As Cystic Fibrosis Awareness Month draws to a close, we reflect on the importance of raising awareness, supporting those affected, and championing the need for continued research into this complex condition.
Cystic fibrosis (CF) is a progressive, genetic disease that causes thick, sticky mucus to build up in the lungs, airways, and digestive system. This leads to chronic lung infections and lasting organ damage.
Today, more than 3,700 Australians are living with CF. With over 2,000 known gene mutations linked to the condition, finding a cure is an ongoing and complex challenge.
Yet, the dedicated researchers at The Kids’ Wal-yan Respiratory Research Centre are rising to the challenge. Their work is crucial in better understanding and treating Australia’s most common life-limiting genetic disease.
As we wrap up Cystic Fibrosis Awareness Month, let’s continue to support these efforts and shine a light on the urgent need for progress.
Here’s a look at some of the Centre’s current research projects:
Understanding lung health in children with CF
University of Western Australia (UWA) PhD student Faith Mhembere is undertaking an important research project to better understand how lung disease develops in young children with CF).
Working under the guidance of Associate Professor Kathryn Ramsey (The Kids/Wal-yan), Honorary Research Fellow Dr David Hancock (The Kids/Wal-yan/PCH/UWA) and Dr Charley Budgeon (UWA), Faith’s study is assessing lung health trajectories in children with CF, including those on new, highly effective Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies.
The project is focused on three key areas:
- Tracking how lung health (structure and function) changes over time in children with CF.
- Exploring how early treatment with CFTR modulators might improve lung health and reduce symptoms and flare-ups.
- Testing how well a special lung function test, called the lung clearance index, can predict how the disease progresses over time.
This research could help doctors better manage CF in children and improve long-term health outcomes.
New clinical trial aiming to improve lung health in children with CF
David Hancock and his team are preparing to launch an exciting new clinical trial to explore a potential new treatment for children with CF.
The trial, backed by Respirion Pharma, will test the safety and early effectiveness of a combination therapy using inhaled EDTA and tobramycin—two medications that may work together to improve lung health.
Currently undergoing ethics approval, the study will take place across multiple sites in Melbourne and Brisbane, with The Kids as the lead site and David as the Chief Principal Investigator. This innovative research builds on an idea originally developed by The Kids’ Barry Clements.
If successful, this trial could pave the way for better treatments to reduce infections and improve breathing for children living with CF.
Giving children with CF access to promising new treatments
The Respiratory Clinical Trials Group is continuing its vital work in CF research by running a range of clinical trials, including studies with Vertex Pharmaceuticals. These trials focus on CFTR modulators – groundbreaking treatments that target the underlying cause of CF.
One important area of research is the use of modulators in very young children aged one-to-two years. These children currently don’t have access to these modulator therapies through the Pharmaceutical Benefits Scheme (PBS), so this research is essential in helping to evaluate the safety and benefits of early treatment.
The results could play a key role in improving early care and long-term health outcomes for children with CF.