Tuesday 16 February 2016

Wind, wind and more wind

Last week in Oxford, the Cystic Fibrosis Trust brought together gastrointestinal (GI) health experts from across Europe to look at the digestive complications that many people experience with cystic fibrosis. Along with the scientists and clinical staff, people with CF and their carers were represented. Below one of those parents, David Turner QC, gives us and insight on the research 'sandpit'.

Storm Imogen was doing its best to bend trees double as 35 scientists and clinicians gathered in Oxfordshire for a research “sandpit” devoted to the problems which cystic fibrosis causes for the digestive tract. An introduction from Dr Jon King (a Senior House Officer at St Mary’s, Paddington, who also has cystic fibrosis) outlined just how debilitating – and embarrassing – these complications can be. For many, digestive complications are amongst the most significant effects of cystic fibrosis.

The sandpit was the brainchild of Dr Janet Allen, the Trust’s Director of Strategic Innovation. 
Her idea was brilliantly simple:
  •     Mix together cystic fibrosis clinicians, gastro-intestinal clinicians, scientists specialising in CFTR defects and leading lights in (non-CF) gastro-intestinal research.
  •     Add a dash of inspiration.
  •     Simmer under significant time pressure.
  •     Remove collective ideas for research priorities and season under the bright light of instant peer review.

The assembled company involved experts from all parts of the UK, Germany, Belgium, the Netherlands and Spain. Discussions were free-rolling, leaping between subjects such as gut microbiota, DIOS, the impact of antibiotics, prebiotics and probiotics, gut acidity, gut motility, bicarbonate channels, chloride channels and methane testing.

The discussion was superbly marshalled and directed by facilitators from Know Innovation. By mid-morning of the second day the attendees were working together in small multi-disciplinary teams to define a novel research question and to justify the question’s relevance by explaining how it would make a real difference to the lives of people with cystic fibrosis. Proposals for further refinement and, potentially, applications for future research funding, included improving gastrointestinal health by manipulating microbiota, identifying biomarkers that could help develop personalised dietary plans, and designing a new supplement to improve fat absorption.

The aim of the sandpit was to stimulate discussion and to provide the foundations for future, multi-disciplinary research collaborations which might ultimately transform the lives of many. Not only was that aim achieved, but a buzz was created which was palpable as everyone left to confront the tail end of Imogen’s winds. All in all, this had been a tremendously exciting and uplifting event!

Friday 5 February 2016

Stopping the Clock - You Voice Matters

With the ongoing success of our Stopping the Clock petition on availability of precision medicines for cystic fibrosis, our Chief Executive Ed Owen gives more insight into why this is such an important issue for the CF community.

When 10,500 people sign up to our petition demanding access to new precisiontherapies like Orkambi and Kalydeco just 48 hours after we announced it, you know that this is an issue which touches people like no other.

Things have come a long way since the 1960s when most children born with cystic fibrosis failed to reach their fifth birthday – and there are now more adults with cystic fibrosis in the UK than kids.

But too many young people are dying early, too many families live with the grief and the fear that cystic fibrosis brings, for us to let up until we have beaten this cruel condition for good.

And this goal is now more than a distant hope. It is one that we now genuinely believe is within our grasp.

Yet the nearer this comes, so it is more important than ever that we increase our efforts to shorten the time it takes to get there, because every day, every week, every year we spend means more lives lost and more suffering families.

The development of a pipeline of new precision therapies that can extend life and reduce the heavy treatment burden of cystic fibrosis is a major step forward, and one that we want to see everyone benefit from as soon as possible.

But it will not happen unless we make it so, and that’s the purpose of our ‘Stoppingthe Clock’ campaign. It will ensure the collective voice of the cystic fibrosis community here in the UK is heard loud and clear by Vertex, the NHS, politicians and others.

Access to therapies that can make a difference to the lives of people with cystic fibrosis is an essential part of our wider Fight for a Life Unlimited.

This fight means we are investing where it matters to generate the best scientific and clinical ideas and to accelerate the process to ensure these benefit people with cystic fibrosis directly.

So, as well as campaigning for vital therapies for today, we are investing in world-class research centres tackling key issues like pseudomonas and NTM infection to help generate the drugs of the future too.

And we are developing plans to provide more capacity for vital clinical trials to take place in the UK and helping to shape ideas to develop SmartCareCF to use new technologies and data to empower people with cystic fibrosis and, where possible, keep them out of hospital.

We are developing new forms of information and support for people with cystic fibrosis and their families – and shaping new programmes to promote activity and empower and create new opportunities for young people living with the condition.

There’s so much we are doing. But we cannot do it alone. We need the funds to support this vital work.

And we need the united voice of all those affected by cystic fibrosis, their families, friends, clinical teams and others to help spread the word, amplify our community’s voice and reach out to new supporters.

Together, we can and will transform the lives of people with cystic fibrosis and achieve our goal of a Life Unlimited. Please get on board and let’s make it happen!

Want to help us do more transformational research and campaigning work? Donate now!