaging, and functional, biological and quality of life measurements that could not be collected in clinical trials.
"Our study will allow us to discover in detail how this powerful new treatment affects the health and everyday lives of people with CF, to understand why different people might respond differently to the drug and to gain insight into how this treatment might affect the very significant treatment burden that people with CF currently endure," explained Prof Paul McNally, a respiratory consultant in Children's Health Ireland and lead researcher on the RECOVER study.
According to Dr Lucy Allen, director of research at the Cystic Fibrosis Trust in the UK, while data from clinical trials has shown that Kaftrio will make a significant difference to people's lives, as CF is a complex condition affecting many parts of the body, "it is important to understand the full impact of this drug".
"In the short term, the results of the RECOVER study may speed up access to Kaftrio around the world. In the longer term, it will provide information on tailoring care for each individual with CF," she noted.
RECOVER has been awarded €2.85 million by the Cystic Fibrosis Foundation in the US, €112,000 by the Cystic Fibrosis Trust in the UK and €100,000 by Cystic Fibrosis Ireland.
"Cystic Fibrosis Ireland is proud to support this major research study that will provide much new detailed data on the impact of new and innovative CF medications across Europe and North America," commented Cystic Fibrosis Ireland CEO, Philip Watt.
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