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Cystic fibrosis breakthrough has given patients a chance to live longer

theatlantic.com

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Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#91

Post by Scoundreller »

vrc wrote: post #86
paulpauper wrote: post #44

Earlier quoted context omitted.

weight gain. With CF the pancreas is blocked, so you lack in digestive enzymes, and it's a struggle to maintain a healthy enough weight to battle respiratory infections. After Trikafta, I gained some 30 pounds, and have a big belly on my small frame 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…

Orlistat. Look at Alli — very available to buy. A very common side effect is steatorrhea. Oily leakage/poops. But not like, “oops I gotta run to the bathroom” but like, a tiny bear down and oil shoots out your bum. There is a clear warning on it about malabsorption as well of fat soluble vitamins.

Having taken orlistat in low/intermittent doses, I can say it really matters what you eat fatty stuff with, on top of quantity. A regular "balanced" low-mid fat diet, and you're (well, I've been) okay.

If you eat a tub of ice cream as dinner, you're going to have a bad time.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#92

Post by optymizer »

This is great news and hopefully with time there will be a definitive cure. I found out about CF when we did genetic testing for family planning. It turned out me and my spouse were carriers of the gene. They said about 25% chance that the embryo would have CF, and since I soon learnt it is a terrible disease I did not want to risk bringing a child in this world with CF.

We did IVF instead and to my surprise, 60% of viable embryos had CF, which I thought was unusually high compared to what I had been told prior to the procedure.

We were very fortunate that we did the testing and had successful IVF pregnancies, but so many other families aren't so lucky. I hope advancements in CF treatment will make it a non-issue for parents in the near future.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#93

Post by [deleted] »

nwiswell wrote: post #75
A_D_E_P_T wrote: post #71

Earlier quoted context omitted.

Yeah. In all seriousness, you can buy the APIs directly from China/India and compound them yourself. It's easy and dirt cheap, in most cases. This is typically how underground steroid labs and other illicit pharmacies work. It's in some cases how legitimate compounding pharmacies work. So how do you know what you're getting is pure, or even real? Simple, before shipping it to the US, you send it over to an analytical…

This feels very Dallas Buyer's Club. I assume communities exist on the Internet for this kind of "drug piracy"?

[deleted]

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#94

Post by Aloha »

bdcravens wrote: post #65
Aloha wrote: post #48

Earlier quoted context omitted.

I suspect very strongly at 300k a year, its still cheaper than treating CF via existing means.

Yes, I (CF patient) just had a one-week hospital stay, and right now the (retail) price of that vacation is around $100k the last time I looked at all of the bills. (fortunately I have amazing insurance)

When I saw you mention multiple hospital stays a year, I went "well a single admission runs about 20k" and extrapolated from there.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#95

Post by Scoundreller »

amplicons4ever wrote: post #52
elektor wrote: post #24

Earlier quoted context omitted.

"In 2037 the patent will run out, and the generic price will likely be 90% less." 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/...

You're talking apples and oranges... Humira is a monoclonal antibody (a biologic drug), not a small molecule. Biologics require cell culture systems and a completely different manufacturing process than small molecules, and are very complex drugs. The "generics" (called biosimilars) aren't significantly cheaper because they're simply very expensive to manufacture at scale. Humira or its "generics/biosimilars" will ne…

What I like to say about pharma, especially small-molecule, is that the first dose costs $billions, and the rest a few cents.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#96

Post by qgin »

forgetfreeman wrote: post #3

I had a coworker with CF that started this treatment. Over the course of their first year of treatment, as it became plain to them just how effective it was and what the implications for their long-term survival might be, their behavior changed drastically. Within 18 months of starting treatment they were showing worrying indications of both budding mental health issues and heavy substance abuse. Their marriage, whic…

Imagine if tomorrow, it was announced there had been a discovery that doubled average human life expectancy to 150. There would be happiness, but there would also be chaos. How many people would quit all the things they'd told themselves they were too old to change? The job they were grinding out until retirement. The marriage they'd resigned themselves to? Imagine billions of people getting pulled backwards back across the midlife crisis line, realizing that yes, maybe there IS more to life than "this". The days and weeks after that announcement would be some of the most chaotic the world has ever seen.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#97

Post by Scoundreller »

refurb wrote: post #79
xenospn wrote: post #25

Earlier quoted context omitted.

In other countries this will be covered by the social safety net (that doesn’t exist in the US, hence the need for “good-enough health insurance”).

It’s not. That’s what a lot of people don’t understand. These drugs from Vertex started being approved in 2014. US insurers pretty much covered them from approval. Even Medicaid (health insurance for low income) started paying for it. Other countries? The UK just started in 2020. In Canada? Last I checked only 5 of the 10 provinces paid for it and only if you’re under 18. The US healthcare is expensive for a lot of r…

Looks like every province pays for Trikafta now: https://www.cysticfibrosis.ca/our-programs/advocacy/access-t...

Of course, it's Canada, so you may have private drug coverage and then it depends on province whether you fall back to the public coverage if the private payor doesn't cover it.

> The third-generation of modulators, Trikafta, is a new transformational drug that can treat up to 90% of Canadians with cystic fibrosis. Trikafta was approved for sale in Canada on Friday, June 18th, 2021, by Health Canada for people aged 12 and up with cystic fibrosis and at least one F508del mutation and on April 20, 2022 for those aged six and up. As of September 13, 2022, every province, territory and federal drug program is funding this drug for those six and up. Unfortunately, it is not accessible to all who can benefit from it, and therefore our advocacy work continues

https://www.cysticfibrosis.ca/our-programs/advocacy/access-t...

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#98

Post by Scoundreller »

ForHackernews wrote: post #29

This is the future I want to live in: one where radical advances in gene therapy are curing (or nearly curing) horrible illnesses humanity has suffered with for thousands of years.

already a thing: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9512634/

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#99

Post by glp1guide »

0xB31B1B wrote: post #42
bruce511 wrote: post #21

Trikafta is a real breakthrough for CF patients. What's not mentioned is the cost - about $300 000 per person per year. [1] In 2037 the patent will run out, and the generic price will likely be 90% less. Fortunately, for Americans with good-enough health insurance, it's covered, so ... yay? For those without insurance, or in other countries where $300k is basically unaffordable, well bummer for you, you'll be the las…

drugs are priced differently country to country and the price has nothing to do with the unit cost to manufacture. The US is the least regulated pharmaceuticals market in the world and drugs here usually cost significantly more than drugs in other locals. For example semaglutide/ozempic costs like 1300/month in the US, but only 100-200/month in European countries. Also, the "1300/month" cost in the US is only paid by…

So note that although Semaglutide/Ozempic cost that amount retail, most are absolutely not paying that price.

https://glp1.guide/content/glp1-pricing-2024-02

Of course, insurers are struggling to not cover the bill, and although it's in the medicaid list it's up to states to choose whether to cover it or not

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#100

Post by joney_baloney »

abhisuri97 wrote: post #55
pedalpete wrote: post #50

Earlier quoted context omitted.

Can you elaborate on the weight gain? I'm curious why that is a downside of the Trikafta? You say with CF the pancreas is blocked, so why did the weight come as a side-effect of the drug, and not with the disease initially?

Med student here. My guess is the following: So with CF your pancreas can’t secrete many of the enzymes that are necessary to actually digest food. You can take medications to help with that digestion, but regardless, you aren’t actually getting all the calories in your food with CF because it isn’t making its way into your body (notably patients with CF have steatorrhea which is fat in their stool because they can’t…

This is exactly right.

Source: have CF.

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