10-01-2025, 12:12 PM
(Ten post był ostatnio modyfikowany: 10-01-2025, 12:16 PM przez subzero.)
"What does the science say about leucovorin and autism?
This treatment is not supported by evidence from large randomized controlled trials establishing effectiveness in treating autism. I could stop right there, because generally, drugs approved by the FDA, with a few exceptions, require large, randomized, double-blind clinical trials to show safety and effectiveness.
And there are no large, randomized, double-blind clinical trials supporting the use of leucovorin in treating autism. The FDA, Trump, and Kennedy are claiming that the drug is a “cure” for autism in the absence of any persuasive and published evidence.
Public health expert Jessica Steier, DrPH, wrote on Substack that there were only five studies, none with more than 80 participants. Dr Steier stated that the effects were “generally modest.”
The five studies are “suggestive,” though hardly gold standard science. A proper clinical trial, which can be done with this drug, would involve thousands of children receiving leucovorin or a placebo.
Ironically, the data for treating CFD itself is weaker.
Dr Steier makes a more important observation:
The proposal of leucovorin as a treatment would only theoretically work for a small subset of autistic people with an antibody against the folate receptor (but these antibodies are not much more common in autistic people than in close relatives who do not have autism, so their meaning is not yet clear).
Summary
I think the observations that leucovorin can treat autism spectrum disorder (let alone “cure”) are intriguing at best. But most drugs that are “intriguing” in Phase 1 clinical trials fail to show any meaningful clinical effect in subsequent larger studies.
I believe that this FDA “approval,” based on very weak science, does a disservice to parents with autistic children. They believe that Trump and Kennedy are claiming science supports the claim that leucovorin can “cure” autism, when the evidence does not support it at all."
https://www.skepticalraptor.com/skeptica...treatment/
This treatment is not supported by evidence from large randomized controlled trials establishing effectiveness in treating autism. I could stop right there, because generally, drugs approved by the FDA, with a few exceptions, require large, randomized, double-blind clinical trials to show safety and effectiveness.
And there are no large, randomized, double-blind clinical trials supporting the use of leucovorin in treating autism. The FDA, Trump, and Kennedy are claiming that the drug is a “cure” for autism in the absence of any persuasive and published evidence.
Public health expert Jessica Steier, DrPH, wrote on Substack that there were only five studies, none with more than 80 participants. Dr Steier stated that the effects were “generally modest.”
The five studies are “suggestive,” though hardly gold standard science. A proper clinical trial, which can be done with this drug, would involve thousands of children receiving leucovorin or a placebo.
Ironically, the data for treating CFD itself is weaker.
Dr Steier makes a more important observation:
The proposal of leucovorin as a treatment would only theoretically work for a small subset of autistic people with an antibody against the folate receptor (but these antibodies are not much more common in autistic people than in close relatives who do not have autism, so their meaning is not yet clear).
Summary
I think the observations that leucovorin can treat autism spectrum disorder (let alone “cure”) are intriguing at best. But most drugs that are “intriguing” in Phase 1 clinical trials fail to show any meaningful clinical effect in subsequent larger studies.
I believe that this FDA “approval,” based on very weak science, does a disservice to parents with autistic children. They believe that Trump and Kennedy are claiming science supports the claim that leucovorin can “cure” autism, when the evidence does not support it at all."
https://www.skepticalraptor.com/skeptica...treatment/