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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

#121
post #117

Earlier quoted context omitted.

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…

> the collective probability of rare, but expensive health issues is basically 100% 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.

If you are referencing this

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”.

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

#122
post #115

Earlier quoted context omitted.

>the cancers were caused by the chemotherapy conditioning regimen for the treatment, not Lyfgenia itself. 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.

The article says "patients must undergo a preparatory treatment with a chemotherapy drug to remove any native stem cells that might remain in their bone marrow." It doesn't make much difference to the patient if it's the Lyfgenia itself or the chemo drug, if the chemo drug is a requirement. Right?

Fair enough, but there remains the possibility of finding an alternate, safer chemotherapy drug.

Does anyone know if changing the drug would require a new FDA approval for the entire regimen, or could the protocol be easily changed?

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

#123

Earlier quoted context omitted.

> which is why this gene has not been bred out of the population over time. Is that why? Or is it just the people with it aren’t sick enough to die before procreating?

It's a recessive/heterozygous thing. If you get the gene from neither parent, you're vulnerable to malaria. If you get the gene from either parent, you're immune to malaria and don't get sickle cell. If you get the gene from both parents, you get sickle cell. A hypothetical future person who's going to be born in an area with a lot of malaria would really want exactly one parent with sickle cell and one parent lackin…

Can they do sperm (or egg) selection to change those odds for IVF?

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

#124

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!

Unless you are diabetic, aspartame is much worse for you than sugar. It causes metabolic issues, such as reduced metabolism (leading to more weight gain than a subjectively equivalent amount of sugar), migraines in some people, interacts with drugs, is bad for your digestive tract, and probably has other side effects.

Even if you are diabetic, you can already eat apples. They have a low glycemic index.

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

#125
post #95

Earlier quoted context omitted.

> which is why this gene has not been bred out of the population over time. Is that why? Or is it just the people with it aren’t sick enough to die before procreating?

Without access to modern hospital treatments it is fairly normal to die very young from sickle cell disease - it causes 100k+ deaths a year. 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 cris…

I think this is right, but just to spell out the recessive gene implications for readers, here's the Punnnett square[1] :

      R  | r
    +----+----+
  R | RR | Rr |
  --+---------+
  r | Rr | rr |
    +---------+
The people with sickle cell disease are "rr" — that's 1/4 the population.

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.

[1] https://en.wikipedia.org/wiki/Punnett_square

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

#126

And US patients won't be able to get it because they're going to be overcharged and drowned in debt to the point of bankruptcy.

Compared to similarly developed countries, Americans are actually in great shape when it comes to debt. See the OECD Data: https://data.oecd.org/hha/household-debt.htm

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

#127

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?

This is the effective altruism / utilitarianism insanity. If we only thought about "what the best use of $2 million is" we would still be living in huts.

What about real people who are living in huts right now? With few million you can drastically improve thousands of lives.

Are they not worth saving? Because they are far away and have small purchasing power there is small sense to help them.

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

#128

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…

Pretty cool, I wonder if it changes the chemical composition of the soil in any way compared to a regular potato.

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

#129
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?

I understand the dilemma, but many of those same children you are thinking of live in poverty in places such as Africa and with the misfortune of sickle cell disease.

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.

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

#130

Earlier quoted context omitted.

[flagged]

> According to Wikipedia, sickle cell disease is a genetic disease meaning that it is caused by mistakes in DNA. If parents did DNA testing and were warned that they are carriers of invalid DNA and decided not to have children, maybe there would be no need to do costly research and expensive treatment. The sickle cell allele is also a malaria resistance allele. Heterozygous = malaria resistance. Homozygous = sickle c…

There are other DNA mutations that give no advantage. For example, a mutation that causes nerves to die gradually.
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