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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

#191

Post by dsmmcken »

mritchie712 wrote: post #146
graphe wrote: post #120

Earlier quoted context omitted.

I would have bred them for more potato protein. It's a very close meat protein substitute by essential amino acids. Might be why it tastes so good. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6245118/ >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…

Not quite vegan, but I like cricket protein.

Cricket protein can trigger shellfish allergies, fyi.

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

#192

Post by eszed »

skissane wrote: post #189
eszed wrote: post #122

Earlier quoted context omitted.

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?

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

What a weird system: there's something better, but the manufacturer isn't allowed to tell you about it. What if they, like, slide the journal article across the desk, whilst holding their finger alongside their nose and winking?

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

#193

Post by eszed »

firejake308 wrote: post #148
eszed wrote: post #122

Earlier quoted context omitted.

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?

It's reasonable to expect small improvements in the risk profile, but I think blood cancer is going to be a side effect for any drug following this basic idea. You will always need some chemo to destroy the defective blood-making stem cells before replacing them with the genetically-modified blood-making stem cells, and any chemo that is strong enough to kill all of the blood-making stem cells in your body is necessa…

That makes sense. Thank you.

In theory, could a separate gene therapy target and knock out the stem cells that carry the mutation?

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

#194

Post by skissane »

eszed wrote: post #192
skissane wrote: post #189

Earlier quoted context omitted.

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

What a weird system: there's something better, but the manufacturer isn't allowed to tell you about it . What if they, like, slide the journal article across the desk, whilst holding their finger alongside their nose and winking?

> What a weird system: there's something better, but the manufacturer isn't allowed to tell you about it.

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.)

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

#195

Post by Lord-Jobo »

hedora wrote: post #124
pastor_bob wrote: post #59

Earlier quoted context omitted.

>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.

Decades of research have found rare, but still very mildly negative health results from aspartame, and an overwhelming flood of direct evidence for strong negative health effects from sugar.

Back up what you are saying with some studies. Because what you are claiming is going against a LOT of modern medical knowledge.

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

#196

Post by refurb »

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?

Sure. It's pretty typical for sickle cell patients to have to go to the hospital severe times per year for "sickle cell crisis".

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.

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

#197

Post by refurb »

1letterunixname wrote: post #47

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.

A very lazy reply.

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.

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