#KaftrioNOW: The Buzz Tag For Cystic Fibrosis That Is Trending On Social Media
Back when a US based medical science company, Vertex Pharmaceuticals took a big leap of faith by investing time into developing orphan drugs for rare diseases such as cystic fibrosis (CF), little did the world know just how life changing their modulator seeds sowed would be.
Recently, the newest Vertex addition, a triple combo CF modulator known as Trikafta was approved for licence by the European Medical Agency (EMA) under the new brand name, Kaftrio and, the big wide web is blowing up about it.
A Wee Recap On Cystic Fibrosis
CF is an autosomal recessive disorder involving the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which causes complications in the lungs, pancreas, and other organs — basically clogging them with thick, sticky mucus.
It eventually eats away at our pretty precious, pomegranate shaped air sacs. You see, when your airways are filled to the brim with sticky mucus, it glues down those super fine hairs in our lungs (cilia) and creates a breeding ground for bacteria to thrive. Fun.
It is really hard to explain CF in just a couple of paragraphs, there is a lot more around salt distribution and keeping airways moist so if you want delve deeper into the science behind the huffers and puffers of the world, click here for a short video created by Demystifying Medicine.
What Cystic Fibrosis Treatment Looked Like Without CFTR Modulators
Up until recently, all the medications used in the management of cystic fibrosis care are only designed to treat the symptoms of the condition. As the underlying cause is genetic, the progression of lung disease can not be stopped by vitamins, antibiotics, steroids and pancreatic enzymes alone although, here’s to trying.
Every CF patient is an individual and their needs will differ slightly but generally speaking, many patients are custom to:
- Taking endless tablets
- Toxic IV antibiotics
- Puffing on electronic nebulisers (our alternative to an e-cigarette that acts as like a modern, steamy sterile salt cave — sexy)
- Enzymes to digest food
- Insulin injections to control blood sugars
- Inhalers
- Non-invasive ventilators to push air into the lungs
- Oxygen supplementation
- High fat dietary drinks or artificial tube feeding
- Hours of grueling chest physiotherapy; every single day.
CF Modulators; Changing The Genetic Face Of CF
The new colourful, little pills being developed by Vertex, are known as CFTR modulators and they work on a genetic level with specific gene types to correct the CF cells thus making the lungs work more effectively.
With the new drugs, we are in a position to change the face of CF. We stop just treating symptoms and we start altering what is genetically wrong below the surface. These tablets are literally capable of building a genetic gateway within a cell. Mind blown.
MEET THE MODULATORS:
- Kalydeco (ivacaftor)
The first to emerge was Kalydeco however this drug only worked on a small select group of rare CF genes (4–5% of CF patients).
- Orkambi (lumicaftor/ivacaftor)
Next was Orkambi, but the results seen in the trial data were underwhelming with small increases in lung function FEV1 of 2.6–3%.
- Symdeco/Symkevi (tezacaftor/ivacaftor)
This was what some might call a combined, enhanced version of Orkambi and Kalydeco and it provided much better trial results — mean value increase up to 6.5% in lung function (FEV1) but it only reached 40% of patients with the correct gene type.
#KaftrioNext
The Triple Therapy: Trikafta/Kaftrio (elexacaftor/ivacaftor/tezacaftor)
The biggest and most promising break-through came with Trikafta/Kaftrio, also known as the triple therapy. Vertex developed Trikafta as a improved version of previous modulators and it targeted 90% of over the 2,000 CF genes alongside exceeding all expectations with the clinical data results.
US TRIAL DATA FOR TRIKAFTA*: Impressive lung function (FEV1) increases with mean values of 13.8% in the first trial which consisted on 403 patients and 10% in the second trial which was a smaller controlled group of 105 patients. Improvements in sweay chloride. Significant reduction in chest infections.
Trikafta was approved by the Food & Drug Agency (FDA) for use in America, in October 2019.
The Proof Is In The Pudding
The success stories being shared globally by CF patients has been substantial and impressive. My personal favourite highlights include US CF patient, Josh, 11 (above), blowing lung function numbers he hasn’t seen in what felt like a lifetime. When I caught up with his Mum, Marissa, she said:
“No longer terrifying to think about all the what if’s that could happen. He also plays outside after school everyday now, which is amazing in itself. He used to hate outside, it was exhausting.
This is one of many victorious stories arising because of Trikafta/Kaftrio. I hve seen CF patients who are waiting for new lungs being withdrawn from the transplant list. Incredible. This drug gives us a chance to slow down the ticking time bomb.
This drug will make “average life expectancy” a thing of the past. CF vlogger, Stephi Lee, shares a candid review of her experience with Trikafta/Kaftrio and what it means for her future, below.
Currently Kaftrio is only available on compassionate grounds for UK patients who are nearing end of life care and out of other available treatment options.
So, It Is Getting Licenced, When Will All UK Patients Get It?
That depends on how well the funding battles go. The licence welcomes the opportunity for all EU countries to buy Kaftrio, some countries such as Ireland and, Denmark already have a deal in place and are awaiting the go ahead.
The UK are yet to make a deal for this new life saving medication, there is a lot of angst among the CF community as Orkambi and Symkevi approval was what some might call a shambles.
Change.org partition to support this can be found here.
We Need To Take Action Now
The National Institute for Health and Care Excellence (NICE) were due to make a decision for UK access to this supercharged triple combo, Kaftrio, in October 2020 but it has already already been pushed back the to September 2021.
We can not afford to lose any more time on this so we continue to be trailblazers among the big pharma politics by campaigning to the UK government and striving to gain nation wide backing for #KaftrioNOW until an imminent deal is made.
Help get a leg behind this campaign by following the link here to the CF Trust website which has an easy step by step guide of how to contact your local parliamentarian, including letter templates and additional information.
You can also show on-going support by keeping up to date with latest news and campaigns on the CF Trust website here or, follow #KaftrioNOW on Twitter and other social media handles.
NHS Egland Vs. Vertex: What We Learnt
It took 4 long years to get Orkambi and Symkevi approved by NHS England. We waited decades for this breakthrough but it wasn’t smooth sailing. As much as I appreciate the risky research, cost and time invested into bringing these new drugs to market safely; the very public battle between Vertex and NHS England (NHSE) was excruciatingly painful to endure.
The National Institute for Health and Care Excellence (NICE) arguing that these modulators were not or, are not cost effective, whilst young people are still suffering or worse, dying from CF felt inhumane and highlighted how outdated the UK protocols around accessing new medications are.
This charade demonstrated the desperation for NICE and NHSE to put a system in place that allows certain drugs to be fast tracked and made available to patients before it is too late.
The current systems that are being used are limiting support of advances in medicine and, creating inappropriate obstacles.
So, that just about encapsulates all there is to know about #KaftrioNOW and why it is trending on social media.
Watching and reading about lives changing daily is not enough. It is time to take action and change the face of CF once and for all.
*All CF Modultor clinical data sourced from the FDA website. Views my own.
