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.

CF in Scotland: SCooP Update

Fresh from speaking at the launch for the Sixty Five Roses Club Scotland, Dr Emma France provides an update on SCooP, the project she is leading to help parents of young children with cystic fibrosis meet their child’s physiotherapy needs. The project is part-funded by the Cystic Fibrosis Trust.

I lead the SCooP (Supporting Children’s Physiotherapy for cystic fibrosis) project, an innovative and exciting project involving a team of researchers from across Scotland, England, and even Australia. Supporters of the Cystic Fibrosis Trust helped to name the project byvoting for their favourite title in an online poll. We have been working with 20 families over the past eight months to create a short film for families who have young children with cystic fibrosis from 0 to 8 years old. The film will encourage and support other families to stick to their child’s chest physiotherapy routine.

Home chest physiotherapy is a major part of the daily treatment to clear the lungs and airways of the thick mucus which can lead to infection. Parents, carers and their young children with cystic fibrosis find it difficult to stick to their home physiotherapy routine for a wide range of reasons. But lots of families have come up with their own ways around the problems they have keeping to their child’s physiotherapy routine. For example, parents told us they visualised the build-up and clearance of mucus to motivate themselves. We are drawing on the expertise and experiences of families to develop the film.

We have just finished filming with real families living in Scotland who have a child or children with cystic fibrosis. The film will show the families telling their stories about having a child with CF, doing home chest physiotherapy with them, and showing us some of the solutions that they have found to help them do physiotherapy.

Now we need 20 families from across the UK with children aged between 0–8 with cystic fibrosis to try out the film. See: http://www.stir.ac.uk/scoop/ for more details.

The 20-month SCooP project started in May 2014 and is funded by the Cystic Fibrosis Trust and the Chief Scientist Office of the Scottish Government. Emma France is based at the NMAHP Research Unit, University of Stirling, Scotland.

Why We Provide Financial Support

Trust Chief Executive Ed Owen talks about why the Trust offers grants to help those with cysticfibrosis most in need.

I am sometimes asked by supporters why the Trust chooses to provide financial grants to people with cystic fibrosis and their families when this diverts money from vital research and care.

It’s an important question, especially from those whose efforts help raise the funds we rely on to carry out our vital work – and one that I am pleased to address as we revamp the way we issue health and wellbeing grants.

I am very clear that supporting those with cystic fibrosis in most need with appropriate, focused help is the right thing for us to do. Whether it is the young person starting out on their own for the first time who needs help to buy a fridge essential for medication, or support for a hard-pressed family to purchase a trampoline to keep their child fit and active, we can make a real and positive difference to people’s lives in this way.

I am also proud that the Trust can provide opportunities to people with cystic fibrosis living on low incomes to take a holiday break which they otherwise could not afford – and to provide funeral grants of £750 to the families of those who have lost their lives to this cruel condition.

We cannot and do not, however, run a benefits service to provide ongoing support for people out of work or unable to work. This is the job of government and the Trust campaigns to ensure all people with cystic fibrosis get appropriate support.

We also do not provide support for clinical equipment such as fast nebulisers. These should be provided by the NHS and the Trust must never become a means by which health providers avoid obligations they have to provide appropriate healthcare to people with cystic fibrosis.

And many particular services required by those affected by cystic fibrosis are best provided by other organisations like the Citizens Advice Bureau. So, through our advice helpline and website, we point families and individuals in their direction.

It is vital that the financial support we do provide is focused on those in most need – and so we have changed the application system to put a particular emphasis on those on low incomes. It is also important that as many people as possible can access the scheme. So we are working closely with CF clinics to promote the grants system to those who might benefit.

Finally, it is essential that the decision on whether a grant is awarded or not is taken fairly and clearly.  So a new grants panel, chaired by Peter Sharp, a former trustee of the Cystic Fibrosis Trust and someone living with cystic fibrosis himself, and including experts in CF and welfare, assesses all health and wellbeing applications.

We are currently spending about 1.5% of the Trust’s income on financial support for people with cystic fibrosis – and it represents about a 20th of our research budget.

But, at a time of welfare cuts and austerity, the system provides an important support lifeline for people with cystic fibrosis and their families who struggle to get by. I am clear that this is the right thing for us to do, and I am hugely grateful for the work of all our supporters that enable us to fund it.

Health & Wellbeing Grants: The Social Worker Perspective

Today the Trust’s grants panel will consider applications for the second round of health and wellbeing grants. In this blog, Anne Calvert and Margaret McCafferty, two specialist CF social workers from Belfast, discuss some of the financial issues that people living with cystic fibrosis can face, and the importance of the Trust’s grants in supporting people during different stages in their lives.

We are from the regional adult CF centre in Belfast, covering the whole of Northern Ireland.  Individuals with CF travel long distances to attend outpatient clinics. Many are not entitled to help via the benefit system and it is costly having to travel so frequently. When someone comes into hospital they are usually admitted for a two-week period and contact with family and friends is extremely important during this time. Families not only incur the additional cost of travelling to and from hospital, but they are also spending money on meals, trying to encourage the patient to eat and supplementing hospital food. 

The first home grant has been extremely helpful to those wishing to set up independently. The availability of small emergency grants is fantastic when, for example, the washing machine breaks down! 

We have also found these grants very helpful when someone needs a one-off payment to help with unexpected costs. Many people attending our unit are parents themselves now. When they have to come into hospital they are extremely worried about the increased cost of child care. We have successfully applied for financial help to meet these costs, which goes a long way to help reduce some of the understandable anxieties for people coming into hospital. We also recently applied for a one-off grant to support someone paying for a professional cleaning service. They were going through a difficult time and felt overwhelmed by the situation. They really appreciated this help and felt better able to “keep on top “of things.

Looking forward to a holiday gives all of us a lift! The holiday grants are very important, especially as insurance costs can be very expensive. It is extremely important that people can go on holiday with peace of mind, knowing they can access medical care if they need to.

When health deteriorates the home care grant has been of significant benefit, especially when someone wants to spend as much time at home as possible. It is important they are not worried about the increased costs such as heating during this difficult time.

Individuals in Northern Ireland go to Newcastle, England, for assessment for transplantation. This is an expensive process for them and the family member who accompanies them. The grant for the assessment period has been very helpful. 

Also, for those families where they have lost someone with CF, they are really appreciative of the £750 grant to help towards funeral costs.  

Our Financial Support for the CF Community

As the Cystic Fibrosis Trust’s grants panel gets ready to consider applications for the second round of health and wellbeing grants, Support Service Manager Becky Kilgariff discusses why the Trust offers grants, and the kinds of financial assistance available.

Sometimes we are asked: “Why does the Cystic Fibrosis Trust offer grants to people and families affected by CF?” The short answer is: “Because they are needed”. Now, in an era of austerity cuts and benefit reform, we know they are needed more than ever. Our Policy and Public Affairs teams continue to campaign for the rights of people affected by CF and their carers and families, but in the current climate, our grants programme is there to help ease the financial burden.

We offer a variety of different grants to meet the needs of the CF community.

Our emergency grants can help at short notice – they are usually paid within a week of the application being received. We offer funeral grants of £750 following the death of someone with CF, hopefully easing some of the worry for families at a very difficult time. We can provide a grant of £250 for those undergoing a transplant or transplant assessment, which can bring unexpected costs. This could perhaps be travel costs for getting to the transplant centre, or for a family member to stay nearby, and these can be applied for up to two months after the costs have been incurred.

We also offer small emergency grants of up to £150 for other unexpected costs or emergencies. This can include urgent household repairs or cleaning to remove damp or mould, or replacement hypoallergenic bedding. We can also fund the first prescription pre-payment certificate for people unable to meet this cost, although we continue to campaign for prescription charges to be abolished for people with cystic fibrosis. Thanks to a generous gift from the Edward W Joseph Trust, we’re also able to offer grants of £450 to promote increased care in the home, rather than hospital, particularly at end of life. Our advice to anyone who needs an emergency grant is to contact our Helpline on 0300 373 1000 or helpline@cysticfibrosis.org.uk.

  

Our programme of health and wellbeing grants of up to £350 for goods or services is designed to help improve the health and quality of life of people with cystic fibrosis. These grants are considered by a panel every two months, and the next panel meeting takes place on Tuesday 13 April. In the past, the panel has awarded grants for gym fees and exercise equipment, fridge freezers and washing machines.

Finally, we also fund holidays for people with cystic fibrosis aged over 25s. From this week onwards, these are administered by the Cystic Fibrosis Holiday Fund – you can apply online or get in touch with them for an application form.

I hope that one day we won’t need to offer grants for people with cystic fibrosis – beating CF for good would mean an end to the financial pressures CF can bring. Until then we will continue to provide financial support where it is most needed through our grants.

Taking on the Atlantic

Later today teams from the universities of Oxford and Cambridge will do battle in the annual Boat Race, televised around the world. But away from the cameras, two groups of friends have been training hard for an even bigger challenge – the Talisker Whisky Atlantic Challenge – to raise money for the Cystic Fibrosis Trust. At 3,000 nautical miles, it’s the equivalent of 821 boat races, non-stop. James Timbs-Harrison from the All Beans No Monkeys team offers an update on the team’s progress.

The All Beans No Monkeys team stepped up our training to a new level over the Easter weekend, rowing our boat from Christchurch over to Cowes on the Isle of Wight.
It was a real challenge at this stage in our training with tough weather conditions, some reasonable sized waves and lots of wind. We tested out a lot of the equipment we will use on the Atlantic row and got to understand how the continuous rowing and resting regime will work on our bodies.
The weekend tied in nicely to other rowing events across the UK including the head of the river race, which the team have taken part in whilst at university and the upcoming Oxford/Cambridge boat race.

The weekend row created a lot of interest from members of the public and we have already had donations made just from people seeing the boat. The team hopes to raise £60,000 for our chosen charity, the Cystic Fibrosis Trust.

Find out more about and sponsor All Beans No Monkeys.

What to know what the challenge is like in numbers? Check out our infographic below:

Singing and Cystic Fibrosis

As Bianca Nicholas gears up to hit the stage for Eurovision 2015, composer Gareth Williams discusses Breath Cycle, a community singing project he developed at Scottish Opera to explore the impact of singing on cystic fibrosis. For Breath Cycle Gareth collaborated with the respiratory ward at Gartnavel Hospital in Glasgow, and worked with a number of people with cystic fibrosis.

“The exercise of singing is delightfull to Nature, & good to preserve the health of Man….
It doth strengthen all parts of the brest, & doth open the pipes.”

These are two of many reasons given by the English Renaissance composer William Byrd (born c.1539) as to why we should all sing. The idea that singing is beneficial to our health and wellbeing has clearly been around for a while, and Breath Cycle was the beginning of our journey to finding out more.

The findings from the pilot project were very encouraging and so over the next few years our plan is to open the Breath Cycle project up across several CF centres and take as many participants through the process of learning to sing as we can.

Before Breath Cycle, I had spent years working with opera singers. They are the professional athletes of singing and breathing – their voices can fill huge auditoriums, carry across full orchestras, and even shatter glass (so the story goes).

I see so many parallels in the daily routines of the opera singers I know and the participants with CF that I worked with. They both have daily rituals, exercises, regimes and routines to keep their instrument/their lungs in good working order. Opera singers work hard so that they can perform, and people with CF so that they can continue to live their lives on their own terms.

When fragility turns up on the opera stage, it is not as we think of it – Violetta at the end of La Traviata, or Mimi at the close of La Boheme, are both characters with chronic lung conditions who nevertheless seem to have the lung capacity to stun a fully grown elephant with a well-aimed top C.

Through Breath Cycle, I came to the conclusion that singing doesn’t have to be fireworks and dazzling displays of pitch-perfect spiralling to be engaging, meaningful and powerful, because it is, at its core, the act of breathing beautifully. 

Listening to the right singer with the right song moves us more than anything else can, because underneath the melodies and the counter-melodies, the harmonies and the rhythms, is a reminder to us all of what we are and what sustains us. Breath.

Here’s a song from Breath Cycle that David Brock and I created for five people with cystic fibrosis. It was recorded and assembled in a recording studio in Glasgow and sung beautifully by Breath Cycle participants. Of all the performances of my music I’ve witnessed, this is the one I am most proud of:  https://youtu.be/vPrZ-aF7Clk.

I might argue that there is only one thing more wondrous than the act of singing, and that is the act of singing together, and this is my aspiration – that people with CF can meet online and sing with one another. There are songs to be written, the technology exists – we just need to find the singers!

In the words of the composer William Byrd: “Since Singing is so good a thing, I wish all men would learn to sing.”

For information, visit www.breathcycle.com.

Making Travel Easier

Our Public Affairs team at the Trust are looking at how people with cystic fibrosis are affected when travelling and in particularly when getting travel insurance to go abroad. Lynsey Beswick, Public Affairs Officer for England, talks more about our work and how CF affects people like her. 

It’s Easter holidays and a time when many people will taken the opportunity to dig out their passport and air those beach towels in preparation for a holiday break. Most people take for granted the ease with which they jump on a plane and fly to far away shores.

However, for many people with cystic fibrosis, myself included, this is not always the case. Finding travel insurance can be a daunting task: insurance cover can be hard to find, the questions are not always relevant and quotes can be ridiculously expensive. 

Then there are the additional costs to consider. Most airlines require a fit to fly test and will charge for oxygen during flights. With numerous medications, travelling light is not an option and extra baggage is required incurring additional charges. 

The Cystic Fibrosis Trust is on a mission to reduce these barriers and make it easier for people with cystic fibrosis and their families to travel. 

We believe it is wrong that it can take hours to find appropriate travel insurance and people with cystic fibrosis often have no choice but premiums that cost hundreds of pounds. As a result many people are forced to travel without insurance or even stay at home. 

The Trust aims to ensure that the industry must have a better understanding of the condition. We have been working with travel insurance companies to help develop a new model of risk specifically for cystic fibrosis, but we need your help to make sure we get it right. 

 If you have cystic fibrosis or are a parent of a child with CF, we are keen to hear from you. Please help us by sharing your experience of travel insurance through our short online survey. 

Over the coming months we will also be challenging the airlines and improving the information we provide to make it easier for you to travel. We firmly believe that everyone deserves a holiday, especially people with cystic fibrosis and their families.

If you would like to be further involved with our travel campaign, as well as completing our survey you can also contact me directly at lynsey.beswick@cysticfibrosis.org.uk.

Things I learned on a summer studentship

Kate Ryan, an undergraduate medical student at Keele University, took part in the Cystic Fibrosis Trust’s Summer Studentships programme in 2014, where she worked on a PhD project on cystic fibrosis-related diabetes. Following her placement in the academic holidays, Kate won first prize for her poster presentation at the UK CF Conference.

Initially I wanted to complete a studentship over the summer as I have been interested in research ever since my first year of medical school. I gained a lot from participating in a summer project funded by the Cystic Fibrosis Trust with regards to lab skills, working as part of a team and becoming more familiar with the process of research; from planning, right through to results and presentation.

This particular project allowed me to present at my first conference which was a great experience for someone considering a career in research and helped me recognise areas I did well in as well as those I could have improved on. I also got the opportunity to meet some of the top names in cystic fibrosis research and to attend workshops about exciting new treatment options. 

Although I have had some experience in medical school, working in a lab was a relatively new experience for me. I was taught about general lab safety as well as the relevant techniques needed for my project. This hands-on approach to research was the part I found most enjoyable.

This research project has led me to consider intercalating next year and possibly considering further research in my career. It has helped me realise the importance of research and how enjoyable it can be.

I would highly recommend this to any medical student, whether you are interested in a career in research or not.

Thanks to the Cystic Fibrosis Trust for such a great opportunity last summer!

Find out more about the studentships programme at cysticfibrosis.org.uk/studentships.

Understanding the VX-661 Phase 2b Results

Pharmaceutical company Vertex, today released the results of a recent Phase 2b trial, which looked at safety of a drug known as VX-661 in combination with ivacaftor (Kalydeco). There has been a lot of anticipation about these results because it is only the second time that the company has issued trial data. Below, Dr Anoushka de Almeida-Carragher, Senior Research Manager looks a what they tell us.

This study involved 39 people with cystic fibrosis who have two copies of the F508del mutation, and confirmed that the treatment is safe and well tolerated.  The stated improvement in lung function, from baseline, within four weeks of treatment is also encouraging. It should be noted that the number of people who took part in the study is extremely small. However the results provide solid support for the next stage of trials – Phase 3 – which Vertex commenced in the early part of this year.

This program of four Phase 3 clinical trials will look at lung function after the administration of the VX-661/ivacaftor combination, but this time involves not only people with two copies of the F508del mutation – who were studied in the phase 2b trial, and make up roughly 51% of the CF population – but also includes people who have one copy of the F508del mutation and a second mutation that is either a gating mutation, residual function mutation or a mutation that results in minimal CFTR function.

This will the first time that the efficacy of this drug regime is being tested in a large population of people with cystic fibrosis (approximately 1150, aged 12 and older). Furthermore, the fact that 4 different ‘mutation combinations’ are being investigated, thus impacting on a larger number of CF patients, means this next-stage trial is vital in enhancing our knowledge-base in striving to reach our goal of beating CF for good.

The Trust will be watching the Phase 3 trials with interest and ensure that our CF community remain updated on the progress in transformational treatments.

Working with Pharma. Ed Owen's US blog part 2

As well as meeting the Cystic Fibrosis Foundation in the US last week, the Trust’s Chief Executive, Ed Owen, and Director of Research & Care, Dr Janet Allen, also dropped in on two pharmaceutical companies of importance to cystic fibrosis, Novartis and Vertex. Here is Ed’s second blog explaining what they find out:

Much is said about the pharmaceutical industry. Some of it is fair, some less so. But the fact remains that only pharma companies have the size, expertise and capability to develop and produce the drugs that can make a difference to people with cystic fibrosis. So it is vital that we have strong relationships with key companies, like Vertex and Novartis, and work with them for the benefit of our community. 

The Vertex story is an extraordinary one, and one in which our sister charity, the Cystic Fibrosis Foundation (CFF), has played a central role. It also tells us much about the industry, its weaknesses and strengths, limitations and potential.

Long frustrated by the lack of industry interest in cystic fibrosis in the late 1990s and early 2000s, the CFF began funding a number of commercial programmes, including one led by a biotech company called Aurora Sciences, later bought by Vertex, to find compounds to develop drugs to tackle the genetic causes of cystic fibrosis.

A decade and a half on, and Vertex is a pharmaceutical trailblazer. Ivacaftor (Kalydeco), is transforming the lives of those with the G551D mutation and a combination therapy of ivacaftor and lumacaftor has been submitted for approval for use in people with two copies of the F508del mutation.

Being a trailblazer however comes at a price, and the high price of ivacaftor has been controversial – with many questions about the likely price of the combination therapy if it is granted approval from the regulators.

With a cash-strapped NHS, and a system of drug appraisal that in England, at least, is ill-suited to assess particular issues relevant to conditions like cystic fibrosis, there is a real risk that this combination therapy is given regulatory approval in Europe later this year for use in four out of 10 people with cystic fibrosis but will not reach those that need it here in the UK for a considerable amount of time, if at all.

Our focus must be to ensure that approved therapies get to those who need them as quickly as possible - and we are discussing these issues now with all key players.

But, when we met them last week, Vertex continued to be tight-lipped about future pricing in advance of regulatory approval. Along with other pharmaceutical companies, the company argues that the price of drugs reflects both the high cost of investment that has been made over many years to develop and trial therapies (estimates vary, but the average cost of producing a new drug is usually well above £1bn) and the relatively small numbers of patients with ‘rare diseases’ like cystic fibrosis.

Like every other pharmaceutical company, Vertex has shareholders who demand an appropriate financial return on their investment. The long-term prospects of the cystic fibrosis ‘market’ means that Vertex is continuing to invest in both their immediate pipeline of potential therapies and in longer-term research to develop what they describe as ‘second generation’ drugs that they believe could be even more effective in future years.

We are delighted they are doing so, and are keen to see other drugs companies invest in cystic fibrosis research too. One such company is Novartis. It already produces cystic fibrosis drugs, most commonly Tobramycin. But we have had concerns about its future commitment following its decision to move its wider respiratory research base from Sussex in the UK to Boston in the US in late 2013.

We were therefore pleased to meet the Novartis team in Boston last week and to receive their reassurance that their cystic fibrosis-related drug programmes were alive and well.

For the first time in decades, the pharmaceutical industry is investing heavily in cystic fibrosis. As President Obama said in January, the condition is at the cutting edge of new advances in ‘precision medicine’ with new therapies being developed focused on defined patient groups with particular genetic mutations.

That investment will only continue for as long as companies believe that health systems will pay for the drugs that follow. Therein lies the challenge for the NHS, ourselves and all those wishing to see innovative treatments that transform the lives of people with cystic fibrosis accelerated as quickly as possible.

High-cost drugs are the future. How they are paid for is the key question that we all need to address. 

My successful SRC grant application

As the Cystic Fibrosis Trust announces a call for applications to form up to three more Strategic Research Centres, Dr David Sheppard of the University of Bristol explains how he went about submitting a successful SRC application.

As a scientist based at a UK university, a significant part of my time is spent applying for research funding. This funding provides salaries and bursaries for the research fellows and students that I have the privilege to work with. These highly creative individuals labour for long hours in the lab to understand better how the cystic fibrosis transmembrane conductance regulator (CFTR) works, how it goes wrong in cystic fibrosis (CF) and how drugs repair the faulty CFTR. Funding also covers the cost of all the “stuff” required to do experiments in the lab. In our case, this involves some very expensive equipment needed to monitor CFTR activity as tiny electrical currents, paraphernalia to grow cells and a battery of chemicals to stimulate CFTR and manipulate its activity. Simply put, without funding there is no research.

With my research focusing on CFTR, the CF Trust is the major funder of our work. I’m extremely grateful to the Trust for the funding that I have received over the last 20 years. The output of our research has included knowledge of how gating mutations (eg, G551D) interfere with CFTR function and the action of chemicals that act as CFTR potentiators. This information has helped the development of transformational drugs that treat the root cause of CF.

When the CF Trust announced its new research strategy in April 2013, I began to think about how to apply for a Strategic Research Centre (SRC) grant from the Trust. SRCs are virtual research centres of excellence that bring together research groups from the UK and overseas to tackle a fundamental problem in CF.

What problem to address? Great progress has been made in our understanding of F508del, the most common CF mutation, leading to clinical trials of CFTR correctors and potentiators. While very encouraging, data from the lab and clinic argue that better drugs are required to fully rescue F508del-CFTR. By focusing on F508del-CFTR, the SRC would have the potential to benefit the largest number of individuals living with CF.

Today, most research is multidisciplinary, meaning that methods from different research disciplines (eg, biochemistry and physiology) are used together to solve specific questions. While some labs successfully apply different research methods, many specialise. My own work is very highly specialised, focusing on how individual CFTR proteins work. To apply successfully for SRC funding, we would need to collaborate actively with other groups expert in studying CFTR structure, how it is made inside cells and delivered to its correct cellular location. We would also need help from pharmacologists expert in testing large numbers of chemicals for drug action.

Early in planning the application, I approached Professor Bob Ford from the University of Manchester. Bob has been at the forefront of efforts to identify the structure of CFTR. This work is fundamental to understand CFTR and its dysfunction in CF. I also approached Professor Ineke Braakman from Utrecht University in The Netherlands. Ineke and I had previously made plans to study how CFTR matures inside cells to become a gated pathway for chloride movement. When compared with other methods to study CFTR activity, those used by my Bristol colleagues are notoriously slow. Two groups studying CFTR activity would therefore be advantageous for an SRC application. Because of her expertise in studying how CFTR flutters open and closed, I approached Dr Paola Vergani at University College London. I was delighted that Bob, Ineke and Paola all readily agreed to participate in the SRC application.

Plans for the SRC were finalised at a conference in Malta organised by the European CF Society last March. Because the meeting was so intense, it was not until the day after the meeting that Bob and I finally found time to discuss, in detail, plans for the SRC, especially how the different project partners would work together to address our research goals. During these discussions, we recognised that the SRC would require a group expert in the pharmacology of CFTR. With his wide-ranging expertise in studies of drugs targeting CFTR, we both agreed that Professor Frédéric Becq from Poitiers University in France was an excellent choice. Returning to Bristol, I telephoned Frédéric to invite him to join; he agreed immediately. With the team recruited, we then set about writing the application. The initial application went smoothly, but the full application involved a lot more work; not for the first time lots of late nights and early mornings close to the deadline!

Bob, Ineke, Paola, Frédéric and I were delighted to learn that our SRC application was successful. We are excited about the studies of F508del-CFTR that we have planned. We believe strongly that they will inform the development of new drugs to benefit the majority of individuals living with CF.

Back from Boston & Bethesda

CEO Ed Owen and Director of Research Dr Janet Allen spent the first half of this week in the US, meeting with colleagues from across the American cystic fibrosis community. Ed fill us in on some of the highlights from meeting with our US counterparts.
Weeks of sub-zero temperatures and Arctic-like weather have left much of the North Eastern coastline of the US encased in ice and snow. Yet our visit this week to see Vertex and Novartis in Boston, and the Cystic Fibrosis Foundation (CFF) in Bethesda, demonstrated that the work to beat cystic fibrosis continues to generate a great deal of heat and light.

What we heard from all those we saw gives me renewed confidence about the future prospects of research. But, while competition in the pharmaceutical industry is an important driver in pushing the development of new drugs, this needs to be matched by new forms of international collaboration and partnership that pools effort and resource in the best interests of people with cystic fibrosis.

Our strong relationship with the CFF is crucial here. We have shared goals, represent communities of people who are themselves increasingly interconnected, and both have access to key clinical, academic and business expertise. We can bring this together on tackling the big issues facing people with cystic fibrosis so the potential for greater and quicker advances becomes a reality.

The Foundation’s success in helping to develop and promote new therapies in cystic fibrosis has been extraordinary.  We in the UK owe a great debt to them – and their $3.3bn windfall late last year resulting from the selling of their Kalydeco royalty rights gives them significant new resource to invest in new research to bring hope to everyone with cystic fibrosis wherever they live.

But, as the Foundation will readily admit, they do not have a monopoly on wisdom or expertise – and are keen, too, to explore areas of collaboration with the Cystic Fibrosis Trust in particular areas that can bring impact.

At our meeting, we therefore discussed a range of areas of potential joint research activity. One was tackling the challenges posed by what are called ‘nonsense mutations’ of cystic fibrosis, and developing novel treatments that would benefit around 1,000 people here in the UK alone. The existing pipeline of small molecule therapies being developed by Vertex and other pharmaceutical companies would not affect this group so it is an important area for ourselves and the Foundation.

A further area we explored was transformational work in gene therapy and stem cell research. The UK has particular expertise here, not least that generated by the Gene Therapy Consortium, supported by the Trust over many years and soon to publish the results of its Phase 2b study. In stem cells too, the UK has a strong scientific base and we are keen to see more of this focused on cystic fibrosis over the coming years.

We also discussed joint approaches to tackling emerging infection threats to people with cystic fibrosis such as NTM and aspergillus. The CFF is supportive of the Strategic Research Centre on NTM being funded by the Trust, and keen to follow its progress.

Finally, we talked about our SmartCareCF programme and working together to harness new technology to give people with cystic fibrosis the tools to take greater control of their lives, as well as increasing access to clinical trials here in the UK.

I believe that in these and other areas we can demonstrate real collaboration over 2015 to bring together the greatest possible expertise and resource in our collective fight to beat cystic fibrosis for good

Moving Mountains - “Making the impossible possible with exercise”

Last Friday saw Churchill College, University of Cambridge, host the Moving Mountains conference on how sport and exercise can benefit those with long-term health conditions, such as cystic fibrosis.

Our own, Paul Rymer, Head of Principal Involvement attended from the Trust and has given us a flavour of the informative, interesting and exciting events there
In the next year fitness and sport will be an increasing focus for the Trust. Last week I attended a conference highlighting the benefits of exercise, including better mental health, less reliance on drugs, and increased feelings of confidence and social inclusion.

The event was organised by Dr Jonathan King, who has CF, in partnership with others living with chronic conditions.

Jonathan used a slide presentation and props (bottles of water) to engagingly describe lung function, his treatment regime, what happened when things went badly wrong during a holiday, and then the months of recovery. With persistence, and support from his care team, Jonathan managed to avoid a lung transplant by gradually improving his lung function though exercise. At first he could barely move, but over a period of months he gradually increased his physical activity, from walking a few steps, to trying a treadmill, to jogging and then to running 4K. Jonathan now uses regular exercise to maintain his health. His next endeavour is to cycle 1,200 miles from London to Barcelona in support of the Cystic Fibrosis Trust.

Nick Talbot gave an extremely funny account first of his experience as a young man with CF, including dating, relationships and work. He then shared his experiences climbing mountains in the Himalayas, and the challenges of doing that with cystic fibrosis. Where Jonathan’s story was one of survival against the odds, Nick’s was an example of someone not accepting the perceived limitations of cystic fibrosis. Nick is aiming to climb Everest in support of the Trust in April–May.

Charlotte Wells, a specialist respiratory physiotherapist, talked engagingly about her experience working with CF patients, and the benefits of exercise from a clinical perspective.

Because of the risk of cross-infection, Jonathan spoke before a break time, and stayed in one area of the large venue, while Nick arrived later on, and aside from when he was speaking, remained at the back of the audience. At one point they were both in the venue, but at the widest possible distance (and it was a very large theatre). They then spoke through microphones asking each other questions. This really brought home one of the saddest aspects of living with cystic fibrosis – people unable to meet in person and share ideas, jokes and experiences.

Following the conference there was a drinks reception with special guest of honour Professor Stephen Hawking, who kindly posed with Nick and a Trust banner. In a short speech, Professor Hawking said: “You have to live life to the full. I am very aware of the preciousness of time, act now however difficult life may be there is always something you can do, be brave overcome the odds, it can be done.”.

Learn more about the Moving Mountains conference.

If you have an interest in exercise, fitness or sport and would like to join me in exploring how the Trust can do more in this area, please email me at paul.rymer@cysticfibrosis.org.uk.