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.