> "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.
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.
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.
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.
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.
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.
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?
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.
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.
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.
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.
> 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
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:
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?
Readit News
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.
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.
The evidence is from a study with N=40. Not 40,000. 40.
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.
* 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.
Do we understand the cause of autism or how this supposed cure works?
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.
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.
I would have liked to see some citations. I’m mostly curious about the sample sizes
Kinda funny how in all this chatter no one is talking about MTHFR etc.
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?