Monday, 3 March 2014

To Petra and Back Again

25 February - 2 March saw 21 intrepid adventurers, including TV's own Sian Lloyd, take on the desert of Jordan in support of the Cystic Fibrosis Trust. We asked Sam Davies, Events Manger for the Trust who was part of the trek to give us a round up of the experience, our first overseas challenge.

Our five-day Petra Trek was a great success. All 19 of our trekkers managed the 27.5km route, including some very steep climbs, thin cliff-top paths hundreds of feet up, and hot desert tracks.

The group soon bonded, helping each other to overcome personal and group challenges, while enjoying spectacular scenery, fascinating history, and the beauty of the ‘lost city’ of Petra.

Our local guides gave us a fascinating insight into ancient and modern Jordanian culture, and we were lucky to experience live Bedouin music around our campfires, while sitting under an incredible desert night sky.

We are on track for raising £50,000 from the event thanks to everyone who has sponsored our trekkers, and are very excited about upcoming overseas challenge events later this year and next.

You can see more about what happened on the Petra Trek over on Storify, where we've collated people's tweets and photos.

Wednesday, 12 February 2014

Unveiling Our First Strategic Research Centres

Following the unveiling of the Cystic Fibrosis Trust's first Strategic Research Centres, Director of Research Dr. Janet Allen writes about what this will mean for progressing research in cystic fibrosis.
On 29 April 2013, we launched an ambitious research strategy,“Investing in research to change lives”, in which we set out a roadmap for the Trust to deliver high quality research that will make a difference to people with cystic fibrosis.

One of the critical components of this new strategy is the formation of Strategic Research Centres/Virtual Centres of Excellence. Through these centres, researchers are encouraged to work together to tackle major research questions in cystic fibrosis. Scientists were asked to bring together the best team of scientific experts, wherever they are based, to form a cohesive group intent on using research to solve problems for cystic fibrosis. Members of these teams form the virtual centre, which can be truly international because, although led from the UK, team members can be recruited to the virtual centre from anywhere in the world. For the first time the Trust will be funding scientists in other countries. These Strategic Research Centres were deliberately set up to provide funding for five–six young career scientists who will work alongside these experts. Our aim is to attract the brightest and best young scientists into problem-solving research within the best teams;  helping to  shape the next generation of leading researchers for cystic fibrosis. Experience shows that investment in these individuals at this stage of their careers results in a long-lasting interest.

I am delighted to say that the first round of Strategic Research Centres has now been completed.  Although this may seem slow, it has in fact been achieved in record time! Following the strategy publication, we invited applications from the research community and then established an independent peer-review process to select those that best fit the overall aims of the Trust for Strategic Research Centres. A number of very high quality applications were received at all stages of the process. In the end, there were three outstanding applications but funds were only available to support two. It was very difficult to select which would be funded but after long, detailed and  careful consideration, the two  have been selected. Both consortia are led by international experts based in the UK but have created teams that bring together international experts to work together to find solutions for people with cystic fibrosis. This is the first time the Cystic Fibrosis Trust has led such an initiative and has funded research beyond the UK.
Briefly, one project is led by Dr Mike Gray at the University of Newcastle and aims to find new ways of treating cystic fibrosis. The second project is led by Professor Jane Davies at Imperial College London and the Royal Brompton Hospital and tackles pseudomonas infections. We will publish full details of both projects on our web site in the early summer.

Tuesday, 31 December 2013

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!

Thursday, 31 October 2013

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.'

Tuesday, 29 October 2013

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.

Monday, 21 October 2013

“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.