$2.1m to beat rare genetic disease cystic fibrosis

Jan 21 2022

University of Newcastle and HMRI respiratory disease expert Professor Peter Wark will lead a new $2.1m project to develop targeted treatments for the rare genetic and life-threatening disease cystic fibrosis, which damages the lungs and digestive system.

One of 27 projects awarded funding through the Federal Government’s 2021 Rare Cancers, Rare Diseases and Unmet Need grant program, the collaborative research project will focus on precision medicine by considering individual genetic variations when tailoring treatments for people with cystic fibrosis.

Caused by more than 2000 gene defects, cystic fibrosis primarily affects the lungs and digestive system because of a malfunction in the exocrine system, which is responsible for producing saliva, sweat, tears and mucus.

Although medications have been developed to help manage cystic fibrosis, they improve outcomes for only 80 per cent of people with the disease.  As part of an ongoing commitment to help people live better, healthier lives, Professor Wark and his team have developed a personalised avatar using cells from each person that can predict accurately who will and will not benefit from these medicines.

The University of Newcastle-led project will involve researchers from six additional Australian universities and research institutes.

The $2.1m grant is part of the Medical Research Future Fund, administered by the National Health and Medical Research Council.