Cystic fibrosis: Triple therapy is particularly effective in early childhood
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…
Cystic fibrosis is a genetic disease that currently has no cure, causing thick mucus in the lungs, frequent infections, and issues with the pancreas, liver, and intestines. The condition arises from a genetic defect that prevents proper functioning of CFTR channels, which regulate water and salt balance in the mucous membranes.
In a study conducted by researchers at Charité – Universitätsmedizin Berlin, triple therapy, a combination of three drugs, has been found to be particularly effective in early childhood. This treatment, previously available to children aged 6 and older since 2022 and to those 2 and older since 2023, restores CFTR channel function to near-normal levels in children aged 2 to 11.
Molecular studies revealed that after four months of treatment, intestinal CFTR function reached 90-100% of normal, significantly more than in adolescents and adults.
Dr. Simon Gräber, head of the Junior Research Group Precision Medicine in Cystic Fibrosis at Charité, emphasizes that these findings support starting triple therapy early to prevent disease progression and irreversible tissue changes, especially in the lungs. Further research is planned to examine the therapy's effects at a molecular level and to investigate the potential benefits of initiating treatment even earlier.
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