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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

#91

Here's basically how the process works: * 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…

is crispr a big improvement here over AAVs or zinc fingies?

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

#92

Earlier quoted context omitted.

The Hep C cure was 100k USD when it released in 2014. 10 years later, it’s 25k USD max before insurance. 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.

And much better than a liver transplant.

> And much better than a liver transplant.

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.

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

#93

Earlier quoted context omitted.

Worse taste but probably healthier in this case.

Yeah potatoes will fill your stomach and better than starving but they aren't really healthy food. Eat them in moderation, and prefer sweet potatoes/yams.

>prefer sweet potatoes/yams.

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.

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

#94
post #53

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.

Of course I will do anything to prolong life of myself and my family, like any human being.

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.

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

#95
post #44

The gene that causes sickle cell anaemia actually provides partial immunity to malaria, which is why this gene has not been bred out of the population over time.

> 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 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.

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

#96

Earlier quoted context omitted.

Worse taste but probably healthier in this case.

Yeah potatoes will fill your stomach and better than starving but they aren't really healthy food. Eat them in moderation, and prefer sweet potatoes/yams.

On the contrary, potatoes are full of good stuff: https://www.mayoclinichealthsystem.org/hometown-health/speak...

Just make sure to go easy on the toppings!

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

#97
post #29
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?

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…

Sadly, unlike tech, the price of drugs doesn't always go down over time.

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

#98

Earlier quoted context omitted.

If it was just that (in the US), then there would be no need to prevent insurers from pricing based on health of the insured. Or legislating a 3x cap on premiums between highest and lowest premium. Or legislating out of pocket maximums. 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.

> If it was just that (in the US), then there would be non need to prevent insurers from pricing based on health of the insured. Or legislating a 3x cap on premiums between highest and lowest premium. Or legislating out of pocket maximums. 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 pe…

Sorry, I don’t really know how else I can explain it. The age rating factor itself is pretty self explanatory.

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.

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

#99
post #73

Earlier quoted context omitted.

> 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.

Except those 10 poor kids aren't spending their own money to save themselves. The patient is though which is the point.

if it's their own money sure.

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

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

#100

Earlier quoted context omitted.

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

Yes, but spending it preventing debilitating disease that would cost about the same amount over the lifetime of the sufferer is a no-brainer, even in net econonic output, terms.

But it is so only for few countries with ridiculously high costs of medical services. What about other? If we are talking about someone from south America?

2m is much higher that either costs or economical output the treated person could deliver through lifetime.

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