Saturday, 4 July 2015

Drawing breath

The last few days have seen some dramatic breaking news for the cystic fibrosis community, with results from the gene therapy Phase 2b clinical trial being published, NHS England announcing funding for ivacaftor for eight rare mutations, and the FDA in the US licensing the combination treatment Orkambi. In this blog, Trust Chief Executive Ed Owen looks back on a momentous couple of days, and how the power of the CF community shines through.

The last 48 hours has proved a significant step forward in our fight for a life unlimited by cystic fibrosis – and one that reinforces the collective power we have as a community, both national and global.

On Friday morning the results of the gene therapy Phase 2b clinical trial were published in the Lancet Respiratory journal. While not a surprise to those who took part in our sneak preview a few weeks ago, the outcome is certainly encouraging.

More clinical trials are needed before we can say gene therapy can be a viable treatment for people with cystic fibrosis, but it successfully establishes proof of concept that demonstrates genuine promise.

Future trials are now needed and that will almost certainly require the sort of resources and expertise that only a pharmaceutical company can provide. We also have to be realistic and accept that this will take some time.

But it is good news and one that has not come this far by accident.

First and foremost, it is a tremendous tribute to Professor Eric Alton and the wider Gene Therapy Consortium team for their ground-breaking work. Yet, as they would be the first to say, this could not have happened without the participation of people with cystic fibrosis, young and old, who have volunteered to take part in this and earlier clinical trials.

And, vitally, it has only come about because of the extraordinary support of the wider cystic fibrosis community raising many millions of pounds over the last 15 years.

This is continuing and the Trust has pledged a further £500,000 to support the GTC’s Wave 2 gene therapy product which – while about five years behind the product that is the focus of this week’s news – has potentially much greater impact.

The Trust will continue to work tirelessly to ensure everyone with cystic fibrosis gets access to life-changing therapies however long it takes. As with gene therapy and other promising scientific development, that means investing funds raised in research.  But it also means lobbying and campaigning to ensure the NHS funds therapies approved by regulators.

So we were delighted with the news on Thursday that NHS England has agreed to extend access to Kalydeco (ivacaftor) to about 40 people who have non-G551D gating mutations of cystic fibrosis.

It has taken far too long, and demonstrates the inadequate system in place to ensure such transformational drugs are appraised. What’s more, a handful of people in Wales are still waiting – and we will continue to fight for them until the right decision is made.

But the voice of people with cystic fibrosis supported by clinical teams and backed by clear evidence of Kalydeco’s transformational impact rightly won the day.

There are many more issues like this to come over the next years and a further big potential campaigning issue on the horizon is Orkambi – the so-called combination therapy eligible for those with two copies of the F508delta gene, the most common mutation of cystic fibrosis.

Orkambi has yet to be approved by EU regulators but it was good to hear this week that the US regulators, the FDA, have given the green light for its use in America.

Its effect is less dramatic than that seen with Kalydeco on those with gating mutations. But the evidence from Phase 3 trials shows that it significantly reduces hospitalisations for those who take the drug and stabilises lung function.

We are expecting a decision from European regulators by the end of this year and, if approved, the key issue will be whether it is reimbursed by the NHS. It is by no means certain that it will, and we will be pushing Vertex and the NHS in the four parts of the UK to do the right thing to enable Orkambi to get to those who need it as soon as possible at an affordable price.

This week has shown that when we come together as one we can change the world for many with cystic fibrosis. The Power of Us will continue to be our key weapon to secure further changes vital to our mission to transform the lives of all.

Friday, 19 June 2015

Cystic Fibrosis Comes to EastEnders

By now, you've either seen or heard that one of the latest storylines on long-running BBC soap EastEnders involves a young girl with cystic fibrosis.

This is the culmination of months of work between the production team and the Cystic Fibrosis Trust to ensure that cystic fibrosis is represented as accurately as possible on-screen. Our own Engagement Director, Oli Lewington, has been one of the key people in these conversations and having already given some insight into how it all came together on EastEnders blog,  Oli expands on the work for us here too.

Back in November I received a meeting request from our Communications Manager, Paul, to accompany him to a meeting at Elstree Studios. All I knew at the time was that it was the same studio where Eastenders was shot.

When we arrived I was hastily asked to sign a non-disclosure agreement forbidding me from discussing any of the contents of the meeting with anyone outside that room.
Today, of course, you are all well aware of Jade, the new character introduced to Albert Square in Friday night’s “duff duff” moment.

Anyone in the CF community is also well-aware of the traditionally sketchy nature of representations of CF on TV, not another well-watched BBC show, Holby City. You’ll understand, then, our excitement to be involved at such an early stage, some eight months before the character first appeared on screen, helping to shape the direction Jade may travel and ensure decision-making about her health was as correct as possible.

We always have to allow for a little dramatic licence and I like to think that some of those minor details that only we would notice aren’t as important as the fact we have a character on a major UK soap opera with cystic fibrosis. Sometimes you have to pick your battles!

Since that first meeting, the team have been sending through scripts for us to review. Clearly, I can’t go into too much detail about things not yet broadcast, but just one example of their willingness to listen to our perspective and adapt their story came up in Jade’s very first scene.

In the final moments of last Friday’s episode, she appears wearing what was originally scripted to be an oxygen mask, struggling for breath and making the impact all the greater on her grandmother, Shirley, who had already lost her son to CF (off-screen, some years ago).

In subsequent scripts, the activity they had Jade doing would clearly have been beyond someone who was so ill. She would also, obviously, have needed to tote her oxygen around with her everywhere she went.

We called the writers and explained that by having her in an oxygen mask for that opening scene, they were committing themselves to one very strict direction that didn’t leave them with many options going forward.

While not as immediately recognisable to a lay audience as an oxygen mask, we suggested that a nebuliser may be a better approach for that first entrance, which is exactly what viewers saw when they tuned in on Friday night.

We’re so excited to finally have this kind of exposure for what we all know is – relatively speaking – quite a rare disease. And we’re even more pleased that the BBC have shown such commitment to getting it right, and are willing to listen to our feedback to make Jade’s story as real as possible not just for our eagle-eyed community, but for the great British public who may be learning about CF for the first time.

I, for one, can’t wait to see Jade’s story unfold, and to finally be able to talk more about it as it does!

Monday, 15 June 2015

The Breathe With Me Strawfie Challenge

We've got an amazing dedicated community out there, especially among those of you raising both awareness and funds through not just individual events but also your own great initiatives and campaign.

A recent example of success these past few months has been the Breathe With Me single and Strawfie Challenge, so who better to ask for inspiration and the story behind it, then the team who made it happen.

The Breathe With Me Strawfie Challenge began with a simple idea – to write a song asking people to try experiencing the struggle to breathe that people with CF live with. The way they could experience this was by breathing through a straw while pinching their nose – an exercise that has been used for many years by CF campaigners.

Inspiration struck and ‘Breathe With Me’ was recorded by annaJo, a songstress from Crawley living with cystic fibrosis who insisted on a group name to recognise the joint effort behind the project. The song was billed as ‘Breathe With Me by TWF featuring annaJo’. TWF stands for ‘Together We Fight’. In December 2014 ‘Breathe With Me’ made the semi-finals of the UK Songwriting Contest in both ‘Music Video’ and ‘Lyrics’ categories.

Several months later, it occurred to the ‘Breathe With Me’ team we could use another modern phenomenon to help spread our message, by asking people to take a selfie doing the straw breathing exercise and develop a social media campaign with people nominating their friends to take part. The description ‘straw breathing selfie’ proved a bit of a mouthful and the Strawfie was born!

The first big event we visited with the Strawfie concept was Butserfest, a music festival aimed at teenagers. This resulted in a fantastic opportunity to have ‘Breathe With Me’ played from the main stage while festival-goers took Strawfies.

Since then, the Strawfie Team has attended many events. The Great South Run was a particular highlight as we were featured in Channel 5’s coverage when Joyce Griffin, a mother of three children with CF, was interviewed at the starting line.

Recently, we gained the support of Miss Hampshire, Larissa Hirst, and through her campaigning, several other Miss England contestants have posted their glamorous Strawfies.

So what’s next for the Strawfie Challenge? We would love the CF community to help us get our first big celebrity Strawfie. The CF community is spread far and wide, with many talented individuals in every field. We know some of you must know a celebrity, but who will be the first to ask that person to post a Strawfie?

Interest in the campaign is building all the time and the Cystic Fibrosis Trust has asked us to share our story as part of their #PowerOfUs campaign. We share their belief that working together and supporting each other is where the strength of the CF community lies.

Recently we have begun to receive Strawfies from as far afield as Australia and the USA. The big vision when we started this journey was to involve the worldwide CF community, because together we can shout louder about our cause.

Hopefully Strawfies will keep coming and we need you to keep working to create opportunities to incorporate Strawfies into ANY kind of event or organisation you have an involvement with, particularly non-CF events as the aim is to spread awareness.

Our heartfelt hope is this campaign will greatly raise the profile of CF among the general public, increasing awareness and fundraising efforts. We share the prayer of the entire cystic fibrosis community – that a cure will result from all of our joint efforts and people will never have to experience breathing in a restricted way. In the words of the song “Together we will find a way, I know we’ll win this fight…”

Saturday, 13 June 2015

The Power of Us is Global - CF Week 2015

While we in the UK have been coming together for CF Week 2015 and sharing our stories through the Power Of Us, there has been another important coming together in the fight against cystic fibrosis as the 38th European Cystic Fibrosis Society Conference took place in Brussels, Belgium.

Our Chief Executive Ed Owen, along with colleagues from our Research and Registry teams, attended and joined many from around the world, demonstrating the global effort in beating CF, and here are his highlights of both ECFS and CF Week 2015.

To get to the old Town Hall for the welcoming ceremony of this year’s European Cystic Fibrosis Society conference I had to weave through the many tourists in the cobbled Grand Place in central Brussels.  As I approached the entrance, a smiling woman bounded up to me to say hello.

“Thank you so much for joining us,” she said.  “It’s a pleasure,” I replied politely and, assuming she was part of the welcoming group, told her how delighted I was to be in Brussels with so many others committed to the fight against cystic fibrosis.

She looked at me quizzically and rather disappointed.  “I am very sorry,” she said, “I thought you were here to join the Tintin tour.”

Like Tintin, created by Herge, one of Brussels’ most famous sons, we in the cystic fibrosis community are on a great adventure to beat a cruel and common villain.  Yet ours is not a work of fiction but a real story of a thousands of people living with a condition that shortens life and limits freedoms and opportunities.

This week saw two different aspects of how that fight is slowly being won.

Here in Brussels, the finest scientists, clinicians, business people and advocates, came together from across the world to share knowledge, ideas and plans.  It is an awesome group working on a range of research projects, trials, innovations and initiatives to beat cystic fibrosis – from tackling new and old bugs to understanding the psychological impact of the condition, to developing breakthrough physiotherapy techniques to correcting the basic genetic defect.

But this global research effort cannot beat cystic fibrosis alone.  It can only do so with the active support and engagement of the wider cystic fibrosis community making its voice heard.  It is the stories and insight of those living with the condition and their carers, together with the collective expression of all supporters, that is helping to change the world of cystic fibrosis.

That is what CF Week in the UK was all about over the last few days, and we have heard some extraordinary insights, from Hannah’s blog onMumsnet to Umar’s story on YouTube. By speaking out, telling our stories, we are helping to make a real difference and demonstrating the extraordinary “Power of Us”.

As Tintin found, the struggle to beat a determined foe is rarely easy.  But, like the intrepid boy reporter too, we in the cystic fibrosis community – bringing together the finest scientific minds, dedicated clinical teams and all those living with cystic fibrosis every day – will win out in the end.

Wednesday, 10 June 2015

'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.

Tuesday, 9 June 2015

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!

Monday, 8 June 2015

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 40th, 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).