The dark side of the Force is strong with me!

The dark side of the Force is strong with me!

Wednesday, 25 June 2014

No Future?

So, yesterday was a pretty significant day in the Cystic Fibrosis community. Vertex pharmaceuticals in the USA announced very promising data from their phase three trials of a combination drug that will treat the genetic cause of Cystic Fibrosis directly. The data claims that if and when the drug should become available in the US and EU markets that it *could* offer suitable patients with the double delta DF508 gene mutation anywhere between a 6-10% (or maybe more) increase in their lung function and see a decrease in the amount of time spent in hospital as it seems to also reduce the amount of exacerbations of CF too.

People with the double DF508 gene mutation make up about 50% of the CF community.

What does this mean? Well, it's a bona fide scientific breakthrough as, since now, all medicines and treatments have been tailored towards treating the underlying symptoms of Cystic Fibrosis - this would be the first drug ever to attack the cause directly. The real world implications of this could be staggering indeed, young children born with CF could now take this drug and live a whole and normal life, requiring much less treatments and time in hospital and experiencing much less serious long term damage to their lungs, perhaps for some, none at all.

And yet, I'm not jumping around with happiness... I don't really feel anything about it. Should I?

Undoubtedly this is a major scientific breakthrough the likes of which have never been seen before and I'm glad that many people won't have to go through many of the horrible things that I did growing up. However, part of me feels angry too. There's a lot of people throwing the words 'miracle cure' around right now, people are elated with the news of the breakthrough, which is completely understandable. There's lots of hope and there's lots of optimism that this drug will lead to other drugs and new treatments and that this whole process could potentially be the first baby step towards an eventual cure for Cystic Fibrosis.

I guess, being the super dooper realist that I am I'm just deeply entrenched in cold, hard reality. If the FDA approves the drug in the US it will probably take between 8-12 months to do, then subsequent approval has to be sought in Europe too which will most likely take another year. Then there's the question of whether the already under strain budgets of the NHS can afford to purchase it. There's all kinds of cost quotes flying round right now, some say in the US it will cost anywhere between $160-250,000 dollars per course of treatment for each patient so it is likely to cost a shitload in the UK.

Another issue is one of what about the people who don't have the double DF508 gene mutation? They continue to wait and hope for a similar breakthrough as that's the problem with genetic diseases like CF - any breakthrough in treatments invariably denies another group within the community as it is just that complex to gain any ground on. Irritating bastard that it is.

My anger is for those of us who the drug offers very little for, the older people with CF who have struggled and fought their way into adulthood. Those of us close, or already on the transplant list, and yes, those of us who have already lost the battle.

Time is always against you when you have Cystic Fibrosis, and discoveries like this are too late for many. I can't help being angry about that. I'm not 100% sure if I qualify for this new treatment *should* it become available, my CF unit are busy attempting to categorise people's secondary mutations if they have them so I'm sure they will tell me once they have assessed the information. I'm more concerned that If I am viable then by the time the drug is approved and if the NHS can fund it what state will my health be in by then? 2-3 years is a lifetime away right now, despite me doing really well and busting my balls to stay that way with another transplant assessment coming up.

Don't get me wrong, I'm happy and recognize the massive importance of yesterday's breakthrough. I just wanted to share some thoughts about the harsh reality of how it will affect the CF community as a whole.

*throws gang sign with hands and struts away from laptop*

1 comment:

  1. Andrew, I was interested to hear your comments on the Vertex drug… Have you heard of the other ongoing trials, such as Bayer's Adempas or Novartis' QBW251 ?