i think we should be talking about the semi-recent advancements in cystic fibrosis treatment like all the time every day. there hasn’t been a drug like this since AZT medications for HIV infection it is truly fucking miraculous and very important
basically: cystic fibrosis is a genetic disease which makes the mucous a person generates extra sticky. it used to kill people in infancy, then with advancements in medical tech it killed people in young childhood, and until very recently cystic fibrosis patients could expect to live until about thirty years old with consistent painful lung infections and complications.
in 2019 the FDA approved a drug called trikafta (which is really three drugs in one) for cystic fibrosis treatment. what it essentially does is patch up the malfunctioning proteins that cause the extra sticky mucus. trikafta is effective on about 90% of cystic fibrosis patients.
people who had spent their entire lives in and out of hospitals, on and off of ventilators, suffering from pneumonia and sometimes treated through painful procedures like intubation took this drug, got out of bed, coughed up an entire lifetimes worth of mucus out of their lungs over the course of a few hours, breathed clearly for perhaps the first time in their lives, and now go on to live well into their seventies.
like isn’t that insane. isn’t that amazing. doesn’t that give you hope for the future of medical advancements and treatment. fuck. i think about it all the time……
There’s a WHAT.
For WHAT.
It's been amazing!
My ward is the respiratory ward - CF is one of the things we specialize in.
Since this med came out we haven't had a SINGLE CF admission to the ward
There used to always be a CF patient spending a couple of months with us at a time
There's a man who is 23 years old who I was sure would not survive his next admission (aim saturations 85% is end stage lung disease)
There's a set of the local frequent flyers that we all know so well
Except
No we don't
On the CF specialist ward (with reasonable staff turnover)
Half the staff have probably never even seen a CF patient
They are going to live
For the people asking "well how do we know people are living that long if it's so new????" Here's a page from the CF foundation about life expectancy.
Additionally, it should be noted that metrics like life expectancy are in no way a guarantee of... Anything. There are significant outlier CF patients who are at an advanced age now despite the odds due to a variety of different factors, having lived the majority of their lives before the development of modulators.
But the fact remains that the odds are better now than they have ever ever been before, by leaps and bounds. It isn't cured, and many patients still need significant treatment in addition to Trikafta, but it is so much better than anyone could have dreamed of twenty years ago, and that is a triumph.
Yes! My sister has a serious form of cf and finally is living a more comfortable and active life. She was also part of many of the clinical trials leading to these breakthroughs due to the nature of her cf. It's been very exciting to see.
SO YEAH HI LMAO I'm a CF patient who's presently taking Trikafta, and I have been since 2022. Before that, I was taking Kalydeco (ivacaftor), which is a component in Trikafta.
These medicines are incredible. My lung function's now basically at healthy-person-without-CF levels, and so's my sweat chloride. I still take pancreatic enzyme supplements, and I still do my respiratory treatments for maintenance reasons, but my overall health is still SO MUCH BETTER NOW. My weight is stable, I don't get anywhere near as many sinus infections, my migraines don't last as long.
Another incredibly cool thing is that they're seeing recovery from lung damage in kids who are on Trikafta and other CFTR modulators - and slight recovery in adults too. My own chest X-rays show that my bronchiectasis has gotten just a little better.
I was born in 1981, and it's wild to see how much the treatments and prognoses for CF patients have improved. Adult CF clinics are a thing now. More and more of us are living longer.
I'll always wish that more of my friends had made it this far, and gotten access to these medications. But I'm so grateful that I have this access, and I'll keep on keeping on in their memory.
