Queensland researchers may have discovered a game-changing treatment for children suffering from cystic fibrosis.
The Queensland Children's Medical Research Institute has released results from world-first clinical trials of a new treatment that combined two existing drugs, lumacaftor and ivacaftor.
Neither had shown significant clinical outcomes when used alone, but QCMRI interim director Claire Wainwright said the combination treatment had resulted in up to 39 per cent fewer lung infections.
"Cystic fibrosis is the most common life-threatening genetic condition affecting children in Australia, so these results are incredibly exciting," Professor Wainwright said.
"As well as the 30-39 per cent reduction in lung infections, our trial participants reported a decrease in events requiring hospitalisation or use of IV antibiotics.
"Results like this mean our novel treatment is a potential game changer for the way we treat CF and other genetic disorders in the future."
Cystic fibrosis sufferers have abnormally thick mucus linings in the lungs, which clog air passages and result in repeated infections and blockages.
It affects the respiratory, digestive and reproductive systems and usually leads to a shortened life expectancy, around 37 years on average.
"Research into novel treatments like the one we trialled is essential to improve the quality of life for CF patients," Professor Wainwright said.
"There is still no cure for CF but with treatments like this proving successful, our research could have positive outcomes for CF patients not just here in Australia but internationally for years to come."
More than 1100 children under the age of 12 participated in the six-month study, which involved two randomised, double-blind, phase 3 clinical trials.
Senior researcher Scott Bell said 187 hospitals in Australia, Europe and the United States collaborated in the trials.
"Nine CF centres in Australia have been involved in the study and patients have been eagerly awaiting the results," Professor Bell said.
"More than 1000 patients in Australia have the potential to benefit from the treatment in the future."
The Food and Drug Administration will make a decision on the treatment in July, after which it will be assessed by the Therapeutic Goods Administration to determine market availability and costs.