Irish study gives cystic fibrosis hope

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A new Irish-led approach to treating cystic fibrosis (CF) reduces inflammation, potentially reducing the need for lung transplants and lowering the risk of death.

The study, led by researchers at the Royal College of Surgeons in Ireland (RCSI), has been published in the American Journal of Respiratory and Critical Care Medicine.

CF is a genetic disease that affects approximately 1,300 people in Ireland and 70,000 worldwide. The main cause of death in people with CF is lung disease, which is the result of severe inflammation and chronic infection in the airways.

While a number of new therapies are aimed at improving lung function and survival, an absence of effective anti-inflammatory and anti-infective treatments remains a challenge.

Researchers found that one of the most aggressive bacteria in the lungs of CF patients causes certain immune cells to change their metabolism. Because of this change, the immune cells produce a protein that causes more inflammation.

High levels of the protein are associated with worse lung function and a higher risk of death or need for a lung transplant.

The researchers used a small molecule, called MCC950, to reduce levels of this protein in a laboratory model of CF. In addition to reducing inflammation, this also cleared bacteria from the lungs.

It is the first time that researchers were able to stop this protein in CF in vivo by targeting cell metabolism. This could lead to a new approach to treating inflammatory diseases.

“This is an important first step to significantly improving patient outcomes for people with cystic fibrosis,” said Gerry McElvaney, the study’s joint senior author.

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.

Oliver McElvaney, the study’s lead author, said that this can lead to “better quality of life.”



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