46 ms·

Cystic fibrosis breakthrough has given patients a chance to live longer

422 points by cowboysauce 3y ago

leetrout 3y ago
I lost a cousin and a family friend to CF. Both in their 20's. One after a lung transplant added some time. It is such a horrible disease and the breakthroughs are amazing. So many families will be much more fortunate than mine and I am happy for them!
forgetfreeman 3y ago
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, which had been stable for years, was in shambles as well. I can only speculate that their exuberance at being given a stay of execution lead them to "oversteer" into some questionable lifestyle choices.
- bane 3y ago
  This section seems to speak to your observation (edited for brevity): 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. For some HIV patients, their reversal of fortune seemed unreal. “He doesn’t trust what’s happening to him,” one doctor said about a patient who had made a dramatic recovery, yet found himself in psychological distress."
- gfodor 3y ago
  I have CF, and my whole life I avoided things like CF communities explicitly because I felt these ties to the disease would lead to a crisis in my life if it was ever something I could stop centering my life around.
  - Aurornis 3y ago
    Chronic illness forums are almost universally terrible. I try to navigate the online communities for a family member’s disease to keep up with new developments. A decade ago I found some value in the way they presented news and research and anecdotes. Now, the forums are overrun by small numbers of constantly online members who feel the need to dominate every conversation. The content has become almost entirely venting and memes, with an unreasonable amount of alternative medicine being pushed as fact. It’s understandable that they’re frustrated, to say the least, but the way their frustration gives way to a communal rage against doctors has weirdly opened doors to alternative medicine peddlers. It’s disgusting to me to see how the alternative medicine pushers have arrived with open arms and comforting smiles for vulnerable communities, which slowly becomes a sales pitch for their products. I’ve seen everting from people peddling custom diet consulting based on your 23andMe results to invitations to private, paid Telegram channels where they supposedly share their secret cures, to doctors from Eastern Europe who claim to have cured the condition (which has eluded many researchers and pharmaceutical companies) with a custom treatment made from the patients’ own urine. The way these communities set themselves up to rage together at modern medicine opens the door for friendly alternative medicine scams. It’s depressing.
    - spondylosaurus 3y ago
      IME one reason (out of many) why chronic illness forums tend to be terrible is that people who are managing their conditions reasonably well don't participate much. Which makes sense—the better you're doing, the less time you spend thinking about it and the less time you're inclined to spend discussing it—but that creates an environment where the most miserable voices become the loudest. And so (1) there's often a disproportionate focus on doom and gloom rather than success stories, which paints a pessimistic picture for anyone joining after a recent diagnosis, and (2) the most prominent voices have a wounded-cornered-animal mentality that makes them defensive and/or prone to lashing out. And I can't totally blame them, given how hard it is to live with a treatment-resistant chronic condition, but it's not the most constructive environment for everyone else.
- bdcravens 3y ago
  Many have reported severe anxiety due to Trikafta. As I mentioned in another comment, I believe I experienced the same, but I also started Lexapro around the same time for other non-CF reasons, so independent test variables and all that.
- qgin 3y ago
  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.
  - dennis_jeeves2 3y ago
    >There would be happiness, but there would also be chaos Which is good in the short term, correct? Long term people hopefully would be wise to settle for stable relationships, jobs etc...
bdcravens 3y ago
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. It can't undo a life of damage to things like my pancreas, vas deferens, etc, and I still take medicine to digest my food, but overall, it's as close to "normal" as I could hope for at this point. The only real downside: 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, and went up some 6 inches in the waist. To add insult to injury, it happened during the spring and summer of 2020, when buying clothes was a challenge due to the pandemic. Speaking of the pandemic, the timing of Trikafta was amazing: it kept the normally full "CF floors" of hospitals empty, opening up those beds for those with COVID and keeping CF patients less exposed. 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. (copied from the last time Trikafta was mentioned here: https://news.ycombinator.com/item?id=37540731 https://news.ycombinator.com/item?id=37540731)
- COGlory 3y ago
  Sorry for the potentially insensitive question, but I really am wondering about this: >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?
  - therein 3y ago
    Just an anecdotal experience so doesn't necessarily mean anything but the only person I knew that had CF had absolutely no anxiety from his condition. If anything he was a very mature guy for his age, having realized he has limited time but of course I wouldn't know the internal struggles he might have kept from us.
bdcravens 3y ago
Interesting note about Trikafta and other advancements in cystic fibrosis: the Make a Wish Foundation announced that children with CF now no longer automatically qualify for their program due to the advancements in care. https://wish.org/cf-update https://wish.org/cf-update
- therein 3y ago
  My friend that had CF was eligible. He was asked by the foundation what he wanted when he was 5-6 years old. He said he wanted to go Zorbing in Australia. 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.
- ForHackernews 3y ago
  This is also mentioned in the article: > 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...
  - tnias23 3y ago
    There’s a sign-up process required.
    - ForHackernews 3y ago
      Ah! Fair enough. I assumed HN links bypassed the paywall.
  - bdcravens 3y ago
    Fair enough. As someone who takes Trikafta, I skimmed the article since it wasn't news to me :-)
bane 3y ago
Oh wow. I remember a kid in elementary school with CF. I didn't understand at the time what he and his family was going through. I remember his personality and intelligence, his skill on the soccer team. He was unusually driven for an 8-9 year old. As I got older and into my 20s, I thought about him a lot -- it was understood that most CF patients don't make it out of their early 20s and I knew even if he were particularly lucky, as time went on and I aged, he was probably gone. My father also recently died from lung cancer, and had a few months where he was effectively drowning in his own lung fluids, requiring doctors to drain his lungs with long needles through his back. That experience also brought me back to that schoolmate, considering what he had had to endure. Recently, I was reorganizing my personal library and came across my elementary school yearbook and flipping through, saw his picture. It's been decades since he's likely passed on. It gave me pause to contemplate certain priorities in my life and try to cultivate greater compassion. It's a particularly cruel disease, and this news is wonderful.
- jhoechtl 3y ago
  Those were wonderful words and moved me a lot.
- dclowd9901 3y ago
  My brother passed about a decade ago from complications from a double lung transplant, which he needed because of CF. 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.
- speedylight 3y ago
  Have you tried looking him up? He could still be alive.
dotnet00 3y ago
I remember studying about how CF was an agonizing death sentence just 10 years ago in highschool. It's both interesting to learn that even at that time the life expectancy info was outdated, and even cooler to hear this news.
varjag 3y ago
It appeared too late to make a difference for my dear friend and the kindest human being I knew. The manifestation of life's injustice drives me mad ngl.
alleycat5000 3y ago
Breath From Salt is a great book on CF and it's history in medicine. https://benbellabooks.com/shop/breath-from-salt/ https://benbellabooks.com/shop/breath-from-salt/
yalok 3y ago
I wonder if this applies to kidneys cystic fibrosis? I friend of mine passed away a few years ago from it, and it was painful to watch slow degradation in his health over the years.
josefrichter 3y ago
Around the time Trikafta became widely available I used to work for a clinical trials company. I remember one guy with CF telling us the full story of his life, how plenty of his friends with CF died in their 20s, and how, after taking the new meds, he felt effects within the first hour. It was like a miracle. If I remember correctly, there’s still some 10% of CF patients for whom it doesn’t work but I hope we will soon eradicate this horrendous disease altogether.
bruce511 3y ago
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 last generation to die of it. I get the insane costs, and risks, of developing these things. I get that the profit motive is what drives there to be any research at all in pharma. I get that the price has to be high for everyone, or insurance companies will balk. And yet, even knowing all that, there's a sour taste when we -could- (literally) save lives, but, well, money first ya-know... I don't have an answer to this issue- there are downsides to all proposals I've heard. But this approach seems, well, pretty harsh. [1] https://www.statnews.com/2023/11/03/trikafta-cystic-fibrosis-price-colorado-prescription-drug-affordability-board/#:~:text=Trikafta%20is%20taken%20by%20only,drugs%20from%20their%20PDAB%20evaluations https://www.statnews.com/2023/11/03/trikafta-cystic-fibrosis....
- elektor 3y ago
  "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/abbvies-humira-gets-us-rival-costs-could-stay-high-2023-01-31/ https://www.reuters.com/business/healthcare-pharmaceuticals/...
  - amplicons4ever 3y ago
    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 never be cheap. It's physically impossible with today's bio processing technology. Trikafta is a mix of three small molecules, which are manufactured in large chemical batches and are the more traditional class of drugs. Many even have total synthesis pathways known which means you can basically make them by the train car scale for cheap. When the patent on Trikafta runs out, it'll be very cheap. I'll note that vanishingly few patients ever pay the full list price in the US--if you have insurance, the copay is small. Your insurance pays (as they should, that's why you pay them premiums!). The company even pays the co-pay in most cases so the actual cost to patients in many cases is $0. If you truly need it and don't have insurance, the company provides it for basically free with a patient assistance program, it's there on their website. Kudos to the scientists who invented this, I don't feel bad for the insurance companies really, they charge their premiums and it's their responsibility to the policyholders to pay the applicable fees for care. *edit: typo
orzig 3y ago
I loved the book The Billion Dollar Molecule (Reviewed by Nature here: https://www.nature.com/articles/nbt0594-521.pdf https://www.nature.com/articles/nbt0594-521.pdf) about the founding of Vertex Pharma. Really drives home the amount of money, time and luck it takes to make a breakthrough - the author needed to write an entire second book to get to the part where they started making real revenue. The second book isn't as good, but if you like the first enough you might try it anyway.
BenFranklin100 3y ago
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 drugs and got them into the hands of patients. Here’s a 2019 STAT article that gives a fuller account of Vertex’s role: https://www.statnews.com/2019/10/23/we-conquered-a-disease-how-vertex-delivered-a-transformative-medicine-for-cystic-fibrosis/ https://www.statnews.com/2019/10/23/we-conquered-a-disease-h...
- jeremiahbuckley 3y ago
  Thanks for this. Very cool to read these types of stories.
- bdcravens 3y ago
  Yes, the CFF has partnered with companies like Vertex to take an almost VC-like approach towards developing new drugs (Trikafta being one of many similar drugs they've developed with Vertex)
- rakejake 3y ago
  The book "The Billion Dollar Molecule" is about the founding of Vertex Pharma. Worth a read.
  - ackbar03 3y ago
    There's a follow up book as well, The Antidote. I read both to learn more about the industry
ForHackernews 3y ago
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.
- Scoundreller 3y ago
  already a thing: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9512634/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9512634/
  - dennis_jeeves2 3y ago
    Summarize it in plain layman's language for me please...
    - Scoundreller 3y ago
      They take out some of your cells, tinker with their genetic sequence, and put them back into you.
kouru225 3y ago
When I was a kid, I was told my cousins would die before they turned 30 because of CF. One of them ended up dying when he was 10, but other is now above 30 and seems incredibly healthy. The amount of development in the treatment of CF over the last 20 or so years is so incredible it’s insane.
clumsysmurf 3y ago
I wasn't able to read the article (behind paywall) but this was also in the news recently, related to CF & Zn. https://newatlas.com/medical/zinc-lung-macrophages-anti-bacterial-cystic-fibrosis/ https://newatlas.com/medical/zinc-lung-macrophages-anti-bact...
- SEJeff 3y ago
  The internet archive has your back. Here is TFA: https://archive.is/gD49J https://archive.is/gD49J
paulpauper 3y ago
If CF like breathing through a coffee straw, as some sources say? I tried it myself, and god I hope not. I lasted a few minutes and was gasping for air.
- bdcravens 3y ago
  I have CF. My lung capacity is around 55% (many have lower, and around 20-30% is when they tend to start talking lung transplant) 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 :-)
- joney_baloney 3y ago
  Once it gets bad, yeah. I was very end stage before my lung transplant and it's an accurate description.
pedalpete 3y ago
https://archive.is/gD49J https://archive.is/gD49J
dm8 3y ago
This is incredible. I wonder if there will be medicine that cures idiopathic pulmonary fibrosis. New class of drugs like Ofev stop worsening of the IPF but doesn't cure it.
optymizer 3y ago
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.
ETH_start 3y ago
If I can be allowed a bit of sophistry: It's a beautiful thing for treatments to be devised for rare conditions, to give people decades more life. It would be more wonderful still if we devised treatments for aging, the universal condition, to grant everyone decades more life. Hardly anyone deserves to die. The vast majority of people are singularly incredible and worthy of life. They deserve to be equally safe from murder, fatal accidents, rare diseases like cystic fibrosis and the universal disease of aging.
- mmustapic 3y ago
  Many incurable diseases come with an awful quality of life, like CF. Even cancer, treated or not, is a terrible experience. Just aging, what you call “a disease”, is nothing like that.
  - ETH_start 3y ago
    Aging comes with a terrible quality of life. By the end, the person is severely debilitated. Osteoporosis can make it so that a fall can lead to major fractures, which leads to a severe reduction in a person's independence. Macular degeneration can make an elderly person blind. Neurological deterioration can take the form of conditions like Alzheimer's, where a person loses their memory of everyone they love. The body, as it ages, becomes much more susceptible to other, terrible, diseases as well, like cancer, heart disease, and diabetes, each carrying its own set of debilitating effects that degrade quality of life.
    - mmustapic 3y ago
      Alzheimer is not aging, it is a specific disease. Same as cancer. Sure, advanced age can make you more susceptible to some diseases, but aging itself is not one.
      - ETH_start 3y ago
        Aging eventually makes you orders of magnitude more susceptible to most major diseases, while coming with its own set of debilitating effects. Aging is the gradual accumulation of cellular and bodily damage. Nothing would do more for human health than having effective treatments for arresting and reversing that degradation.
warion 3y ago
This sounds bad and can give edge to republicans as generally rightist people have more incidence of CF

422 points by cowboysauce 3y ago

I lost a cousin and a family friend to CF. Both in their 20's. One after a lung transplant added some time. It is such a horrible disease and the breakthroughs are amazing. So many families will be much more fortunate than mine and I am happy for them!

forgetfreeman 3y ago

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, which had been stable for years, was in shambles as well. I can only speculate that their exuberance at being given a stay of execution lead them to "oversteer" into some questionable lifestyle choices.

bane 3y ago
This section seems to speak to your observation (edited for brevity): 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. For some HIV patients, their reversal of fortune seemed unreal. “He doesn’t trust what’s happening to him,” one doctor said about a patient who had made a dramatic recovery, yet found himself in psychological distress."
gfodor 3y ago
I have CF, and my whole life I avoided things like CF communities explicitly because I felt these ties to the disease would lead to a crisis in my life if it was ever something I could stop centering my life around.
- Aurornis 3y ago
  Chronic illness forums are almost universally terrible. I try to navigate the online communities for a family member’s disease to keep up with new developments. A decade ago I found some value in the way they presented news and research and anecdotes. Now, the forums are overrun by small numbers of constantly online members who feel the need to dominate every conversation. The content has become almost entirely venting and memes, with an unreasonable amount of alternative medicine being pushed as fact. It’s understandable that they’re frustrated, to say the least, but the way their frustration gives way to a communal rage against doctors has weirdly opened doors to alternative medicine peddlers. It’s disgusting to me to see how the alternative medicine pushers have arrived with open arms and comforting smiles for vulnerable communities, which slowly becomes a sales pitch for their products. I’ve seen everting from people peddling custom diet consulting based on your 23andMe results to invitations to private, paid Telegram channels where they supposedly share their secret cures, to doctors from Eastern Europe who claim to have cured the condition (which has eluded many researchers and pharmaceutical companies) with a custom treatment made from the patients’ own urine. The way these communities set themselves up to rage together at modern medicine opens the door for friendly alternative medicine scams. It’s depressing.
  - spondylosaurus 3y ago
    IME one reason (out of many) why chronic illness forums tend to be terrible is that people who are managing their conditions reasonably well don't participate much. Which makes sense—the better you're doing, the less time you spend thinking about it and the less time you're inclined to spend discussing it—but that creates an environment where the most miserable voices become the loudest. And so (1) there's often a disproportionate focus on doom and gloom rather than success stories, which paints a pessimistic picture for anyone joining after a recent diagnosis, and (2) the most prominent voices have a wounded-cornered-animal mentality that makes them defensive and/or prone to lashing out. And I can't totally blame them, given how hard it is to live with a treatment-resistant chronic condition, but it's not the most constructive environment for everyone else.
bdcravens 3y ago
Many have reported severe anxiety due to Trikafta. As I mentioned in another comment, I believe I experienced the same, but I also started Lexapro around the same time for other non-CF reasons, so independent test variables and all that.
qgin 3y ago
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.
- dennis_jeeves2 3y ago
  >There would be happiness, but there would also be chaos Which is good in the short term, correct? Long term people hopefully would be wise to settle for stable relationships, jobs etc...

bdcravens 3y ago

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. It can't undo a life of damage to things like my pancreas, vas deferens, etc, and I still take medicine to digest my food, but overall, it's as close to "normal" as I could hope for at this point. The only real downside: 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, and went up some 6 inches in the waist. To add insult to injury, it happened during the spring and summer of 2020, when buying clothes was a challenge due to the pandemic. Speaking of the pandemic, the timing of Trikafta was amazing: it kept the normally full "CF floors" of hospitals empty, opening up those beds for those with COVID and keeping CF patients less exposed. 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. (copied from the last time Trikafta was mentioned here: https://news.ycombinator.com/item?id=37540731 https://news.ycombinator.com/item?id=37540731)

COGlory 3y ago
Sorry for the potentially insensitive question, but I really am wondering about this: >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?
- therein 3y ago
  Just an anecdotal experience so doesn't necessarily mean anything but the only person I knew that had CF had absolutely no anxiety from his condition. If anything he was a very mature guy for his age, having realized he has limited time but of course I wouldn't know the internal struggles he might have kept from us.

bdcravens 3y ago

Interesting note about Trikafta and other advancements in cystic fibrosis: the Make a Wish Foundation announced that children with CF now no longer automatically qualify for their program due to the advancements in care. https://wish.org/cf-update https://wish.org/cf-update

therein 3y ago
My friend that had CF was eligible. He was asked by the foundation what he wanted when he was 5-6 years old. He said he wanted to go Zorbing in Australia. 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.
ForHackernews 3y ago
This is also mentioned in the article: > 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...
- tnias23 3y ago
  There’s a sign-up process required.
  - ForHackernews 3y ago
    Ah! Fair enough. I assumed HN links bypassed the paywall.
- bdcravens 3y ago
  Fair enough. As someone who takes Trikafta, I skimmed the article since it wasn't news to me :-)

bane 3y ago

Oh wow. I remember a kid in elementary school with CF. I didn't understand at the time what he and his family was going through. I remember his personality and intelligence, his skill on the soccer team. He was unusually driven for an 8-9 year old. As I got older and into my 20s, I thought about him a lot -- it was understood that most CF patients don't make it out of their early 20s and I knew even if he were particularly lucky, as time went on and I aged, he was probably gone. My father also recently died from lung cancer, and had a few months where he was effectively drowning in his own lung fluids, requiring doctors to drain his lungs with long needles through his back. That experience also brought me back to that schoolmate, considering what he had had to endure. Recently, I was reorganizing my personal library and came across my elementary school yearbook and flipping through, saw his picture. It's been decades since he's likely passed on. It gave me pause to contemplate certain priorities in my life and try to cultivate greater compassion. It's a particularly cruel disease, and this news is wonderful.

jhoechtl 3y ago
Those were wonderful words and moved me a lot.
dclowd9901 3y ago
My brother passed about a decade ago from complications from a double lung transplant, which he needed because of CF. 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.
speedylight 3y ago
Have you tried looking him up? He could still be alive.

dotnet00 3y ago

I remember studying about how CF was an agonizing death sentence just 10 years ago in highschool. It's both interesting to learn that even at that time the life expectancy info was outdated, and even cooler to hear this news.

varjag 3y ago

It appeared too late to make a difference for my dear friend and the kindest human being I knew. The manifestation of life's injustice drives me mad ngl.

alleycat5000 3y ago

Breath From Salt is a great book on CF and it's history in medicine. https://benbellabooks.com/shop/breath-from-salt/ https://benbellabooks.com/shop/breath-from-salt/

yalok 3y ago

I wonder if this applies to kidneys cystic fibrosis? I friend of mine passed away a few years ago from it, and it was painful to watch slow degradation in his health over the years.

josefrichter 3y ago

Around the time Trikafta became widely available I used to work for a clinical trials company. I remember one guy with CF telling us the full story of his life, how plenty of his friends with CF died in their 20s, and how, after taking the new meds, he felt effects within the first hour. It was like a miracle. If I remember correctly, there’s still some 10% of CF patients for whom it doesn’t work but I hope we will soon eradicate this horrendous disease altogether.

bruce511 3y ago

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 last generation to die of it. I get the insane costs, and risks, of developing these things. I get that the profit motive is what drives there to be any research at all in pharma. I get that the price has to be high for everyone, or insurance companies will balk. And yet, even knowing all that, there's a sour taste when we -could- (literally) save lives, but, well, money first ya-know... I don't have an answer to this issue- there are downsides to all proposals I've heard. But this approach seems, well, pretty harsh. [1] https://www.statnews.com/2023/11/03/trikafta-cystic-fibrosis-price-colorado-prescription-drug-affordability-board/#:~:text=Trikafta%20is%20taken%20by%20only,drugs%20from%20their%20PDAB%20evaluations https://www.statnews.com/2023/11/03/trikafta-cystic-fibrosis....

elektor 3y ago
"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/abbvies-humira-gets-us-rival-costs-could-stay-high-2023-01-31/ https://www.reuters.com/business/healthcare-pharmaceuticals/...
- amplicons4ever 3y ago
  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 never be cheap. It's physically impossible with today's bio processing technology. Trikafta is a mix of three small molecules, which are manufactured in large chemical batches and are the more traditional class of drugs. Many even have total synthesis pathways known which means you can basically make them by the train car scale for cheap. When the patent on Trikafta runs out, it'll be very cheap. I'll note that vanishingly few patients ever pay the full list price in the US--if you have insurance, the copay is small. Your insurance pays (as they should, that's why you pay them premiums!). The company even pays the co-pay in most cases so the actual cost to patients in many cases is $0. If you truly need it and don't have insurance, the company provides it for basically free with a patient assistance program, it's there on their website. Kudos to the scientists who invented this, I don't feel bad for the insurance companies really, they charge their premiums and it's their responsibility to the policyholders to pay the applicable fees for care. *edit: typo

orzig 3y ago

I loved the book The Billion Dollar Molecule (Reviewed by Nature here: https://www.nature.com/articles/nbt0594-521.pdf https://www.nature.com/articles/nbt0594-521.pdf) about the founding of Vertex Pharma. Really drives home the amount of money, time and luck it takes to make a breakthrough - the author needed to write an entire second book to get to the part where they started making real revenue. The second book isn't as good, but if you like the first enough you might try it anyway.

BenFranklin100 3y ago

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 drugs and got them into the hands of patients. Here’s a 2019 STAT article that gives a fuller account of Vertex’s role: https://www.statnews.com/2019/10/23/we-conquered-a-disease-how-vertex-delivered-a-transformative-medicine-for-cystic-fibrosis/ https://www.statnews.com/2019/10/23/we-conquered-a-disease-h...

jeremiahbuckley 3y ago
Thanks for this. Very cool to read these types of stories.
bdcravens 3y ago
Yes, the CFF has partnered with companies like Vertex to take an almost VC-like approach towards developing new drugs (Trikafta being one of many similar drugs they've developed with Vertex)
rakejake 3y ago
The book "The Billion Dollar Molecule" is about the founding of Vertex Pharma. Worth a read.
- ackbar03 3y ago
  There's a follow up book as well, The Antidote. I read both to learn more about the industry

ForHackernews 3y ago

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.

Scoundreller 3y ago
already a thing: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9512634/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9512634/
- dennis_jeeves2 3y ago
  Summarize it in plain layman's language for me please...
  - Scoundreller 3y ago
    They take out some of your cells, tinker with their genetic sequence, and put them back into you.

kouru225 3y ago

When I was a kid, I was told my cousins would die before they turned 30 because of CF. One of them ended up dying when he was 10, but other is now above 30 and seems incredibly healthy. The amount of development in the treatment of CF over the last 20 or so years is so incredible it’s insane.

clumsysmurf 3y ago

I wasn't able to read the article (behind paywall) but this was also in the news recently, related to CF & Zn. https://newatlas.com/medical/zinc-lung-macrophages-anti-bacterial-cystic-fibrosis/ https://newatlas.com/medical/zinc-lung-macrophages-anti-bact...

SEJeff 3y ago
The internet archive has your back. Here is TFA: https://archive.is/gD49J https://archive.is/gD49J

paulpauper 3y ago

If CF like breathing through a coffee straw, as some sources say? I tried it myself, and god I hope not. I lasted a few minutes and was gasping for air.

bdcravens 3y ago
I have CF. My lung capacity is around 55% (many have lower, and around 20-30% is when they tend to start talking lung transplant) 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 :-)
joney_baloney 3y ago
Once it gets bad, yeah. I was very end stage before my lung transplant and it's an accurate description.

pedalpete 3y ago

https://archive.is/gD49J https://archive.is/gD49J

dm8 3y ago

This is incredible. I wonder if there will be medicine that cures idiopathic pulmonary fibrosis. New class of drugs like Ofev stop worsening of the IPF but doesn't cure it.

optymizer 3y ago

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.

ETH_start 3y ago

If I can be allowed a bit of sophistry: It's a beautiful thing for treatments to be devised for rare conditions, to give people decades more life. It would be more wonderful still if we devised treatments for aging, the universal condition, to grant everyone decades more life. Hardly anyone deserves to die. The vast majority of people are singularly incredible and worthy of life. They deserve to be equally safe from murder, fatal accidents, rare diseases like cystic fibrosis and the universal disease of aging.

mmustapic 3y ago
Many incurable diseases come with an awful quality of life, like CF. Even cancer, treated or not, is a terrible experience. Just aging, what you call “a disease”, is nothing like that.
- ETH_start 3y ago
  Aging comes with a terrible quality of life. By the end, the person is severely debilitated. Osteoporosis can make it so that a fall can lead to major fractures, which leads to a severe reduction in a person's independence. Macular degeneration can make an elderly person blind. Neurological deterioration can take the form of conditions like Alzheimer's, where a person loses their memory of everyone they love. The body, as it ages, becomes much more susceptible to other, terrible, diseases as well, like cancer, heart disease, and diabetes, each carrying its own set of debilitating effects that degrade quality of life.
  - mmustapic 3y ago
    Alzheimer is not aging, it is a specific disease. Same as cancer. Sure, advanced age can make you more susceptible to some diseases, but aging itself is not one.
    - ETH_start 3y ago
      Aging eventually makes you orders of magnitude more susceptible to most major diseases, while coming with its own set of debilitating effects. Aging is the gradual accumulation of cellular and bodily damage. Nothing would do more for human health than having effective treatments for arresting and reversing that degradation.

warion 3y ago

This sounds bad and can give edge to republicans as generally rightist people have more incidence of CF