in the airways is less sticky, and lung inflammation is greatly reduced: In individuals withcystic fibrosis
(CF), triple combination treatment can produce these long-term benefits.
The findings ofCharite Universitatsmedizin Berlin
and theMax Delbruck Centre
were recently published in theEuropean Respiratory Journal
. This type of drug, according to their research, relieves the symptoms of CF in many individuals.
Two years ago, a Charite-led study found that a three-drug combination therapy involving elexacaftor, tezacaftor, and ivacaftor is effective in a large proportion of patients with cystic fibrosis, a hereditary disease, meaning that the treatment improves both lung function and quality of life. Now, a team led by Prof. Marcus Mall, the principal researcher in both trials, has explored for the first time if this type of treatment is also beneficial in the long run, i.e. over a period of 12 months or longer. To investigate this, the researchers examined sputum, or secretions from patients' respiratory tracts. "In patients with cystic fibrosis, the mucus in the airways is very sticky because it doesn't contain enough water and the mucins, the molecules that form mucus, adhere too much due to their chemical properties. This results in thick, sticky mucus, which clogs the airways, making it harder for patients to breathe and leading to chronic bacterial infection and inflammation of the lungs," explains Mall, Director of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and the Christiane Herzog Cystic Fibrosis Center at Charite.
In the current study, the researchers show that a combination of elexacaftor, tezacaftor, and ivacaftor results in less viscous respiratory secretions and decreasing inflammation and bacterial infection in the lungs of cystic fibrosis patients. "What's more, the effects lasted over the entire one-year study period. This is really important because previous medications caused a rebound in the bacterial load in the airways," explained Dr Simon Graber, who also works in the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charite and was one of the co-leaders of the study. 79 adolescents and adults with cystic fibrosis and chronic lung disease participated in the trial.
"This is a major step forward in treating cystic fibrosis," Mall says. "At the same time, it would be premature to say that patients have been normalised, let alone cured. Chronic lung changes arising over many years of living with the disease cannot be reversed, unfortunately." This means patients with advanced lung disease will still need to rely on established treatments involving inhaling mucus-thinning medications, taking antibiotics, and physical therapy.
"We plan to forge ahead with our research on how to make treatments that address cystic fibrosis via the molecular defects that cause the disease -- like the triple medication combination studied here -- even more effective. This includes starting treatment in early childhood with the goal of preventing chronic lung changes wherever possible," Mall notes. "Aside from that, this therapy is not available to about ten percent of our patients right now due to their genetic conditions," Graber adds. "That's why we are also hard at work on research involving new molecular treatments so we can treat all people with cystic fibrosis effectively."
The researchers are also working to advance their understanding of mucus defects in cystic fibrosis and develop new mucolytics, drugs that thin and loosen the mucus. This research could also benefit patients with common chronic inflammatory lung diseases such as asthma and COPD.
Cystic fibrosis is one of the most common fatal hereditary diseases worldwide. As many as 8,000 children, teens, and adults are living with the disease in Germany today. An imbalance in salt and water transport across mucosal surfaces of the body causes people with cystic fibrosis to produce thick, sticky secretions that harm organs such as the lungs, intestine and pancreas. This leads to progressive loss of lung function and shortness of breath, which still significantly lowers life expectancy despite advances in treatment. Some 150 to 200 children are born with this rare disease in Germany each year.
- Published On Jul 10, 2023 at 12:57 PM IST
Join the community of 2M+ industry professionals
Subscribe to our newsletter to get latest insights & analysis.
Download ETHealthworld App
- Get Realtime updates
- Save your favourite articles