Recent research into cystic fibrosis (CF) at Leeds Teaching Hospitals and the University of Leeds has found that highly effective new drugs, called CFTR correctors, are having a positive effect on lung -damaging inflammation. This is very positive news as inflammation in key driver of lung damage in CF. So, is it possible that there will be a cure for CF in the next ten years?

Cystic fibrosis is caused by a genetic mutation that disrupts the body’s ability to regulate salt and water transport in the body. This results in a mucus build up that causes damage to the lungs and other organs. The median age of death of a sufferer is currently just 31 years old.  

The research, funded by the UK Cystic Fibrosis Trust and Leeds Hospitals Charity, has shown that inflammation is overactive in CF and this is likely to be partially responsible for disease progression.

New drugs, such as Kaftrio, which have recently been approved by the NHS, increase the function of a key protein that is wrong. The Leeds team found that these drugs had a profound effect on downregulating damaging inflammation both in the laboratory and in real life. 

The means there is a real possibility that with these new treatments, inflammation in the lungs can be brought fully back to normal, and that by rectifying both the salt and water transport and normalizing the inflammatory response, a potential cure can be achieved within the next decade.

Professor Daniel Peckham, director of the adult cystic fibrosis unit at Leeds Teaching Hospitals said: “In the past, we've always thought that inflammation was due to infection and so we've attempted to treat that with antibiotics. In Leeds, we have shown that it's inflammation that appears to be causing a lot of damage in terms of the destruction of the lungs.

The research has also shown that the sodium channel is one of the mechanisms that's driving inflammation, and drugs that block that can reduce inflammation.

We think that this work is going to really contribute to the clinical improvement. We believe that within the next ten years there will be accelerated developments in new drug developments which will lead stabilization of disease. An effectual cure is in sight as long as drug therapies prove safe and are administered early in life as structural damage is unlikely to be reversible. 

We've already seen amazing improvement with Kaftrio, but what we don't know is how this new drug affects the cellular functions, and that's what we're going to be doing in Leeds - to find out whether we need to use other drugs to maximize the benefit and thereby get the eventual cure.”

Research into the disease is happening at an international level, with researchers in Leeds collaborating with researchers across the United Kingdom, as well as Switzerland and Australia. 

Daniel adds: “I think the way we're going to get this cure is if we all continue working together. Each little project is going to feed in, but what is important, is this is not just going to feed in for cystic fibrosis research but impact on other disease. Good quality research also has a massive impact on the quality of care that we provide across the board.