Clinical trials in cystic fibrosis – time to up our game, by Janet Allen

On 11 December, Professors Alan Smyth and Simon Langton Hewer ran an innovative meeting on “Clinical trials in cystic fibrosis – time to up our game”. I went along and listened to the range of stimulating speakers talking about the value of clinical trials in advancing how we manage cystic fibrosis and the challenges of running multicentre trials, including the immense bureaucratic hurdles organisers have to jump.

But what marked this meeting out was less the interesting and important subject matter, and more the innovative way in which people could participate. About 80 people attended, physically seated in the auditorium, but the talks were streamed live on the internet allowing anyone with internet access to hear what they had to say. The panel of experts answered questions both from those in the auditorium and from the online audience.

Dr Matthew Hurley, Wellcome Fellow at the University of Nottingham organised the streaming. The Wellcome Trust has funded him to set up online conference streaming to allow people with cystic fibrosis, who would otherwise be unable to attend because of the risk of cross infection, to participate fully in this sort of event. Gilead also generously supported this “first”. There were some teething problems and the technology is still being developed, but the team worked hard on the day to sort the problems out and will continue their work to allow those with CF to participate fully in conferences about their condition.

Ed Nash-Steer's Marathon Challenge!

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 April this year he ran the London Marathon and then began looking for a bigger challenge, which could raise more sponsorship.

So I have finally announced to the world that I am running five marathons in five days…

Back in April I proudly ran the London Marathon for CF which was one of the best experiences of my life. I have never tried so hard or prepared for so long for anything before. 

Now I needed a bigger challenge, bigger for me and for those sponsoring me. I am running from Porton, Wiltshire, to London in four days and then running the London Marathon on the fifth day, with the aim of raising over £10,000.  This will be over 130 miles in five days.

I have been training since early September and I’ve already had some massive highs and lows. I was running better and quicker than I ever had but I went through a rocky patch for a few weeks when my motivation was very low. I guess it had something to do with running 45 miles per week without any company and having no smaller targets to aim for. But since I ran the Cardiff Half back in October I have found my mojo again. It’s a great course and I ran my best time ever (1hr 35 minutes). Since then I made a better training plan and have started to spread the word. In my first week fundraising I’ve raised £1,300 and have had pledges for more.

My next marathon is on 23 December in Portsmouth and my current training is focused on completing this whilst still being in a decent shape to enjoy Christmas. My mind wanders across many subjects when I have a few hours of running ahead of me. Sometimes I think about the amazing crowd last year, about Hannah or my work; but most of the time I simply zone out and think about nothing, which when you lead a busy life is pure bliss. I never thought when I started running last August that I would enjoy each and every run I go on. 

People think I am nuts to be running so far and I always get asked why am I doing this? There are many answers I give but the truth is… because I can, because I should and because it makes a difference. 

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

“Living into adulthood is now expected.”

Last month a cystic fibrosis course was held at the Royal Society of Medicine in London. Dr Nicholas Simmonds, Consultant Physician in adult CF at the Royal Brompton Hospital in London gave a fascinating presentation on ageing and long-term survival in CF, and has kindly summarised his talk for the CF Trust blog.

Dr Nicholas Simmonds

The demographics of cystic fibrosis, particularly median survival, continue to improve at a rate unparalleled to most other chronic conditions in medicine.

Within a few decades average survival has more than doubled and now stands at 41.4 years (CF Trust Annual Data Report 2010).

Children born with CF from the early 2000s are expected to live beyond 50 years of age and over 55% of the current UK CF population are adults. Living into adulthood is now expected – currently over 22% of UK CF individuals are in their 30s and over 7% aged at least 40! In paediatrics, the emphasis is now very much on when a patient will transition to the adult clinic (not if) and the aim is to transition with normal lung function with planning for a full and active life (e.g. college, work and relationships etc). 

My presentation at the Royal Society of Medicine highlighted this change in demographics and outlined some of the important reasons behind the improvements (e.g. keeping lung function and weight optimal from an early age, keeping Pseudomonas infection at bay by effective treatment, etc.). 

It also highlighted that all this good work is not straightforward to individuals with CF and comes at a cost – time, energy and effort – as treatment/physiotherapy is intensive and needs to be sustained throughout life. As clinicians and researchers we recognise this and are continuously looking for new and innovative ways to help (faster nebulisers, for example). 

Currently the majority of our adult patients are co-habiting/married and working – as a specialist adult clinic we aim to do all we can to support our patients to progress in life while at the same time providing effective specialist healthcare. The next few decades are likely to continue to bring exciting and important challenges – new drugs are being developed and the adult population will continue to increase. With this we recognise the need to continue to adapt and develop for the CF adults of the future.

An insight into the Research Advisory Committee

Dr Janet Allen, the CF Trust's Director of Research, provides an update  following the Trust's biannual research meeting.

This week I attended my first Cystic Fibrosis Trust Research Advisory Committee (RAC) meeting. This was a special meeting because we wanted to consult with RAC expert members and ask them to contribute to our early thinking on the new research strategy, due to be launched at the end of April 2013. 

As this was a special meeting we invited Professor Danny Altmann from the Wellcome Trust to start the event. Professor Altmann spoke about the different ways that research can help improve the lives of people living with cystic fibrosis. 

Members of the Committee were then split into three groups and challenged to provide innovative comments to two challenging research questions. I stayed up until 5am to collate all these ideas! I used them as the starting point for a more formal discussion the next day. We also shared with the RAC some of the early thinking on the research strategy that we have developed within the CF Trust and had some very useful and constructive feedback.

This was an excellent meeting and the members of RAC, led by Professor Elborn, worked hard together with senior members of the CF Trust (Ed Owen, CEO, and George Jenkins, Chairman) to provide me with some inspiration as I develop the strategy. 

What is really encouraging is the value placed on the RAC’s contribution to the research strategy. We had a “full house” for these discussions and the senior team at the CF Trust were present throughout.  
 
Watch this space for further information about the exciting developments in CF research.

Sadie's Marathon effort

Sadie Mulvey has CF and is running the Virgin London Marathon 2013 in support of the CF Trust. She will be blogging regularly about her experiences so keep checking back to follow her progress!

To be running the most famous marathon in the world for Team CF with my family and friends there to watch me will be a phenomenal experience. Getting a Golden Bond Entry into the marathon was the easy part: suddenly the training I have always done to make sure that I keep my lungs as fit as possible just became very serious.

I want to give myself the best chance possible in not only finishing this bucket list entry, but to remain as injury free as possible. I emailed James Buckingham, owner of Precision Fitness and Performance in Exeter, asking if he would be able to support me with any training and nutrition advice and I could not believe it when he replied to say he would be delighted to help. I start with my personal trainer, Billy Wakeley, this week and he will be getting me ready for race day. No pressure Billy and thank you!

I was diagnosed with CF at the age of four, my younger sister, brother and cousin also have CF and the latter, Neil, is on the transplant list for new lungs. I will be 34 on the day of the Virgin London Marathon. If anyone had told me in my early days that I would be able to contemplate this sort of distance, I would have told them they had the wrong girl.

After long periods of illness as a child, I found exercise turned my health around and my trainers, the open roads and fresh air became my panacea. I am a huge advocate for physical activity in the battle against the bugs and in keeping a strong mind and body. This is going to be the biggest test I have ever put my fitness to but I am confident my determination will see me trotting over the finish line, and then people, it is party time!

I am overwhelmed with the generosity I have encountered on my journey to raise £5,000 for the CF Trust, and the encouraging messages. All news of my fundraising events will be posted on my charity page. http://uk.virginmoneygiving.com/SadieMulvey
 
Sadie is being sponsored by Precision Fitness and Performance 
www.precisionperformance.co.uk

An insight into the Medical Advisory Committee

The Cystic Fibrosis Trust Medical Advisory Committee (informally known as MAC!) meets twice yearly to advise the CF Trust on clinical and medical issues relating to cystic fibrosis, and guide our work in these areas. The Committee comprises expert clinicians – CF adult physicians and paediatricians, specialist CF nurses, physiotherapists, dietitians and pharmacists, and also patient and parent representatives. We highly value this input and are grateful to all of those who give their time on MAC and our other advisory committees to help ensure that our work is relevant and impactful.

The Committee met last week at our Bromley HQ, and a diverse range of topics were discussed and debated.

Director of Marketing Tamsyn Clark presented a preview of the CF Trust's new brand, and sought feedback on the general direction in which we're heading. The Committe's feedback was constructive; ensuring that the clinical community is on board and understands and appreciates our brand and messaging is crucial, as our relations with clinicians are vitally important to our work.

Our annual medical conference is well-attended by clinicians from CF Centres and Clinics across the UK. The next conference will take place in the spring of 2013 and MAC discussed how the event could be enhanced to become more useful for those attending. As Committee members highlighted, study leave is becoming more scarce in the NHS and there are also a number of other conferences competing for delegates, so it is important that we continually evaluate and improve the events that we hold for clinicians.

Our consensus documents provide valuable guidance to clinicians treating patients with CF in critical areas such as antibiotic treatment and general standards of care. MAC identified which documents are due to be updated and also agreed two new documents to be produced; next year work will begin on consensus documents for management of infants diagnosed with CF through screening, and CF-related liver disease.

The Clinical Care team reported that peer review pilot visits have gone ahead and feedback so far on the new peer review process has been very positive. The CF Trust will aim to facilitate around ten peer reviews a year and to have a six week turn around from the official date of review to the publishing date of the report. The CF service will be given six months notice of a peer review and most of the data will be inputted online via an extranet server similar to the NHS system. However, patient sensitive information will not be included in the server.

Dr Diana Bilton, Chair of MAC, reported that the UK CF Registry, which is funded by the CF Trust, currently contains complete clinical data for an impressive 89% of the UK CF population. New data from the Registry will be released before the end of the year.

There was also discussion around the current challenges facing the various specialist groups involved in the provision of CF care (such as physiotherapists and pharmacists). One of the key priorities of the Trust's clinical care programme is ensuring that specialist care is available for everyone with CF, and we work closely with clinical teams to achieve this.

This week our Research Advisory Committee (RAC), which advises the Trust on scientific and research issues in CF and also reviews our research grant applications, is meeting over two days. We'll provide an update soon after the meeting.
 
Read more about MAC and RAC.

Research Conference

As I settle in to the world of research in cystic fibrosis, I am finding out just how exciting some of the work in this field is. All of this will help me when I plan the CF Trust’s research strategy, due to be released in late spring next year.

I went to a conference on Wednesday (7 November) called "Expert View Points on CF Pulmonary Disease - State of the Art", where over 150 healthcare professionals gathered to hear and debate state of the art treatment for lung disease in cystic fibrosis. 

A stellar international expert faculty had been brought together to provide a cutting-edge overview of the different treatment approaches. Experts were drawn from the UK, Canada, Europe, Australia and the USA. Topics covered ranged from the new therapies (those being developed to treat the root cause of cystic fibrosis) to the management of Pseudomonas infections and new infectious agents. 

Highlights from the meeting were the ways the experts provided research evidence to support their presentations. This demonstrated the need for meticulous, well-constructed clinical trials to inform treatment practices, something the CF Trust supports.

In fact I found the meeting so inspiring it was difficult to choose one stand-out talk amongst a series where each one was of such a high standard.  But for me the presentation on adherence was enlightening and demonstrated that development of “personalized medicines” is not just a tailoring of drug therapies. 

Congratulations go to the organisers, Drs Charlie Haworth and Andres Floto, for pulling together such a high-quality expert faculty that the vast majority of the audience stayed on beyond the last session to continue the discussion and debate. 

Watch this space for more research news!

Janet

Read more about cystic fibrosis research and the role of the CF Trust