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FDA approves a CRISPR-based medicine for treatment of sickle cell disease

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Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#51

Post by JumpCrisscross »

csdvrx wrote: post #8
trident5000 wrote: post #2

Pretty big news. I believe this is the first gene editing therapy approved by the FDA and theres a large backlog thats been in the works for many years. Id like to see the flood gates really open up for gene editing for diseases, preventative treatments, and even cosmetic.

> Id like to see the flood gates really open up for gene editing for diseases, preventative treatments, and even cosmetic. 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!

> some people are ethically opposed to that

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.)

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#53

Post by esturk »

ponector wrote: post #37
ericmay wrote: post #29

Earlier quoted context omitted.

This is Day 1 so the price and how well it works today is almost certainly the worst it will ever be. Insurance will likely cover the cost. It's a very bad, painful, and outright deadly genetic mutation and $2.2 million is practically nothing compared to doubling someone's lifespan or giving them an extra 10 years. More info I found relevant regarding cost for typical treatment and out of pocket estimated costs: http…

I don't agree that it is nothing. 2 millions, if applied properly, could do good for many people. Take ten children from poverty, give ten children chance to get a good education, etc. 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?

Such a crass statement. What if you're the patient? Would you spend 2 million to live 30-40 more years? It's so easy to step back and weight the lives of other as if you're making the decision for others.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#54

Post by lotsofpulp »

esturk wrote: post #53
ponector wrote: post #37

Earlier quoted context omitted.

I don't agree that it is nothing. 2 millions, if applied properly, could do good for many people. Take ten children from poverty, give ten children chance to get a good education, etc. 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?

Such a crass statement. What if you're the patient? Would you spend 2 million to live 30-40 more years? It's so easy to step back and weight the lives of other as if you're making the decision for others.

The $2M represents a certain portion of society’s productivity, which is not unlimited.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#55

Post by huytersd »

jabbany wrote: post #31
mritchie712 wrote: post #25

Ohalo (the company Dave Friedberg is now CEO of) recently got approval for a potato edited by CRISPR: > 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…

Hmmm. That second one reminds me of the Flavr Savr ( https://en.m.wikipedia.org/wiki/Flavr_Savr ). More shelf life in exchange for likely worse taste...

Worse taste but probably healthier in this case.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#56

Post by EvanAnderson »

The mechanism by which this treatment works is really neat (some notes in [0]). The treatment increases the production of a fetal version of hemoglobin. That fetal hemoglobin is unaffected by the sickle cell mutation. Presumably everybody who is alive (and not a fetus) has a good copy of this fetal hemoglobin gene and it "just" needs re-activated.

[0] https://sciencebasedmedicine.org/first-crispr-treatment-appr...

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#57

Post by lotsofpulp »

ajross wrote: post #41
SamBam wrote: post #21

> Vertex set the price of Casgevy at $2.2 million > 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?

Collectively paying for rare but expensive treatments is literally the problem that insurance solves. This isn't wildly out of the expected range for this sort of thing. And it will surely get cheaper as it evolves.

Since the collective probability of rare, but expensive health issues is basically 100%, I would describe it less as insurance and more as wealth redistribution. Hence the (typical) requirement to purchase insurance and lack of ability to price it based on risk.

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.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#58

Post by huytersd »

lotsofpulp wrote: post #50
dopa42365 wrote: post #45

Earlier quoted context omitted.

It's possible to do both :)

How? Society does not have unlimited resources. Especially the one that extremely ill people need, humans.

People with sickle cell already cost the taxpayer a lot of money over their lifetimes. I wouldn’t be surprised if it cost more than this treatment.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#59

Post by pastor_bob »

mritchie712 wrote: post #25

Ohalo (the company Dave Friedberg is now CEO of) recently got approval for a potato edited by CRISPR: > 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…

>and another which results in reduced glucose and fructose content in the potato,

That's pretty amazing. Imagine if we can change apples to produce Aspartame instead of sugars!

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#60

Post by soulbadguy »

esturk wrote: post #53
ponector wrote: post #37

Earlier quoted context omitted.

I don't agree that it is nothing. 2 millions, if applied properly, could do good for many people. Take ten children from poverty, give ten children chance to get a good education, etc. 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?

Such a crass statement. What if you're the patient? Would you spend 2 million to live 30-40 more years? It's so easy to step back and weight the lives of other as if you're making the decision for others.

> What if you're the patient?

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.

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