It isn’t often that Cystic Fibrosis gets to hit front-page news, but for a while in 2019, we did. After the production of three modulators with low success rates came the triple-combination modulator that would revolutionise our world; otherwise known as Trikafta in the US, or in Europe as Kaftrio. In laymen’s terms, the last recipe was… mix most of’em together, slap another one on top, and call it a day. And it rallied the crowds.
Whether it be Trikafta or Kaftrio, I hope your faith in this deity is strong, as my belief in it crumbled a little with every bit of research I did to write this piece. If you haven’t heard about it, you’ve been living under a rock. And if you have heard about it, chances are you are of two minds. Either you believe this is what will save the CF community from burning in the fiery pits of lung failure. Or, you see it as a legend that’s quickly becoming a tall tale.
The hype behind this therapy is pretty simple; it’s the first time we have had pills that correct the basic functions of the CFTR protein (the faulty gene that causes Cystic Fibrosis in the first place). Trikafta or Kaftrio, depending on where you are in the world, is that triple-combination modulator.
It is this incredibly non-invasive and – supposedly – very efficient treatment, that almost regenerates the whole body, before giving it a newfound breath. So far, it’s been shown that as soon as the magic pills are stopped the functional fantasy world slips away. Definitely becoming a treatment and not a cure.
The science: there are two morning pills that contain elexacaftor, ivacaftor and tezacaftor; binding agents that work with the faulty protein to help it get to the top of the cell membrane. Correcting this issue is a big deal, as it plagues a big percentage of the CF population. The evening pill and second part of this treatment is an ivacaftor pill, otherwise known as Kalydeco. What this molecule does is help the CFTR protein stay open longer at the surface.
drawings by Tristan Castella
All of this means that, technically, this drug wouldn’t just affect one part of the body. But instead, it would target the source of the problem that created this incredible domino effect of sickness. It promises a lot, but can’t assure much.
Interestingly, Kalydeco was the first modulator, created back in 2012. However, it only helped 6% of the CF population so it did not go very far. And now, it’s having a comeback as the VIP. Since 2012 there was the creation of Orkambi in 2015, and the creation of Symbdeko in 2018, before this final revolution that seems to have stolen the spotlight.
And that is why I am curious.
I resisted both Orkambi and Symbdeko but felt somewhat childlike and scrutinised when I hesitated to accept Kaftrio. In my doctor’s eyes, there was truly a sense that the bad outcomes were a 1 in a million chance. Perhaps it had been the eight years of experience with modulators, or maybe it was my doctor’s lack of patience in waiting for me to be ready for this logical next step that was quickly becoming the status quo.
It’s true, my objections are because I worry about the worst-case scenarios… but a lot of my worries actually are about the worst-case scenarios doctors haven’t dealt with or even met yet. The fact is, we still know so little about it.
What You Can Hope To Change…
Welcome to the purge. CF Style.
Or maybe not. Who knows. Because that’s how medicine works nowadays.
But more specifically; this triple-combination modulator targets to change the mucus we create. Turning something sticky into something friendlier to the body. Many people have said they have had a purge-like episode that lasted anything between hours to weeks, where they coughed or snorted all the excess shit that’s been holding them back.
It is difficult to quantify or even pinpoint exactly where this medication could change our bodies and consequently, our lives. And that is because of our illness structure itself. CF is a domino effect of symptoms and collateral damage from years of intense medication mixed with genuine dysfunction. But the glimmer of hope that is Trikafta, is that it changes our molecular function. It’s not gene therapy – the utopic cure – but CFTR modulators are treatments that target the root problem.
So, even if it isn’t a cure, it has the potential to help a multitude of problems. What is scary about this medication is exactly that; the fact that it could effect anything and everything.
For CFers, our primary battle is with this stuffy mucus that ends up clogging our bodies over time. So, if this medication allows a different transaction to happen between our cells, then that mucus itself could change. I’m not a doctor, but in my humble opinion, I don’t think we realise how much this medicine will change our bodies.
The point is; this treatment is an opportunity. Not a miracle.
The politics behind Trikafta are an important part of this story because I wanted to note how challenging it was to be someone who wasn’t convinced and asked a few too many questions in face of this life-changing event.
In preparation for this post, I did some research and found other people’s testimonies about how they had lived this change in colour. That’s when I realised I wasn’t alone in my worries. But more importantly, I found that I hadn’t been the only one to be shut down either… by both doctors and other CFers. It broke my heart to see there wasn’t a safe space to express our worries about gambling with the little bit of health we had left… all of this simply because propaganda is powerful.
I kept getting angrier and angrier with every new bit of reading I found on Kaftrio. It was portrayed as this full-proof make-over pill that made us normal. And that is just wrong on so many levels. Headlines read the words “game-changer”, “transformative”, “life-changing” and even “miracle”. Which, just to be clear, is laughable. Any doctor knows that uses the word miracle is just sensationalism. They know that that word has no hold in science or medicine. And to say something like that to a community of kids that have been dying since birth is gaslighting if even remotely untrue.
and it is untrue
Trikafta only has a slightly higher success rate than previous modulator and the possible side effects are far greater. Far darker too. So, why such noise?
Because this modulator has the biggest reach in our community. And for a small community divided by over 2,000 mutations, finding a united treatment is a big deal. Regardless of the treatment itself. And that is what we should remember.
The average lung increase (FEV1 in our charts) with this medication is around 15%, from what I could find online. Let’s unpack why that is not life-changing.
The fact that the average is that low is surprising because there are some patients whose lung function goes up by 40- 50%. So let’s just take a second, to consider how many people only had a 5% or less FEV1 increase – that’s if we’re not even mentioning the people that didn’t see a change at all – to balance out the ones who had such a remarkable jump and made such noise.
The second reason why this medicine does not qualify as a game-changer is that despite supposedly resolving our lung issues, the medication comes with clear instructions not to stop respiratory therapies. It says it is up to our personal physicians to make that choice after seeing what happens. So, just to put it in black and white, this treatment is just another pill in my pillbox. The way the treatment is managed and handed out is not in a way to replace already existing therapies.
Another interesting thing I came across was that a specific possible side effect of Kaftrio was “upper respiratory infection”. I found this shocking and surprising seeing as the point of this medication was to prevent said infections. Doctors claim this is just medical talk for little colds. But I wasn’t convinced… so, getting a little bit more curious, I checked on the other modulators. Assuming that America always listed an enormous list of side effects as legal offense, I was expecting to find it on the other modulators. I only found it on the Kalydeco list; which makes sense considering we are talking about Ivacaftor and it is also in Trikafta. I’ve been fighting colds turned lung infections since birth naturally, and now I will be fighting them because of my medication? Fab.
How is this life-changing again?
The Unheard Truths.
Amidst all this golden smoke, we have lost important bits and pieces of the story.
Patients are noticing more creative side effects, that either wouldn’t typically be associated to CF issues…
despite there being so many options to choose from
… or symptoms that aren’t obviously linked to what is known about Kaftrio. And when spoken of, there seems to be a strong wave of denialism within our care. So much so, our Facebook group created a poll to keep track of how often and how many things were being brought up.
I haven’t been around the Facebook group for long, but it’s the first time I’ve seen a poll to map what’s happening to us medically. Or more importantly what’s being pushed aside.
The leaflet that came with my Kaftrio has an impressive warning list already, but in this post, I also wanted to bring out of hiding the list the CF community created. The ones who are on Facebook at least. It deserves to be heard by people who don’t have CF. As we all tether on that line of hypochondria, however, I will be grouping the poll choices together to make it easier to understand. I think showing the truth about our erratic thinking might just scare you all off.
Emotional & Personality Changes
To start off on a sensitive note; here I grouped together anxiety, depression, restlessness (sometimes even to the point of not being able to sit down for more than a few minutes), increased anger and/or spikes of irritability, and finally insomnia. There were also quite a few votes for a category quite simply named “weird dreams”. Which I thought was unpleasantly threatening.
As far as I know, doctors are simply blaming these issues on the global pandemic or seen as an expected consequence of our illness. Ignoring the fact that CFers are well aware of their baseline going into this treatment. Not acknowledging, that perhaps this meant they had fallen into a deeper rabbit hole that was unusual even for them.
think of it as our tolerance for darkness is high… so, our complaints shouldn’t just echo as empty laments
There are quite a few testimonies that expose the extent of this emotional hurricane and those are the people who said they had suicidal thoughts because of this treatment. And it is not as uncommon as you’d want it to be. The reason some feel that Trikafta is the source of the problem is that upon stopping it, things changed back to what they had once been. Some were able to pick up the treatment back up at a later date, others weren’t.
This is a big category, big because of its importance in regard to how dangerous these side effects could be. But also, important because I have yet to hear a doctor admit they’ve heard ANYTHING about this. The reason I’m putting it in quotation marks is because CFers have named these “neurological symptoms” but it’s to encapsulate many things we don’t understand and can’t explain.
Symptoms have been described as a brain fog or a feeling of having been slowed down in your own thought process. Others feel it as forgetfulness and memory issues. Some describe it as having forgotten their words mid-sentence. Reporting an actual inability to communicate and think sometimes. Which inevitably affects our ability to do take care of ourselves too.
Muscular & Physical Effects
Surprisingly, “increased fatigue” was high on the poll. Because the medication is intended to allow us to breathe better to then become more active, it seemed illogical to me. This umbrella term included arthritis, which was somewhat common pre-Kaftrio. But then again, this point could be taken as a symptom that was present before and has now gotten worse. There were also a few votes for tremors, ringing in the ear, and general muscle weakness. Building to more dangerous symptoms, like swelling of the legs and arms.
Some people even noticed hair loss, whilst others noticed complete hormonal changes that messed up menstrual cycles.
Lastly, there was a poll option simply named “general body pains increased”. As I’ve stated before, we love being extra, but my heart broke a little for the people who voted for this one. Because it was so easy to understand what they meant and what that statement signified. If you looked closer… you would see it was a resignation. A resignation to find where the pain starts and where it ends.
Whether it had become a maze or a distorted watercolour painting… sometimes we can’t make sense of our pain because it’s just everywhere.
Effects on Chronic Illness
I created this sub-group for the symptoms and issues that were already frequent and typical to CF before Kaftrio, but are still described as having gotten worse or harder to manage. Issues like constipation, night sweats, water retention issues and dehydration, oily stools, increased reflux, and sun sensitivity.
When something happens almost daily, you need to trust the person you’re talking to when they say that the messed-up thing actually got worse. Just like we trust the doctors to tell us what to do. Sometimes it’s about awareness and feeling, not knowledge. Sometimes it’s just a gut feeling that forces you to start drawing an x on your calendar when something feels off. Confirming that something has in fact goten worse only with time .
Other side effects that are perhaps more troubling include new volatility in blood sugar, higher blood pressure, and even heart palpitations. I remember at the end of 2020, a few posts even appeared talking about strokes. It was believed to be a possible turning point when hitting the one-year mark of being on Trikafta. Even if no doctor has acknowledged this either, I was happy to know about it.
What is not being taken into consideration truthfully, is the life people may have when on this medication, in the other sense.
More than one person voted for each of these symptoms and side effects. And in no way was this poll compulsory! I showed you this side of the coin so that somewhere on the internet, people could find the truth about what it’s like to take this medication.
The difference between giving or taking Trikafta is that all of these things – even if some are slight in intensity – start to weigh on the body and mind when they accumulate. So, even if there is that lung function increase and shorter antibiotic treatments, it’s still worth asking how the rest of the body is doing. And yes, if it is actually worth taking.
I really wish that the medical world didn’t have to deny any truths to claim that this medication was good. Just like I wish it didn’t have to claim to be life-changing to be good. It feels like a sort of eyes wide shut situation to get as many people to take it. There will always be more medical intervention to prevent treatments from truly negatively affecting a patient’s health. But I’m not sure doctors would put that much effort into describing all of our self-assessed symptoms to colleagues. Or perhaps it is Vertex that is refusing to acknowledge more symptoms.
Who knows. But I have asked my doctors about these possible consequences… and no one knew what I was talking about. No one had even heard anything.
It has potential.
However, the reality is that we are just in the second year of commercial use. Side effects are being denied or blamed on things that are outside of our control. Which will always be easy with a chronic illness in case anyone was wondering! We don’t know what will happen in the long term. Will improvements plateau, like with the other modulators?
Vertex has been trying to make the perfect modulator since 2012, so why is it acceptable to label this treatment a miracle? A treatment that achieves on average a 15% lung improvement? A medication that creates whole new problems? Problems that are truly frightening if you were to imagine it happening to your body. But most importantly, not all CFers can take it. Why is that a miracle drug, for us?
One of the articles I read actually quoted Doctor Leiden (a Vertex biologist) saying that the birth of Trikafta meant he had conquered a disease. And oh boy, was that triggering.
No, you did not.
stay in your lane.
Coming from an educated man that should know better, this statement truly seems to have come off an ego high. Because who the fuck are you to say that? If you haven’t lived a day with this illness, you haven’t conquered anything. Never experienced our battle and fought for your breath. For your life. This isn’t your battle so don’t claim our struggle as your victory. You are no conqueror.
Especially considering your medications is average, sir.
now, putting the guns away…
I wanted to write this post before I started my own Kaftrio treatment because I wanted to remember how I walked into it. It made me sad to see other CFers or families of CFers get aggressive with people who were questioning this new treatment. Because I wish they understood that questioning something isn’t the same as an accusation.
When I had objected to these treatments in the past, it was blamed on my lack of perspective. As belittling as it was, doctors allowed for my decisions to reign. But in 2021, when asked why I wasn’t taking modulators, my being healthy enough to not want to risk was not an appropriate answer anymore. It was met with such a blank stare that it made me wonder if I had mooed at them.
Truthfully there was no discussion. Kaftrio was on the table now. The ultimate modulator. The UK had been waiting to catch up with the US for a year, so what the doctor found to tell me was “well, we usually get people begging us to get them on the medication. So, this is new.” As per, she was more amused at getting her way than concerned about my feelings.
All of this indoctrination, in just one year and a half, is truthfully impressive. But with everything going on in the world on top of our usual fighting, I just don’t understand why hesitation is so surprising. Despite the truth being that plenty of us are willing to take it… plenty of us are also worried and hesitant. And I’ve seen that. I’ve also seen people decide that the bad side effects weren’t worth the boost and they stopped. Even if the doctors wish they hadn’t.
So, my real and honest question is… is this simply a good medication, that the doctors and pharmacists are treating as a golden ticket?
Regardless of what our certified doctors said, the good Samaritans of our community took the time to warn us. The whispers of a crowd can be powerful. And it’s a shame, for everyone, to turn their nose down on it just because they can. It’s the snobbism of the medical world.
My Pandora pills arrived just as I finished typing this up. I only realised how tense I had been that morning after my Kaftrio was delivered and in my hands. After having put my whole day on hold to be there when the doorbell rang, I forgot how quick it was to pick up a parcel and just start freaking out.
I can’t say I’m excited and it’s because I am playing offense right now. Without focusing on who or what, I felt pushed into this medication. A medication that feels different – more aggressive – and in a time where chronically ill patients are not getting the care they usually do. It sucks that we’ve been waiting for a new treatment to change something for us for over two decades and that it came out just a few months before a worldwide pandemic. It’s understandable that some of us are in a state of hypervigilance.
But hey, I’ll be happy to be proven wrong on this one.
subscribe below to receive our next post directly into your inbox!
please leave a comment, like or share!
it would be greatly appreciated!
or if you would like to read more…
As someone who spent her life in and out of hospital consultations without truly getting better, I wanted to explore the idea that medicine and health are a fad. This is a tiny piece of the puzzle but it is where a discussion can start.Keep reading
After 17 weeks of being cooped up in my home, I have finally stepped outside again. But not for a simple walk, more like a very long car drip across the Channel and France. I have finally gotten to Switzerland, and the trip was so different to any I have gone on in the past.…Keep reading