Four years of campaigning, protesting outside Westminster and writing to MPs about the immense and immediate need for these new small molecule drugs (that treat the underlying cause of CF, rather than only its symptoms, as Isaac does) that have been licensed but remained unavailable to patients across the UK, due to COST. From Kalydeco, Orkambi, Symkevi and now Trikafta (the UK brand will be called Kaftrio) - yesterday a deal for the whole portfolio of drugs has been agreed with NHS England.
This will prolong lives; this will improve the quality of lives; and for young children with CF, this should prevent the lung scarring, colonised infection and chronic inflammation the CF brings about.
I am so proud of the CF community and the CF Trust that have helped bring this about. There is no doubt that the campaign and publicity have driven this decision in a way that the government and NHS England have not.
So many happy tears for those who can now start this amazing treatment. But also tears for those that this drug comes too late for. There are too many.
For us in that last 10% that these drugs will not work for (I explain about this here) we hope not to be forgotten, and that these breakthroughs in medical research will open up new avenues for treatments that will work for the minority too. When Isaac was born, we were told that gene therapy was our best hope, but that it may come too late to help him. Still waiting on that! That these treatments have come to fruition at all gives us incredible hope for a better future.
Isaac is not well right now. Chest full of treacle. Hoping oral antibiotics may help, if not, will need to kick off IVs shortly. Tomorrow we head into hospital for the first time since lockdown began (which is an amazing stretch for him!). Mask up. Drop off sputum. X-ray, and get out of there. Follow up by telephone. So thankful for our incredible CF team that have enabled us to stay safe at home so long x
Local art. Life is beautiful.
