Wednesday 31 December 2014

Looking Back, Moving Forward.

Ed Owen, Chief Executive of the Cystic Fibrosis Trust, looks back on our 50th anniversary and the successes of those 50 years, and how the Trust will build on them as we enter 2015 and beyond.

The Trust's 50th anniversary was never cause for celebration. How could it be when so many with cystic fibrosis can still only dream of reaching such a milestone, and when the lives of so many young people like Emily Thackray, who died this week , are so terribly cut short?

But 2014 has enabled us both to mark the contribution of all our supporters and, most importantly, to redouble our effort to beat this cruel condition for good.

The decision of HRH The Prince of Wales to take on the Patronage of the Trust this year was a fitting reward for the extraordinary commitment and dedication of all parts of the cystic fibrosis community - people with the condition, families, supporters, fundraisers, campaigners and many others .

The tireless efforts of so many over five decades has helped ensure vital research is funded, NHS care has improved and over time, turned what was exclusively a childhood disease in the 1960s into one where the majority of those with cystic fibrosis in the UK are adults.

And I want to add my tribute too. So, on behalf of all of us who work at the Cystic Fibrosis Trust, I send a massive thank you for everything you, our supporters, do on behalf of those we are here for.

Yet with so many young people dying early, and with the lives of all those affected being limited so severely by the daily physical and psychological burden of cystic fibrosis, our collective community activity, unity and voice are as critical today as they have ever been.

We are at the beginning of a new era of opportunity in the treatment and care of cystic fibrosis. Research breakthroughs offer the prospect of gene-modifying treatments for many with the condition, and new technology promises new ways of improving health and wellbeing.

2014 saw further steps forward with the continued use of Kalydeco showing extraordinary results for those with the G551D mutation, and results of Vertex's Phase III trial into its ‘combination therapy‘ focused on those with two copies of DF508. The latter is soon to be considered by European regulators with a decision likely in late 2015.

The Phase IIb trial of a gene therapy product was also completed in the summer of this year. This is the latest development of work which has been generously supported by the cystic fibrosis community in the UK over many years, and we await the results with keen anticipation.

After a short delay caused by further work processing the trial data - this is the largest research study of its kind in the world - we expect the Gene Therapy Consortium to publish the final results early in 2015.

Alongside these promising developments, the Trust has been investing in other vital research projects aimed at tackling issues of real concern to those with cystic fibrosis. Three new Strategic Research Centres were announced early in 2014 focused on vital issues like pseudomonas and NTM - and we are due to announce a further three new SRCs in early 2015 ensuring that the brightest and best scientific efforts we can find are focused on the issues that really matter.

2015 will also see the development of the Trust's SmartCareCF programme to bring industry and academic experts together with people with cystic fibrosis, their carers and clinicians to develop new forms of care using smart technology and big data.

SmartCareCF is one of a number of ambitious programmes we at the Trust are developing as part of our renewed determination to beat cystic fibrosis. These include new research projects, better support for people with cystic fibrosis and more effective ways to campaign for new drugs, tackle funding shortages in the NHS and raise wider awareness of the condition.

In order to deliver these ambitious plans we must ensure the organisation of the Trust is fit for the task. So we are making a number of internal changes including the introduction of new skills and expertise in specialist areas of fundraising, research and communications; investing in new IT and looking at new accommodation options; and bringing in new talent at trustee level to ensure the Trust is being led effectively at this important time.

We are also improving the way we communicate, support and learn from our supporters, particularly those living with cystic fibrosis - and sharpening our message to raise wider awareness and impact. 

This recent film produced for our partnership with the British Comedy Awards is an early example of how we can do so.

50 years on and, with your support, the Trust continues to play a major role in improving the lives of people with cystic fibrosis in the UK. But we must now seize the opportunity to make transformational change over the next few years. With the help of the wider community here and overseas we can and will ensure that cystic fibrosis is barely more than a distant memory when the next 50 years comes around.