Cystic fibrosis: Triple therapy is particularly effective in early childhood
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Cystic fibrosis is an incurable genetic disorder. Since 2020, a treatment known as triple therapy has addressed the underlying cause. Researchers at Charité – Universitätsmedizin Berlin found that the therapy is particularly effective in early childhood: Molecular channels in the mucous membranes, which do not function or function poorly in people with cystic fibrosis, reached nearly normal functional levels in children ages 2 to 11 following treatment.
The researchers say the results support starting treatment early. The study was published in the journal European Respiratory Journal.
People with cystic fibrosis struggle with thick mucus in their lungs, frequent respiratory infections and problems with the pancreas, liver and intestines.
"The reason for this is that, due to a genetic defect, certain molecular structures in the mucous membranes—known as CFTR channels, which regulate the balance of water and salt—do not function at all or do not function properly," explains Dr. Simon Gräber, head of the Junior Research Group "Precision Medicine in Cystic Fibrosis" at Charité's Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine.
"Until a few years ago, life expectancy for those affected was significantly reduced. Thanks to the triple therapy, which has been available since 2020, both life expectancy and quality of life for patients have improved dramatically."
The therapy—a combination of the three active ingredients elexacaftor, tezacaftor and ivacaftor—has been approved for children ages 6 and older since 2022 and for children ages 2 and older since 2023.
First indication in the sweat test
Studies in adolescents and adults have shown that triple therapy can restore CFTR channel function to about 50%. Until now, however, there had been no direct measurements of CFTR channel function in children.
Clinical studies showed that the combination drug led to greater improvements in children on the so-called sweat test than in older patients. The sweat test is a common procedure used to diagnose cystic fibrosis. It measures the salt concentration in sweat; a high salt content indicates that the channels are not functioning properly.
"With our study, we wanted to investigate this finding and examine, at the functional level, the extent to which the triple therapy restores CFTR channels in children, and whether this effect is more pronounced than in adults," says Prof. Marcus Mall, director of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité.
Functional impairment in children has been nearly completely corrected
Under the leadership of Charité, 26 children with cystic fibrosis, ages 2 to 11, who had at least one of the most common disease-causing genetic defects (the F508del mutation) were examined.
Before treatment began and again after four months, researchers measured lung function, performed a sweat test and assessed nutritional parameters. They also collected small samples of the intestinal mucosa using a painless procedure. The researchers transferred the samples to a culture medium and measured electrical current to investigate the function of the CFTR channels in the mucosa.
"A functional CFTR channel transports negatively charged chloride ions. We can detect and quantify this transport by measuring the current," explains Gräber. "The result was astounding: The triple therapy restored CFTR channel function to 90% or even 100%."
"We really hadn't expected that the children treated with the triple therapy would have values close to normal—that is, values similar to those of children without cystic fibrosis," adds Mall. "Such positive findings regarding the functional efficacy of triple therapy in children are truly remarkable and offer hope."
The younger, the more effective
The researchers compared the data collected in the study with results from a previous study they conducted using the same methodology among adolescents and adults.
"In comparison, the restoration of CFTR function was significantly more pronounced in younger children than in adolescents and adults. We were able to identify a clear correlation with age here: the younger the person, the more effective it is," says Gräber.
"If early treatment in childhood can prevent disease progression and the associated irreversible tissue changes and functional impairments—especially in the lungs—in the future, that would be wonderful."
The results are consistent with those of a previous study in which the research team led by Mall showed that children benefit greatly from triple therapy, with improvements in both lung function and changes in lung tissue.
"With our latest study, we now provide a mechanistic explanation—based on the functional improvement of the CFTR channel—for why starting treatment early is particularly promising," says Mall.
In further studies, the Charité team plans to use single-cell analyses to examine mucosal cells and investigate how triple therapy works at the molecular level. If the therapy is approved in the future for children as young as 1 year old, the researchers want to investigate whether starting treatment even earlier might be more effective at preventing disease-related changes.
Publication details
Jasmin Berger et al, Elexacaftor/tezacaftor/ivacaftor improves CFTR function to near-normal levels in children with cystic fibrosis, European Respiratory Journal (2026). DOI: 10.1183/13993003.00422-2026
Journal information: European Respiratory Journal
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PediatricsChildren's healthPulmonary medicineCommon illnesses & Prevention Provided by Charité - Universitätsmedizin Berlin Who's behind this story?
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