The European Cystic Fibrosis Society Conference: Day 2

Director of Research Janet Allen gets an update on trials of a new drug for treating people with cystic fibrosis who have the F508del mutation, and then attends a mini symposium on ivacaftor (Kalydeco).

At the ECFS today, the most recent results for a new drug with the potential to influence the function of F508del were presented. Lumacaftor (VX809) is being developed by Vertex Pharmaceuticals to restore CFTR function to people with cystic fibrosis who have F508del. The early Phase 2 results were shown last October and based on these results, Vertex announced the launch of a Phase 3 study of lumacaftor in combination with ivacaftor (Kalydeco).

This Phase 3 trial will study two different doses of lumacaftor, 600mg once a day compared to 400mg twice a day. Both doses will be given in combination with ivacaftor (Kalydeco). A number of cystic fibrosis clinics in the UK are participating in this trial, which is recruiting people who have two copies of the F508del mutation. At the presentation today, results clearly demonstrated that both doses of lumacaftor in combination with ivacaftor had positive effects on FEV1 (increase ~8%) in the Phase 2 trial. It was also announced that the first patient had started the Phase 3 trial.

At a later session, this evening I attended a mini-symposium on ivacaftor to review the experience of physicians who have been prescribing Kalydeco for people with G551D.

ECFS Conference, Day 3 Influence in Europe

Ed Owen, CEO at Cystic Fibrosis Trust reports on day 3 of the EU Cystic Fibrosis confernece.

Improving and transforming the lives of people with cystic fibrosis is our mission and the Cystic Fibrosis Trust has been hard at work in Lisbon at the European Cystic Fibrosis Society (ECFS) conference on ways to help achieve it.
Elaine Gunn, our Registry manager, has been working closely with international colleagues here on developing an effective data registry for Europe to help drive research and clinical treatments.
And Janet Allen and I have been meeting researchers, international partners like the Cystic Fibrosis Foundation (CFF) and industry representatives to develop support for new initiatives to drive innovative science and improve treatment regimes for people with cystic fibrosis.

Janet, who as our Director of Research has built a strong global reputation after just nine months in the job, took delegates through our new research strategy at a specially convened event at the conference. She explained to an assembled group of clinicians, scientists and pharmaceutical companies our desire to collaborate on joint projects to boost the overall research effort in cystic fibrosis.

The strategy has been very well received and Janet, together with Professor Stuart Elborn, ECFS President and one of our trustees, explained how our new funding arrangements would work and how we wanted to ensure Trust funds were used to ’leverage‘ resources from others.

Later at the same meeting, I was joined by Dr Diana Bilton, the lead consultant at the adult centre at the Royal Brompton Hospital, to talk to UK consultants about issues relating to clinical care, including the Quality Improvement Programme to raise standards of cystic fibrosis care within the NHS.

Tomorrow, Nick Medhurst, who heads our public affairs activities, will be helping to enhance our relationships with fellow European cystic fibrosis patient organisations so we can learn from each others' experiences and enhance our ability to secure EU funding for research.

One of the important strengths of the Trust is our ability to work with many different partners to improve the lives of people with cystic fibrosis. Collaboration in so many forms is helping to deliver our mission.

Ed

The European Cystic Fibrosis Society Conference: Day 1

Janet Allen, Director of Research at the Cystic Fibrosis Trust, discovers the incredible opportunities the ECFS conference creates for networking and sharing ideas and knowledge with colleagues from around the world.

I am attending my first ECFS meeting here in Lisbon. There are many presentations studying the protein CFTR which harbours the mutations that lead to cystic fibrosis. The rate of scientific progress is impressive. We are learning so much about how the mutations affect CFTR function and how drugs such as Kalydeco and VX809 help restore function. The more we understand CFTR the better chance we have of finding new treatments that tackle the basic defect in people with cystic fibrosis.

These meetings are valuable not only for hearing of the latest science but also a wonderful opportunity to network with colleagues across the world. Yesterday, we had very valuable discussions with the leaders of other cystic fibrosis organisations, including the CF Foundation (USA), CF Canada, CF Europe and many other countries. These meetings are useful to understand what is happening across the world and identify areas where we can better work together.

Today, we are holding a "fringe" meeting where we will present the research strategy that was launched by the Trust at the end of April. This is an opportunity to provide more details for our research call and also to engage with researchers around the world.

The European Cystic Fibrosis Society Conference: Day 1

Cystic Fibrosis Trust Policy Officer Nick Medhurst gets ready for a packed conference.

When I arrived this morning at the conference hall in Lisbon that is playing host to the 36th European Cystic Fibrosis Conference, I was struck by the scale of this event.

It is quite remarkable that the ECFS attracts so many clinicians and professionals, not just from Europe but from all over the world. I think it is testament to what an exciting time we in the cystic fibrosis community find ourselves in, but also to the dedication and commitment to sharing and learning together that these people have.

I have a packed agenda hearing from leaders in the field on subjects from adherence to resilience, care models to treatment innovation. I only hope I can take it all in!

This afternoon the Trust is hosting its own seminar, where we will be gathering together centre directors from across the UK to discuss new research opportunities and ways of partnering together to improve the lives of people affected by cystic fibrosis.

With so many eminent people here, I can't really fail to learn a lot and I'm looking forward to bringing this learning back to HQ to continue the excellent work of the Trust's Policy team.

The European Cystic Fibrosis Society Conference: Day 1

Registry Manager Elaine Gunn reports on the first day of the ECFS in Lisbon.

Here in Lisbon the sun is shining but the meeting rooms are air conditioned. As part of my role I sit on the European Registry Data Quality Control Group. There are approximately 21 countries who enter data to the European report which looks at country comparisons. With all these different countries it is important that data is compatible so that this work can be carried out.

Today we are finalising the latest data fields. Later this evening it is the Welcome Reception where everyone meets to listen to the opening plenary, this year given by Prof Kevin Webb from Manchester. Tomorrow is another day with European meetings and a great symposium about data and registries.

Will tell you all about my day tomorrow. 

Elaine Gunn RGN DPA Registry Manager

The welcome reception thanked the European CF associations. A cheque was presented to the ECFS for 120,000 euros.

The European Cystic Fibrosis Society Conference: Preview

Cystic Fibrosis Trust Chief Executive Ed Owen gets ready for the ECFS conference in Lisbon.

Later today the start of the European Cystic Fibrosis Society conference gets under way in Lisbon. It is warm and sunny here but there's no time for sunbathing you will be pleased to know. It's a packed programme with seminars on all aspects of cystic fibrosis care and research - from correcting CFTR to the role of parents in CF care.

All of Europe's top scientists and clinicians are here, along with other patient-based groups like us and big pharmaceutical companies investing in cystic fibrosis research.

It is a good opportunity to share experience to help improve outcomes for people with cystic fibrosis. I am also keen to explore where we can collaborate more with others in Europe and elsewhere to the benefit of people in the UK.

Many of the clinical trials bringing new drugs and treatments for people with cystic fibrosis are taking place in more than one country, and it makes sense for us to work with our colleagues in Germany, France, Netherlands and others - and to share costs - where this makes sense.

The Cystic Fibrosis Foundation (CFF) in the US always sends people to the ECFS conference too and Janet Allen, our research director, and I met up with two of its senior people over breakfast this morning to discuss areas of research we can work on together.

We heard their views on the latest developments in the Vertex pipeline of small molecule treatments in clinical trials. This pipeline only happened because of "seed funding" provided by the CFF a decade ago. It is a stark example of the benefit of patient-based organisations in drug development, and our new research strategy aims to draw on the best of their experience.

We are talking to delegates about our strategy tomorrow afternoon to help promote it among clinicians, researchers, other CF bodies and industry. Their collaboration with us is vital and we want them to work with us over the next few years. I will let you know how we get on later in the week.

Making Great Strides for the Cystic Fibrosis Trust

David Turner writes about the the Cystic Fibrosis Trust's Great Strides 65 challenge.

Dawn broke over the North Downs on Saturday 18th May to the sound and smell of bacon being fried on a camping cooker to feed the intrepid walkers participating in the second running of the “Great Strides 65” – a 65km (40 mile) supported team walk from Newlands Corner, just outside Guildford, to Shoreham-by-Sea.

Eight teams took part in the 2013 event, with the aim of bettering the £20,000 plus Gift Aid raised on the inaugural running in 2012. Remarkably the Dorset Dawdlers and Somerset Strollers came back for more, having successfully completed the previous year’s event: the Dawdlers’ Mike Rutter was determined to finish in better style than he had 12 months ago – when he walked the last five miles wrapped in a space blanket.

The Dawdlers were joined by teams from Surrey, London and as far afield as the West Midlands, Bristol, Somerset and northern Cambridgeshire. All of the teams had some connection with CF, whether through family friends or as parents of children or adults with the disease. Walkers ranged in age between their late-20s and 70 and included siblings, parents, uncles, aunts and grandparents of those with CF, and even a vicar who had once baptised a girl with cystic fibrosis! The remarkable Sue Ward from March was walking in memory of her son, motivated by a burning determination to help fund the research which will improve the outcome for current and future generations of patients

Ready for the “off” – Newlands Corner at 7am

After setting off at 7am, and an initial leg which involved a couple of gentle drops and climbs as the walkers moved away from the North Downs, the teams settled onto the Downs Link – a long distance path linking the North and South Downs which largely follows the route of the disused Cranleigh and Steyning railway lines. There were pre-arranged checkpoints every five to seven miles for the teams to meet up with their support crews so that they could refill their camelbacks, top up on Mars bars and bananas and change their socks and T-shirts. Despite earlier forecasts of incessant and heavy rain, weather conditions on the day were kind: dry, reasonably cool and no wind.

The teams made good progress, with the leaders reaching the third checkpoint at Slinfold (about 17½  miles into the route) at about midday. However, by the time that the teams reached Southwater (just five miles further on), many were beginning to suffer from sore feet, and the paramedics assigned to the event were earning their money as they performed a series of essential running repairs to keep people going. One walker’s feet were so badly blistered by this stage that she completed the walk wearing flip-flops!

For many, the final checkpoint at Upper Beeding was a welcome respite: conveniently located in a pub, participants were able to replenish depleted carbohydrate reserves with a pint of Sussex or a plateful of home-cooked chips before embarking on the final five-mile slog to the finish. Unfortunately, half the teams bypassed the pub (through inadvertence not design) and so denied themselves the simple pleasures on offer.

The posse was led across the finish line by the SW15 Ramblers at 7.30pm. The other teams followed, with the last team hobbling over the finish line at 9.50pm after nearly 15 hours of walking. Some of the walkers had pushed themselves to the point of exhaustion and beyond, and there were some spectacular blisters on display well before the end of the walk. The extraordinary determination of all of the walkers was both inspiring and humbling, and has helped the Trust raise a huge sum of money: to date almost £30,000 has been pledged. With gift aid, the total could exceed £34,000. We owe a huge “thank you” to all of the walkers and their support crews; to Katie Burr of the CF Trust who has organised the event for the last two years and who leaves the Trust shortly to go travelling (perhaps she will be back in time to “walk the walk” in next year’s iteration) and to the volunteers who helped the event to run so smoothly (most of all Mel Urwin, another walker from last year, who volunteered to help for a few hours but ended up staying the whole day!).

The date for next year’s “Great Strides 65” will be announced shortly. If you fancy doing something a bit different and surprising your family, friends and even yourself, then give it a go!!

The Mail Publisher Solutions team, and paramedics, at the finish