Live data from Hacker News

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

statnews.com

181–190 of 228 posts

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

#181

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?

Malaria often kills people before they reach the age of being able to procreate.

[deleted]

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

#182
post #124

Earlier quoted context omitted.

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.

> * reduced metabolism (leading to more weight gain than a subjectively equivalent amount of sugar),* Doesn't that mean aspartame somehow contains more energy than sugar? I mean, assuming the increased weight is fat, that is stored extra energy.

There is some evidence that artificial sweeteners increase insulin resistance: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7014832/

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.

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

#183
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…

1. Soy, brown rice, pea, corn, and potato all hit their EAA/total protein cut-off. 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…

Don't compare whole potato, it should be to potato protein extract.

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

#184
post #122
post #115

Earlier quoted context omitted.

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?

[deleted]

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

#185
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?

There is active research on doing gene therapy without requiring chemotherapy (or radiation) first. It has been shown to work in mice, and they may eventually get it working in humans too. It would likely require a significant modification to these gene therapies though, since one approach is to alter the method of growing the stem cells to produce a significantly higher number, and then transplant that, with the hope that the transplanted edited cells outnumber and outnumber the original unedited ones. So very likely the FDA would treat that as a new therapy requiring a new approval process.

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-...

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

#186
My neighbor has 2 adult children with sickle cell. Its very tough watching what the have to go through. Frequent ambulance visits followed by multiple days in the hospital. Lots of pain in everyday life. The daughter is legally blind from complications.

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.

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

#188

Earlier quoted context omitted.

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

Don't be facetious. Parent was obviously referring to medical debt, which most first worlders never have to think about.

I wasn’t being facetious, I (wrongly) assumed you could infer the argument:

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.

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

#189
post #122
post #115

Earlier quoted context omitted.

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?

> 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 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.

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

#190

Earlier quoted context omitted.

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 h…

As mentioned above—this is day 1. 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.

I wrote the OP :) I agree with you. What I was trying to highlight is that many of the people the person who originally responded to me was concerned about have the exact disease! And we need continued development with initial high costs to hopefully bring the costs down.
Post reply on HN