FDA takes action to make a treatment available for autism symptoms
103 comments
·September 23, 2025Mizza
perching_aix
Unpaywalled: https://archive.md/FpHmz
estimator7292
I'm about 98% certain this is simply a ploy to put autistic people on a list for future abuses.
In today's political climate, nobody should be revealing their autism status to anyone remotely connected to any level of government. Nor should one trust any sort of medical advice or reporting from same. Our government is explicitly targeting minorities of all sorts for abuse and persecution.
Now is, unfortunately, the time to hide and weather the storm. That or flee to another country with a functioning society.
Please protect your autistic children and friends. Times are real, real bad for them right now.
incomingpain
>I'm about 98% certain this is simply a ploy to put autistic people on a list for future abuses.
I'm a foreigner, not even in the USA. Your politics are melting down but I feel like the FDA isn't tracking or seeking to harm you.
>In today's political climate, nobody should be revealing their autism status to anyone remotely connected to any level of government. Nor should one trust any sort of medical advice or reporting from same. Our government is explicitly targeting minorities of all sorts for abuse and persecution.
Pretty strong argument against socialized medicine. Here in Canada the government actively tracks each of these. For example to get the covid shot, they wanted to know about my autism, non-binary/trans etc. They werent just asking everyone this, I didnt bring it up because why would it matter? I was in their system. I also happen to know this data is being transferred out of country to CDSI; ASN 23498.
>Now is, unfortunately, the time to hide and weather the storm. That or flee to another country with a functioning society.
I dont think you need to flee the usa. Canada has drastically reduced migration, you dont want to come here. Europe maybe?
leakycap
> "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.
dsr_
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
Can you link to the study? The release implies there are multiple but doesn't link to any
bunderbunder
Here's a PubMed search. https://pubmed.ncbi.nlm.nih.gov/?term=leucovorin+cerebral+fo...
It's worth noting that, in the absence of pre-registration, one should should assume that the body of literature (and any systematic review or meta-analysis that relies on it) could be significantly influenced by the file drawer effect.
Anyway, I looked at a recent systematic review from those search results (https://pmc.ncbi.nlm.nih.gov/articles/PMC8622150/), and it found exactly one double-blind RCT (https://pmc.ncbi.nlm.nih.gov/articles/PMC5794882/) that seemed to support what the FDA is saying here. It had a fairly short duration (12 weeks) and a small cohort (48). I'm not medical expert but I do read medical literature as an amateur, and I'm pretty sure this is nowhere close to the standard of evidence for establishing safety and efficacy that the FDA used to demand. It feels like we may be reverting all the way back to the evidentiary standards that allowed crap like thalidomide onto the market.
metalcrow
Thank you! That's useful. And yeah i agree, this and the aducanumab scandal make it seem the FDA is going away from "too strict" back to "too loose".
OkayPhysicist
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.
thaw13579
I reviewed the linked studies of folinic acid treatment in ASD, and they uniformly say that larger studies are needed before considering this as a widely available treatment. The main issues that need to be worked out are:
* who this applies to (some studies suggest genotype and autism subtype matter for getting positive outcomes)
* what the side effects are (12 week studies of <100 people are not enough to safely deploy this as a long-term treatment at scale)
* how this compares to behavioral treatment (ABA and sensory interventions have reliable positive outcomes as well)
I think there's a useful signal there, but we need to be cautious rolling things out at a national scale without bigger studies.
bunderbunder
It does tell you something. But that kind of sample size is arguably underpowered for anything but a preliminary study. It's the statistical equivalent of Hubble before it had its corrective optics installed: still fine for seeing big unsubtle things like whether parachutes are warranted when jumping from airplanes, but unable to resolve all the details you want to know before concluding that a medication is safe and effective.
yibg
This gets brought up often and while true isn't very useful. We know jumping out of plane is dangerous because we have a good understanding of the physics and we have many many comparable examples (jumping out of buildings etc). We understand the mechanism of action of parachutes, so we also know for example a tiny little parachute made of paper towel won't work, and we don't need to test it to know that.
Do we understand the cause of autism or how this supposed cure works?
OkayPhysicist
The key is effect size. We didn't know the exact method of action when we started injecting comatose diabetic children with insulin, but when the coma patient sits up and starts chatting with you, you can establish that a drug is effective with very small sample sizes.
Glyptodon
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
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
The dose in the pill is very high, it's not something you can just let everyone take.
incomingpain
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.
sys32768
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
You can have anecdotal about anything in any direction. Not a great way to come to a scientific conclusion.
reissbaker
Can you also have clinical trials performed under the Biden administration that "significantly improve communication with medium-to-large effect sizes and have a positive effect on core ASD symptoms and associated behaviors" about anything in any direction?
yibg
I'm not sure how that's related. Some research is flawed and statistics provide misleading or incorrect data so we just... don't care about data?
lupusreal
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".
incomingpain
>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.
newobj
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
Perhaps because B12 supplementation isn't a cure for autism?
tonmoy
> 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
tencentshill
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.
rdtsc
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?
formerly_proven
I bet it’s a really good sign that the history section of Novo Nordisk on Wikipedia skips directly from the 1920s to the 1980s.
rdtsc
Yikes, indeed
reissbaker
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.
vkou
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?
ryandrake
If the electorate wants a circus, they'll elect clowns. There's really not much non-clowns can do about it if elections are to be maintained and fair.
mpalmer
Until elections become something else entirely, and then the non-clowns can do even less.
RobRivera
The first line of defense is the power of NO as a patient.
Easier said than done and requires staying on top of literature and sourcing multiple opinions.
Maybe trips abroad to get that alternative opinion for peace of mind?
All spoken from an understanding that the average person cannot afford the time or expense associated with this idea.
throwup238
A never ending vigilance against corruption. That’s the only real solution — any other system or set of rules can be undone.
atmavatar
Practically speaking, no amount of punishment for fascists will ensure that another set of fascists won't rise up again in the future. There's far too much to gain by taking over political power for any deterrent to work effectively.
The best solution is to improve education to help people see through the bullshit that got us here. In particular, teach people that campaigning through memes, emotional appeals, and demonization of minority groups are big neon signs that the campaigning group does not have your best interests in mind.
xnx
Educating people to resist fascism is at odds with educating them to be obedient/subservient worker bees.
cbeach
Which "fascists" are we talking about, here?
Be careful around powerful words with meaning (see https://en.wikipedia.org/wiki/Fascism ). If we casually use these words to refer to people we simply disagree with - one day, when actual fascists start rising, we'll have taken all the meaning out of the language we rely on to identify them.
billy99k
You mean the 'adults in the room' that pushed an unproven Covid vaccine from those same corrupt companies?
The Democrats wouldn't take the Covid vaccine when it first came out and called it the 'Trump vaccine' on CNN...until they came into power. Then they made a 180.
During the Covid era, you most likely lost your job and were censored from all major platforms if you had an opinion about the Covid vaccine that didn't match exactly what the government told us.
What would you think about getting this same treatment for this?
esseph
The biggest block of voters in this country are independents, not Democrats or Republicans.
vkou
> unproven Covid vaccine
Please inform yourself. Both approved vaccines were rigorously tested. Unlike other vaccines, multiple phases of those tests were done concurrently, which is why it shipped in 6 months, instead of 3 * 6 months.
> The Democrats wouldn't take the Covid vaccine when it first came out
This begins to cast doubt that you will inform yourself, but from day 1, vaccine uptake was much higher, and continues to be much higher in blue states.
The only person who flip-flopped on vaccines was Trump, because he realized that his base is far too stupid to appreciate what Operation Warp Speed did for them.
danorama
“The Democrats wouldn't take the Covid vaccine when it first came out and called it the 'Trump vaccine' on CNN...until they came into power. Then they made a 180.”
[citation needed]
MSFT_Edging
1: Police unions: Police in this country hold a monopoly on violence granted by the state. Police unions in turn have created a political bubble around the holders of this monopoly. By politicizing what should essentially be a public servant, we have made any accountability for these public servants very difficult to perform.
2: Breech of public trust can never be punished with a slap on the wrist. There cannot be "get out of jail free" sentencing for people of note. That deconstructs any trust anyone can have in the system, if there exists a class who cannot have the law applied to them.
3: A bill of Human rights, designed to encompass attempts to remove rights from certain people. A focus on positive rights, ie "the right to live", rather than negative rights "the right to cause damage to others via an existing right(property rights)".
4: Overturning of citizens united or the ability to punish corporations in an equivalent measure to how an individual can be punished. The fines must always be greater than what can be saved by exploitation via crime.
That's more or less a start. I'm no political scientist but to me these are big points of gridlock.
lupusreal
[flagged]
nineplay
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.
morkalork
Apparently Dr Oz stands to profit from one of those approvals..
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.