Friday 12 October 2012

Let's mention the K word!

Ed's second blog from the NACFC

There's one word on the lips of most people here at the North American CF conference. It is Kalydeco - or, to give it its scientific name, ivacaftor.

The CF Foundation - our sister organisation in the States - describe it as "a game-changer" as it's the first CF drug that addresses the basic genetic defect.

As we know, Kalydeco is effective for those with the G551D mutation - about 4 per cent of the UK CF population - and we are calling on Vertex, the drug's manufacturer, and the NHS to do the right thing and ensure all those who need it receive it as soon as possible.

If you haven't already, help us put pressure on both by signing up to our petition here which we will present before their meeting on 22 October.

We met representatives of Vertex in Orlando today to reinforce this message, and they assured me that they would do everything they could to achieve this outcome. The Trust and the CF community expect nothing less.

Vertex also used today to present to the conference new findings from the clinical trials they have conducted on a drug to treat those with the F508del mutation, the most common form of CF in the UK and worldwide, Vertex have published data about this on Vertex’s website.

The drug is a combination of Kalydeco and a substance called VX-809, and Phase II trials in the US have shown significant impact on lung function for those with two copies of the gene.

Larger Phase III trials are due to begin early next year, and it is likely that UK patients will be included. Vertex are also doing further Phase II trials to see if a higher dose of the combination drug has an impact on those with a single copy of the F508del.

And there are final trials beginning using Kalydeco on young children under 6, and on those with the R117H mutation.

These are exciting times for CF care and research. There is real potential that, for the first time in 50 years, we are on the cusp of transformational treatments for CF.

There is much to do to extend and improve the lives of all people with CF. But, together with the advances in other treatments and care, many at this conference are beginning to believe that the future will be one that few dared even hope for just a few years ago.

No comments:

Post a Comment