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.