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.