Following recent news
reports of a ‘cure’ for cystic fibrosis being within reach, Cystic Fibrosis
Trust Chief Executive Ed Owen discusses the promising research at the centre of
the story, and the need to think big but stay grounded.
Nothing
is more guaranteed to get all of us excited than stories of a possible ‘breakthrough’
or ‘cure’ for cystic fibrosis.
So
a news item on Channel Four News this week heralding a research project led by
Dr Anil Mehta in Dundee as a possible cure for cystic fibrosis has
understandably caused a wave of interest among many of us affected by the
condition here in the UK.
In
a blog on Tuesday
accompanying the news piece, the veteran broadcaster Jon Snow described the research
as a “Eureka moment” that offered the hope of a “cystic fibrosis cure”. Many
people called our office to find out more, dozens tweeted and hundreds posted
messages on Facebook at what appeared to be a remarkable moment.
So amid all the
excitement, what’s the reality?
First,
it is important to note that no research paper has yet been published so we at
the Trust have not been able to see the detailed analysis. But the facts
according to the University of
Dundee’s press statement are that in an early stage clinical study undertaken in
Italy, medics trialled the combined use of two drugs already licensed for other
conditions - Cysteamine, and epigallocatechin gallate (EGCG) – involving 10
people with cystic fibrosis with two copies of the DF508 gene.
The
University statement says that the study found that the two drugs given
together reduced inflammation in 9 out of 10 of the patients’ airways and also
dramatically reduced their sweat chloride levels. Obviously, then, the research
looks promising. But it’s at an early stage trial and used only a very small
sample.
I
had the pleasure of speaking with Dr Mehta on Wednesday and he agreed with me
that talk of a potential cure was misplaced. He acknowledges that his work
is at an early stage but wants it to now go forward to a larger Phase 2
research study involving 120 patients. He is planning to submit an
application to the Government’s Medical Research Council (MRC) which supports
this kind of clinical research in the UK, although there are a number of
regulatory hurdles the work needs to overcome first.
As
a result of the extraordinary support we receive from thousands in our
community, the Cystic Fibrosis Trust is currently investing in a wide
range of research projects aimed at improving and transforming the lives of people
with cystic fibrosis. This has helped fund earlier stages of Dr Mehta’s work
and our new Venture and
Innovation Awards
created last year are targeting funds at projects like this one as a way of
bringing in even larger sums of money from the Government and other quarters.
Dr
Mehta and I discussed these funding arrangements and – once we have analysed
the full details of this work – we stand ready to offer such an award as part
of the process of bidding for MRC funds.
But,
three days on from the story being broadcast, I confess to feeling disappointed
at the way it was covered.
Talk
of breakthroughs and cures make great headlines. But such hyperbolic coverage
risks the unwitting manipulation of the emotions and passions of those of us
desperate to believe that there is an imminent prospect of being freed from the
terrible clutches of cystic fibrosis. Put bluntly, such stories bring into
sharp focus the stark contrast between longer life and an early death.
I
say this not to pour cold water on all our hopes because this particular
project looks promising – and I am hugely optimistic about the wider future for
cystic fibrosis. Kalydeco is already correcting the basic genetic defect
of cystic fibrosis for four per cent of people with the condition in the UK –
and, with the active support of the cystic fibrosis community, I believe we can
help ensure that new transformative therapies and treatments will be introduced
over the next decade that move us closer to our goal of beating cystic fibrosis
for good for all.
But
developing these drugs is expensive and often a long slog – and what might look
hopeful at an early stage of development cannot be assumed to be a guaranteed
success in clinical terms further down the line.
So
the lesson to us all from this week is let’s keep thinking big, but with our
feet on the ground – even if, sometimes, media headlines tempt us to do
otherwise.