CF Week 2013: Ed's Blog

Cystic Fibrosis Trust CEO Ed Owen looks back on an incredible week raising funds and awareness for cystic fibrosis, and the controversial but critical subject of transplantation.





We're into Day 5 of CF Week 2013 and together we've been making a real impact across the UK, which has only been possible because of our amazing supporters. 

Thank you to everyone who has supported our transplant campaign by speaking so eloquently about the difference a transplant makes. By sharing your stories you've enabled us to raise awareness of the issues surrounding transplantation in every BBC and ITV region.

Transplantation is now firmly established as a health concern within the media, at a time when there is immense competition for space and airtime. In many of the media interviews journalists asked about the crucial issue of the shortage of organ donors.

This week we have also been raising awareness of the issues with politicians across the UK, helped by parents and relatives affected by cystic fibrosis, and clinicians.

We met the Welsh Health Minister, Mark Drakeford, during the week to discuss transplantation policy. The Welsh Government is currently considering moving towards a soft opt-out system of organ donation.

And finally, a massive thank-you to everyone who has been fundraising, raising thousands of pounds for the Trust. We've been posting your photos and highlighting your CF Week activities on Facebook, and we really appreciate all your support. We are fortunate to have such a dedicated and committed community of fundraisers who turn out time and again to support our vital work. We can't achieve anything without you, and we are truly grateful to you all.

You can learn more about the human side of transplantation from the films and stories some of our supporters have shared for CF Week.

CF Week 2013: Sam's Blog

Sam appeared in our CF Week campaign in 2011, when he was on the waiting list for a lung transplant. After coming through five years on the waiting list, the operation itself and some post-transplant health scares, he is now enjoying being a teenager and looking to the future.


Hi I'm Sam, I am 14 years old and am one of the feature case studies in this year's CF Week. You can watch my video here.

I was pleased that the topic of CF Week this year is transplantation as this is a theme close to my heart.

I waited for five years for my transplant call to come. I was 13 when my call came and the operation was carried out at Great Ormond Street. When the call came we were blue lighted to the hospital, I remember feeling really excited but really scared at the same time! We got final confirmation of the transplant at 03.00 in the morning.

I was lucky to get my transplant in time and now I'm looking forward to the future. I will be choosing my GCSE options soon; I'm joining in with sports again, going out with family and friends and being a teenager. I can't say it’s been an easy year and we've had some health scares post transplant but I'm here and cystic fibrosis is not gong to stop me.

I am pleased that the Cystic Fibrosis Trust is highlighting the problems for people in getting a transplant in time, and starting a consultation on to how to improve rates of transplant and informed choice for children and adults who find themselves on the transplant list. I hope that everyone gets their call in the end. 

So please get involved with CF Week. I will be doing my own big bounce and there are lots of other ways to raise funds too. Visit www.cysticfibrosis.org.uk/cfweek for ideas and help the Cystic Fibrosis Trust carry on its work to improve the lives of people like me.

Sam x

CF Week 2013: Mike's Blog

Mike Mackay, from Witham, Essex, is living life to the full after transplant.





Mike here, I hope you are enjoying CF Week this year, my film is available to watch now.

When I was younger, I decided that a transplant wasn't for me; it seemed like a lot of “hassle” to go through. Yet, when the time came, it was something I wanted more than anything in the world. The more I began to struggle, the more I wanted it. Only I never ultimately knew whether I would have one or not...

I consider myself extremely fortunate to be here now, writing this. I've known many who haven't been so lucky and I feel it's only right that I make the most of this second chance at life. Before my transplant I was wheelchair bound, eventually bed-bound on a ventilator; I wasn't really living but just existing. I thought my time was up - I never thought I'd be doing silly things like completing the London Marathon or working in a full time job. In September I'll be climbing Ben Nevis to celebrate my sixth transplant anniversary. All of these achievements highlight just how much a transplant can change a life.

Without the generosity of my donor, none of this would be possible. Without donors, many people representing CF Week this year wouldn't be here. Organ donation is so important and the work the Cystic Fibrosis Trust does reflects that.

Without people like you, there wouldn't be people like me.

www.cysticfibrosis.org.uk/cfweek

CF Week 2013: Philippa's Blog

Philippa, who was one of the first people to receive a transplant in the EVLP pilot, talks about the new lease of life her transplant has given her.



The Ex-vivo Lung Perfusion (EVLP) technique uses a modified dialysis machine to turn lungs that were previously deemed unfit for transplant into suitable donor organs.

For about a year or two before my transplant I was very poorly, I didn’t really have a life; I was just existing. I hardly went out anywhere as it was such a huge effort to get ready, and by the time I got dressed I was exhausted. I didn’t want to catch any new infections either. My lungs were functioning at just 17% and I would watch my friends and family live their lives from the side lines. 

I was on oxygen 24 hours a day, taking a massive cocktail of drugs, including continuous intravenous antibiotics and a range of nebulisers. I had to use a wheelchair if I did go out anywhere as I couldn't walk very far and I used a ventilator overnight to give me a rest from breathing. I couldn't do anything for myself such as having a bath or getting dressed as I just didn't have the energy or the breath. I was very weak. I also had to drop out of university as I was too ill to finish the course. That was a huge blow for me.

But now, I have an incredible new lease of life. Since my transplant I have been on holiday a few times, got myself a full time job, found love, moved into my own home and now I am planning my wedding, which is next year. My lung function is 126%, which I still can't believe! Two years ago when I was so ill, I never once imagined that life would be this fantastic after transplant. I still have to pinch myself to make sure this is not a dream.

I strongly support CF week because I want to help raise awareness of cystic fibrosis, but I also want to raise awareness of transplantation. A double lung transplant is very much the last resort, when your own damaged lungs are failing. Yes it is a massively scary time, one in three people with CF waiting on the transplant list don’t make it as there is a huge shortage of donor lungs. The wait itself is exceptionally difficult, but it was the only way forward for me.

Put simply, I wanted to live – so I had to do this. Because there is such a shortage of donor lungs that are suitable for transplant, doctors have recently been pioneering a new treatment where previously unsuitable lungs are treated to make them usable – this is the type of transplant I had, it is known as EX-VIVO, and was funded by the CF Trust. And I can honestly say my lungs are amazing, my life now is wonderful and I have no regrets whatsoever.

Watch Philippa's video about her experiences.

CF Week 2013: Emily's Blog

Emily, who has been waiting for a lung transplant since December 2012, describes life on the waiting list.



Hi I'm Emily and I'm the first of this year's CF Week case studies. You can watch my video online at www.cysticfibrosis.org.uk/emily. I am supporting CF Week this year because the focus is on transplants. I have been waiting for my all-important call for over eight months now, I have had a false alarm and been very ill. I really want to raise awareness about the need for more lungs to be made available. Here's what waiting on the transplant list is like:

As most people with cystic fibrosis will tell you, you start off in your early years coping with all medications, or rather your parents do! You start taking the nebs and feeling like you really are beating cystic fibrosis, you get to your teens and start to think about your future, then suddenly, infection after infection slowly takes away your lung function. I had a terrible time last year and ended up in hospital for 10 weeks with a collapsed lung. I was put on the transplant list in December 2012, and my lung function is now at 25%. I am using oxygen 24/7 and a machine to give my lungs a rest called Bi-Pap. It’s exhausting.

To raise my chances of getting that all-important call I am signed up to receive reconditioned lungs through Ex-vivo Lung Perfusion, this is just one of the research projects funded by the Cystic Fibrosis Trust, with support from the Robert Luff Foundation. It is just one reason why donating money during CF Week or any other time of the year to the Cystic Fibrosis Trust is worth your while. If it means I and others waiting for lungs get their call it will be worth it.

My friend Tom Chalk is running a 250 mile ultra-marathon for the Trust to help fund vital research. Visit his fundraising page if you want to support him: http://uk.virginmoneygiving.com/TomChalk.

Don't forget to read more about the consultation being launched today, this is now open for people to respond to, you can find this here. Hope you enjoy the video and the Week.

Emily

x

Watch a video about Emily's story.

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.

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.