FDA approves a CRISPR-based medicine for treatment of sickle cell disease
statnews.com
statnews.com
> Ohalo had two RSRs under consideration this year for its potato, one which focuses on higher concentrations of beta carotene – enhancing the overall health and nutrition value of the potato – and another which results in reduced glucose and fructose content in the potato, which, according to Ohalo, will reduce the adverse side effects that lead to significant spoilage during cold storage of potatoes.
https://thespoon.tech/gene-edited-food-startup-ohalo-emerges...
More shelf life in exchange for likely worse taste...
Sweet potatoes, as you might expect, have more sugar in them. As do garnet yams.
The issue with potatoes isn't really their (low) sugar content.
Just make sure to go easy on the toppings!
“ true that potatoes are high in starch or carbohydrates, the nutrients that cause spikes in blood sugar. But pairing them with foods high in protein, fiber and unsaturated fats can slow digestion and lead to a steadier release of glucose into the bloodstream.”
Which suggests you should add toppings or else potatoes are not very healthy if eaten on their own. (I think they’re fairly high up on the glycemic index).
But also yes exactly, the whole family can cause inflammation and difficulties in people sensitive to them (which tragically because I love spicy food, includes me D-:). So that means that any cool stuff they do I won't be able to try.
That's pretty amazing. Imagine if we can change apples to produce Aspartame instead of sugars!
Even if you are diabetic, you can already eat apples. They have a low glycemic index.
Aspartame is safe, and very well tolerated.
You’re spreading misinformation.
Here, have a read. I have more if you would like them.
https://journals.plos.org/plosmedicine/article?id=10.1371/jo...
https://www.sciencedaily.com/releases/2022/03/220324143800.h...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1392232/
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1964906/pdf/ehp0...
http://www.mpwhi.com/soffritti_2010_20896_fta.pdf
https://ehjournal.biomedcentral.com/articles/10.1186/s12940-...
https://annalsofglobalhealth.org/articles/10.5334/aogh.4163
http://ajcn.nutrition.org/content/96/6/1419.long
https://www.ncbi.nlm.nih.gov/pubmed/24436139
https://www.pnas.org/doi/10.1073/pnas.2213120119
https://news.fsu.edu/news/university-news/2022/12/08/fsu-res...
https://www.nature.com/articles/s41598-023-41213-2
https://www.ncbi.nlm.nih.gov/pubmed/8939194
https://www.youtube.com/watch?v=yCoBuTr0Or0
http://www.cmaj.ca/content/189/28/E929
https://www.statnews.com/2017/07/17/artificial-sweeteners-we...
https://blogs.wsj.com/experts/2017/09/14/why-one-cardiologis...
https://www.philly.com/philly/health/this-cardiologist-wants...
https://www.ncbi.nlm.nih.gov/pubmed/27129676
http://stroke.ahajournals.org/content/early/2017/04/20/STROK...
https://www.bumc.bu.edu/busm/2017/04/20/daily-consumption-of...
https://www.washingtonpost.com/news/morning-mix/wp/2017/04/2...
https://www.ncbi.nlm.nih.gov/pubmed/24787915
https://www.ncbi.nlm.nih.gov/pubmed/24787917
https://www.ncbi.nlm.nih.gov/pubmed/1579221
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1474447/pdf/env...
https://www.ncbi.nlm.nih.gov/pubmed/28198207
https://www.ncbi.nlm.nih.gov/pubmed/28049023
https://www.ncbi.nlm.nih.gov/pubmed/17684524
http://www.sciencedirect.com/science/article/pii/S1043661805...
https://diabetesjournals.org/care/article/doi/10.2337/dc23-0...
https://www.ncbi.nlm.nih.gov/pubmed/27997218
For the author, try reading some of these papers. Also don't bother linking to press releases or newspaper articles, you're just wasting other people's time. You probably want to skip the animal studies too. Go for no more than three links, preferably meta-studies -- at least if your intention is to be taken for something other than a crank.
Doesn't that mean aspartame somehow contains more energy than sugar?
I mean, assuming the increased weight is fat, that is stored extra energy.
I have also heard that because artificial sweeteners increase insulin levels without increasing blood glucose to the same extent that sugars would, this leads to a blood sugar drop which induces increased eating.
Back up what you are saying with some studies. Because what you are claiming is going against a LOT of modern medical knowledge.
>Of the ten plant-based proteins included in the current analysis, potato protein is the only protein source containing the WHO/FAO/UNU requirements for all essential amino acids. Thus, when consuming potato protein as the only dietary protein source at the recommended adult protein intake level of 0.66 g/kg/day, sufficient amounts of all essential amino acids should be consumed. It remains to be investigated whether the ingestion of a single meal-like amount of potato protein has the capacity to stimulate muscle protein synthesis.
Anyone know about vegan protein profiles and best ones now?
They don't have to use any chemical solutions more exotic than water and air. I bungled the explanation with "centrifuge", it's more complicated than that, but the fiber content is removed in that step.
[1]: https://gogood.co.nz/blogs/news/how-is-pea-protein-manufactu...
> Soy, brown rice, pea, corn, and potato protein have essential amino acid contents that meet the requirements as recommended by the WHO/FAO/UNU (WHO/FAO/UNU Expert Consultation 2007) (Fig. 2).
2. I don't see how amino acid % of total protein is a useful way to gauge a food's protein especially in this context. Wouldn't you want to look at protein per calorie or EAA per calorie?
For example the paper's chart might make you think that you should eat potatoes and corn if you want to maximize plant-based protein, but that's not the case at all.
- 500 calories of potatoes boiled: 10g protein (650g of food)
- 500 calories of rib-eye steak: 54g protein (200g of food)
- 500 calories of soy chunks (TVP): 75g protein (btw hits all EAA objectives for the day) (150g of food)
- 500 calories of wheat gluten (seitan): 101g protein (135g of food)
Potatoes are dense in other nutrients and a great part of a healthy diet, but you definitely wouldn't use potatoes as a "meat protein substitute". Even broccoli is 3x as protein dense as potatoes.
On the other hand, seitan, tofu, and TVP are the trifecta of plant-based protein that can actually substitute for meat.
PDCAAS is a reasonable standard to use, pro-rated against total calories.
https://en.m.wikipedia.org/wiki/Protein_Digestibility_Correc...
A food with 1000x the EAAs per calorie of every other food, but 0 of one EAA would score below the other foods even though you could get the day's worth of EAA on 1/1000 the calories + one serving of an additional food that can give you the missing EAA (i.e. eat more than one food every day).
But your comment on the EAA/totalProtein ratio (when potatoes are very low in protein) possibly being responsible for why potatoes taste good made me chime in for clarification.
I am certain some media group is going to conveniently leave this part out of the title of their article, and surely no one is gonna waste time reading the actual article and the rumors will take off.
Does anyone know if changing the drug would require a new FDA approval for the entire regimen, or could the protocol be easily changed?
In theory, could a separate gene therapy target and knock out the stem cells that carry the mutation?
The FDA-approved prescribing information will recommend a particular chemotherapy regimen, but clinicians will be free to substitute alternatives if they believe those are clinically superior. They won't need permission from the FDA or the manufacturer to do that; clinicians deviate from the FDA-approved manufacturer recommendations all the time ("off-label prescribing").
If the manufacturer wants to update the official recommendations in the prescribing information, then they'll need FDA approval for that. But it is possible for clinicians to publish their own treatment guidelines (e.g. in medical journal articles), independent of the manufacturer, and the FDA has no control over those.
It is the way medicine works – not just in the US, in most countries worldwide. Not just about gene therapy, about all drugs and devices.
The FDA and its international equivalents (the EMA in the EU, the TGA in Australia, etc) regulate the manufacturers, not the clinicians. They control what the manufacturers sell and even what the manufacturers are allowed to say about their products (in product packaging, prescribing information, advertisements and marketing collateral). They don't control what the treating clinicians do with those products – to the extent that is regulated, it is the job of other regulatory agencies (e.g. professional licensing boards, civil courts hearing medical malpractice claims, etc)
> What if they, like, slide the journal article across the desk, whilst holding their finger alongside their nose and winking?
What they'll do instead: there will be a conference where (among other things) the journal article author will present their findings/recommendations, and the manufacturer will sponsor (and hence help pay for) the conference. They never actually said anything, they just made sure you were there to hear about it.
I'm not a doctor but my mother is. When I was a teenager, she'd be invited to these free dinners at fancy restaurants paid for by pharmaceutical companies, and a couple of times they allowed her to take me along (she was allowed to bring her spouse/partner to some of them, so she just asked "can I bring my teenage son instead"?). During the dinner, some academic would do a presentation on their research into how wonderful one of the company's drugs was, and also do some Q&A. So the manufacturer wasn't technically saying anything, everything was said by some academic (whose research they were funding). I didn't understand it all, but I found it rather interesting. Still didn't follow her footsteps into medicine though (although my younger brother has).
But, she tells me the regulators have cracked down on free perks from pharmaceutical companies, so they are forced to be a lot less generous nowadays than they were back in the 1990s. (This is not the US though, this is Australia.)
https://med.stanford.edu/news/all-news/2019/05/radiation-fre...
There is also ongoing research into immunotherapy for killing stem cells, as an alternative to the existing methods of chemotherapy and radiation. Potentially, immunotherapy could have significantly reduced secondary cancer risk.
https://jhoonline.biomedcentral.com/articles/10.1186/s13045-...
https://clonk.me/nft/137/0x8abd8d9fab3f711b16d15ce48747db496...
If we could eat different things, we could give up agriculture and save a bunch of land and energy.
This was a recurring theme in Lucky Luke [1]. The titular hero cooks and eats his boots when he faces starvation, usually when he gets lost in the desert.
They are apparently unrelated, it is 2022 that I was referring to.
Is that why? Or is it just the people with it aren’t sick enough to die before procreating?
An in-law of an ex has it, and regularly spends days in hospital during crises. Without access to a high quality hospital he'd have been dead a long time ago.
The average life expectancy for someone with sickle-cell disease in developed countries is 40-60 years, and serious crises tend to start from childhood.
That said, it's recessive, and so it's likely the reverse of what you think: It's not primarily the people with full-blown disease who contributes most to the long term survival of the trait, but that the trait alone confers fairly significant advantage in regions where Malaria is huge killer mostly without causing health problems. So across the combined set of carriers and those with the full disease, the life expectancy in Malaria stricken areas tends to be higher.
Pattern of change of the prevalence of the trait correlating with changes in prevalence of Malaria has been observed many places. E.g. the prevalence among US black people is significantly lower and dropping than in the areas their ancestors came from.
R | r
+----+----+
R | RR | Rr |
--+---------+
r | Rr | rr |
+---------+
The people who have some malaria resistance are all of the ones with "r". In particular, the "Rr" folks have the resistance, but not the anemia.
So basically, this gene screws over 1/4 of the population and benefits 1/2. In areas with lots of malaria, this tradeoff is worthwhile, evolutionarily speaking.
One of those harsh cases where evolution (if we personify it) does not care about individuals — only the species.
Although sickle cell does seem to be one of the rare cases where they work out.
But practically, it'd be a huge challenge. Nigeria's one of the main victims of malaria and, not by coincidence, one of the main victims of sickle cell. There are IVF clinics in Nigeria, but they're very expensive even before you consider sickle cell testing. It likely wouldn't scale to all of the births per day, and something like a quarter of the country would need it.
But it's not IMPOSSIBLE. You'd need to do maybe 75 or so per day to cover the 25% or so of the country that have the gene and would need it. Hard and expensive and impractical, but perhaps possible?
Prevention is the preferred method of passing this trait on however.
The train doesn't make you immune to malaria but it does increase resistance after infection.
The FDA-approved treatments reactivate the fetal HBB gene in adults and this change in gene expression control effectively prevents SCD.
Very cool and transformative work. Now we have to get the price tag down from seven figures to four or five figures so that it will be used widely. That may be a few decades. Let’s hope that more efficient alternatives are developed soon.
Note: I am not a doctor, but I do have a genetic condition that might benefit from CRISPR therapies, hence my interest.
[1] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10034092/#:~:te....
* Harvest stem cells from the patient.
* Prepare a DNA plasmid with the Cas9 gene, guide RNA for the desired genetic modification, and an antibiotic resistance gene.
* Electroporate the plasmid into the harvested stem cells. Grow the electroporated stem cells in antibiotic-containing nutrient media. Only cells with the plasmid (and thus antibiotic resistance) survive.
* Expand and freeze the genetically modified cells.
* Administer chemotherapy to the patient to eliminate defective bone marrow stem cells.
* Inject the modified stem cells back into the patient, where they repopulate the bone marrow with the CRISPR edits, aiming to correct the genetic mutation.
This process isn't new but one of the biggest challenges is propagating genetic modifications to all effected cells in the body. This is why it's much easier to GMO an egg / sperm because once the change is made there, it's replicated in every new cell thereafter.
Other techniques utilize harmless viruses to transfect genetic modifications to the body, but this has other trade-offs. mRNA vaccines don't propagate to every cell, but the cells which do successfully transcribe the mRNA are able to generate enough of the target protein that the body can recognize it and develop an immunity to it. Eventually, the modified cells will die and no cells will be left to produce the mRNA vaccine protein.
In this specific experiment they chose to transfect cells with plasmid directly rather than transduce with virus.
https://en.wikipedia.org/wiki/Fetal_hemoglobin#Treatment_of_...
Me too, because it's fun to consider DNA as some code we can edit to get outcomes we want!
However, some people are ethically opposed to that - but piggybacking on the preference people have for having children should be able to move the Overton window!
I doubt it. One of the root causes of “race” issues is humans using prior probabilities. Unless that changes, then the priors will simply move on from being skin tone based. I would suggest they already have for some portions of the population.
Body autonomy may be a fundamental right requiring legal recognition. It curiously cuts across many protracted debates: abortion, vaccine requirements, transgender rights and now gene editing. (I suppose abortion and germ-line edits are a special case.)
FDA considers first CRISPR gene editing treatment that may cure sickle cell - https://news.ycombinator.com/item?id=38354939 - Nov 2023 (124 comments)
> Patients must spend weeks, even months, in the hospital before and after the therapy is administered.
Yoiks. So how many actual people are going to be able to get this treatment?
"""Each treatment is an individualized “one-off” treatment. For this reason, a single treatment for a single patient is expensive. At present it is estimated that in the UK treatment will cost £1 million or more. In the US the estimated cost is $2 million.
That may seem prohibitive, but we need to consider the overall cost-effectiveness of the treatment, which means comparing the cost of treatment to the cost of managing each disease without the treatment. Sickle cell patient require frequent hospitalization, which can be very expensive. One analysis found that Casgevy can be cost effective at £1.5 million or $1.9 million. This is in range of the estimated cost. Also, the longer the treatment benefits last, the more cost effective the treatment becomes. A lifetime of transfusions or hospital admissions adds up."""
https://sciencebasedmedicine.org/first-crispr-treatment-appr...
If I were a betting man I'd wager the house that the above is exactly why it costs what it does. 'Pay 2 million now, or pay 2 million over the rest of the patient's life as they suffer' is a pretty inarguable value proposition.
Of course once patents expire and processes refine prices will come down. The wheel of progress rolls on (more of less) as intended.
More info I found relevant regarding cost for typical treatment and out of pocket estimated costs: https://www.hematology.org/newsroom/press-releases/2022/the-...
There is always a some kind of moral dilemma: should you spent millions to try to extend extremely I'll person or help with that money to some healthy poor children?
Especially the one that extremely ill people need, humans.
2m is much higher that either costs or economical output the treated person could deliver through lifetime.
What if you are on those 10 poor kids he mentioned ?
I don't agree that OP statement is "crass". It's a very pragmatic and important question we have wrestle with.
But almost all care services end benefiting from some sort of subsidies. Even if just by increasing the cost of inssurance for the rest of the population
So in essence, you'd be trading the equivalent of one person's entire lifetime of productivity in exchange for the first generation of a radical new medicine whose outcome is unknowable.
I don't think it's crass to err on the side of caution for such a scenario.
[1] https://en.m.wikipedia.org/wiki/Per_capita_personal_income_i...
So if there even is an increase in the total cost of treatments, it's not at all a given it's a a net increase once account for decades of additional working life.
But as a society with limited resources we need to set priorities. I hope everyone will be able to receive treatment.
However, such treatment is only for rich people, or from rich countries.
Even some countries in Europe are not reach enough to pay for such medicine. Like Zolgensma, which also costs around 2 millions USD to cure SMA.
Not sure it's really easier though, economics and emotional affect are often at odds. Ask people if a hospital administrator should spend 100k on either a single liver transplant for an 11 year old girl, or spread over 100 less expensive life saving interventions for 50 year olds, most people will say save the girl and demand the administrator be fired for even needing to think about it.
(Half remembered but apparently real scenario, though I'm not sure where from)
Its very close to the lifetime average financial cost of medical services related to sickle cell disease for those with it, from things posted elsewhere in the thread. So its literally just paying the same (loosely) financial cost up front and then not having them suffer through the disease.
An incentive structure that encourages mostly making the wrong decisions on things like this when it comes to cost/quality-of-life is why the US has the most expensive healthcare system in the developed world on a per capita or per GDP basis, and doesn't have better-than-typical general outcomes to show for it.
Over-under on when the NHS agrees to pay for this?
Many societies with excessively strong opinions on morals however are literally living in huts.
Setting aside that the US government is deeply influenced by Christian conservatism and the culture by Puritan ideals, to the point that no American President can be elected without vocally professing faith in God, Christ, or being seen with a Bible in hand, and thus is the most moralizing culture within Western civilization by far (particularly where sex and gender are concerned,) which hut-dwelling societies are you talking about, specifically?
https://www.pewresearch.org/short-reads/2019/05/01/with-high...
The US can be said to be both. E.g. you won't find many "huts" along the northeastern coast, but search and you'll find them elsewhere:
https://en.wikipedia.org/wiki/List_of_U.S._states_and_territ...
I'll leave it as an exercise for the reader to determine where the most "huts" are found.
So it's flat out wrong to claim that some sort of perceived moral bankruptcy with regards to the value of human life has left our society in the stone ages, when all evidence points to the contrary.
Under utilitarian capitalism people are expendable, but in the meantime they are less likely to dwell in huts than morally superior societies.
Note that I've blatantly equated religion with moral here.
Are they not worth saving? Because they are far away and have small purchasing power there is small sense to help them.
If we prevented treatment because the money could be used elsewhere, we likely wouldn’t/won’t develop a drug that we could eventually[1] make cheap enough to cure these kids and give them longer lives too. We can do better!
[1] There is a cynical take here about drug costs, geopolitics, etc. but I am rejecting that cynicism.
How much did the first human genome sequence cost? (Effectively several billion dollars—now $1000.) How much did the first organ transplant cost? How much did the first electronic computer cost?
Yes, cost will slow widespread use but it will spur the. next wave of innovation—-some motivated by profit, some motivated by social altruism.
Healthcare? People pay to be and stay healthy. The money was earmarked specifically for this purpose. Also, in the long run, you will be able to cure diseases even those "healthy" children have.
The price you see now will likely shrink in the coming years. Pretty good opportunity for an analysis on CRISPR pricing if you have a well-trafficked blog and are willing to track this for the next five years.
There are a lot of steps in between "acute HCV infection" and "requiring a liver transplant", and many insurers, even today, will require you to go through some or all of them before considering paying for HCV antivirals.
That's not a great comparison. There was a previous cure for hepatitis C before the first antiviral-based cure 2013, and the initial treatment regiment for the antiviral based regimen was a hybrid of the two, before they settled on a fully antiviral-based treatment.
The reason that the antivirals came down in price so quickly was because so many nearly-identical ones came on the market within a couple of years. That's due to the discovery of a particular protein and corresponding class of inhibitors some years earlier, which was not patented, opening the door for a flood of drugs which are all functionally identical in purpose and mechanism of action, but chemically distinct and eligible for separate patent protections.
That came at a time when political and other pressures made some private insurers more willing to approve treatment (usually after a few rounds of denials and appeals) - but again, with an emphasis on some, because there are large classes of people for whom it is difficult or impossible to get treated for HCV today. (They're just not the ones likely to comment on HN).
Contrast to this treatment, which is for a congenital condition that does not have the same political pressure to address, and for which a significant financial barrier to access is not merely the costs of the drug, but the cost of the associated care (chemotherapy, etc.) which is not included in the quoted price. In addition the patent laws function differently in this case, to the detriment of patients.
The history of hepatitis C and its treatment is fairly idiosyncratic and it would be a mistake to use the price trajectory of HCV antivirals as a predictor for any other treatment.
Of course, insurance and taxation can be viewed as similar things anyway, but it is different from things like term life insurance or motor vehicle insurance or home owners insurance.
Humm no... It's just risk amortization...
The premiums are very explicitly a subsidy from young to old, which I view as a tax by a different name. Except instead of it being based on one’s income/wealth, it is based on age.
Health risks in general are very predictable and very high, especially as one ages.
I do not follow the point you are making here. The regulation and legislation around health insurance do not change the nature of it.
I think you are assuming that insurers have perfect risk assessments power and thus regulating them should be unnecessary. But they don't and we have to.
> The premiums are very explicitly a subsidy from young to old
You are just repeating your assertions here. I would love some arguments.
> which I view as a tax by a different name. Except instead of it being based on one’s income/wealth, it is based on age.
Sure, as long as we agree that is just your point view and nothing rooted in reality.
Instead of charging a sicker or older person $10,000 per month and healthier or younger people $100 per month because that is close to the expected loss in the calendar year for the insurer, they are mandated to charge younger/healthier people $1,000 per month so the older person can only be charged $3,000 per month.
Imagine a similar law for motor vehicles. The car insurance companies can only charge the worst and riskiest drivers 3x what the safest driver pays. Basically, you can keep getting into collisions and at some point your premium will stop increasing. Where will the money to pay for all the damages come?
> I think you are assuming that insurers have perfect risk assessments power and thus regulating them should be unnecessary.
I do not assume this. Insurance and tax/wealth redistribution is a spectrum.
When we offer a service to a group of person, and somehow mandate a flat price for that service. The people using the service less are subsidizing the cost for the people who use the service less.?
> The people using the service less are subsidizing the cost for the people who use the service more.?
Also, it is not a flat price, it is a capped price.
As an aside, think about how the optics would have been if the politicians were transparent that a significant portion of the tax liability to pay for the healthcare would be levied based on age.
Then think about older, rich people taking advantage of this and retiring early (between age 50 to 65), and because they can afford to have very low income (but a lot of assets), they qualify for even more subsidies during their most expensive years to insure, without negatively affecting their lifestyle.
The trouble is, as social cohesion breaks down, and demographic cliffs approach, people lose faith that long-term programs will still be there for them as they age. Perceptual time horizons shrink, and the arguments that you have made begin to resonate.
I don't have a good answer for either of those problems. Immigration solves the demographic cliff, but appears to threaten social cohesion. We can get into tedious and repetitious arguments about why that is, but let's please not?
When you (or your employer) buys a policy from UNH, Elevance, Cigna, CVS, Humana, etc, part of what they pay for is access to the MCOs pricing services. And vetting services to minimize errors/fraud (a process which itself is ridden with errors/fraud).
Not really, no. Most people will die of something expensive, but not $2M expensive. A quick google says that per-capita lifetime health care expediture is ~$300k.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1361028/
That data is from the late 1990s, before the Affordable Care Act greatly expanded access to healthcare, and many new treatment options have become available since then.
What I meant, though, is that across a big population’s entire lifetime, there will be a ton of high healthcare cost events. And with technological progress, new treatments will always be coming out. Which is a great thing, just not what is typically thought of as an “insurable risk”.
Sorry, how does that follow? Insurance works any time you have a function with predictable average but high variance. Is the total health care expenditure across a relevant subscriber base in 2023 very close to 2022? Then you can make insurance work. It's just math.
Non insurable risk as in charging someone a premium unrelated to their specific expected loss (which is what health “insurance” is).
What you're saying doesn't make sense. There's no difference between health insurance and any other insurance in the way it works. You collect reliable and regular premiums from everyone, pay out unreliable/bursty (but statistically very predictable in aggregate!) losses as contracted, and pocket the remainder as profit. And it works.
Really, I don't know what you're talking about here. Health insurance is "expensive" in the US, sure. But it's not failing.
For example, if you carelessly drive and get into car collisions where you are at fault, your premiums go up, because your expected loss goes up.
In health “insurance”, it does not matter what you do, because your premiums are not based on your expected health costs. Hence it is more akin to a tax (or subsidy).
In practice, car insurers are allowed to partition their customers this way because it's felt to be "fair" and because it encourages safe driving. Trying to partition health insurance customers like that feels "unfair", and has minimal net benefit as health expenses aren't as controllable-by-the-subscriber as car accidents are. So we pass laws about how the partitioning gets done.
But again, "insurance" as a business model (and mathematical model) works EXACTLY THE SAME WAY. The only difference is how you draw the lines around who gets insured at what rate.
I do not dispute this. As I wrote in a sibling comment:
>Insurance and tax/wealth redistribution is a spectrum.
If this is a cure (I haven't looked at the data), imagine the NPV of a lifetime of multiple hospitalizations per year. It's likely in the millions.
[0] https://sciencebasedmedicine.org/first-crispr-treatment-appr...
The son tried for a long time to hold a job but couldn't because of how much time he would miss.
I hope they are able to get this.
“Editing Humanity” by Kevin Davies covers the history and near future of CRISPR and includes a great chapter on describing both of these therapies.
As breathtakingly advanced and unprecedented as CRISPR treatment is, the execution is still radical and crude by necessity. Not to diminish the accomplishments but to note that we still have a great deal of room to grow.
Hopefully knowledge will eventually advance so that less extreme and unpleasant methods will take its place. But that would be a tough nut to crack.
Americans have significantly less debt than Western Europeans. Their average appears to be 50% higher than ours. Including medical debt.
The idea, as per GP, that this treatment will inexplicably drive Americans to bankruptcy, is stupid. It’s offensively dumb. It’s the kind of uninformed doomer nonsense that runs rampant online progressive echo chambers, but has no basis in reality.
> …which most first worlders never have to think about.
Those lucky bastards. With 50% more debt.
Look at access to the cystic fibrosis therapies ($300,000 per year). The US (including Medicaid) were paying for them from launch in 2012.
The UK just came to an agreement for NHS to pay for it in 2020. 8 years later.
In Canada, only 5 of the 12 provinces/territories pay for it and only if your a child. Adults don't get it.
You actually stand the highest chance of getting it in the US.
"An August report from the nonprofit Institute for Clinical and Economic Review found that the treatment and similar gene-editing therapies for sickle cell disease would be cost-effective if priced between $1.35 million and $2.05 million. In the U.S., patients with the condition and their insurers pay on average between $1.6 million and $1.7 million for disease management over the course of a lifetime." Source: https://www.politico.com/news/2023/12/08/fda-gene-editing-th...
I imagine an expensive part of this process is incubating the treated stem cells to increase their numbers, and then just the cost of the hospital time while the new cells establish and the patients immune system recovers from the chemotherapy.