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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

#81

Post by A_D_E_P_T »

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"?

Yeah, IRL too. 5-10 years ago there were some biohacking collectives around the Bay Area that did everything from pharmaceuticals to experiments with CRISPR. I don't know if they're still around -- I left that scene when I left the USA a few years ago -- but if you're in the area you might want to give it a look. (As an aside, I heard that a few scene-adjacent people have recently gotten gene-mod therapy; Patri Friedman, for instance, received follistatin therapy.)

Nootropics were a pretty popular trend around the same time, and I knew a lot of guys who would import weird drugs and peptides from Russia and China. It's the same sort of procedure I outlined in my last post, in that you're ordering drugs or drug-active-ingredient-powder from abroad... And then hopefully testing it.

Roughly 10 years ago, when [redacted breakthrough drug] was released, I helped some friends set up a pirate pharmacy. Those were good times.

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

#82

Post by patmorgan23 »

paulpauper wrote: post #44
bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

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…

Appetite suppression/regulation seems like a much better route to pursue than intentionally inducing malabsorption.

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

#83

Post by nwiswell »

A_D_E_P_T wrote: post #81
nwiswell wrote: post #75

Earlier quoted context omitted.

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

Yeah, IRL too. 5-10 years ago there were some biohacking collectives around the Bay Area that did everything from pharmaceuticals to experiments with CRISPR. I don't know if they're still around -- I left that scene when I left the USA a few years ago -- but if you're in the area you might want to give it a look. (As an aside, I heard that a few scene-adjacent people have recently gotten gene-mod therapy; Patri Fried…

That's really cool. I don't have a bio/pharma technical background but I'm very sympathetic to the scene and the ethos so I'll have to check it out.

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

#84

Post by cogman10 »

bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

> The only real downside: weight gain.

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.

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

#85

Post by kelnos »

cbeach wrote: post #66
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…

I've seen conversations like this before, regarding the heavy cost that Big Pharma levies on treatments for major diseases. And US companies frequently come up in these discussions. 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 p…

What about direct government funding? Isn't that a reasonable alternative? Maybe not a politically-popular one, but I wouldn't be surprised if health care overall would be cheaper under a system where pharma research is done with public money, and then isn't allowed to be patented. Might even in save enough money to avoid much in the way of extra taxes to fund it.

Hell, a lot of medical research is funded (in part) by public dollars, and then the results get locked up behind patents for years, which is gross.

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

#86

Post by vrc »

paulpauper wrote: post #44
bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

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.

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

#87

Post by vrc »

derefr wrote: post #56
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…

They sort of tried this in the '90s — not in the form of suppressing the required enzymes for nutrient absorption, but rather in the form of diet foods that contained inherently non-absorbable nutrients. The downsides were basically the same — the non-absorbed nutrients had to come out. People didn't generally respond well to these products. See e.g. https://web.archive.org/web/20060113084223/http://www.zug.co...

Olestra! I ate a whole bag of the Lay’s with Olean as a kid. They had the audacity to say that they assumed that people would only eat a handful at a time. Of near zero calorie potato chips that taste like potato chips…

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

#88

Post by krooj »

gcanyon wrote: post #38
krooj wrote: post #31

Earlier quoted context omitted.

I'll echo this - I have had two left leg DVTs, spaced about 7 years apart, and after the second event, really started diving into medical publications - surgical journals, medical textbooks, clinical trials - as a means to better understand the condition, it's pathology, etc. I ultimately submitted to testing and discovered a congenital stenosis of the left iliac vein with heavy retroperitoneal collateralization that…

This is interesting -- I have Factor V Leiden (heterozygous) and have had one DVT. It never would have occurred to me to seek out a support group.

I also have the same mutation, as does my wife. From what I've been told by various hematologists, vascular surgeons, and interventional radiologists, it's a very weak clotting disorder, but you do have to keep an eye on certain environmental factors: smoking, hydration, movement, and trauma/surgery. To put it another way, FVL is fairly benign until you're already way into Virchow's danger done, and at that point it's gonna work against you. When it comes to VTE in the presence of ONLY FVL, I would shoot serious side-eye at a doc that chalked it up to the mutation - there's usually something else going on.

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

#90

Post by rakejake »

BenFranklin100 wrote: post #23

This article gives short shrift to the role the pharmaceutical industry played in making this life-changing treatment a reality. It barely mentions Vertex. Vertex is the biotech company that made the big bet on cystic fibrosis, a rare disease that most companies wouldn’t touch because of the small market and unknown biology made it too risky. They were the ones that believed in the science and developed multiple CF d…

The book "The Billion Dollar Molecule" is about the founding of Vertex Pharma. Worth a read.
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