An Irish-led study has found that a new approach to treating people with cystic fibrosis (CF) could in the future reduce the need for lung transplants and lower the risk of death among those affected.
CF is Ireland's most common life-threatening inherited disease, affecting around 1,300 people here and 70,000 worldwide.
The condition affects the regulation of absorption and secretion of salt and water in various parts of the body including the lungs. This defect inhibits the flow of salt and water through the body's cells, causing a build-up of thick, sticky mucus, which can clog airways and harbour harmful bacteria.
The main cause of death in people with CF is lung disease, which is driven by severe inflammation and chronic infection in the airways.
In recent years, a number of new therapies have emerged, which are aimed at improving lung function and survival. However, the lack of effective anti-inflammatory and anti-infective treatments remain a major challenge.
This new study, which was led by a team at the Royal College of Surgeons in Ireland, found that one of the most aggressive bacteria found in the lungs of people with CF caused certain immune cells to change their metabolism.
This change caused the immune cells to produce a protein that causes more inflammation. The researchers identified that high levels of this protein were associated with worse lung function, and a higher risk of death or need for a lung transplant.
They then used a small molecule called MCC950 to reduce levels of the protein in a laboratory model of CF. They found that in addition to reducing inflammation, this also helped clear the lungs of bacteria.
This marks the first time that researchers were able to stop this protein in CF by targeting cell metabolism. This could potentially lead to a new approach to treating inflammatory diseases like CF.
"This is an important first step to significantly improving patient outcomes for people with CF. While more testing is required before delivering this to patients, we believe these results are very promising and could make this molecule a candidate for clinical trials," explained the study's joint senior author, Prof Gerry McElvaney, of the RCSI.
The researchers pointed out that previously, people with CF had a very low life expectancy.
"Due to improvements in medical treatment, these individuals are now living longer. However, they still suffer from a very severe disease. We hope that this advancement can lead to further improvements in outcome, better quality of life and eventually a normal life expectancy for our patients," commented the study's lead author, Dr Oliver McElvaney.
Details of these findings are published in the American Journal of Respiratory and Critical Care Medicine.
Discussions on this topic are now closed.