Ed Owen, looking forward to CF Week

I am looking forward to this years CF Week. Here at the Trust we are having an event every day.  It promises to be as lively as ever with over 100 events across the UK during the week. As we embark on our fundraising events there is also a serious side to the week, the campaign on transplants. This area forms a major part of the Cystic Fibrosis Trust's work this year. With improved treatments, better understanding of cystic fibrosis and continued research, people with cystic fibrosis are living longer than ever before, and often with a better quality of life. There has never been a more positive time. However, sadly, people with cystic fibrosis will still succumb to the effects of the condition, which does the most damage to the lungs. It is still the case that the biggest cause of death in people with cystic fibrosis is due to lung failure.

For some the only course of treatment is to receive a transplant, but once accepted on to the transplant list the wait can be as long as 412 days, or 3 years.

For 1 in 3 that call never comes.

Too many people are waiting too long for transplant, there are still issues with a lack of available lungs and a lack of facilities to successfully retrieve lungs.

This is a hard reality to face, but the devastation for families who lose a loved one to cystic fibrosis is mine and our driver for change. People who need a lung transplant with cystic fibrosis are usually under 40 years old. For younger people the possibility of many more years of life after a successful transplant gives hope to them and their families. People waiting for a transplant should be able to receive one. That is why we are starting CF Week this year with a strong message. We want the policy makers, transplant and clinical community to join us in a consultation to find a solution to the problem of people with cystic fibrosis not getting the lungs they need.

The Trust will be highlighting to the media the cases of people with CF who have agreed to share their experiences of being on the waiting list.

Log on to www.cysticfibrosis.org.uk/cfweek and find out more about the issues around transplant, join in with our consultation, we want to hear your views.

Have a good CF Week.

Ed.

The European Cystic Fibrosis Society Conference: Day 3 session on NTM

Janet Allen, Cystic Fibrosis Trust Director of Research, attends a special session exploring the NTM bug, and discovers how organisations across the world can come together to fight for a common cause.

A whole session at ECFS was dedicated to a discussion on NTM, the bug that is causing some physicians concerns. The Cystic Fibrosis Trust has set up a working group to look at our cross-infection guidelines.

Meanwhile, a partnership between ECFS and CFF has been working hard to develop a series of guidelines on how to identify that someone has NTM, how to find out exactly which bug is responsible (as NTM represents a number of subtypes) and importantly, recommendations on which antibiotics should be used to treat it.

This working party is led by cystic fibrosis physicians in the UK and is a wonderful example of how the different CF organisations can come together across all of Europe and the USA to create a unified set of guidelines on diagnosis and treatment.

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.