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Highlights from the European CF Society conference
The ECFS Conference was an impressive and important event. What really struck me was the sheer scale and scope of the research efforts that are taking place in Europe and beyond. It was great to see further evidence of the impact of Kaftrio and the emphasis placed on what that means for the changing needs of people who can benefit and the treatments and support they will need. It was also fantastic to see how central the needs of those who can’t benefit were and the energy to make a real difference for all was in the sessions and in the conversations.
David Ramsden, Chief Executive of Cystic Fibrosis Trust
Closing the gap – treatments for everyone with CF
It was exciting to see that there were lots of sessions discussing treatments for those who are unable to benefit from Kaftrio. There were presentations from lab-based researchers, like Trust-funded Professor Steve Hart who is working out the practicalities of delivering genetic therapies into the lung; to ‘theratyping’ studies, testing whether people with very rare forms of CF could benefit from Kaftrio. (The number of people with these forms are too small to test Kaftrio in clinical trials). Researchers also spoke about genetic therapies that are being or about to be tested in early phase clinical trials.
Knowns and unknowns of taking Kaftrio
Last week marked the three year anniversary of access to Kaftrio being approved in the UK for those aged 12 and over. For everyone it was a ‘where were you when you heard’ moment when the news came through.
A lot has happened since then, and this was reflected in the talks and discussions at the conference. Many interesting studies were shared that added weight to the early findings that modulators are significant and positive in terms of overall improvements in health outcomes.
For example, we heard the results of the extension clinical trials, showing the sustained benefits in lung function for people with CF on Kaftrio. There are many other ongoing studies to understand more about the long-term effects of these medicines in all parts of the body.
Some researchers showed how modulators are turning typical CF symptoms on their head. For example, clinicians are now starting to see increases in people’s body mass index (BMI), which is prompting them to rethink the CF diet. “We need to be supporting people with CF who are navigating that change” explained Ali Taylor our Information Manager who attended talks on this topic at the conference.
Mental health challenges linked to Kaftrio were also discussed, these included observed changes in behaviour, reports of depression, reports of insomnia, and the challenges of adjusting to a longer life expectancy with CF. The conference provided an important opportunity for doctors, researchers and CF teams to share their experiences of these symptoms and the best ways to manage them.
“The conference showcased the volume and depth of research and clinical innovation to understand and respond to the changing landscape for people with CF following the introduction of modulators and as new treatments, including genetic therapies, are developed.” said Clare Corbett our Director of External Affairs. “From new information resources, to the launch of our Work Forwards programme, we are responding to these changes at the Trust. Insights from clinicians, researchers and the CF community are critical as we develop our information, support and advocacy”.
Inequity of access to medicines
Doctors and researchers know that there is so much more research to be done to improve the lives of people living with CF. However, there are many people with CF across Europe and the rest of the world who are still not benefiting from the advances in knowledge, care and access to medicines for non-medical reasons, for example economic reasons. It’s a topic that was passionately and movingly raised at the conference. Access is limited to CFTR modulator medicines such as Kaftrio, and also to other medicines and services that could significantly extend and improve the lives of people with CF.
“These talks were very sobering”, commented Connie Takawira, Programme Manager for CF AMR Syndicate based at Medicines Discovery Catapult. “As we do work in the Syndicate to develop novel medicines to treat CF lung infections and better ways to diagnose them, it made me question ‘is there an opportunity for us to be thinking about inequity of access too?’ ”.
Understanding more about CF lung infections
To effectively detect and treat CF lung infections, it is important to have a good understanding of the lungs themselves. The lungs are made up of smaller and smaller-sized passageways that branch out (literally like branches of a tree), making some parts of the lungs quite separate to other parts. At the conference, scientists shared their new results on how the structure of the lungs contributes to how infections develop – the challenge is how this new information can be applied to treating infections in the future.
Many people with CF will have more than one bug growing in their lungs at the same time, in what is known as the ‘lung microbiome’. A greater understanding how these bugs live together will help doctors and researchers find better, more effective ways to treat infections. For example, at the conference we heard about the aggressive competition between infection-causing bugs for the best growing conditions within the lungs, and how the action of some bugs can protect others from the effects of antibiotics, contributing to a reduction in effectiveness of antibiotic treatments.
Establishing new partnerships and collaborations
The ability to meet virtually is a huge benefit for the whole of the CF community. But the face to face conference gave people the opportunity for spontaneous collaboration with international colleagues. Whether it was saying hello to researchers we’ve worked with and funded previously, or whether it was meeting someone for the first time. For many, the opportunity to discuss new partnerships and cement existing collaborations was an important reason to attend the conference. Whether it was unplanned clusters of people around the speakers at the end of the session, or the roar of conversation during coffee breaks – everyone was networking! Every meal time (yes, including breakfast!) was an opportunity for planned meetings, to take advantage of everyone being in the same place at once.
My role is all about talking to doctors, researchers, representatives from companies and other charities, to spot and make the most of opportunities to work together, share resources and fund research together. The conference was an important place to do this. This helps us make the most of the funds we have available to make the biggest difference to the lives of people living with CF.
Dr Lucy Allen, Director of Research and Healthcare Data at Cystic Fibrosis Trust
Cystic fibrosis (CF) is a genetic condition which causes sticky mucus to build up in the lungs and digestive system. It affects more than 10,900 people in the UK. One in 25 of us carries the faulty gene that causes it, usually without knowing.
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