Reflections From Gothenburg

After four action-packed days in Gothenburg at the European Cystic Fibrosis Society annual conference, Ed presents his ‘top ten’ reflections of the last few days:

We are in a new era of cystic fibrosis research

There is belief among the world’s best scientists and clinicians that cystic fibrosis research has entered a period of extraordinary opportunity with the prospect of drugs and therapies focused on correcting the basic genetic defect. As Jerusalem’s Professor Eitan Kerem said in his inspiring speech at the opening session, "Ivacaftor [Kalydeco] provides belief there is light at end of tunnel for cystic fibrosis." The conference showcased a range of other ongoing research, including a drug made from Norwegian seaweed due to be trialled in the UK and elsewhere this year!

But let’s keep our feet on the ground

It is an exciting period but we must ground our expectations in reality. Even if the results of the Phase 2b gene therapy trial, expected in October, are positive, there is a long way to go before it can be assessed for wider clinical impact. And no one expects the data from Vertex’s Phase 3 trial of a combination therapy of Kalydeco and lumacaftor (VX809) for those with two copies of the D508F gene, due to be published in a few weeks’ time, to be as dramatic as Kalydeco for those with G551D. There is much to be done, but this conference has reaffirmed my belief that we are making significant progress towards our goal of beating cystic fibrosis for good.

Don’t forget the simple things

While much of the attention of the cystic fibrosis community is on this transformational research, there is much more we can achieve by helping to prevent and manage the symptoms of cystic fibrosis. A very good session I attended talked about the importance of exercise for all people with cystic fibrosis. The US’s Anne Swisher said that kids and teenagers with cystic fibrosis should be aiming to do at least one hour of moderate to vigorous activity a day, while adults should aim for 150 to 300 minutes a week. Sweden's Margareta Sahlberg urged parents to start their children doing regular physical exercise as soon as possible – and always with a smile!

We have fantastic health professionals in the UK

Seeing British clinicians and health professionals like Stuart Elborn, Di Bilton, Jane Davies and Tim Lee leading discussions and debate on the major issues at the conference this week, and the esteem in which they are held across the world, reminds me how lucky we are to have such a group of committed and dedicated professionals working to improve and transform the health of people with cystic fibrosis.

But those with cystic fibrosis and their carers are experts too

Those living with cystic fibrosis every day have extraordinary knowledge – and the NHS needs to do more to ensure people are shaping the way their care is provided. Dutch psychologist Yvonne Prins, who also has cystic fibrosis, gave a thought-provoking talk about creating “person-centred” care to put the needs, wellbeing and interests of those with the condition centre-stage rather than as merely recipients of clinical treatment. She suggested that clinicians and health professionals needed to ask themselves if, when they saw someone with cystic fibrosis in clinic, they saw a patient, a pair of genes or a person. Only if it was the latter could they begin to properly help develop a form of care that understands the emotional complexity of living with this lifelong condition.

We can be proud of what the Cystic Fibrosis Trust is doing

The Trust needs to constantly improve what it does to ensure it has the maximum impact on those we are here for. It was heartening to speak to a range of cystic fibrosis organisations from across Europe that want to learn from what we are doing, particularly on research and clinical care. The UK CF Registry is a great example, and the work to use the registry in partnership with companies like Vertex and Pharmaxis to help study the safety of new drugs being introduced in UK clinics got a great deal of interest at the conference.

But there is much more we can do

I was able to use the conference to meet a number of companies who we might work with to help develop new research opportunities for people with cystic fibrosis.  The Cystic Fibrosis Foundation in the US – who we are working with on a number of fronts - has led the way in this regard, and there is more we can do. It was good to see Novabiotics represented in Gothenburg. They are a UK-based bio-tech company we are now in partnership with to trial a drug to treat lung inflammation, and I want to see more of this as we deliver our research strategy.

We need to co-operate and collaborate to achieve more

These conferences help build and cement important relationships. There is a great deal that the Trust can do alone, but there is so much more we can do working with others – whether cystic fibrosis charities or companies and other groups. If we can use our expertise and resources to leverage additional funds and know-how into cystic fibrosis then we will. So expect more cooperation at a European level to access vital EU funds to research key areas of cystic fibrosis, and help ensure more clinical trials can take place when needed.

The voice and experience of those with cystic fibrosis can change the world

We must never forget that for all the extraordinary expertise within the clinical and scientific world, it is often the voice and stories of those with cystic fibrosis that make the difference. That was what made the difference on ensuring Kalydeco was made available to all eligible across the UK, as well as Manitol, Colobreathe and other new therapies. It will be so again when other drugs appear – and as we work to shape a healthcare system that puts the interests of people with cystic fibrosis first.

Let’s look forward to next year

A lot will happen in the 12 months before the next ECFS conference. We will be working very hard to make a significant contribution to the change occurring in cystic fibrosis and to improve our work for people here in the UK. With the tremendous support of the wider cystic fibrosis community, I am confident we can achieve a great deal. I am also relieved that the next conference is in Brussels which is much easier to get to!