Great research rarely happens in isolation, and a new study relating to cystic fibrosis is a powerful example of what can happen when clinical expertise and specialist research capabilities come together.

Researchers studied children with cystic fibrosis before and after starting elexacaftor-tezacaftor-ivacaftor (ETI) triple therapy, looking beyond clinical improvements to understand what was changing inside their cells.

That’s where Children’s Medical Research Institute’s (CMRI) expertise came in. Researchers Dr Mark Graham from CMRI’s Biomedical Proteomics Facility and Dr Nader Aryamanesh and Dr Lee Marshall from CMRI’s Bioinformatics team helped analyse thousands of changes across genes, proteins and phosphorylation pathways to provide a detailed picture of how children’s cells were responding to the treatment.

The study found that after treatment, several molecular abnormalities associated with cystic fibrosis began to reverse, including changes linked to inflammation and protein kinase activity.

Published in iScience, the findings could ultimately help identify blood-based biomarkers to monitor how children respond to treatment.

CMRI worked alongside clinicians and scientists from The Children’s Hospital at Westmead, the University of Sydney, Westmead Institute for Medical Research (WIMR) and the Australian Genome Research Facility (AGRF).