This blog is a bit different – but please bear with me. Some of you know, and some of you don’t know, that I have cystic fibrosis – an inherited condition that I was born with, diagnosed at age 10, and have lived with for 54 years of my life. For most of my life, by making a huge effort to keep fit and exercise almost every day (except when injured or ill), I have managed to keep symptoms to a minimum, and stay as well as I possibly can. Working at the woods has played a huge part in keeping me well.
However over the last couple of years, things have been heading inexorably downwards, despite every effort I could make. This year, things have been much worse. I have struggled to breathe walking round the woods, and have found I have to stop a lot. I couldn’t chase the butterflies that I love to photograph. I couldn’t do as much of the work as I would have liked. I was struggling to talk while showing groups around. And at home, I had to have a rest after having a shower, and was breathless after getting dressed, or cleaning out the chickens. My life was becoming a lot more constricted, which, for somebody who loves to be outside in the woods doing things, and a lover of sport, was not a good thing. In reality, my life expectancy was down to a few years, with the prospect of being permanently tied to oxygen looming large, and beyond that, the trauma of considering a lung transplant, should I be eligible.
This year, a new medication has been licensed. It treats a mutation that 5% of people with CF have – one of many thousands of mutations that occur in this condition. Amazingly, that is one of the mutations I have. What luck! Except, of course, that the medication is very expensive. There are relatively few people in the whole World who could benefit from it, but the costs of licensing and regulation to take it to market are the same whether 100 or 10,000,000 can take it. There is also the issue of producing a return on investment, and making a profit. These two, combined, have set the price extraordinarily high – about £15,000 a month.
The medication works in a clever way. CF is a condition caused by an absent or malfunctioning chloride channel called CFTR that sits in the apex of cells lining the lungs and other organs, and actively transfers chloride ions from one side to the other. Along with the chloride goes water, and the absence of water in secretions causes the lungs to block up with sticky mucus that then gets infected with unusual and unpleasant organisms which cause inflammation, lung damage, and declining lung function. Treatment of these organisms is unpleasant, involving daily nebulisers, physiotherapy and periodic admission to hospital for intravenous antibiotics, which also have nasty side-effects – in my case, I’m also allergic to most of them. The new medication, called ivacaftor, opens the gate in this chloride channel, allowing it to function. It doesn’t treat the symptoms, it treats the disease itself, turning a non-functioning protein into one that works normally. It effectively converts you from somebody with CF to somebody without CF, although still with the lung damage brought about by years of having CF. But this is a much better state of affairs.
In trials, people have had average improvements in lung function (FEV1) of around 12-15% – this is enough for some people to come off oxygen, off the transplant list, start exercising again, and enjoying a full life.
But the NHS in England still haven’t reached a decision on whether to fund this drug. In the meantime, the drug company, Vertex, have instituted a free access programme for patients whose lung function is poor, allowing them to get the drug free for as long as it is needed. Your FEV1 had to be below 40% for six months – and mine wasn’t. It was hovering around 40%, but after recent IV treatment it went up to 47%, just for a few weeks, so I felt I wasn’t eligible and didn’t want to ask for it, in case I was turned down.
However Stephen decided otherwise, and asked for my case to be considered. On 26th November, I heard the amazing news that I could have this treatment, and I took my first pill on 29th November. And the effects have been amazing. The World has Changed.
I noticed it on holiday – I was able to walk further, much faster, and up hills, all at the same speed as Stephen. We went fell walking. I went swimming and managed 40 lengths without difficulty. My chest feels clear and quiet, not constantly inflamed. My racking cough has disappeared. I have more energy. I am not always hungry. I can get showered and dressed without even thinking about it. I can even do a session of spinning on my indoor bike.
I am often asked what it is like to have cystic fibrosis, and I answer that I don’t know, because I don’t know what it is like NOT to have cystic fibrosis, so I have nothing to compare it with. Well, now I do, and I have to say that I definitely prefer NOT to have cystic fibrosis! If this is what it feels like to be normal, then bring it on! The World has Changed for me, and I am extremely grateful. I have been given my life back.
And the woods? Well, I hope that a rejuvenated me will be launching vigorously into the winter work very soon now, able to play my full part again in the management of the woods, all the activities that we do there, and enjoy being outdoor, and free, in a beautiful wild place again. Now that really is worth it!
We are expecting an announcement on whether ivacaftor will be funded for all patients with the G551D mutation on 18th December in England – decisions in Wales and Scotland will be later. I hope all patients will be able to benefit from this amazing, life-changing drug.