Cystic fibrosis breakthrough has given patients a chance to live longer

He would take his enzymes, do the vibrating jacket treatment. He graduated and got a full time offer from a nice company. As he was relocating, he picked up some infection and passed away in a few weeks. None of us expected it. This was months before I heard of the approval of Trikafta.

My personal situation is probably pretty unique, in that I have CF as well, but had a double lung tx many years ago. A couple years ago my transplant team (actually it may have been the CF people.. fuzzy memory) asked me if I was interested in getting on Trikafta and after thinking it over for about 30 seconds I told them I'd probably pass. It seemed like it wouldn't have any beneficial effect on my lungs, as they no longer "have CF", and that I assumed it would just make extra weight for my decently healthy but still under par lungs to have to drag around. They didn't disagree.

>One side effect I should mention: many report extreme anxiety. However, I was starting an anxiety medication for the first time (something I should have done 20 years ago, but alas ...) and so those effects were muted or hidden to me.

I would have imagined that most people with CF already had extreme anxiety - wondering when the infection that is going to end one's life will arrive. Is it really possible that Trikafta is causing noticeably worse anxiety?

This seems like a no-brainer way to treat obesity, by restricting or blocking such enzymes. Obviously the downside is diarrhea and other malabsorption symptoms, but obese people have too much nutrition. Blocking absorption seems easier than dieting, as no willpower required. You shit out the undigested food.

Hearing about any kind of advancement is huge. I can't even begin to understand what you've gone through, but am so happy to hear there is a new medicine to help with such a cruel disease and that it's made a world of difference to you. Sometimes it feels like medical science is still in the dark ages and I get saddened that we haven't got flying cars or Dr. Beverly Crusher can't wave some device over me and fix all the ailments I now have. Other days I hear about these kinds of advancements and it makes me smile!

So I just wanted to say: it was an impactful piece of writing, enough so that I remembered it quite clearly after a single read these few months ago.

Interesting. I'm an uncle to a family member with CF (and I'm a carrier) and one of their long battles has actually been weight. To the point of doing things like downing olive oil to try and keep up the weight. They've eventually had to take the fairly drastic measure of getting a feeding tube installed. Getting enough calories in just hasn't been possible until the feeding tube.

It seems like something every day reminds me of him. I’m really glad there’s hope for people with it. It’s a particularly shitty disease and he was the nicest most thoughtful person I’ve ever known. I miss him dearly.

Bittersweet. Glad there’s progress, wish it were earlier.

Unfortunately, that's not guaranteed to bring a price drop. Humira is a good example of that; it recently went generic but much of the savings went to higher rebates to pharmacy benefits managers.

Some reading for anyone curious:

https://www.reuters.com/business/healthcare-pharmaceuticals/...

In the online CF communities I'm a part of, I've never heard of anyone who couldn't get access to the medication (aside from the country they're in not approving it) (I'm in the US so my perspective is probably skewed)

But the alternative, without the massive cashflows, is a world where the research never happens, and the diseases haunt us forever.

The Big Pharma treatments will eventually become public domain, and may inspire other cheaper treatments prior to that.

The fact that these conversations come up frequently is a symptom of the fact that Big Pharma is frequently doing fantastic things. Curing diseases that have plagued humanity for millenia and killed millions.

We have a very bright future. But the future is not free of charge, as we'll continue to require a lot of medical research, and medical researchers have families to feed, like the rest of us. Medicine costs money - there's no escaping it.

In the UK, we sometimes mistakenly imagine that our socialised healthcare system is "free." It's not. It's one of the most expensive in the world, and employs over 2 million people. The component of tax that I paid to fund the National Health Service was £8919 last year - the largest single expenditure of my taxes, and vastly more than I'd need to spend to get private healthcare.

So while American medical pricing is scary and sometimes ruthless, I think we need to put things in perspective and recognise their leadership in medical research - without which, we'd all be in a worse situation.

Since Trikafta is a blend of 3 small-molecule drugs, is it possibly feasible to publish a do-it-yourself synthesis process?

Or perhaps the community could even come together to set up a lab, where individuals could travel and go through the motions to produce their own?

I recognize this isn't ideal, but it might be legal...

> Or stopped being covered by insurance? Trikafta’s sticker price is more than $300,000 a year. Insurance typically covers most of that cost—minus what can be significant co-pays and deductibles—and Vertex offers co-pay assistance. But patients’ lives ultimately depend on decisions made by nameless bureaucrats in rooms far away: Insurance plans can suddenly change what they cover, and in 2022, Vertex announced that it would substantially reduce its financial assistance.

They dragged it until he was 18. He always jokingly said "make a wish foundation is waiting, hoping I'll die".

He finally got to go Zorbing when he was 19. Passed away a few years later.

Not surprised the foundation jumped on Trikafta to remove eligibility.

> Recently, Make-A-Wish announced that children with CF would no longer automatically be eligible for the program, because “life-changing advances” had radically improved the outlook for them.

I know this is a long piece, but half the comments in this thread seem like people didn't even bother to read it before commenting...

Doctors told me they could think of only one other comparable breakthrough in recent memory: the arrival of powerful HIV drugs in the 1990s. Like Trikafta, those drugs were not a cure, but they transformed AIDS from a terminal illness into a manageable chronic one. Young men got up from their deathbed, newly strong and hale. ... This was a remarkable turn of events. But it elicited a complicated mix of emotions, not all of them joyful. Some patients who were no longer dying grew depressed, anxious, and even suicidal at the thought of living. This phenomenon became known as “Lazarus syndrome.”

Death is an end, after all. Life comes with problems...the writer Andrew Sullivan, who is HIV-positive, described life after the advent of the HIV drugs in his essay “When Plagues End”:

   When you have spent several years girding yourself for the possibility of death, it is not so easy to gird yourself instead for the possibility of life. What you expect to greet with the euphoria of victory comes instead like the slow withdrawal of an excuse. And you resist it.
   The intensity with which you had learned to approach each day turns into a banality, a banality that refuses to understand or even appreciate the experience you have just gone through.

Well, people are people. Somehow the life of a 90 year old is less worthy that than that of 20 year old. As to how they arrived at the magic number of 90 remains a mystery to the fictitious society where individuals routinely live for 9000 years.

I wouldn't use that analogy. It's more like breathing in a deep swimming pool or a very humid sauna. Slight physical exertion (like stairs or hills) can be like most people doing strenuous exercise. Laziness tends to be a way of life :-)