Ed Nash-Steer's Marathon Challenge - blog three

Ed Nash-Steer’s niece Hannah was diagnosed with cystic fibrosis four years ago, and seeing what people with CF go through everyday motivated him to start fundraising. In 2012 he ran the London Marathon and then began looking for a bigger challenge, which could raise more sponsorship. In 2013 Ed is running five marathons in five days!

Finally the hard work feels like it's starting to pay off...

January's winter weather made training conditions difficult. While everyone else enjoyed building snowmen and sledging, I dreaded going out for my daily runs.

Just finding the time is getting difficult too. I am having to find innovative ways of fitting in the exercise; last weekend I ran to my niece's third birthday party while my wife drove our children.

Over the past month I have gradually increased the distance and last week I ran over 61 miles. Weekday training runs are just over 5 miles and I am running 15 miles on Saturdays and 25 on Sundays.

The long runs are hard but the sense of accomplishment when you achieve what you set out to is difficult to match. Someone once told me that successful people are individuals who decide to do something and just do it. I have taken this maxim into running and daily life.

My nutrition is getting better. I have stopped drinking alcohol, which is great: it's been over a month since my last hangover. I am eating more healthily, cutting out fat and focusing on slow-release carbs and white meat. My issue has actually been eating enough food as I’m not hungry after exercising and I should snack as often as I can. I am still eating chocolate - obviously can’t give that up!!

I am now at my lightest since I was a teenager and the fittest I have ever been but my big concern is staying injury free. Sometimes it’s more helpful not to run but it doesn't stop me feeling guilty if I miss a session.

It's time to start fundraising with a vengeance. So far I have raised £1,850 which is great but I have ambitious ideas of raising over £10,000. Last year my colleagues and I organised a "pub quiz" for our clients and this year we are running a bigger event with over 120 people attending. I am also raising money through family and friends and asking complete strangers. Hopefully my story is compelling enough for people to donate.

Everything's starting to become very real now... it's just over two months away. I need to stick to my plan as the next month's training will be very gruelling.

You can donate to Ed's amazing fundraising campaign here: www.virginmoneygiving.com/edwardnash-steer

The Cystic Fibrosis South West meeting - road testing our research strategy

Janet Allen, Director of Research at the Cystic Fibrosis Trust, braves the elements with members of the CF community to discuss our new research strategy.

I travelled to Bristol for the Cystic Fibrosis South West meeting on Tuesday, together with CF Trust CEO Ed Owen.

This was my first regional meeting and my first opportunity to explore some of our ideas for the Trust’s new research strategy with the CF community. Any strategy needs to be “road tested” before you roll it out, so we held our discussions in confidence.

I was very nervous – what would people think? Were we on the right track?

The weather was atrocious, with sleet and snow, and as it got worse Ed and I were worried that no one would be able to make it to the meeting, including ourselves! Audrey Williams, our South West Regional Fundraising Manager, who put all the hard work into arranging the event, had to turn back when she hit a blizzard en route. 

About 30 intrepid travellers turned up for the meeting, reflecting the commitment of the CF community. There were parents and family members of people with CF and scientists and physicians from Bristol University.

Ed spoke about the Trust and “where we are”, and then I talked about the process of building a strategy and what we wanted our strategy to look like. We then opened the session for feedback, questions and comments.

There was lots of constructive feedback and debate – we even ran over schedule. We had an open and frank discussion and there was a lot of support for the new research strategy approach and how we have gone about the process. Over the next couple of months we are holding more consultations, refining the strategy ready for publication at the end of April.

Thank you Audrey for arranging the meeting and fantastic venue, and above all, thank you to everyone who braved the weather to take part.

CF Unite - one participant's account

As Dr Matthew Hurley discussed in his blog post last week, the first Cystic Fibrosis Unite event took place on Saturday. This was a virtual conference hosted by the University of Nottingham and funded by a Wellcome Trust People Award, discussing Kalydeco. Rose Donnelly, Executive Assistant to Chief Executive Ed Owen, tells us all about it.

The conference was moderated by Professor Alan Smyth, who leads the paediatric specialist cystic fibrosis service in Nottingham, and David Noonan, who has cystic fibrosis. The speakers were Dr David Sheppard from the University of Bristol, Professor Stuart Elborn from Queens University in Belfast, Dr Michael Boyle from Johns Hopkins in Baltimore and Ed Owen, CEO of the CF Trust.

Dr Sheppard began by giving an overview on CFTR (cystic fibrosis transmembrane conductance regulator), explaining what it does, why it doesn't work in people with CF, and what ivacaftor (Kalydeco) does to it. I particularly liked his analogy that ivacaftor affects CFTR like oil on a rusty turnstile - it makes it easier to open and helps it to stay open for longer.

Stuart explained that previous research had shown that milder symptoms of CF are associated with mutations where there is some CFTR function leading to a stratified medicine approach, where specific therapies are designed for specific patient cohorts. The Phase III ivacaftor studies showed very encouraging results in terms of lung function, sweat chloride, weight gain and frequency of pulmonary exacerbations.

Mike's presentation focused on F508del, the most common mutation in people with CF, and the research being carried out by 23 centres worldwide combining ivacaftor with VX-809. Approximately 87% of people with CF worldwide have this mutation, and a therapy successfully treating it could have an enormous impact. Vertex plans to start clinical trials in 2013.

Ed feels the success of ivacaftor represents a paradigm shift in terms of discussion and discourse in scientific approach. Forbes has described ivacaftor as the "most important drug of 2012" and the rapid approval and positive funding position will create more investment. The strategic decision made by the Cystic Fibrosis Foundation was instrumental in developing the drug and the role the patient voice has played cannot be underestimated.

Finally, David Noonan gave a personal account of Kalydeco. After 64 days David has gained 20% lung function, and now feels energetic and has put on weight.

Throughout the session questions were submitted by the virtual audience.

If you were unable to join the event online, you can see a recording on the CF Unite website: www.cfunite.org.

CF Unite - a chance to get involved in research

Dr Matthew Hurley, a Clinical Research Fellow at University of Nottingham, discusses why CF Unite is a great opportunity for people with CF to get involved in research.

I don't have cystic fibrosis. I've met a few people that do, but I don't have the foggiest idea what it's like to have to count out the creon before each meal or find the time in the day to take all the meds and nebs.

I'm just a junior doctor that pretty much fell into doing CF research. But, after 'falling into CF' and after hours of experiments and plugging numbers into a spreadsheet, I have felt the buzz of seeing a beautiful graph showing a clear result. Sometimes there are enough of these rare moments to make a story to take to a conference to tell other scientists. That feels good too. 

The problem is though, researchers can talk to themselves to their hearts content, but if it doesn't make the blindest bit of difference to those with CF, what’s the point? So imagine what it would be like if you - someone with CF or a parent of a child with CF - could make that call: "does this bit of research make any difference?" More importantly, what if you could help plan those studies so that more people would feel able to participate or to help the final result be more relevant in the real world?

I also don't know what it's like to hold a CF meeting and have to put on the advert that cruelly ironic phrase "due to cross-infection concerns we cannot allow people with CF to attend". Sadly it’s a very bad idea to get a group of people with CF in the same room because the bugs would have a field day.

That's why Cystic Fibrosis Unite was formed. It's the 21st century and (almost) everyone has access to a computer, a smartphone, or both. So we can get the people who are doing the exciting research and sit them in front of their computer so that they can tell you what they've done. And what's more you can ask questions and tell them what you think.

So that's what will happen on our first event on 12th January 2013. Dr David Sheppard from University of Bristol will tell us all we ever needed to know about CFTR - the channel in cells that's 'wrong' in cystic fibrosis. Prof Stuart Elborn from Queens University in Belfast will talk us through the first Phase III trial of Kalydeco for people with the G511D mutation. Dr Michael Boyle from Johns Hopkins in the US will tell us the results of the Phase II trial of the combination of Kalydeco and 'VX-809' for people with the most common mutation, dF-508. Ed Owen will also be there to let us in on how stressful it was as the powers that be decided if they would fund it or not, as will David Noonan who is now taking Kalydeco.

So join us online on 12 January and take part. If you can't make it, we're putting a recording of the event on the website and you can ask your question or make your comment on the forum.

To keep in touch with updates and get the link to the event register for updates on the website (www.cfunite.org) or follow us on Twitter @cfunite or Facebook /cfunite. If not just send us an email on info@cfunite.org. If you think you can help, it would also be great to hear from you.

Ed Nash Steer's Marathon Challenge - blog two

Ed Nash-Steer’s niece Hannah was diagnosed with cystic fibrosis four years ago, and seeing what people with CF go through everyday motivated him to start fundraising. In 2012 he ran the London Marathon and then began looking for a bigger challenge, which could raise more sponsorship. In 2013 Ed is running five marathons in five days!

Christmas Running - What's the point...?

Running has taken over my life for the past two years in a very positive way but every Christmas I have found it hard to train effectively.

As the nights draw in I find my motivation to leave my warm house wanes. Offers for a Christmas drink are all too easily accepted and Christmas parties can clearly not be avoided.

This year my excuses included a stag do, a skiing holiday, a wedding and illness. The first week in December was my most successful yet, with over 45 miles covered. Between then and the new year I only covered 23 miles in 5 runs. Those I do manage I find harder due to the cold, but incredibly rewarding physically and mentally.

Someone advised that running in the cold reduces your time by 20%. I don’t know if this is true but the thought helps when I make it outside.

I believe December, Christmas and New Year are to be enjoyed. Train hard in November but December is about damage limitation before training starts with a vengeance in January.

January 2013 is an important month as I gear up for my five marathons in April. My daily runs have moved to the morning so I get up at 5am, which is proving "interesting". I think I am developing a new category in the sport called "sleep running".

The plan is to build recovery in long weekend runs. This weekend I have a 15-mile run on Saturday and a 9-miler on Sunday, increasing to 21 and 11 miles by February. I have a regime where I eat more healthily more frequently; a minimum of six "meals" per day. One of these is an afternoon cake however, so hardly a hardship!

While training takes up most of my free time I need to remember why I am putting myself through this - fundraising!! I plan to run a networking event, ask everyone I know to sponsor me, stand outside shops, and get some press coverage. The CF Trust is a great charity and I need to maximise this opportunity.

I am ready to take myself to the next level. See you on the other side!

You can donate to Ed's amazing fundraising campaign here: www.virginmoneygiving.com/edwardnash-steer

A new year's blog by Ed Owen

Ed Owen

One year ends and another begins in the fight to beat CF

Kalydeco might be difficult to pronounce (try saying ker-lie-di-co) and, confusingly, is often referred to as ivacaftor (its clinical name) too. But this little blue pill takes the award for the stand-out star of the last 12 months.

Put simply, it represents the most important breakthrough in the treatment of cystic fibrosis for many years. For the first time ever, following licensing decisions in the US in February and Europe in July, there is now an approved treatment that directly affects the genetic defect that causes cystic fibrosis.

For those with the G551D mutation of cystic fibrosis - for which Kalydeco is targeted - its impact is potentially life-changing. But its development brings real hope to everyone that further transformational treatments can be discovered for other genetic types in the next few years.

But licensing doesn't automatically mean access for what is a very high-cost drug. That is why we have been leading a campaign to ensure that the drug is funded within the NHS - and just a few weeks ago, we received the excellent news that all eligible patients in England will have access to Kalydeco from the beginning of 2013. We are pushing hard to ensure that Scotland, Wales and Northern Ireland follow England's lead soon.

That Kalydeco exists owes much to the foresight and imagination of our sister charity in the US, the Cystic Fibrosis Foundation (CFF). The CFF funded its early development work in partnership with a commercial company - and we need to learn from this as we improve our own approach to research here in the UK.

There have been research advances here in the UK too, and in June the first doses in the Gene Therapy Consortium's Phase 2b clinical trial were administered to patients. This trial will continue to the end of 2013 and we should have results sometime in 2014. The trial is a major milestone in the development of gene therapy research and all those in the cystic fibrosis community who have raised money for this work over the last decade can feel enormous pride in what has been achieved.

Going forward, we are going to be much more clever - and hard-headed - about how we fund research to ensure that the work we support has the best possible chance of getting into clinics as soon as possible. And in 2012 we took the first steps towards this with the appointment of Dr Janet Allen as our new Director of Research. She is working hard on developing an innovative and ambitious strategy to be published at the Wellcome Trust in April 2013.

Janet's arrival was only one of a number of changes at the Trust in 2012. In July, George Jenkins became our Chair and - following five months as an interim - I was made the permanent chief executive in October. It's a genuine privilege to do this job, and we are all focused on promoting the interests of everyone with cystic fibrosis and their families.

Improving the way we communicate with our core supporters, as well as to clinicians, politicians and the general public, is a key part of further changes we are making - and in the spring we plan to launch a new brand that will demonstrate the inspiration, passion and determination that lies behind everything we all do.

There is a great deal of other work going on too, not least the roll-out of our new Quality Improvement Programme in 2013 to drive up standards in CF care across the UK.

But I want to take this opportunity to thank you, our supporters, for your tireless efforts raising money for and awareness of cystic fibrosis. The Trust can only exist with your energy and generosity, and we are determined to repay that commitment by making 2013 a year of real achievement and progress.

Have a very happy new year!

Ed

Microbe management in CF

Pseudomonas bacteria -
a hot topic at the recent
CF Microbiology
Consortium meeting

From 2005-2008, the CF Trust funded the development of the UK CF Microbiology Consortium to bring together research activity from four universities (University of Liverpool, Queens University Belfast, University of Edinburgh and University of Cardiff). The funding has ended, but the Consortium continues to meet annually to promote better understanding of CF microbiology and its third annual meeting was recently held in Liverpool. Professor Craig Winstanley and Dr Jo Fothergill of the University of Liverpool give an insight into what these issues are.

The third annual meeting of the UK Cystic Fibrosis Consortium brought together over 80 CF researchers and clinicians from throughout the UK. There were 16 presentations and lively discussion around each of the four major themes addressed (diversity and adaptation, pathogenicity, therapeutics and epidemiology).

The opening session, chaired by Dr Diana Bilton (Royal Brompton Hospital) and Professor Stuart Elborn (University of Belfast) emphasised the diversity of microbes in the CF lung, and how disease-causing bacteria adapt to the CF lung environment. A better understanding of these issues could lead to novel approaches to therapy. There are many things can influence bacterial communities in the lungs. For example, Dr Will Flight (Manchester Adult CF Centre) spoke about the impact of respiratory viruses on the make-up of the bacterial community. Because it is difficult to do these studies in patients, model environments that resemble the CF lung can be used. Damian Rivett (Kings College Hospital) and Dr Chloe James (University of Liverpool) both described studies using model systems to study bacterial communities. The focus of Dr Volker Behrends’ (Imperial School of Medicine) talk was the metabolic adaptations of the bacteria (the way that bacteria alter their metabolism).

In the second session, chaired by Professor John Govan (University of Edinburgh) and Professor Eshwar Mahenthiralingam (Cardiff University), various aspects of pathogenicity (how micro-organisms cause disease) were discussed. Megan Jackson (Queen’s University Belfast) talked about the role of obligate anaerobic bacteria (bacteria that cannot survive in the presence of oxygen) and Ian Passmore (University of Cambridge) presented work on Pseudomonas aeruginosa type III secretion systems and biofilm formation (two important mechanisms contributing to how Pseudomonas causes damage). Sonali Singh (University of Nottingham) focused on host responses (such as the immune system), and the role of an immune system cytokine that can contribute to inflammatory responses, IL-17. The session ended with a talk by Dr Cristobal Mujica Tronconso, who described the role of two cell wall enzymes in Burkholderia cenocepacia.

In a session on therapeutics (treatments for infection), chaired by Professor Miguel Camara (Nottingham) and Dr Jane Davies (Imperial College, London), we heard talks about novel therapeutic targets (bacterial structures or activities that we can design drugs against) from Dr Matthew Robinson (University of Exeter) and Dr James Lazenby (Nottingham), whilst Dr Nick Tucker (University of Strathclyde) described a new group of compounds with potential as anti-Pseudomonas agents. Dr Rishi Pabary (Imperial College, London and Royal Brompton Hospital) presented a study where phage therapy (the use of viruses to attack bacteria) was used against P. aeruginosa in a rodent model.

In the final session, chaired by Dr Juliet Foweraker (Papworth Hospital) and Dr Martin Walshaw (Liverpool Heart and Chest), issues relating to epidemiology (patterns in populations) were discussed. Jane Turton (Health Protection Agency) presented an overview of P. aeruginosa genotypes infecting UK CF patients and Richard Barton (Leeds General Infirmary) talked about the epidemiology of Aspergillus in cystic fibrosis. The growing threat of non-tuberculous mycobacteria was addressed in the presentation of Dr Dorothy Grogono (University of Cambridge). The final presentation, by Dr Laura Thomas (Cardiff University), discussed approaches to testing preservatives with activity against pathogens important in cystic fibrosis.

In addition to the short talks, there were 16 posters presented at the meeting, covering a range of topics. This growing meeting provides a platform for researchers to present and discuss their ideas. As well as demonstrating the breadth and depth of expertise in the area of CF microbiology in the UK, it also highlights a real enthusiasm and willingness to work together to provide high quality research and practical solutions in this important area.

The meeting was sponsored by Forest Laboratories Inc.