Cure4CF Update

Reflections from the 48th European Cystic Fibrosis Conference

In June 2025, Milan played host to the 48th European Cystic Fibrosis Conference (ECFS), drawing together the brightest minds in cystic fibrosis (CF) research, innovation, and care. The event was not only an opportunity to stay abreast of cutting-edge developments but also a chance to build valuable relationships and elevate Australia’s presence in the global CF research community.

Here, are some of the key scientific insights and strategic connections we made during this transformative week.

Science in Action

Held annually, ECFS showcases the best of European CF research. This year’s opening plenary was particularly powerful, shining a spotlight on efforts to improve access to modulator therapies for people with rare gene variants. Historically, these therapies were only approved for those with at least one F508del mutation — a standard that left many out, despite the potential for benefit.

Change is happening. The European Medicines Agency has recently approved a broader range of gene variants and, crucially, endorsed new ways to assess individual treatment response without relying on large-scale clinical trials. These changes mean that even people with extremely rare variants — fewer than 100 known globally in some cases — may now have a pathway to treatment.

Countries are taking different approaches. France trialled compassionate access for everyone. The Netherlands adopted a more targeted method, granting therapy access based on lab-confirmed cellular response. The Dutch model in particular stands out as a pragmatic, cost-effective path forward. They’re now leading a world-first trial using this approach — a true example of personalised medicine in action.

So what does this mean for Australia?

Only last week the announcement and approvals were finally forthcoming which now allows more people in Australia with CF to access Trikafta. We’re already equipped to test response using the same organoid technology as the Dutch study, with capabilities at UNSW and the University of Newcastle. Cure4CF-funded researcher Dr Gerard Kaiko is partnering with Dr Peter Wark on a clinical trial exploring modulator responsiveness in currently ineligible patients — a critical step toward more inclusive treatment.

Connecting with Our CF Research Community

Throughout the conference I met with Australian researchers presenting their work, including Dr Keith Ooi, Ms Tamara Katz, Dr Shafagh Waters (UNSW), Dr Nikki Reine (University of Adelaide), Dr Elena Schneider-Futschik (University of Melbourne), and Dr Claire Wainwright (University of Queensland).

We also heard from Professor Scott Bell (Gold Coast Hospital), one of our IRAC members, whose presentation challenged us to consider how climate change is impacting people with CF — from increased infection risk to hydration concerns. He also advocated for greener clinical trials and sustainable healthcare models, a timely and urgent message.

Shifting Focus: From Lungs to the Whole Body

The lungs often take centre stage in CF research, but this year, there was a strong focus on the gut, pancreas, and systemic effects of CF. One of the most compelling sessions addressed pancreatic sufficiency — a growing issue due to modulator therapies. While many people with CF have pancreatic insufficiency and require enzyme supplements, those with restored function are now facing a new challenge: pancreatitis.

Sydney Children’s Hospital dietitian Tamarah Katz gave a standout presentation on evolving dietary needs. For decades, people with CF were encouraged to follow a high-fat, high-calorie diet, but the modulator era is shifting that paradigm — and the research to guide these changes is still catching up.

Katz highlighted the importance of the gut microbiome, explaining how diet can influence gut bacteria that produce short-chain fatty acids — compounds with anti-inflammatory and even anti-tumour properties. Practical solutions were discussed (hint: baked beans might be more helpful than you think!), and she also stressed the need for dietary changes to be acceptable and sustainable for the CF community.

One sobering reminder from the session was the significantly increased risk of colorectal cancer in people with CF. By age 40, about half of individuals are likely to have precancerous polyps, and their overall cancer risk is 5 to 10 times higher than the general population. Early and regular screening is essential.

Looking Forward: Building Global Partnerships

On the strategic front, I spent time exploring partnership opportunities with the CF Trust in the UK. Their impressive track record in funding and clinical trial infrastructure offers a potential pathway for Australian researchers seeking international collaboration.

This conference reaffirmed the importance of Cure4CF’s role in connecting, funding, and advancing CF research in Australia. We came away with new ideas, fresh partnerships, and a renewed commitment to ensuring every person with CF — regardless of their genetic variant — has a chance at better treatment and better outcomes.

Stay tuned as we continue to bring these global insights home and translate them into real impact for Australians living with cystic fibrosis.