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