New drug shows potential to treat cystic fibrosis patients

  • Research

Cystic fibrosis (CF) is a multi-organ disease that is characterised by excessive inflammation and mainly affects the lungs and digestive system.

There are over 70,000 people living with CF worldwide and each year a further 1,000 cases are diagnosed. Ireland has the highest incidence (per head of population) of CF in the world with approximately 1,300 children and adults in the country living with the condition.

For a person with CF, their body produces a thick and sticky mucus that clogs and obstructs their lungs, pancreas, intestines and other organs in the body. This mucus builds up in the lungs causing bacterial infection and inflammation, leading to progressive lung damage and respiratory failure.

New research from RCSI explores why the immune system causes excessive inflammation and how it can be reduced without impacting the body’s natural processes to control inflammation.

To study this, patients were recruited from the Beaumont Hospital CF Clinic. They were observed before and after three months of treatment with Kaftrio, a new drug that can be used on patients aged six years and over.

Kaftrio is known to have potential benefits for patients in terms of improvement of lung function and quality of life. This study found an additional benefit of Kaftrio,in its ability to reduce inflammation in CF patients. This finding could impact thousands of patients with CF in Ireland and across the world.

The research is published in the American Journal of Respiratory and Critical Care Medicine and also highlighted in an editorial in the journal. This study was carried out by an active and dynamic group of scientists led by Prof. Emer Reeves, and with clinical leads Prof. Gerry McElvaney and Dr Michelle Casey, from the Department of Medicine at RCSI and Beaumont Hospital Dublin.

Conducted in collaboration with the Data Science Centre in RCSI; the Irish Centre of Genetic Lung Disease, Department of Medicine, Department of Clinical Microbiology and the Cystic Fibrosis Unit in Beaumont Hospital, the research was funded by US Cystic Fibrosis Foundation (REEEVES21GO) and StAR MD Programme, 2020.

RCSI is committed to achieving a better and more sustainable future through the UN Sustainable Development Goals.