Ed Owen's New Year Message

2014 is set to be a very important year for all of us committed to improving and transforming the lives of people with cystic fibrosis.

This summer we receive the results of international Phase III trials of a therapy aimed at treating those with two copies of the DF508 gene, the most common CF mutation.  The treatment - which combines Kalydeco (or ivacaftor) with a new compound VX809 (or lumacaftor) - is the latest in the pipeline of drugs from Vertex focused on tackling the basic genetic defect of cystic fibrosis.

We will also hear the outcome of the year-long Phase IIb study of gene therapy being conducted by the Gene Therapy Consortium here in the UK. This programme has received extraordinary support from the CF community over the last decade or so, and we are eagerly awaiting these results too.

And 2014 marks the 50th anniversary of the Cystic Fibrosis Trust too.

The last five decades have seen enormous changes.  In 1964, cystic fibrosis was almost exclusively a childhood condition.  Today, there are more adults with cystic fibrosis in the UK than kids, with survival rates increasing year on year.

This progress is an extraordinary tribute to the families, clinicians, scientists and, most importantly, those with cystic fibrosis themselves who, throughout the last 50 years, have refused to accept that nothing could be done - and who have fought with commitment and determination to improve the lives of those touched by the condition.

It is down to the efforts of the wider cystic fibrosis community that, today, we have specialist CF centres in the NHS, newborn screening across the country and new drugs and treatments widely available.

And this fight is one we must continue. Even with all the improvements we have seen in recent years, a half of all people with cystic fibrosis are still not reaching their 40th birthday and the daily burden of treatment is imposing heavy burdens and restrictions on all.

So we will not be sitting on our laurels during our 50th year. Given the extraordinary "window of opportunity" being created by developments in research, I believe the next few years have the potential to transform the lives of people with cystic fibrosis.  We must seize this unique opportunity and, with the support of our fantastic community, we will.

On behalf of all at the Cystic Fibrosis Trust, I want to thank you so much for all your support over the last year and to wish you a very happy new year!

Behind the Lens of the Cystic Fibrosis Trust 2014 Calendar

Along with a great collection of cards and gifts, our Christmas Shop is also selling our 2014 calendar, containing photos taken by the Trust's own Dominic Kavanagh.

We've asked Dominic to give us an insight into the story behind some of the photos included.

It's a privilege to help out with the 2014 calendar It's exciting to think my efforts will help support clinical care, campaigning, raising awareness and funding of vital research within cystic fibrosis. 

My creative expression has always been through art; through watercolour painting and drawing as a child and young adult, but a love of wildlife and the natural world and interest in cameras led me into wildlife and landscape photography as a hobby.

Importantly, this has kept me relatively fit over the years - carrying heavy lenses to photo destinations is a great form of airway clearance! Although my health has deteriorated in the last two years, I've not let CF beat me or my photography. I have to compromise, doing morning physio whilst other photographers are capturing great sunrises; stopping short of the highest vantage points as others carry on for the best landscape views. But whilst other photographers list their achievements in terms of awards or competition prizes, I feel my achievement is managing to get out there and capture an evocative image in spite of the discomfort and breathlessness CF causes.

Some of my favourite images have been the hardest to achieve. A trip inside the Arctic Circle in March 2013 was physically punishing for me, in temperatures of -25 °C in Finland and a balmy -7 °C in Norway. I dosed up on steroids and took all the advice from my CF team to counter the low temperatures. I battled my way through Heathrow with camera bag, tripod, suitcase, rucksack of meds and compressor/nebs, as well as my in-flight oxygen. Hours later, the snow scene (January) of pine trees greeted us, as we headed into the woods for Finland's colourful birds. I stayed out until 3am to photograph the Aurora Borealis (Northern Lights), although haemoptysis cut my last night short.

The image of two puffins (July, pictured) on the Shetland Islands reminds me of the challenge of wildlife photography for someone with cystic fibrosis. I had to get up at 4am to do physio and nebs before our gruelling walk, with kit, to the cliffs at Hermaness. The experience provided everything: beautiful natural morning sunlight, undisturbed great squa birds breeding in the moorland and the melodious call of ring plovers which look like Faberge eggs in golden sunlight. To top it all, the return from sea of colourful, characterful puffins to feed their young, at almost arm's length in front of us. We lay in wait for hours for a handful of puffins, kept awake by 'the Stones' on a photographer’s iPhone.

Photography continues to be an incentive for me to challenge myself, pack as much as I can into my life, to achieve the unachievable, and to keep as well as possible for as long as possible. There are some images I still haven't managed to achieve, but I guess, 'you can't always get what you want.'

Engaging MPs in Cystic Fibrosis

The Cystic Fibrosis Trust has recently begun a project of getting Members of Parliament to visit their local cystic fibrosis units. To give an insight into how such visits support the Trust's work, Public Affairs Officer Lynsey Beswick has written about the first two visits:

This month as part of my role as  Public Affairs Officer for England, I invited Luciana Berger MP; Shadow Health Minister for Public Health and Rachel Reeves MP Shadow Secretary of State for Work and Pensions to visit cystic fibrosis units in the North.

Last Friday, the Cystic Fibrosis Trust team and I met with Ms Berger, who is MP for Liverpool Wavertree, at Alder Hey Hospital in Liverpool to discuss cystic fibrosis care with the clinicians there.

The staff there spoke of their excellent network of care structure in Liverpool which spans across 12 smaller hospital clinics locally. They discussed the importance of sustaining this model of care within the region.

We were also invited to enjoy the spectacular view of the new hospital building site, which is being built adjacent to the current hospital. The new hospital promises improved facilities including ensuite rooms on all the wards, which will be important to help to segregate children with cystic fibrosis for cross-infection purposes.

As a result of the visit, Ms Berger will champion the work of the local service in Westminster by highlighting concerns raised by the service around the payment by results tariff and psychology provision.

In West Yorkshire, Rachel Reeves who is MP for Leeds West visited the adult cystic fibrosis unit at St James's University Hospital in Leeds the previous Friday. Ms Reeves met with the multidisciplinary team there and the Cystic Fibrosis Trust team.

The key focus was around capacity issues in adult cystic fibrosis care., relating to the rise in the number of adults with cystic fibrosis, due to better life expectancy and treatments. This means that as the population grows, there is an urgent need for the development of new adult cystic fibrosis care services.

The staff  gave a presentation on cystic fibrosis care with an emphasis on the importance of home intravenous antibiotic care at the unit.

We were also keen to discuss the new Personal Independence Payment (PIP) welfare system, which is being rolled out later across the country in stages this year and will affect thousands of people with cystic fibrosis

As a result of the meeting we were delighted that Ms Reeves would like ongoing information on how the new PIP welfare system is affecting people with cystic fibrosis so that she can represent her constituent's interests in Westminster as the rollout takes shape.

“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

Day Two in Salt Lake City

The conference starts for real today (Thursday) with sessions on a range of topics including latest developments in CFTR research, innovative approaches to improving adherence and ways of supporting the growing number of adults with the condition.

Later today there is also a plenary session entitled ‘Roadmap to a Cure’, which I am particularly looking forward to attending. People in the UK can tune into this session via live streaming at http://www.prolibraries.com/cff/events/2013annual although it is not due to start until 11.20pm UK time, I'm afraid.

Through the day we are planning to catch up with colleagues from Canada, France and Germany to discuss ways of collaborating on common challenges, and we are due to meet the Vertex team too. This conference will be the venue for further information on the latest clinical trials of Vertex’s combination therapies which are generating a lot of interest within our community, and we will make sure we communicate any information we receive on these.

Yesterday, Janet Allen and I had a series of meetings with partners, including two pharmaceutical companies, Gilead and Forrest. We are very keen to develop closer relationships with industry to help drive our research and clinical ambitions.

We also spent a couple of hours talking to Dr Diana Bilton, the lead adult cystic fibrosis consultant at the Royal Brompton Hospital in London. Di is a fantastic champion for the cystic fibrosis cause and chairs the NHS’s Clinical Reference Group that helps shape NHS policy in England on key issues relating to cystic fibrosis care.

We discussed NHS funding of cystic fibrosis centres, the program we have launched to provide genotype testing for people with cystic fibrosis, and innovative ways to develop new models of care.

The Trust works closely with Di on a range of issues and it is one of the bizarre features of conferences like this that they provide rare opportunities – 6,000 miles from home - to have in-depth discussions with people who may work only a few miles away from us in the UK.

Just after lunch, I took the opportunity to take a quick look at the Salt Lake Temple, the spiritual home of the Mormons. Salt Lake City was founded by the Mormons more than 150 years ago and their headquarters is only a few hundred yards from the Convention Centre where this conference is being held.

One of the legacies of the Mormon pioneers is the extremely wide streets here. So to ensure that pedestrians are properly protected as they make the long trek to cross the road, red flags are provided at some crossings to warn oncoming traffic. It’s a bit over the top in a city where traffic is relatively light but I was happy to try it out and you will pleased to know that I arrived safely at the other side of the street!

I’m looking forward to our meetings today and we’ll keep you posted on what we learn from the sessions we attend.

Greetings from the North American Cystic Fibrosis Conference

This week sees the 27th Annual North American Cystic Fibrosis Conference take place in Salt Lake City, Utah and we've sent three delegates to represent the Cystic Fibrosis Trust and on the world stage. Over the week we'll be posting various blogs rounding up the many interesting things which happening over in the US, starting today with this update from Cystic Fibrosis Chief Executive Ed Owen.

The home of the Mormons, Salt Lake City is the venue for this year’s North American Cystic Fibrosis Conference organised by the Cystic Fibrosis Foundation.

More than 4,000 people from scientific research, clinical care, industry and groups representing people with cystic fibrosis are arriving from across the world to discuss the latest developments in drug discovery, medical practice and wider research.

I am here with Janet Allen, the Trust’s Director of Research, and Elaine Gunn, our Registry Manager – and, although it’s a long way to come (it took us 15 hours to get here from the UK!), it is worth it. Many of the contacts and much of the vital information we need to enhance the Trust’s research and clinical work are here – and we will be spending every possible hour seeking them out for the benefit of people of cystic fibrosis.

Although the meeting does not officially open until later today, we have already had a very good meeting with Bruce Marshall, the Cystic Fibrosis Foundation’s clinical director, to discuss peer review, registry and wider clinical issues – and, yesterday evening, we met up with the Cystic Fibrosis Foundation’s President, Bob Beall, and his number two, Preston Campbell.

The Cystic Fibrosis Foundation has led the way in the US charity sector in acting as an engine for new drugs and treatments. The Vertex small molecule pipeline, including Kalydeco, was made possible because of its seed-funding through the 2000s.

We have much to learn from our American colleagues as we improve our performance and impact – and it was very useful to catch up and hear their latest plans. We were able to tell Bob and Preston of the key research projects we are funding and to explore areas of future cooperation.

International collaboration is a must if we are to deliver real benefits to people with cystic fibrosis in the UK. We will be playing our part this week and beyond.

We will also be updating you on meetings and interesting facts and insights that come from the conference through the week. But goodbye from SLC for now!

Follow Ed over on Twitter for live updates from the conference at @Ed_Owen. Don't worry if you miss any due to the seven hour time difference, we will be retweeting Ed over on our own Twitter @cftrust in during UK office hours.

Cystic Fibrosis Trust Clinical Conference 2013: Ed Owen's Speech

On 25-26 September we held our annual Clinical Conference in Manchester. For the first time we combined the medical conference for cystic fibrosis clinicians with the exclusive Centre Directors meeting into a two-day event. Ed Owen, our Chief Executive, opened proceedings on the first day with a welcome speech in which he highlighted the close relationship between the Trust and the clinical community, and spoke of the need to better engage the cystic fibrosis community. Here is the text for Ed’s speech:

It gives me great pleasure to open the 2013 Cystic Fibrosis Trust Clinical Conference here in Manchester. It is great to see so many clinicians and health professionals here. This is my first conference as Chief Executive and I look forward to meeting old friends and making new ones today.

First, I want to say “thank you”. On behalf of the thousands of people personally affected by cystic fibrosis, thank you for all of your commitment, dedication and purpose. Here in the UK we are extremely fortunate to have such an extraordinary group of clinical professionals as represented here, working every day to improve the lives of those we are here for.

Thank you also for your support for the Cystic Fibrosis Trust and our work. The partnership between the clinical community and the Trust is a vital one, and has been hard-wired into what we do since our foundation, 50 years ago next year.

This partnership is as important today as it was then; yet it has adapted through that time and needs to continue to adapt as we face new challenges and new opportunities.

I believe there is a fantastic ‘window of opportunity’ for us in the wider cystic fibrosis community today. The developments in transformational treatments has changed the weather and brought global interest in cystic fibrosis never witnessed before. We now have a drug in clinics for the first time that targets the basic defect of cystic fibrosis.

There are other opportunities too, with the changes in health systems in the UK and the rapid developments in technology.

We need to seize this window of opportunity – partly because it will not be open forever, but primarily because it offers a unique opportunity to shape new approaches that will transform the lives of people with cystic fibrosis.

We at the Trust are changing to help us seize this opportunity. We have a new brand to raise more funds and build greater support, better communications and advocacy. And we have a new research strategy focused on impact and leverage.

I believe that a more defined partnership with the clinical community, building on the success of the last 49 years, can achieve great things too.

Together, we can help develop personalised care shaped around the needs and requirements of people with cystic fibrosis to enable them to control a condition that has for too long controlled them.

You can therefore expect us to become more active across our activities as we seek to improve and increase our real impact, and I am pleased to tell you today of a number of initiatives from the Trust which will, I believe, help in that mission.

First, personalised medicine requires better information about genotyping, and I am pleased to announce that we will soon be launching a project to assist clinics in ensuring that they and their patients have complete information about their genotype.

It will start as a pilot project over the next month with support from Vertex Pharmaceuticals to provide testing for a little over 200 people over the next six months. If it is successful, we hope to have a larger project to follow.

The Trust will also soon be announcing a new fund to assist clinicians and health professionals to attend vital conferences. I can confirm that the Trust will be restoring funding of clinical fellowships to ensure that we maintain the flow of clinical expertise in cystic fibrosis care for the future.

Further details of all these projects will be provided on our website over the next few weeks.

Finally, but most importantly, I want to say something about what will be a defining part of our mission over the next period.

A vital component to getting the changes we want is the voice and experience of patients and their families. They have for too long been the silent voice in what we do, and that is the case here today too – although I am delighted that Oli Rayner joins us today. Oli has joined the Trust as an adviser and you are most welcome today.

We all know the issues of cross-infection. But that can no longer be used as an excuse for us. We have to work harder to hear and understand the views, experience and opinions of those with cystic fibrosis and to articulate and promote them in everything we do.

Please bear this vital issue in mind throughout our discussions today. I look forward to your positive and constructive contribution. I hope you are stimulated by the discussions. And I hope you enjoy it too.

If you’d like to know more about what happened at our Clinical Conference, then keep an eye on this blog where we will be posting a round-up of the event next week.

In their own words: The stars of '...the rest is up to me'

Yesterday we released ‘…the rest is up to me’, a film we made about teenagers with cystic fibrosis. They spoke in their own words about cystic fibrosis and how it affects their lives.

We asked some of the teenagers why they took part in our film and what they hope it will achieve:

Holly Van Geffen

When I tell people I have cystic fibrosis their immediate reaction is “You don’t look ill”. They’re right, from the outside I don’t, I look normal, but the inside of my body tells a very different story. My ‘normal’ appearance is one of the reasons raising awareness of cystic fibrosis is so hard and why when I saw the opportunity to be in this film I jumped at it!

Most of my treatments go on behind closed doors, it is rare that people get a full insight into the everyday routine we have to carry out to ensure we look as ‘normal’ as we do. I hope this film opens people’s eyes to how difficult living with cystic fibrosis is and how much determination and strength it takes to carry on the fight. My life motto is ‘always have hope’, and I am always hoping for improvements in treatments and one day, a cure.

Ben Witham

I hope this film makes people aware not only of what cystic fibrosis is like for teenagers but also for what it is like for everyone else with cystic fibrosis, regardless of age. It's a great way to make people aware. I wanted to take part in the filming for many reasons and doing so for a charity that I was a part of was even better. I feel cystic fibrosis isn't a widely recognised condition, even though it's fairly common and the Cystic Fibrosis Trust isn’t that well known. That was why I wanted to take part in this, more people need to be made aware of cystic fibrosis and the work the Trust does. They do a really good job.

The Trust has helped raise awareness as well as provide support for people like me and when it’s just a small amount, I'm happy with that, that's the best part of it (as well as being filmed, that was fun too).

Cicely Matthews

I really enjoyed doing the film, it's great to know this will help raise awareness of cystic fibrosis. I'm very excited to show it to my friends as I finally have an answer to the 'What is CF?' question: I can just let them watch the video and all will be answered! I think they may be in shock about how much stuff I have to do and what I have to go through. As much as they try to understand I don't think they ever really imagined how much I have to do. It will finally feel like a weight off my shoulders and I won't fear the 'What is CF?' question any more.

Jordan McKinlay

"I’d originally approached the team for this film hoping to gain knowledge in the media aspect of theatre. Theatre is very important to me, particularly sharing stories about CF; and I hope to one day create my own work – with my own company. I hope that other CF patients, like me, will find this a relief in some sense; so that their friends will be able to understand fully all the different ways different patients are affected. I’ve always been treated as an average person by friends and family, and I hope this film will teach others that that’s the way we want to be treated: just like everyone else."

Holly, Ben, Jordan and Cicely are just three of the thousands of teenagers with cystic fibrosis, each with their own lives and their own story to tell

We want you all to share your story, too.

If you’ve been inspired by ‘…the rest is up to me’ we’d love it if you could do your own short videos about your own experience with cystic fibrosis. Upload them to YouTube, share them on Twitter with the hashtag #cfteens and we’ll collate them all on our YouTube channel.

They can be as short as 10 seconds, but please no longer than three minutes! And film them on your mobile, camera, however – it’s what you say about your own experience that is important.

We’re shouting loud about cystic fibrosis and with your voice, we’ll be shouting even louder.

Why we made ‘…the rest is up to me’ about teenagers with cystic fibrosis

Jacqueline Ali, Head of Information,
Support and Conferences,
Cystic Fibrosis Trust

Sometimes the Cystic Fibrosis Trust is perceived as an organisation that just exists to help parents of children with cystic fibrosis, rather than people who have the condition themselves, but that is not the case. It is important that we provide information and support to everyone affected by cystic fibrosis, whatever their age.

The aim of ‘…the rest is up to me’ is to show young people with cystic fibrosis that they aren’t alone; that there are lots of other young people dealing with the same issues they have (over 2,000 in fact!) and juggling the demands of cystic fibrosis and treatment with education, work and a social life. Cross-infection means cystic fibrosis can be isolating, and film is a great medium for overcoming this; we can show young people with cystic fibrosis in their every day lives. We also wanted to reach out to young people who don’t have cystic fibrosis, to help them better understand the condition and how it could affect someone they know.
Most importantly, we wanted to make a film that really represented young people with cystic fibrosis, and we got a fantastic range of responses when we put out the call. It was hard to try and include everything, but some key themes did emerge. From the feedback, it was clear that we had to strike a balance between presenting a positive message to young people, whilst not shying away from the more serious aspects of the condition. Showing young people with cystic fibrosis living life to the full and discussing the various issues in their own words enabled us to do this.

We hope that you will be inspired by the six young people in the film. Each of them has a very different experience of cystic fibrosis, but they all have one thing in common: as Holly says at the end of the film, while cystic fibrosis is definitely part of their lives, it doesn’t stop them living life to the full. The rest is up to them!

Please do let us know your thoughts on ‘…the rest is up to me’ by leaving us comments on YouTube or over on Facebook and Twitter. We’ll be following up this blog tomorrow with the experiences of some of the stars, so stay tuned.

As well as our fantastic actors and everyone that auditioned and provided suggestions for the script, we are very grateful to Genetic Disorders UK the charity behind,  Jeans 4 Genes Day, which provided the funding for us to make this film, and of course the production company Once We Were for putting it all together. We would also like to give special thanks to Mandy Bryon, Consultant Clinical Psychologist at Great Ormond Street, and Sejal Patel, Clinical Psychologist at Barts Health NHS Trust, for their helpful input on the script, and to Great Ormond Street for allowing us to film in the hospital.

Introducing Oli Rayner

Oli Rayner speaking at the launch of
our research strategy at the Wellcome Trust

When Formula 1 teams like McLaren or Ferrari design and test a new car, they need lots of input from their drivers. When it comes to designing and testing new cystic fibrosis treatments, we are the drivers and we need to speak up.

Over the next six months, I will be working as the Cystic Fibrosis Trust’s Special Adviser on Research and Patient Involvement. It is my job to enhance the involvement of people with cystic fibrosis in research in line with the Trust’s five year research strategy.

Why get involved in research?

We know much more about cystic fibrosis and how to treat it than when I was born in 1975. Survival has improved dramatically and people with cystic fibrosis are able to live much fuller lives. Most of the advances have resulted from research. 

There are many different types of research, including clinical trials. No drug can be licensed without robust data from proper clinical trials but a persistent shortage of volunteers slows progress.

Studies that don’t involve a new drug can be just as important in teaching us how to use what we have more effectively or revealing new things about the disease.

Currently in the UK, only about 50% of trials meet their recruitment targets and only around 10% of people with cystic fibrosis participate in clinical trials. Patients pay the price for under-recruitment because it means fewer treatment options.

We want to see research embedded in the culture of all cystic fibrosis clinics. This will encourage us all to think about how we can do things better in the future and how we can use the tools we already have more effectively.   

With potentially transformative treatments in the pipeline, increasing knowledge of the genetic and molecular basis of the disease and unprecedented commercial interest in cystic fibrosis, this is an exciting time and it is important that we make the most of the opportunities ahead.

As patients we have a responsibility to speak up and explain what is important . Cystic fibrosis is a complex disease and researchers will not necessarily understand what it is like to live with the disease, what is important to patients in the real world or what are the most relevant outcome measures unless we tell them.

What do I want to achieve?

My key objectives are to:

·        make sure people with cystic fibrosis have an easy way to find out what clinical trials are open to them and access to the information they need; 

·        put mechanisms in place to make sure decisions about the commissioning and design of clinical studies take into account the views of people with cystic fibrosis;

·        make sure the community is aware of the projects being funded by the Trust, why they are important and how they fit into the broader strategy; and

·        work with the Trust to establish systems and make better use of technology to engage people with cystic fibrosis and make sure their views feed into every aspect of research.

I will also be helping the Trust with a broader long term initiative to give patients more control over their own care and better tools to manage their own health. We want to make the best use of new technologies to:

·        allow people to manage their own health from home with minimal disruption to life;

·        identify early warning signs so we can take action before things get too bad; and

·        systematically learn what works for individuals and feed this back into the system in a way that helps us see broader patterns.

I will be speaking to a broad range of people in the cystic fibrosis community including others with the condition, parents and carers, clinicians and other experts; and looking at what works well elsewhere.

What do I bring to the table?

I graduated with a 2:1 in Law from University College London in 1998 and, as part of this, I studied Medical Ethics. I then spent 12 years working in investment banking and venture capital, advising fast-growing companies in the media, technology, life sciences and renewable energy sectors.

Recently, I have had to step back from full-time work so that I have more flexibility over my time and can prioritise my health. I currently have a number of roles which each take up small amounts of my time.

Among these is consumer referee for Cochrane’s Cystic Fibrosis & Genetic Disorders Group, which carries out systematic reviews of the evidence available to support current treatments, and author of a regular column on drug development and clinical research for CFUnite.org.

In June 2013, I was selected by Eurordis to attend their highly-regarded Summer School for Patient Advocates in Drug Development, Clinical Trials and Regulation.

This experience has given me the ability to understand and process technical information, think critically, interrogate experts and communicate results in non-technical terms. On top of this, I have my own experience of living with cystic fibrosis and taking part in clinical research.

I am excited about this opportunity and interested to speak to people across the cystic fibrosis community. If anyone wants to contact me they can email me at oli.rayner@cysticfibrosis.org.uk or talk to me on Twitter: @oli_rayner.

Find out more about patient involvement in cystic fibrosis research when Oli joins Janet Allen (Director of Research) and Ed Owen (Chief Executive) for Ed’s Question Time live on our forum at 1:00 pm on Wednesday 11 September.