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FDA takes action to make a treatment available for autism symptoms

52 points| amai | 6 months ago |fda.gov | reply

123 comments

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[+] leakycap|6 months ago|reply
> "The U.S. Food and Drug Administration today initiated the approval of leucovorin calcium tablets for patients with cerebral folate deficiency (CFD) ... Individuals with cerebral folate deficiency have been observed to have developmental delays with autistic features (e.g., challenges with social communication, sensory processing, and repetitive behaviors), seizures, and problems with movement and coordination."

The wording, my emphasis added, certainly suggests this is a new med for CFD even though the title mentions Autism symptoms, not CFD

Search for the word autism in the release and tell me if you think this treats what they suggest in the title. It would be every other word if they believed it.

[+] Mizza|6 months ago|reply
WashPo article on this was interesting: https://www.washingtonpost.com/health/2025/09/22/leucovorin-...

Seems like it works only for a very specific type of childhood autism, but if my child had this I would be kicking down doors to get it. The article has some good insight into how honest researchers feel about their work being trumpeted by the scientifically illiterate carnival barkers in charge of things.

[+] dsr_|6 months ago|reply
This is absolute corruption of the FDA's mission to accurately label and appropriately market drugs.

The evidence is from a study with N=40. Not 40,000. 40.

[+] metalcrow|6 months ago|reply
Can you link to the study? The release implies there are multiple but doesn't link to any
[+] OkayPhysicist|6 months ago|reply
Sample size doesn't tell you everything about a study. You don't need to throw 40,000 people out of an airplane to determine it's safer with a parachute.
[+] Glyptodon|6 months ago|reply
I'm surprised they didn't just "partner" with General Mills or some such to turn this into an advertisement for folate fortification in sugary breakfast cereals.

For those replying: I did not mean this would seriously work, but that it would be par for this administration to basically bring cereal ads from Saturday morning cartoons into the CDC.

[+] trehalose|6 months ago|reply
Leucovorin works (at least for some cases of CFD) because it bypasses a folate transport protein that can be nonfunctional and fail to transport ordinary folic acid into the brain. I guess that doesn't necessarily mean they couldn't do what you're suggesting though.
[+] reenorap|6 months ago|reply
The dose in the pill is very high, it's not something you can just let everyone take.
[+] incomingpain|6 months ago|reply
The typical folate in food or even the pregnant women folic acid supplement is good at preventing these birth defects; but for someone with NDD it would actually not help and depending on dose block up the pathways for it to heal.
[+] nineplay|6 months ago|reply
Looking for The Cause of autism and The Cure for autism is exactly as absurd as looking for The Cause of cancer and The Cure for cancer. I think most people understand that there are many causes of cancer and many treatments that cover a range of different use cases.

The whole concept of a cause and cure is really damaging to the autistic community and just flying in the face of any sort of intelligent diagnosis and treatment.

[+] tonmoy|6 months ago|reply
> The FDA has conducted a systematic analysis of literature published between 2009-2024, including published case reports with patient-level information, as well as mechanistic data, and has determined that the information supports a finding that leucovorin calcium can help individuals suffering from CFD.

I would have liked to see some citations. I’m mostly curious about the sample sizes

[+] newobj|6 months ago|reply
I know they're not serious because they didn't recommend methylcobalamin.

Kinda funny how in all this chatter no one is talking about MTHFR etc.

[+] malfist|6 months ago|reply
Perhaps because B12 supplementation isn't a cure for autism?
[+] amai|6 months ago|reply
I wonder about the following: Leucovorin is most often given in the form of Leucovorin calcium. That is interesting, because calcium is needed to regulate the release of dopamine in the brain. And this might also help with autism:

https://news.ki.se/new-study-links-dopamine-to-autism-sympto...

However there is also Leucovorin sodium.

Are there any studies that compare Leucovorin calcium vs. Leucovorin sodium in autism? Maybe it is in fact the calcium that helps with autism and not the Leucovorin part?

[+] codedokode|6 months ago|reply
Wikipedia says that autism is caused by DNA errors, so the only perfect cure would be, as with many other incurable diseases, DNA editing? I wonder if we come into the future where parents will be able to buy DNA fragments for example, for certain eye color or size, or for thin hands and legs, and collect future child's DNA from pieces. Or DNA will be considered non-copyrighted and owner of elf ears won't be able to sell his unique feature? Seems not fair to me. Also whether there will be pirate sites with DNA.
[+] notacoward|6 months ago|reply
It's also worth noting that leucovorin is an important drug for counteracting the effects of methotrexate (chemotherapy drug), and this thinly veiled attempt to drive profit for Dr. Oz's company could create a shortage. This kind of thing has happened before, eg. with some of the bogus COVID treatments. In other words, this is not just unhelpful. It might actually kill people.
[+] sys32768|6 months ago|reply
For the non-partisans who actually care more about autism in children than they do throwing ideological jabs, there are numerous interesting anecdotal reports from parents of children who have been trying leucovorin in the past year or so:

https://www.reddit.com/r/Autism_Parenting/search/?q=leucovor...

My hope is that this does lead to breakthroughs in understanding the mechanism for some types of autism, and perhaps even a "miracle" drug akin to how stimulants can radically transform the brains of many with ADHD.

[+] yibg|6 months ago|reply
You can have anecdotal about anything in any direction. Not a great way to come to a scientific conclusion.
[+] incomingpain|6 months ago|reply
>For the non-partisans who actually care more about autism in children than they do throwing ideological jabs

It's interesting how USA politics has polarized the autism community so heavily.

Leucovorin only helps in 1 of 6 ways the prenatal damage occurs.Sucks as well that it's prescription.

L-Methylfolate might work as well, as a supplement alternative? Havent tried it. Same with NAC, havent tried it.

CBD will help, no thc allowed, no alcohol allowed.

Omega 3 fish oils will help.

Hot showers will help.

Whole foods, low carbs will help.

[+] lupusreal|6 months ago|reply
You can find Facebook groups filled with thousands of parents who report success with literally any wacky "treatment" you can think of, from chiropractic cures to cancer to making their children drink bleach to "cleanse parasites".
[+] morkalork|6 months ago|reply
Apparently Dr Oz stands to profit from one of those approvals..
[+] tencentshill|6 months ago|reply
The cure is a simple pill, provided by a company with a long history of corruption, prosecuted for "promotion of drugs for unapproved uses, failure to report safety data and kickbacks to physicians in the United States".

A company that operates like that sees opportunity in this US administration.

https://en.wikipedia.org/wiki/GSK_plc

[+] rdtsc|6 months ago|reply
Wondering which drug companies are trustworthy. Let's say if this was Eli Lilly or Pfizer, would that make the announcement blurb better? As in, we see it and say "oh wow, that's sounds great". I imagine if we insert any name in there it would still read just about the same?
[+] reissbaker|6 months ago|reply
GSK is one of the largest pharma companies in the world, created the malaria vaccine, the first HIV antiretroviral, amoxicillin, valacyclovir, and like a zillion other important drugs.

It's also not even an American company, despite your insinuation: it's British. This isn't some fly-by-night operation created by Trump or whatever.

How well will this pill work? I don't know. There is reasonably good research [1], (carried out during the Biden administration btw!) that a large percentage of autism is linked to folate deficiency due to autoantibodies that wreck your folate pathways, and that d,l-leucoverin bypasses those and restores folate to developing brains.

Complaining that it's "a simple pill" to me feels pretty anti-intellectual. So were antibiotics, so were antivirals, so were many other treatments for horrible diseases that just... are solved now. The fact that it's pill-shaped tells you nothing about the research it took to develop.

1: https://pubmed.ncbi.nlm.nih.gov/34834493/

[+] vkou|6 months ago|reply
If the adults ever return to the room, what exactly should be done by everyone responsible, to make nobody else even dream of doing this?
[+] kg|6 months ago|reply
I worry that once it turns out this doesn't actually work, there's going to be a revolving door of the 'next new Autism fix' and gradually kids with autism are going to be loaded up with more and more drugs of unproven effectiveness based on low-quality studies.
[+] jmclnx|6 months ago|reply
Does anyone with knowledge still pays attention to .gov sites ? Seems they are all filling up at different rates with pseudo science, or in other cases, removing science facts.
[+] jkaplowitz|6 months ago|reply
.gov is available to nonfederal US governmental entities as well. Those .gov sites run by states and cities which still believe in science are still worth paying attention to. Also even federal .gov sites remain reliable for certain topics like giving current statutory or regulatory text.
[+] josefritzishere|6 months ago|reply
This is the Amway administration. I am running out of words.
[+] silverquiet|6 months ago|reply
That was the last Trump administration, where the Secretary of Ed was married to the CEO of Amway. The current one is the former CEO of World Wrestling Entertainment, so I'd say it's more the Pro Wrestling administration.
[+] lupusreal|6 months ago|reply
This pill is an obvious scam. Legos are the treatment. Give autistic kids more legos.
[+] bobmcnamara|6 months ago|reply
No Lego. Only TRAAAAIIIINS