I took a look at some of the aftermath reports (i.e. https://pmc.ncbi.nlm.nih.gov/articles/PMC10638066/ and some others) which get into specific details about the course of treatment in several patients who died from this complication. The through-line is an aggressive use of several immune suppressing and modulating therapies to calm the cascade.
I have to admit I can't find any specific discussion about dialysis in that context, so I can only assume that removal of the viral particles would be a case of closing the barn door after the horse escaped.
Maybe a better phrasing of your question would be:
> Why is hemodialysis ineffective for this?
(because, it definitely look like gambling, like "investors are behind us right now, so we have the money to do it, so let's do it before money runs out")
They’re not trying to kill people. There is a hell of a lot more money in _not_ killing people.
I'd be curious what the numbers are for the "good" that this therapy does; is there any way that this therapy is still "worth it" at any scale? but I know little about this area so that's a fairly naive question.
It seems to me to be similar to the approval of the three Alzheimer's drugs which don't really show improvement either- it seems like over the past decade the FDA has wanted to approve drugs that might work for diseases where there was no treatment at all (while saying things "delivering hope"). And it's not gone well, and has not been a good idea.
"The other problem is with viral vector based gene therapy is you can’t have it again. You develop antibodies which prevent it from working again, and it could cause a dangerous immune response."
Just wondering - would it make sense to immune-suppress the patient for a short period of administering of the viral-based therapy.
And as they describe that most gene therapies affect only extra-nuclear DNA, and thus have no permanent effect, wouldn't mRNA work better then in such cases - naturally the tech wasn't there 10+ years ago, yet today thanks to COVID it is here.
Edit (due to posting rate limit) in response to comment below:
I was thinking about mRNA coding dystrophin like it was coding COVID protein - should be cheap and easy (well, for some definition of easy in that context) doable, and it would be like a weekly self-injection - no toxicity, etc. Of course fixing the issue once for life would be better, once such cure becomes available, yet for now it would be similar like diabetics have with insulin - hassle for sure, yet it works.
Certainly the vaccine's mRNA sequence breaks down into separate nucleotides. If it did not, continued production of the antigens would cause a chronic immune reaction and/or immune exhaustion that would make the vaccine ineffective.
I don't know what happens to the N1-Methylpseudouridine though. That's an interesting question.
The modifications to increase mRNA half-life concerned mostly the caps and poly(A) tail. But even with those the persistence was in the range of days (sort of depending on how sensitive a method you picked).
mRNA is in comparison very transient (in the range of days, and that's being charitable), even when modified (5' cap, uridine analogs, poly(A) tail) as it was in COVID vaccines. This is fine for vaccines, as you essentially want just a single exposure to the protein with each vaccine dose. You do need dystrophin continuously though (even though the cells are not dividing much, they are still recycling it).
You could argue for delivering gene therapy with mRNA/NLPs in multiple doses over the course of patient's life but that would likely 1) exacerbate toxicity and 2) be super-expensive
https://medcitynews.com/2025/07/sarepta-gene-therapy-fatalit...
https://www.axios.com/2025/04/18/rfk-jrs-potential-future-ta...
https://kffhealthnews.org/news/article/nih-grants-mrna-vacci...
> National Institutes of Health officials have urged scientists to remove all references to mRNA vaccine technology from their grant applications, two researchers said, in a move that signaled the agency might abandon a promising field of medical research.
Reasons to use AAV: they're going for sustained production of the therapeutic gene, and AAVs are better at doing that than LNPs. LNPs were used in the mRNA COVID vaccine, because they're great at transient production.
To get stable production from an LNP you'd likely have to integrate into the genome, which risks cancer from disrupting oncogenes. You'd also need to package the therapeutic gene with a mechanism of integrating into the genome, like recombinase.
Thoughts from Derek Lowe (In The Pipeline).
https://www.science.org/content/blog-post/sarepta-s-approval... ("Sarepta's Approval Woes" (2013))
https://www.science.org/content/blog-post/sarepta-s-duchenne... ("Sarepta's Duchenne Therapy Is A Lot Further Away" (2014))
https://www.science.org/content/blog-post/sarepta-s-day-fda ("Sarepta's Day at the FDA " (2016))
https://www.science.org/content/blog-post/sarepta-gets-appro... ("Sarepta Gets An Approval - Unfortunately" (2016))
https://www.science.org/content/blog-post/gene-therapy-duche... ("Gene Therapy for Duchenne" (2018))
https://www.science.org/content/blog-post/opening-lid-sarept... ("Opening the Lid on Sarepta's Drug Approvals" (2020))
https://www.science.org/content/blog-post/sarepta-tries-agai... ("Sarepta Tries Again" (2023))
https://www.science.org/content/blog-post/sarepta-why ("Sarepta. Why?" (2024))
That happens to anything recombinant we produce: crops, cattle, bacteria. That even applies to dog breeds.
"Advantageous" changes as the environment changes. Being a large long lived strong dinosaur is advantageous until meteorite? Then being a small mammal is advantageous. A population explosion of locusts ravages all plants successfully, until a fungus infects and kills almost all, thankfully for locusts some of them which were not "the best" survived that one infection.
And the same homogeneity means that developing a defense for any future hostile organism is much more straightforward, just like e.g. developing software that works on windows is much easier than developing software that works on windows, Linux and mac.
This is also happening with other types of pathogens - antibiotic resistant illnesses are on the rise because we used quickly created defenses to eliminate all but the strongest versions of them. We have very few effective anti-fungal medications, and most of those are very risky.
If we were good at developing defenses for homogeneity, farmers all over the world wouldn't be fungi destroying the monocultures we depend on for modern agriculture (bananas and corn are really great examples). Estimates are that as much of 30% of global crops are lost to fungal infections; I sincerely doubt that homogeneity is the panacea you assume it is.
> Making fungus-resistant agriculture is challenging because fungi share many cellular similarities with humans, making it difficult to develop fungicides that target fungi without harming plants or humans.
Think again about your statement, what you're saying is the fitest is the easiest to manipulate? Thats just mindboggling bad, cause you'd also be a honey pot for all the other bacteria and viruses out there.
When people die, we change policy. When people feel like they cannot get the treatment they need, we change policy.
Unfortunately, this is very complicated and emotionally heavy, and it is much easier to set down the burden on someone else's shoulders, in the form of blame.
We want the FDA to do life critical, complicated and contradictory things, so it's easy to create a narrative that blames the FDA.
The other option besides blame is shared responsibility and humility, but it feels like people are not very good at thinking that way right now.
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