“I have never felt more alive” - Running the London Marathon

Ballot places in the Virgin Money London Marathon 2014 were released last month, and we at the Cystic Fibrosis Trust would love those of you who got in to run for us.

It is always inspiring when anyone runs a marathon for us, but even more so when that person has cystic fibrosis. People like Alastair Mavor, who has told us his story in the hopes that you all out there will come and be a part of Team CF.

“I have never felt more alive”.

As I stepped off the train the realisation hit me. The sponsorship, the training, had come down to this one day. I breathed in as deeply as I could. No crackles, no phlegm. I felt so incredibly clear. In body and mind. I sent a text to Mum and Dad.

“Thank you so much for all your support. I feel so excited right now. I love you so much. This really is what living is all about.”
I hated running at school, yet every year from the age of about six, I sat rooted in front of the TV watching the London Marathon, dreaming that one year I could run it.

So it became my gap year goal! The day itself was agony, but at the same time fantastic. A year later, at just 19, I had run my second London Marathon and managed to raise more than £30,000 for the Cystic Fibrosis Trust.
I’m 24 now. I have cystic fibrosis, and in April of this year, I ran my third marathon. I have, on the whole, been lucky with my health. I was probably fortunate to be diagnosed in my first three weeks, meaning I could begin the daily rigmarole of physio without my lungs degrading too greatly at the start. My parents were also incredibly disciplined about giving me my treatments, and it’s something I’ve managed to carry on throughout boarding school and university as a result. One thing that has helped me maintain my lung function over the last few years is my love of running.

I have found few better ways of clearing my lungs than a hard run to shake things up. This winter was one of the wettest and coldest for many years, yet running for my own health as well as training for the event provided another motivation to get out in the snow and churn out the miles. Hitting the trails of the Peak District also gave me relief from the stresses of University Finals!
But the thing which fired me up most was running and raising money and awareness for a charity that is incredibly close to my heart. Without the efforts of the Cystic Fibrosis Trust, and all those who have supported it, I would not be alive today.

Before this year’s race, I kept my condition quiet from all but my closest friends at University. I suppose I was scared it would shape people’s opinions of me. I decided to be more open about it; to raise awareness of the disease, and to show people that I might happen to have a nasty condition, but it wasn’t going to stop me achieving all the things I wanted. I needn’t have worried. The support that came as a result was incredible. People who I barely knew started flooding my Justgiving page with donations. By the time the race began, I had reached over £5,000 and more than 150 donations. 
That instilled me with an unflinching desire to be on the start line, despite hobbling around on crutches due to an achilles injury three weeks before the race!


If you are thinking about a marathon, stop thinking and start doing. The feeling of elation and emotion when you cross that finish line is like nothing else. There were great miles, and horribly painful miles, but the overriding memory of making the final turn around Buckingham Palace, seeing 3 hours 50 on the clock, and running on through a wall of cheering and applause, is one I will never forget.

Have Alistair's words spoken to you? Then please use your ballot place and run for us in the Virgin Money London Marathon 2014. Details are on our website.

Research News from NACFC

Janet Allen, Director of Research, gives her final research perspective on the North American Cystic Fibrosis Conference

Despite it being the last day there is still a buzz around the conference centre, with people scurrying between sessions. The day started off with an excellent plenary talk on cystic fibrosis-related diabetes. It provided a superb example of how to integrate basic science and clinical research to get the best value. 

I spent the rest of the day getting my "fix" on the science around the structure of the CFTR molecule. There is an amazing amount of work across the world trying to understand how a complex molecule like CFTR folds within cells to allow it to function and how mutations in the gene affect that folding pathway. Although this research may seem esoteric, it is this detailed understanding that is the foundation for the discovery of new drugs that will help people with cystic fibrosis.  

One highlight of the morning session was the presentation of a small clinical trial for ivacaftor (Kalydeco) in people who have genetic mutations in CFTR that affect its gating. These are very rare mutations and so standard Phase 3 trials are difficult to perform. Only 39 patients were involved in this trial but the data is promising as the results were very similar to those for people with G551D taking ivacaftor.

You can find out more about cystic fibrosis research and our involvement on our website and through the Cystic Fibrosis Trust Research Strategy 2013-2018.

All Nations With One Goal

With the North American Cystic Fibrosis over and the 4,000 delegates from around the world making their way home, UK CF Registry Manager Elaine Gunn rounds up the success it has been.

The end of the North American Cystic Fibrosis Conference has arrived after a final day of excellent seminars, workshops and a final plenary session.

It has been a fabulous conference, lots of hard work, early starts and numerous out of session registry meetings but the overall feeling of passion to work together no matter where you come from to ensure people with cystic fibrosis get 'gold standard' care is palpable. The research going on around the world is outstanding and listening to researchers passionately describing their work is amazing. 

For me the use of the CF Registries, not only the US one but the UK Registry, in various talks was fantastic. They offer a wealth of data and it is great seeing it being used.

Tomorrow we start the long journey home. I will miss the mountain view walking to the conference centre and the clear, crisp, sunny weather – but feel excited about what is happening in the world of cystic fibrosis. It is a fight we must win, not just in the UK but around the world. All nations, with one goal

Goodbye To Mountain Country!

Chief Executive Ed Owen offers his final thoughts on the North American Cystic Fibrosis Conference

The 10,000-foot, snow-topped mountains that provide such a dramatic backdrop to Salt Lake City seem appropriate as I leave this year's North American Cystic Fibrosis Conference.

Fifty years ago, the challenge facing the families, clinicians and others affected by cystic fibrosis must have seemed Himalayan in scale. Those born with the condition were not expected to live into double figures, and precious little was known about its cause or how to combat its devastating effects.

Today, in the view of many of the scientists gathered here in this frontier city, the summit is now in reach.

Of course, there is considerably more work still to do to reach our goal to beat cystic fibrosis for good. Too many young lives are ended early, too many futures are blighted by this condition, to suggest otherwise. We certainly cannot afford to let up now.

After all, the pipeline of transformational small molecule treatments showing such promise and, in the case of Kalydeco, already making such a difference to some, will not in themselves get us all the way to the top.

New challenges are appearing too, such as 'superbugs' like NTM. Vital issues raised this week also reflect the increasing need to support people with cystic fibrosis pursuing careers, starting a family or coping with the many psychological pressures imposed by their condition.

And improving standards of care so that everyone receives the best quality, personalised care with equal access to drugs and treatment they need will continue to be a driving mission for many years to come.

But the extraordinary progress of the last few years in what people here call "disease modification" must give us fresh hope - and new impetus to increase further our efforts to conquer the cystic fibrosis mountain.

We in the UK, people with cystic fibrosis, their families, clinicians, health professionals, scientists and industry, are playing an important part in this extraordinary challenge. And we at the Cystic Fibrosis Trust are determined to raise our game further at this critical time.

This week has reinforced my view that the global cystic fibrosis community faces a unique window of opportunity at this time. We must seize it now and push on to achieve our goal.

Some years and many obstacles still lie ahead. But we must hope, believe and expect that we will get there.

So it's a fond goodbye to Salt Lake City from me with a huge thank you to the scientists, clinicians and, most of all, those personally affected by cystic fibrosis, for your monumental efforts and achievements.

Janet and Elaine are here for the last day today and will provide you with final thoughts of what has been an inspirational and successful conference.

Learning From Others At NACFC

The North American Cystic Fibrosis Conference is organised by the Cystic Fibrosis Foundation, who are the US counterpart to the Cystic Fibrosis Trust.  Like many other organisations we work with they have become an excellent and indispensable partner in the fight against cystic fibrosis. Ed Owen's latest blog from Salt Lake City gives an insight into the valuable work they have been doing and what we here in the UK can learn from them. 

"There have been tremendous improvements in cystic fibrosis in recent years. But now we need to close the deal."

So said Preston Campbell of the Cystic Fibrosis Foundation at the opening of the North American conference

here in Salt Lake City yesterday.

He was reflecting the great progress that has been made in recent years to increase life expectancy and the quality of life for people with cystic fibrosis - but also the single-minded determination that exists here among scientists, clinicians and others to end cystic fibrosis for good.

We at the Cystic Fibrosis Trust are equally ambitious and we all need to redouble our efforts to achieve this ambitious goal.

Like the Cystic Fibrosis Foundation here in the US, we are currently working on ambitious and inspiring plans that will bring us closer to this ultimate aim.

Bob Beall, the President of the Cystic Fibrosis Foundation, articulated in his speech yesterday what he believed were the three key challenges facing the cystic fibrosis community today:

- to develop transformational, disease-modifying treatments for all people with cystic fibrosis whatever their mutation;

- to ensure those with cystic fibrosis have access to these new therapies and to makes sure their wider value is properly recognised by healthcare systems; and

- to ensure all people with cystic fibrosis have the best quality of care available.

Bob has been with the Cystic Fibrosis Foundation for two decades and has provided inspirational leadership for the cystic fibrosis community here in the US and across the world.

The Cystic Fibrosis Foundation's seed-funding of small molecule work more than a decade ago has produced the tremendous breakthroughs we are witnessing today with Kalydeco and other potential therapies in the pipeline.

There is much we in the UK can learn from the Cystic Fibrosis Foundation’s experience - the need for clear focus and purpose, the value of targeted collaboration with industry and an impactful fundraising offer.

Earlier in the day I attended two sessions - the first focused on supporting the growing number of adults with cystic fibrosis, and the second on what works to improve adherence to treatment.

The morning session covered a wide range of issues including career development, parenthood and depression. The afternoon saw presentations on motivational therapy, the value of social networking and a novel supervision programme in the US called iCARE.

There are many fantastic things going on for people with cystic fibrosis on both sides of the Atlantic. Together we can both improve and transform the lives of all people with the condition.

Research at North American Cystic Fibrosis Conference

Dr Janet Allen, the Trust's Director of Research, has been  liaising with colleagues from all over the world at the North American Cystic Fibrosis Conference. Here she tells us what she has been getting up to over in Salt Lake City

This is my second North American Cystic Fibrosis Conference. My first was in Orlando last year just after I had started at the Cystic Fibrosis Trust and it was a fantastic introduction to the whole area of research in cystic fibrosis. This year is an opportunity to build on the networks that the Trust is developing internationally across Europe, North America and elsewhere and to hear of any new developments in the field.  

Since arriving on Monday we have been very busy networking with many different people and organisations. Yesterday there were clearly many more people travelling in from across the world to attend what is a truly international meeting. Attendees are easily spotted on the streets of Salt Lake City as we have all received bright blue conference bags. Today (Thursday), the scientific sessions started and I have been trying to juggle the various talks in the scientific programme. This evening the first plenary talk was given and it is only when everyone is sitting (or standing) in the main hall to hear the formal opening speeches and the plenary talk that it really drives home how large the conference has become. 

The plenary was the first of three, all subtitled ‘Roadmap to a Cure’. This one was delivered by Scott Donaldson from University of North Carolina. His first slide was titled ‘The Audacity of Hope’ and this set the tone of his talk. He reviewed how far cystic fibrosis research has come, and highlighted how there is much more to do. His analogy was one of a journey of discovery – and posed the question: how do we know where we are on that journey and how do we know when we have arrived? His talk then led through the extraordinary discoveries that have been made and what we need to do to deliver more. He drew on research from around the world to give examples and it was particularly pleasing to see the work of Professor Mike Gray acknowledged as part of this. Mike is based at the University of Newcastle and is now the chair of the new Research Strategy Implementation Board for the Cystic Fibrosis Trust. There was a real sense of people working together and sharing information to find ways to benefit people with cystic fibrosis.  

Another Take on NACFC

Our Chief Executive, Ed Owen, isn't the only person from the Cystic Fibrosis Trust out in Utah at the North American Cystic Fibrosis Conference. Along with Janet Allen, Director of Research, Elaine Gunn, UK CF Registry Manager has also made the 15 hour, 6,000 mile journey to Salt Lake City. Here's her take so far

The weather here is clear and crisp from being surrounded by mountains, which is great after the soggy weather in the UK.

On Wednesday I attended the US Registry Coordinators Meeting with all my US Registry Colleagues. This meeting is always a highlight of the conference as not only do I meet with my colleagues, but the US Registry team presents its CF Registry data and any changes it has made. This year was different in that there was a ‘live’ poll going on throughout the meeting where we had to text comments and you could see the results live on the screens.  It really kept us focused and caused some amusement as some of the questions were frivolous. The US CF Registry reports will be available on the Cystic Fibrosis Foundation website next week.

Today the full conference starts in earnest and I will be attending several really exciting sessions, including the plenary which is always an engaging one. There are lots of my UK colleagues here and I am engaged in conversation at almost every corner. As Ed said, 6,000 miles is no barrier!

I too walked to the Mormon Temple yesterday and it is an amazing feat of architecture.  As a musician though, I was particularly interested in the Mormon Tabernacle Choir, which I had heard of since my childhood. Tonight I am going to their open rehearsal in the Tabernacle, so a great day ahead, with excellent sessions, and some music in the evening.

The plenary session mentioned by Elaine is being live streamed, starting for UK viewers at 11:20pm. You can view it over at http://www.prolibraries.com/cff/events/2013annual