Following a US federal press conference hinting at leucovorin’s potential in autism treatment, prescriptions for the drug surged by 46%, despite limited evidence and warnings from health authorities.
A sharp rise in leucovorin use among children followed a federal press conference in September 2025 that cast the decades-old drug as a possible autism treatment, according to research led by Michigan Medicine and published in The New England Journal of Medicine. The study found that the number of children ages 0 to 17 with a filled prescription climbed 46% in the week of the announcement, even though the evidence for autism use remained limited.
The federal event, organised by the U.S. Department of Health and Human Services, was meant to highlight a label update for cerebral folate deficiency, a rare genetic disorder that can include autistic traits. But officials also publicly suggested leucovorin could help autism, a claim that has not been backed by large randomised trials. The American Academy of Pediatrics has since said the evidence is too thin to recommend the drug for routine use in autistic children, and its interim guidance advises against broad use.
The Michigan Medicine team analysed a national pharmacy database that captures nearly all U.S. retail prescriptions. In 2025, it recorded 150,026 leucovorin prescriptions for 47,394 children, with weekly dispensing rising from 734 to 5,858 by the end of the year. The biggest increase came after the press event, and the sharpest gains were seen in children aged 6 to 11 and in the South. Adult use did not show the same jump.
Researchers said prescribing was already trending higher before the conference, possibly because of news coverage featuring family accounts. Still, they warned that the surge could create false hope, crowd out proven therapies and expose children to a treatment whose long-term safety remains unclear. Separate reporting by FactCheck said the FDA’s approval was limited to the rare folate disorder, not autism, underscoring the gap between the regulatory action and the public messaging surrounding it.
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