‘A game-changer’: How Vertex delivered on cystic fibrosis

I never knew anyone with CF until college. Didn't even know how serious it was.

First day of the orientation, at our dorm I made a new friend who I later found out has CF. He would take medication and enzymes every day and do his CF treatment with the vibrating jacket. He seemed very healthy and energetic otherwise, except for the occasional cough.

I stayed in touch with him over the four years. I've witnessed him go to parties, sing, study, help his friends. He was always very public about how he has CF.

I remember at one point a mutual friend of ours told me "I really like him, I'd love to be in a relationship with him but chances are he won't be with us in a few years, I am not sure if I want to get myself involved knowing that's what's going to happen". I didn't think too much of it, maybe thought she was worried too much.

This friend went on to get a few great internships, first one nearby so no flight necessary but the second was in Texas, flying from Illinois to Texas a few times, he must have been exposed to some pathogens in during the flights and this guy whom I knew to have had his CF under control for 4 years, whom had his chance to "make a wish" and had his wish despite make a wish foundation dragging their feet for years hoping maybe he would die before getting his wish, as they didn't expect him to live this long to begin with, he basically worsened and passed away in 4 days, literally a few weeks after graduating college.

Not sure if the recent advancements would have helped but whenever CF is mentioned, I think of him.

He lived his entire life with doctors telling him he will die young or he won't live to finish elementary school, high school and then college. He finished college.

The 14% number they give in the article is wrong in a subtle but very big way. They describe it as an increase in volume of 14%, but it's actually an increase in the FEV1 score by 14 percentage points. FEV1 is calculated by dividing the volume of air you can expel in one second by the amount they figure a normal healthy person could expel, based on your height, weight, and probably some other factors. So if you expel 50% of what a healthy person could, then your FEV1 is 50%. Trikafta increased that FEV1 value by an average of 14 points over placebo, in only a few weeks. That's massive.

I'm 32 years old, diagnosed with CF at 6 months old, and before starting Trikafta at the beginning of November, my FEV1 was 24%. (That's incredibly low. I was in the hospital on IV antibiotics, and we were all set to put me on the waiting list for a double lung transplant, when news of Trikafta came in and put the transplant on hold. At the time, I would get winded just by taking the trash down the hall to the trash chute, and would usually start hacking up a lung by the time I finished walking back to my apartment.) I go back to the CF clinic in just a few days to see just how much I've improved, but judging by just the way I feel now after about 2 months, I would not be at all surprised if I'm now pushing 40% for my FEV1. (And at the very least, I don't expect to hack up a lung after every pulmonary function test, which has been the norm for the last decade of my life.) If I go from 24% to 40%, that would be over a 50% increase in actual air flow volume. That's why this is a game changer.

The other big thing the article fails to mention is that the rate of exacerbations (which usually result in hospitalizations and a few weeks or more of IV antibiotics) dropped by about 60% over placebo. Exacerbations are where you see the biggest and sudden drop in lung function, so reducing the occurrence of those is really key to stabilizing our health.

Lastly, I want to mention that, while this drug isn't yet approved for children under 12, they're working hard on that. Theoretically, if a child were start taking this very early on, it's very possible that they would never experience any symptoms of CF. Unfortunately for me, I've had 30-odd years of CF that has permanently wrecked my lungs and pancreas (probably my bones too), but I still find it crazy awesome to think that I might be the last generation that has to deal with this.

The $311k per year price tag is still an unbelievably large amount, but the article certainly doesn't make any attempt to justify that price tag.

I can’t help but notice the $311,000 price. What if you are not covered by insurance?

If embryos are screened for CF in IVF..what happens in a natural pregnancy? Would it still be legal to terminate the pregnancy?

What about other countries? I understand life changing medications are expensive. But shouldn’t the govt just pick up the tab? Imagine we have universal health care...wouldn’t they just do it?

I have CF and have been taking Trikafta for just over a month. AMA.

Does this drug have a positive impact on the problems of CF other than lung function?

> Van Goor’s fix used cells derived from human bronchial tubes to test the potential of drugs.

Anyone know the back story on this detail? I wonder what cells specifically we're talking about.