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.
That's absolutely incredible. Don't get me wrong. It's miraculous from a clinical standpoint. But, uhh. Not to be a downer but I need people to see this so they stay angry and stay real about what medical breakthroughs actually mean for patients. When I call something "survival gatekeeping", this is what I mean:
That is per month with the most common coupon people are likely to use.
But don't worry, there's grants and patient assistance programs you can apply for. 🤞🫠 Most people in high income countries like the USA can get it "covered" through insurance for fewer thousands of dollars. Or even less if your insurance is good or the manufacturer likes how poor and/or on Medicaid you are! A good social worker will help you with the process, and make sure your yearly reapplications and PAs are done a little early so they have time to think about it before you run out. Jesus Christ.
NEVER look at something like this and navigate away feeling better about things without asking how much it costs and who can get it. NEVER. It isn't revolutionary until poor people can access it without a struggle.
