2014 is set to be a very important year for all of us committed to improving and transforming the lives of people with cystic fibrosis.
This summer we receive the results of international Phase III trials of a therapy aimed at treating those with two copies of the DF508 gene, the most common CF mutation. The treatment - which combines Kalydeco (or ivacaftor) with a new compound VX809 (or lumacaftor) - is the latest in the pipeline of drugs from Vertex focused on tackling the basic genetic defect of cystic fibrosis.
We will also hear the outcome of the year-long Phase IIb study of gene therapy being conducted by the Gene Therapy Consortium here in the UK. This programme has received extraordinary support from the CF community over the last decade or so, and we are eagerly awaiting these results too.
And 2014 marks the 50th anniversary of the Cystic Fibrosis Trust too.
The last five decades have seen enormous changes. In 1964, cystic fibrosis was almost exclusively a childhood condition. Today, there are more adults with cystic fibrosis in the UK than kids, with survival rates increasing year on year.
This progress is an extraordinary tribute to the families, clinicians, scientists and, most importantly, those with cystic fibrosis themselves who, throughout the last 50 years, have refused to accept that nothing could be done - and who have fought with commitment and determination to improve the lives of those touched by the condition.
It is down to the efforts of the wider cystic fibrosis community that, today, we have specialist CF centres in the NHS, newborn screening across the country and new drugs and treatments widely available.
And this fight is one we must continue. Even with all the improvements we have seen in recent years, a half of all people with cystic fibrosis are still not reaching their 40th birthday and the daily burden of treatment is imposing heavy burdens and restrictions on all.
So we will not be sitting on our laurels during our 50th year. Given the extraordinary "window of opportunity" being created by developments in research, I believe the next few years have the potential to transform the lives of people with cystic fibrosis. We must seize this unique opportunity and, with the support of our fantastic community, we will.
On behalf of all at the Cystic Fibrosis Trust, I want to thank you so much for all your support over the last year and to wish you a very happy new year!
Sending some smiles!
4 weeks ago