Friday, 24 July 2015

Welcome to Hack It Up!

Yesterday, we launched Hack It Up!, our new online colloboration and innovation platform designed to bring the CF community together so we can all work on the challenges cystic fibrosis brings.

Today our Community Manager, Emma Lake, who will be acting as the facilitator on Hack It Up! introduces herself and the platform



Hi, I’m Emma, the facilitator at Hack It Up! I have cystic fibrosis and I believe passionately that everyone with the condition and those who help and support us, both at home and in hospital, should help shape the future, through the discussions and decisions that affect our lives.

Our insight and experience is uniquely valuable, we are the ones who are truly experts in living with cystic fibrosis and we need to drive change, delivering impact based on our needs and in our interests.

That’s why I’m thrilled to be a part of Hack it Up, an innovative platform to make our voices heard on the issues that matter.

I am really excited about the first two challenges we have to discuss, and can’t wait to start exploring ideas and sharing our collective expertise.


In the first challenge, we want to find out the tricks and techniques people have developed to manage their CF while still living the life they want to lead.


Together with people with CF, healthcare professionals and industry, the Cystic FibrosisTrust is developing a project called SmartCareCF, to find better, smarter ways of managing care. Before we look ahead though, we need to look at what’s happening right now. What works for you? How do you use technology to keep one step ahead?-


At the same time, we are exploring how being active can help keep lungs healthy for longer. In our second challenge we want to know what activity means to you. What do you do to try and stay healthy? How does it make you feel?


So, get stuck in! Join us in Hack it Up and give us your views and share your experience– there’s a whole community waiting to listen, to learn and to connect.


Once you’ve joined, please invite other people you know who understand life with cystic fibrosis, such as parents, partners, friends, employers and colleagues. I’ve already invited my mum, my husband and my best friend!



Together, we can change the world of cystic fibrosis and shape a better tomorrow.  We just have to get stuck in!

This is an edited version of a blog post from Hack It Up! Join the conversation at www.hackitup.org

Tuesday, 21 July 2015

The Drop in Donors: What the organ donation statistics tell us

Yesterday NHSBT published figures revealing the first overall drop in organ donation in the UK for more than a decade. Public Affairs Manager Darren O’ Keefe talks about what these figures tell us about soft opt-out, public attitudes and the need to talk to your family and friends about your wishes.

New statistics released by NHS Blood and Transplant yesterday for 2014/15 show a 5% decrease on organ donation across the UK since 2013/14. This is the first decrease in 11 years, showing there is no room for complacency when it comes to ensuring as many people as possible receive this amazing gift of life.

Interestingly, the only nation that has not shown a decrease in donations is Wales, where the move to soft opt-out for organ donation in December this year has been preceded by years of high-profile debate about organ donation. The lesson here is that we must keep people talking about organ donation and sharing their wishes to donate with their families. Especially given that figures show family consent rates are below 60% and families are much more likely to agree to donation going ahead if they know it is what their loved one wanted.

The subject of soft opt-out stimulates conversation and it’s a policy we support. We are encouraged to see Wales take the lead on it and Northern Ireland and Scotland exploring legislation on the issue. However, it is not acceptable to "wait and see" what happens in Wales; this will take years – years that the many people with cystic fibrosis and other conditions across the UK waiting for a donor just don't have. This is why we will continue to support the soft opt-out legislation in Northern Ireland and Scotland, as well as urging England to explore the issue as well.


The Cystic Fibrosis Trust is working tirelessly to ensure that people with cystic fibrosis are able to receive the lung transplant they may come to depend on. We have made a great deal of progress through the ‘Hope for More’ campaign to ensure the most is made of the donated lungs that are already available. This work risks being undermined if donation rates fall, so please sign up to the organ donation register if you haven’t already, make sure you share your decision, and encourage others to the do the same.

Organ donation features in tonight's BBC Two documentary on Great Ormond Street. Focusing on children who need double lung-transplants, tonight's epidsode those with cystic fibrosis. Watch it live on BBC Two, 9pm or catch up on BBCiPlayer

Friday, 10 July 2015

What the DLA ruling means for CF

Policy Manager Nick Medhurst takes us through Wednesday's landmark ruling on DLA and why it matters for people with cystic fibrosis.

On Wednesday the Supreme Court ruled that the government’s decision to stop Cameron Mathieson’s Disability Living Allowance (DLA) payments, as he lay in the hospital where he would eventually pass away, was unlawful,  with judges labelling it “grossly unfair”.

The government had argued that they were “paying twice” for Cameron’s care.

Cameron had cystic fibrosis, Duchenne muscular dystrophy and other long-term health issues, and passed away in 2012 at the age of five, having spent over two years in hospital.

Cameron’s family continued to fight an injustice that hit them in their darkest hours, on behalf of everyone who has and will face the same situation.

The case revolves around a government directive, which demands that an individual’s benefits are stopped after they spend over 12 consecutive weeks in hospital. This is apparently “to prevent double provision” – in other words, the government don’t believe they should have to support a family financially, if they are already picking up the medical bill in hospital.

This fundamental misunderstanding – that an individual’s or a family’s need for financial support vanishes as soon as you step on to hospital grounds – is obviously wrong, something supported by research, conducted by Contact a Family and The Children’s Trust with families who found themselves in the same circumstances:

  • 99% said they provide more or the same level of care when their child is in hospital compared to when at home.
  • 93% said that their costs relating to their child's disability increase when their child is in hospital.
Increased travel, accommodation and food costs are simple examples but the reality is more complex and many families will find themselves significantly harder-up in such periods.

So Wednesday’s ruling is a very important victory in the fight to recognise the true costs of living with a long-term health condition or caring for someone with one.

Cameron’s father, Craig, told the BBC that the ruling “is a fantastic legacy for Cameron.” And its ramifications could be hugely significant for us as a community.

Cameron Mathieson was very young and very unwell when he passed away but his short life and his family’s courage to take forward the fight in his honour, struck a blow for everyone living with a long-term health condition who says that life is worth more than the cost of medical care.; We, as a society, must work hard to remove barriers to leading a happy and fulfilling life.

So when we hear nameless DWP spokespeople telling us that:

"Up to now, DLA has been suspended when a child is in receipt of long-term NHS inpatient care in order to prevent double provision - the taxpayer paying twice for the same thing. This has been the case for more than 20 years"

– we can say they were wrong and  that was 20 years too long, denying the basic support that can go some way to meeting an individual’s or a family’s non-medical needs, often when it was needed most. Their careless and miserly policy-making caused unnecessary pain and hardship.

For many people with cystic fibrosis – children and adults – welfare support is a vital lifeline, enabling work, socialising, education, exercise and physically getting to clinic. Cameron Mathieson’s legacy for the CF community is a renewed strength and resolve to tell society that we can turn a little basic support into much more and we can achieve great things.


For information on benefits, contact our helpline on 0300 373 1000.

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.