[HN Gopher] Cystic fibrosis breakthrough has given patients a ch...
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Cystic fibrosis breakthrough has given patients a chance to live
longer
Author : cowboysauce
Score : 192 points
Date : 2024-03-07 13:49 UTC (2 days ago)
(HTM) web link (www.theatlantic.com)
(TXT) w3m dump (www.theatlantic.com)
| leetrout wrote:
| 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 wrote:
| 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 wrote:
| 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 wrote:
| 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 wrote:
| 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 wrote:
| 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.
| krooj wrote:
| 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 necessitated a stent to keep that
| iliac vein open.
|
| I also had a quick look into the social media (primarily
| reddit) aspect of these vascular conditions, and it's a
| pile of dogshit. Most of these patient communities bill
| themselves as "support groups", but there's never any real
| discussion on meaningful research, drug, or device
| advancements. They places serve primarily as "pity pits"
| for chronic moaners and scammers selling alternative
| medicine.
| gcanyon wrote:
| 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.
| bdcravens wrote:
| 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)
| COGlory wrote:
| 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 wrote:
| 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.
| mason55 wrote:
| > _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?_
|
| There's a discussion of this in the article. Essentially, a
| bunch of people have said that they started experiencing
| extreme anxiety when they started Trikafta, and that stopping
| or lowering the dose helped immensely. However, clinicians
| have said that there's no actual evidence that the Trikafta
| is causal.
|
| So it seems to be a bit of an open question and I'm sure an
| emotional one on both sides.
| vkou wrote:
| People generally get acclimated to the situation they are in.
|
| If you have anxiety over money, for instance, you'd probably
| still have it, regardless of whether you had $15,000 in your
| bank account, or $15,000,000.
| pfannkuchen wrote:
| You might even have more with 15M! It's much harder to
| replace if you lose it.
| therein wrote:
| One of the first friends I made in college was a friend with
| CF. I didn't know what CF was. He was very mature for our age
| and generally a very nice, happy, caring person. Got
| internships, studied with us. Went to bars with us but never
| drank because he couldn't. He had stories about how Make-a-Wish
| foundation has been dragging their feet not to give him his
| wish since his childhood.
|
| 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.
| paulpauper wrote:
| _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 people
| have too much nutrition. Blocking absorption seems easier than
| dieting, as no willpower required. You shit out the undigested
| food.
| bdcravens wrote:
| 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
| therein wrote:
| 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 wrote:
| 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 wrote:
| There's a sign-up process required.
| ForHackernews wrote:
| Ah! Fair enough. I assumed HN links bypassed the paywall.
| bane wrote:
| 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 wrote:
| Those were wonderful words and moved me a lot.
| dotnet00 wrote:
| 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 wrote:
| 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 wrote:
| Breath From Salt is a great book on CF and it's history in
| medicine.
|
| https://benbellabooks.com/shop/breath-from-salt/
| yalok wrote:
| 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 wrote:
| 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 wrote:
| 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....
| elektor wrote:
| "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/...
| xenospn wrote:
| 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").
| ploika wrote:
| Not necessarily. I don't know about other countries, but I
| remember there being a campaign here in Ireland (with one of
| the highest rates of cystic fibrosis in the world) to get
| Orkambi covered under the Drug Payment Scheme when it was
| new.
| BurningFrog wrote:
| Or the country decides to not import the medicine due to the
| cost.
| ForHackernews wrote:
| It is mentioned. Quoting directly from this piece...
|
| > 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.
| nwiswell wrote:
| In many other countries, patents do not apply to personal use,
| where there is no sale.
|
| 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...
| deadbabe wrote:
| If you're going to do all that why bother with legality, just
| push pills on a black market.
| 0xB31B1B wrote:
| 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 people who are
| paying cash and not covered by insurance, if the drug is
| covered by the patients insurance than the Pharmacy benefits
| manager usually pays a fraction of the 1300/month price as it
| is a "bulk negotiated discount, so they often pay 200-300 USD,
| the actuall price in these deals is a closely guarded trade
| secret.
| orzig wrote:
| I loved the book The Billion Dollar Molecule (Reviewed by Nature
| here: 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 wrote:
| 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-h...
| jeremiahbuckley wrote:
| Thanks for this. Very cool to read these types of stories.
| ForHackernews wrote:
| 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.
| kouru225 wrote:
| 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 wrote:
| 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-bact...
| SEJeff wrote:
| The internet archive has your back. Here is TFA:
| https://archive.is/gD49J
| paulpauper wrote:
| 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.
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