'Hope For More': Transplants & The Power of Us - CF Week 2015

A common story shared by our community is that of transplantation. Still 1 in 3 people with cystic fibrosis on the transplant waiting list will die before they receive a lung transplant.

Since last year, following the publication of our 'Hope for More' report and subsequent campaign, the Trust has continued to lobby for change and improvements to transplantation in cystic fibrosis. Public Affairs Manager Darren O'Keefe has been leading on much of this work and today updates us on some of the developments and progress made since last Spring.

It is always humbling to meet the people directly affected by the fight to improve transplant rates: people on the waiting list, desperately waiting for the call that might just be the difference between life and death; people who’ve been lucky enough to get the call, and of course the families and friends who’ve been there every step of the journey.

Last week I was in Scotland in a room full of these incredibly brave people as Anne McTaggart MSP (pictured) followed Northern Irish politician Jo-Anne Dobson and the Welsh parliament in introducing legislation to make soft opt-out organ donation a reality.

With 70% of the national population not registered as organ donors, this policy is a no-brainer. I hope the whole country follows the example set in Wales without delay and will continue to pressure Westminster.

However, these things inevitably take time and the Trust is doing everything we can to make sure that the organs already available are put to the best possible use. A year ago, in our ’Hope for More’ report, we exposed the tragedy that one in three people with cystic fibrosis waiting for a lung transplant dies before they can receive one, while 75% of donated lungs go unused.

Our campaign resulted in the Government admitting there was a huge problem and they have agreed to trial our proposed system of national allocation for donated lungs. This system is based on need rather than location and we believe it could greatly reduce these needless deaths. We understand that such a radical change must be tested and eagerly await the outcome of the trial.

So for now we shift our focus to other issues:

·   Why are so many donated lungs rejected by surgeons and patients? Are the criteria for what makes these lungs acceptable suitable?

·   Are people given enough information to make a truly informed choice as to whether to accept a pair of lungs that while not perfect, may be their only chance?

·   Do our hospitals and surgeons have all resources and skills needed to make the very best of what they have?

We have a host of MPs supporting our fight to have these questions answered. We will not let the government rest in campaigning for improved facilities and will soon get some of the finest surgeons together to explore the best transplant practices from around the world. We have partnered with NHS Blood & Transplant to establish surgeon and patients attitudes to risk.

‘Hope for More’ was so effective because of the stories shared by you, our community. We were able to show the real impact these issues have on real people, each and every day. Ultimately we will fight for as long as it takes because those brave people and their families deserve hope for much more. So keep those stories coming. And we will win this fight together.

The Power of Knowing Your Genotype - CF Week 2015

Personalised medications like Kalydeco (ivacaftor), which work with specific genotypes, are transforming the lives of people with cystic fibrosis. Knowing your genotype has therefore become crucial in allowing those with CF to access the right drugs for them.

But many people still don't know theirs, or if they do, what treatments could become available to them. That was the case for Jessica Didlick, who through our Genotype Matters campaign learnt that she's eligible for Kalydeco. Here's her story of how knowledge became power.

I found out that I could benefit from Kalydeco with my genotype, if it becomes available on the NHS (it isn’t available for people like me yet, despite evidence showing it could transform my life), when the Cystic Fibrosis Trust posted on Facebook about the Genotype Matters campaign. I asked my hospital about it but they didn’t know anything about my genotype and the fact that Kalydeco could become available for my mutation. We still don’t know if or when it will become available, I feel like I’ve been left in the dark, when I know how much it will help my health and my lifestyle.

My life with cystic fibrosis at the moment is borderline but when I get bad chest infections I'm worse than ever and feel my life can't get any better. I can't even run after my nephews and nieces without being out of breath afterwards and having an attack. I'm trying my best to live life to the fullest and get my health to the best it can be; I want to become a mother, even with a challenging illness holding me back. IVF is my only option right now. My lung function is only 59% at its highest, and that’s without infection.

Kalydeco would change my life. I would be free to do a lot more physical activity without being so out-of-breath and I’ve heard stories about female patients with CF getting pregnant naturally. Kalydeco has reversed the thick mucus and the resulting drop in lung function. Realising my dream of becoming a mother becomes more and more difficult as my health deteriorates. It will give me a few more extra years of living a life I’ve always wanted – I believe Kalydeco is my best chance to feel like I can live a normal life. 

I really wish the drug was available now to those who could benefit: we’re missing out on a wonderful drug that is restricted because of funding arrangements and bureaucracy. Our lives shouldn't be a price tag and shouldn’t be caught up in endless paperwork and committees: save us from a painful, daunting death. At the end of the day, none of us want to die young or in pain. 

It's important for people to know their genotype, and to share their stories, because otherwise you may not get the right drugs; you won’t know whether you are able to receive them. Please find out about your genotype or ask your CF centre if you don’t know it. If they don’t have a record, the Cystic Fibrosis Trust will pay for them to test if for you.

I hope my story will help more people with cystic fibrosis to come forward to share their stories and find there genotype. I am quite passionate when it comes to this kind of thing, especially when it comes to my health and other CFers!

Opening Up the Power of Us - CF Week 2015

London-based Tim Wotton marked his milestone 40th birthday with the publication of his blog, ‘Postcards from Earth’, which in turn led to him writing his memoir: 'How Have I Cheated Death? A Short and Merry Life with Cystic Fibrosis'. Now 44, Tim has just won a Best Achievement book award. Here he explains the power of the CF community opening up…

As cystic fibrosis is not well known or understood, cannot be easily seen or ever properly imagined by most people, I tended to suppress my emotions about CF and was very guarded about disclosing it to strangers. I never wanted to be defined by my condition and I certainly never wanted anyone to feel sorry for me or be pitied.

When I turned 39 with the big and almost mythical 4-0 looming, I had an epiphany that surviving CF had been the biggest achievement in my life but it was hidden away and I wasn’t talking about it or using it for my own or others’ benefit.

I decided to write a diary of the year leading to my 40th to properly reflect my trials, tribulations, happy times and what it has taken for me (physically and mentally) to survive this chronic illness for so long.

I wanted to increase awareness and understanding of CF and share my bigger picture of life perspective, perseverance and optimism with a wider audience. One prime driver was to inspire people that even at your lowest ebb with the odds stacked against you, there is usually a way to overcome.

One additional challenge and cruel twist is that CF can be a lonely and solitary condition as us CFers cannot support each other physically as we’ve been advised not to mix face-to-face. I felt the written word could help bridge this gap for the CF community.

Actually reaching 40 in 2011 was such a dramatic, life-affirming landmark for me that I felt I needed to share my experiences and survival lessons. Still being alive with CF was not a fluke and I had many useful strategies and anecdotes that needed to be offered to others. Personally, I had an overriding desire to reduce the burden of carrying this horrific condition on my shoulders by opening up more.

The reaction to my blog was nothing short of spectacular with over 60,000 global hits. Family and friends wowed me with how much they were enjoying my writing style and close friends confided they didn’t know half of what I had to deal with on a daily basis to still be alive.

The biggest reactions I received were from the adults with CF, and the parents of young sufferers.  Indeed, the moment when I really knew I was making a difference was a blog reply from a mum, which stated “Reading your blog gives me hope for a future for my CF son, when sometimes there seems no hope at all.”

By opening up through my blog, I noticed the new transparency about my CF struggle was being reciprocated by many people. This felt empowering and I was motivated to take it to the next level. As I had written a diary during the year leading to my 40^th, I decided to embellish that into a full-blown book.

As I am a full-time business consultant I had to write the book in my spare time and would often use my underground journey on the Northern line to edit chapters and my lunchtime and evenings to type up the text.

I went through an emotional process with writing this book as I dredged up my experiences and associated feelings. I would always revert back to asking myself ‘What do I always think, feel or say about this?’ Once I’d landed the right narrative I instinctively knew and it always felt cathartic.

I had a deep desire to write profoundly about CF with candour and lack of sensationalism or pity, which is in keeping with the mind set of people with cystic fibrosis.

As I began to delve deeper into my daily health challenges and uncover my personal demons it became obvious to me that being authentic meant bringing out my inner feelings on every facet of what I’d endured to try to leave a normal life. The health battle I had always struggled to convey verbally to people felt easier to expose in words.

Writing ‘The Hardest Part’ chapter about the loss of my friends  with CF was the toughest to write by a distance. Many a tear was shed onto the paper as I tried to unearth the raw emotion of how it felt to lose fellow sufferers who had every right to live as long as me.

The final book is a thought-provoking and amusing memoir, which elaborates on my extensive medical regime, going to university, getting a job, the importance of alternative therapy, faith and a positive mental attitude to counteract the multitude of dark moments. It also highlights the significance of family support, my marriage to Katie and the rollercoaster journey we undertook to start a family which finally delivered our awesome son Felix.

I am already reaching a global audience with this book. The CF community are deriving some hope and survival strategies from my story while the wider audiences are definitely understanding CF better and appreciating what it takes to combat it on a daily basis.

In this brave new virtual CF world, where CF folk can’t openly meet up and chew the fat, the power of the written word in the form of social media, forums, blogs and books is filling a void and allowing us to open up in ways unimaginable even 10 years ago.

Having a book published and available for anyone in the world to read feels awesome, humbling and bewildering in equal measures. Judging from the feedback received so far, my memoir is making a real difference. To win an award at the UK’s People’s Book Prize has given me the extra motivation to keep writing, being honest and to help others with my life insights.

The book, which features an introduction by actress and long-time Trust supporter Jenny Agutter, is available to buy from online retailers Austin Macauley Publishers, and Amazon (ISBN 9781849637190). 

The Power of Us - CF Week 2015

Today marks the start of CF Week 2015, and this year our theme the Power of Us looks to bring the whole CF community together to help us spread the word and work as one in the fight against cystic fibrosis. To kick things off Emma Lake, our Community Manager who also lives with CF, tells of her experience and how sharing her story (and those of others) has played a key role in achieving great things for the CF community.

My husband thinks I am a super hero. He calls me sputum girl. Sometimes he calls me a mutant. I'm not sure what my superpower is, but if the X-Men give me a call I won't say no.

In a way my husband is right. I do have a superpower. So does he. So do you.

Our superpower is our stories. Having worked at the Cystic Fibrosis Trust for nearly 12 years, I know that it is our stories that are the real drivers for change.  The Power of Us is the greatest tool we have.

The fact that Kalydeco is now funded is not just down to the Cystic Fibrosis Trust. True, they played their part, but it was our stories, our lives, our voices that made the difference. In this new future of personalised medicines we have a vital role to play.

The Enough is Enough campaign would have achieved nothing, without our community behind us every step of the way. From signing the petition and writing letters to talking to the media and waving placards, we are unstoppable when we use our own experiences to generate a change.

I sit on the Clinical Reference Group for Cystic Fibrosis as a patient representative (or “person representative” as I prefer to call myself). Our community is also represented by parents of people with cystic fibrosis and we are more than just a tickbox exercise. We are part of a group who essentially decide the future of CF care and I and the parent reps are influencing those decisions, using not just our knowledge and experience, but yours too.

There are countless other examples that I (and you) could mention, such as approval of Mannitol, Colobreathe and the Tobi Podhaler. We have access to all these inhalers, because we used the power of us. We were so good at using it that people had to listen.

So now it’s time for our community to put on our capes and manifest our superpowers. Words are powerful. Don't be afraid to shout them loud!

Join in with CF Week 2015 by sharing your story across social media (and beyond) with the hashtag #PowerOfUs.

We're also challenging you, our community, to talk to a person a day about CF. From someone new to someone you may have mentioned it too in the past.

Orkambi in the UK: Our work begins

Following the Trust taking part in a meeting with National Institute for Health and Care Excellence (NICE) to discuss the process for appraising the new cystic fibrosis drug Orkambi, Chief Executive Ed Owen, who attended to represent the Trust, looks a the road ahead for getting the combination therapy to people with CF.

The news two weeks ago that Vertex’s latest drug, Orkambi (a combination of ivacaftor or Kalydeco and lumacaftor), has demonstrated impact for the most common mutation of cystic fibrosis was greeted with enthusiasm and excitement.

TheBBC called it a “breakthrough drug” and it is the first therapy that targets the basic genetic defect of those with two copies of the F508d mutation affecting about half the cystic fibrosis population in the UK.

It is a significant development. But we should be under no illusions that this is anything but the beginning of what is most likely to be a tough fight to ensure that all those who might benefit from Orkambi in the UK get access to it.

First of all, it has to be licensed for use by the European Medicines Agency, which is the primary body responsible for granting drug authorisation across all EU member states, including the UK. This decision is expected in the autumn, two or three months after the equivalent decision in the USA by the Federal Drugs Administration.

Then, if the EMA does grant a licence to Orkambi, it will need to be assessed by each of the four devolved NHS systems in the UK (in England, Wales, Scotland and Northern Ireland) and each will need to decide whether and how to allocate funding to pay for it.

In Scotland, the drug will be assessed by the Scottish Medicines Consortium while in England it is likely to be subject to an appraisal by NICE, the National Institute of Health and Care Excellence.

Yesterday, in Manchester, I attended a meeting organised by NICE and including cystic fibrosis clinicians and health professionals along with NHS commissioners and others to discuss possible options of how it might undertake such an appraisal. If it does do so, NICE will look at the clinical effectiveness of the drug as compared to existing therapies available and, even if that is accepted, then take a view on whether the likely clinical impact justifies the likely cost to the NHS.

Of course, calculating the cost means knowing the price – and Vertex has yet to announce the price it will charge for Orkambi. Most experts assume it will not come cheap.

As my colleague, Nick Medhurst, said yesterday, it is encouraging that NICE is engaging early on this issue, in advance of the EMA’s decision. We do have concerns, however, that the methods it uses to carry out drug appraisals are not particularly well-suited to assessing potentially transformational drugs in relatively rare conditions like cystic fibrosis.

And an appraisal can often take six months or more with no guarantee of a positive recommendation at the end of it.

At the appropriate time, Vertex is likely to be asked to submit detailed evidence to NICE in order to demonstrate that Orkambi is both effective and affordable. The Trust will also submit evidence to communicate the voice and experience of people with cystic fibrosis themselves, and their carers.

This process is unlikely to begin much before the end of this year in the event of a positive decision by the EMA. Yet we are already listening and engaging with the wider cystic fibrosis community to ensure we are able to speak clearly and forcefully on behalf of everyone with the condition in this forthcoming process.

So, it will not be an easy ride. But we will always be committed to getting therapies that make a difference to people’s lives to those that need them as soon as possible. In order to do this we will need both Vertex and the NHS to do the right thing, and we will work hard to ensure they do.

As a community, we will need to show patience, resilience and determination throughout if we are to help get the right outcome for people with cystic fibrosis on this issue and other potential therapies further down the line targeting other mutations.

And, most of all, we will need unity. By sticking together and making our voice heard as one we can change the world of cystic fibrosis. We have done it before and we can do it again.

My Marathon Journey

There's 24 hours to go until the ballot for next year's Virgin Money London Marathon closes.

It's the leading mass-participation charity fundraising challenge event in the UK, and this year we had more #TeamCF runners than ever!

We'd love 2016 to be even bigger, with even more of you joining #TeamCF but we know it can be a daunting experience, especially for first timers. To help you all get a feel for what it's like, Libby Sears, a first time London Marathon runner this year has shared her journey, from spectator to marathon finisher.

My marathon journey has been one unforgettable experience and one of the most memorable days of my life (so far). It has taken more courage and willpower than I thought possible.

It all started after being a supporter and spectator at the London Marathon 2014. Having watched a very good friend go through her journey, I felt inspired to take on the challenge myself. Thankfully with the help of my wonderful bosses, I secured a charity place with the Cystic Fibrosis Trust early on in the year, so I had plenty of time to get my training into action.

Arriving at the expo on Friday morning to collect my number and time chip I felt completely overwhelmed and inadequately prepared for what lay ahead of me. I was pretty much an emotional wreck, even the gentleman handing me my pack expressed concern at the look of fear on my face. I was petrified, not only at whether I could actually complete the full 26.2 miles but also the thought of letting everyone down. There was no going back.

I woke on Sunday 26 April 2015 with excitement (much to my amazement). I arrived at the Green Start in the drizzly rain and headed for the changing tent to find it full of ladies sat on the floor, in pretty much the same state as myself, constantly feeling the need to wee and looking at their phones. The time passed quickly listening to stories of training disasters (great!) and tips to get through. I was inspired once again at what these people were willing to put themselves through to raise money for charity.

The race got underway, I threw off my jumper, which unfortunately landed on some poor lady’s head!   I was swept along with the crowd of runners and found myself running at a faster pace than I had planned, but decided to go with it, even though in my head I had my friend’s words telling me not to start like a racehorse. 

The first five miles quickly passed and I found myself excited to be seeing family and friends with the CF supporters by the Cutty Sark at mile seven, and there they were just as planned, I felt great and uplifted, it was an amazing sight, full of people cheering, I was feeling great. I had no idea where I was until I saw the Rotherhithe sign and realised Sam and Michelle had planned to watch at this point, as I ran on I heard them shout my name, it made me smile from ear to ear.  

As I approach Tower Bridge I could hear the roar from the crowd, I spotted a friend from holiday who screamed my name and the CF guys going wild; people were running backwards taking selfies, I was tempted to offer to take it for them but was reluctant to slow my pace, I was on a mission!

On I went, through a tunnel before mile 15 where men and women were having a quick unseen wee and a sneaky walk, and a few meters on were Dave, Imogen, Jamie and my wonderful friends. I was so excited to see them, I felt my heart swell with pride, “I can do this”, I was feeling good. I wanted to get to mile 18 so I could see them again; all I was thinking about was who I had managed to see as I’d sped past and who I needed to make sure I saw next time. By now my knee was starting to let me know that it wasn’t happy and my pace was slowing, but thankfully I saw my support group and I managed to fight back the tears as I ran past waving like a lunatic.

Having past mile 20 and getting a phone call from Dave, all I could make out was "see you at the finish”. The next five miles were the hardest I have ever experienced; doubt was creeping in as my knee was unforgiving, the painkillers making little impact. I focused on counting down the miles, knowing I was on the home straight and trying to work out where on earth in London I was; I never have had a sense of direction. I attempted to take my jelly beans out of my pocket but ended up losing the lot in a comedy style throw them in the air attempt.

I had one goal in my head now: “Don’t stop. Whatever you do, keep running.”

The crowds were huge and the noise immense, I could no longer hear the music playing in one ear, so turned it off completely, it was time to dig deep.

Turning the corner at the Houses of Parliament everything changed, my pain had gone, I had beaten my demons, my pace increased and I sped down Bird Cage Walk excited to see Michelle, Lisa and Maria at the 600m mark. There they were, I felt exhilarated, I’d done it. Now to the finish, this was the easy bit.

I waved frantically at my mum and mother-in-law who had sat patiently in their grandstand seats, and crossed the line. The feeling of achievement was overwhelming, waiting for my photo to be taken I was trying not to cry. What an incredible day.

I have raised a whopping £25,500+ for the Cystic Fibrosis Trust thanks to the fabulous Christine and Berenice, my amazing family, wonderful friends and other incredibly generous people. Thank you CF for your support and encouragement throughout.

To Dave, Imogen, Jamie, my parents, Jamie and Hilary Gray, Michelle - Thank You.  I couldn’t have done this without you.

26.2 Smashed

Inspired to join #TeamCF? Enter the Virgin Money London Marathon 2016 ballot today!

Three marathons, three cities, three countries – one cause

This weekend, runners from all over the UK will descend on Greenwich for the Virgin Money London Marathon, running for Team CF to raise money for the fight to beat cystic fibrosis. Among those lining up will be Kyle Parsad, completing a trilogy of marathons in just five weeks, for the Cystic Fibrosis Trust.

Rome was beautiful, I loved the city when I went last year and that's what made me sign up to the marathon there for this year. I didn't quite realise it would be so difficult though, and the cobbled streets really took it out on my body! Having said that, I got through it and the first one out of three was done in a time of 4hr 08mins.

After going through the pain and how hard Rome was, Paris was playing on my mind and there was a part of me that was worried that it would be as tough. But it was a stunning route, and the organisation was second to none. I enjoyed every minute of it... up until the last few kilometres when it started to hurt, but the support from the crowds kept me going and the atmosphere makes you want to finish. I loved it, and would definitely do Paris again in a heartbeat. This one I finished in a time of 3hrs 47mins... a new PB!

Finally, at the time of writing this I will be 11 days away from the final one of my three marathon challenges... London! I finished this in 2011 and again it was the best thing, but the hardest thing I have ever done in my life. However I am more prepared, mentally and physically, for this one then I was previously.

The other two marathons have trained my mind and body to be prepared to tackle the streets of my home country! It seems a long time ago that I started all this training and a while ago that I did Rome but in the space of five weeks I will have completed my trilogy of marathons, all for such a worthy cause... cystic fibrosis. I'm very much looking forward to pounding the streets and making this my marathon, my race and the one that I will remember forever! I am someone who is lucky enough to be able to do this, I am physically healthy and CAN run...therefore it is my responsibility as someone who CAN to help those who CAN'T!

Sponsor Kyle at www.virginmoneygiving.com/kyleparsad.