A 2016 Unlimited

The New Year is here! We're ready and raring to go in our fight for a life unlimited for everyone affected by cystic fibrosis!

Our Chief Executive, Ed Owen, lays out what will be an exciting year ahead and how you can resolve to help us throughout the year.

We are at a unique moment in the history of cystic fibrosis – and 2016 must be a year of progress towards our ultimate goal.

New transformational therapies, exciting research programmes and breakthroughs in genetic science are bringing closer the day when we will beat this cruel condition for good.

And we, the Cystic Fibrosis Trust, will be unveiling new ambitious plans to help ensure our united CF community seize the extraordinary opportunities that exist to remove the limits CF imposes on all those whose lives are affected.

But, as all of us touched by cystic fibrosis know, with hope comes grief and pain.

Last year began for me with the funeral of Emily Thackeray, a shining star who dedicated her life to campaign on issues like organ donation – and ended with the dreadful news that 14-year-old Dylan Samuels had passed away.

These, and all the other individual stories of those who have lost their lives to cystic fibrosis, are the reasons we enter this year with even more determination and focus to seize this special moment than ever before.

2015 saw major steps forward.

The decision of the European Medicines Agency in November to approve the use of Orkambi for those aged 12 and over with the most common CF mutation in the UK (F508del) was very good news – as was its decision to extend the use of Kalydeco to children under six and to adults with the R117H mutation.

But this does not guarantee access, and this year our ‘Stopping the Clock’ campaign will need to step up a gear to ensure both the NHS and Vertex do the right thing so more people with CF are given access to these gene-modifying therapies.

It is encouraging, too, to hear of the progress other pharmaceutical companies are making to develop transformational drugs, as well as further Vertex work to bring forward treatments affecting other mutations.

2015 also saw the long awaited results of the Phase 2b clinical trial of a gene therapy developed by the Gene Therapy Consortium (GTC) here in the UK. This study was only possible because of the extraordinary support of the CF community over the last 15 years, and the results demonstrated for the first time the potential for gene therapy as a potential cystic fibrosis treatment.

We hope that a commercial partner can be found to take on this work in 2016 and, as a result of the continued generosity of all our supporters, the Trust is continuing to fund the GTC’s longer term ‘Wave 2’ work to develop a potentially more impactful gene therapy product using a viral agent to deliver the genetic material to the lungs of people with cystic fibrosis.

2015 saw extraordinary developments in gene-editing technology as a potential for treating, and perhaps curing, conditions like cystic fibrosis.

The Trust is already investing in this field of research and looking to build new collaborations to exploit the opportunities it presents. We are also joining other groups to ensure we have the right regulations in place to enable more vital research to take place to advance gene-editing work.

We continue to invest in research across a range of vital areas – from transformational science to beat cystic fibrosis at source to work confronting challenges like pseudomonas and NTM infections.

We will be expanding our research portfolio in 2016, and looking to develop our commercial collaborations with industry to ensure good ideas have every chance of progressing to real-life treatments for people with cystic fibrosis.

And we are working well beyond research too.

We continue to work closely with clinicians and CF teams across the UK to ensure all with cystic fibrosis have access to the best possible care in the NHS.

To do so, we will continue to lobby to ensure proper NHS funding is directed to support specialist CF centres – and we are soon to announce new funding to support initiatives that promote innovation and excellence in CF centres.

These will all be part of a range of new activities we will be unveiling in the next few months as we seek to raise our game and our ambitions to improve and transform the lives of those we are here for.

But we cannot do it alone. We need your support whether through fundraising or donating, through campaigning or giving up your time to volunteer.

2015 saw too many young people dying of cystic fibrosis. We have no time to lose. The support of the CF community is extraordinary. But there is much more to be done if we are to move closer to beating CF and ensure a life unlimited.

As part of this challenge, we are asking you to make resolutions for the New Year that can help make a difference. I, for one, will make a clear commitment that the Trust will build on the improvements we have been introducing to make 2016 an historic year in our work to create a step-change in the impact we make.

I will also resolve to try and find the time to see more of the greatest football team in the world. That’s Manchester City, just in case you didn’t know!

Find our more about how you can use your New Year's resolutions to join the fight for a life unlimited at www.cysticfibrosis.org.uk/resolutions