Sisters ❤️

I will scream my lungs out till it fills this room

Today NHS England finally came to a deal with the pharmaceutical company Vertex that means the 90% of CF patients who have the most common CF mutations will have access (within 30 days) to the first drug that they will ever take that treats the underlying cause of the disease, rather than only its symptoms. Read more here. 

Orkambi, Symkevi and Kalydeco are the first iterations of these small molecule drugs that will limit disease progression by allowing the normal movement of salt and water between cells. These drugs are not a cure, and the long term positive impact of taking them is not fully known, only time will tell, but even newer iterations of these drugs (like Trikafta) are already known to have a more dramatic impact and should be licensed here soon too. Today’s deal should pave the way for these future treatments. For children born today, the hope is these drugs will halt progression before the irreversible damage to their lungs ever occurs. 

This news (after 4 years of intense and passionate campaigning by people with CF and their families) is long overdue, and very, very welcome. 

Isaac is not a candidate for this drug, or others in the same pipeline. Unfortunately he is in the 10% minority that this drug will not work for (see blog below which explains why). But there is other research ongoing and pending clinical trials that may work for his rare mutation, as well as other drugs that are not mutation specific; but these are frustratingly a few years behind. We need to keep him well until these are available, so we continue to fight the progression of this shitty disease. 

If the momentum behind the research seemingly slows, then you will hear my screams. #don’tforgetthe10% 

Today is a very happy day. Next week, we start IV antibiotics again in the next round against the bastardly bugs camping in his lungs x 

I will light the match this morning so I won't be alone
Watch as she lies silent
For soon light will be gone
Oh I will stand arms outstretched pretend I'm free to roam
Oh I will make my way through one more day in hell
How much difference does it make?
How much difference does it make yeah
I will hold the candle till it burns up my arm
Oh I'll keep takin' punches until their will grows tired
Oh I will stare the sun down until my eyes go blind
Hey, I won't change direction
And I won't change my mind
How much difference does it make?
How much difference does it make?
I'll swallow poison, until I grow immune
I will scream my lungs out till it fills this room
How much difference?
How much difference?
How much difference does it make?

How much difference does it make?

Pearl Jam. 93. 

Not having IVs, and time.

Despite a funny week where Isaac’s chest has been up and down (interspersed with some delightful vomiting episodes) his lung function at clinic was surprisingly stable. This is good news, and gave his argument to delay IV antibiotics some pretty punch. In the end, his consultant agreed that he can delay, but only by a week or two. This means our plan to time this extra treatment over school holidays is scuppered (he would miss less school had they been over half term, and we would enjoy a calmer week, balancing treatment and life, as I am on leave for half term anyway). Instead, we’ll being having them very soon anyway, with the added stress of super early mornings before the school rush. 

But I get it. He deserves to enjoy his half term. And while it is a bit scary for us, he does need to be more active in decisions about his own care. We hope now that he stays well enough in the interim that home IVs, when he does start them, are still an option, because if he is not well enough we will end up on the ward again, which no one wants. 

In other news: This week has been filled with incredible news for the CF Community (not that you would know it here, while Brexshit is going on) as a new wave of drugs are gaining approval around the world that treat the underlying cause of CF, and have the potential to dramatically change the course of Cystic Fibrosis in up to 90% of patients. I cried hearing that the US FDA have now approved Trikafta, the stories I have heard from people already on this drug and have felt the impact within mere hours or days (from a pill!!!) are pretty frickin amazing. Each iteration of these small molecule drugs is proving remarkably more powerful than the last. Given that treatments in CF tend to be of a more time or medically intensive type (multiple daily nebulised drugs, and frequent IV antibiotics), the idea that an oral drug each day can have such an impact is pretty mind blowing.  

Then I had a good cry last night, thinking of all those sufferers lost (friends included) that this has come too late for. And I must admit, that as Isaac is in the 10% of patients that these drugs will not work for, a renewed scream for us; the last 10% must not be forgotten (Isaac’s mutation is better explained here.) 

I am so filled with hope, anticipation (mixed with a smidgen of distrust and worry) for what might come for us, and (crucially) when. But bloody hell.... I know this is not a cure by any means, but it is definitely the closest thing to a cure the CF Community have ever come close to! 

So we wait in exciting times. 

It has been 15 odd years now since these kids of ours exploded into my heart... lately I’ve been feeling that time has suddenly seesawed such that I have less time left with them as kids than I’ve already had. I am a sentimental fool at the best of times - but the swing from wishing for more sleep and less worry that they will choke on a grape, or lick a slug... to ‘will they even want to hang out with me much longer?’ has swung too suddenly. 

If you have little ones, enjoy them while you can guys x 

Anouk. 

Rosa (insisting that her baby cardigan still fits as a crop top).

And Isaac. Since he hardly lets me point a camera at him these days, enjoy a mini-Ise x 

Rebel and flu.

We had a double whammy hospital trip yesterday.

Dan went in for a procedure I do not know the name of, but sounded something like cavity wall insulation to me. He had thrombosis earlier this year, which was treated, and then came back, and so they squirted a kind of foam into the now rubbished vein to stop it occurring again. Our total reliance and ongoing adoration of the NHS knows no limits..... right now he is also waiting on a hernia op and knee surgery! 

At the same time Isaac went in for the lineogram to check his portacath, which as suspected revealed he has a *fibrin sheath. The test involved his port being injected with dye while a CT type scanner zoomed in close above his chest him. I stood there in my heavy lead apron, as I always do, making sure that he was OK during the tests. Only now, my murmurs of encouragement are met with a ‘shush Mum’. My not so little boy needs me less and less. 

It was fascinating to see exactly how far down into his chest the port line goes (his port is sited close to his collar bone, but the line reaches near the bottom of his rib cage), to follow the dye moving down the line as they picture it, and to see where it goes wrong. Our consultant still needs to review the images, but initial thoughts are that the port can be used; the sheath means the line won’t bleed back, but the IV drugs are entering his bloodstream properly. We want to use this port for as long as possible, both to avoid more surgery and to prolong the time we use each port-site available to us. 

Another new; chatting to Isaac yesterday about his impending IVs (due to kick off next week) he is now adamant that he doesn’t want or need these. In true teenage style, he won’t really explain his thinking about this, other than...

‘NO, I’m not doing it, I’m fine, I don’t need them’

Only he is not fine. He is not bad, but he is not fine. And yes, we could negotiate with the doctors to delay this course (I’ve left it with Isaac that this is a decision he needs to make with his doctors next week) but I’ve reminded him that delaying further will only increase his risk of getting too sick that the IVs need to happen in hospital rather than at home. Or worse, delaying and he gets so sick and he ends up needing more than a two week course and it drags into Nov, Dec... (Christmas?). A comfortable couple of weeks at home, half of which is over half term where we can just chill - which makes the early mornings, the schedule, the side effects so much easier to manage, seems like the better plan to me. 

But I get it. It means no showers, no football, no swimming. Being tied to a pump. Your Mum coming into your room in the night, rustling in her weird  plastic apron. Waking up to the smell of alcohol wipes. The sore port site. The constant reapplication of tegaderms. The visible lump under your shirt at school. The fatigue. The coughing MORE as a result of the treatment, before starting to feeling better... 

This is the first time Isaac has ever come close to refusing a treatment plan.... yes, he moans at times about the daily grind (six lots of nebulisers, two to four lots of physio and a ton of tablets 365 days a year will do that to you) but ultimately he has always complied after a short grumble, and understood why he needed to (the threat of a PS4 ban usually covers us).

I’m fairly sure we can convince him, or compromise and push IVs back a little, but not much. Plus I know that he needs to become more active in this decision making process as he approaches adult care (in less than two years) and that the push back is understandable... but my worry is he rebels further (from delaying to ‘I don’t need these at all!’). Non compliance is very common in CF, as the burden of treatment is so high. Plus, I rebelled as a teenager. Why would I expect any less from my bright, strong minded boy? I just hope he never stops listening to me. 

One last thought, please consider having the flu jab this year. If you are not eligible for it on the NHS, you can have it at most supermarkets for less than a tenner. You cannot catch flu from a vaccine. It is safe. You are not using up supplies that are better saved for the vulnerable (they will be covered by the NHS). It may save you a lot of suffering. And even better, YOU WILL HELP STOP THE SPREAD which will protect those more vulnerable around you. 

Have a great day peeps x 

* Fibrin sheaths are a heterogeneous matrix of cells and debris that form around catheters and are a known cause of central venous stenosis and catheter failure.