FDA Reverses Course on Leucovorin as Autism Treatment

On March 10, 2026, the U.S. Food and Drug Administration (FDA) approved leucovorin calcium (Wellcovorin) for the treatment of cerebral folate deficiency (CFD) caused by a variant in the folate receptor 1 gene (CFD-FOLR1), a rare neurological condition affecting fewer than 1 in a million people. This decision marks a significant departure from earlier claims made by the Trump administration, which had promoted leucovorin as a potential treatment for autism, suggesting it could benefit "hundreds of thousands" of children.

FDA Rescinds Broad Autism Claims, Approves Leucovorin Only for Rare Genetic DisorderFDA authorizes new use for leucovorin as first treatment for rare neurological condition CFDUSA TodayDisability Scoop

In September 2025, FDA Commissioner Marty Makary and Health and Human Services Secretary Robert F. Kennedy Jr. announced that leucovorin would be reviewed for its potential to treat autism symptoms. This announcement led to a 71% increase in prescriptions for the drug among children aged 5 to 17, as families sought what they believed could be a breakthrough treatment. However, subsequent reviews revealed insufficient evidence to support the drug's efficacy for autism, leading to the FDA's retraction of broader claims.

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FDA officials clarified that their focus shifted to the strongest scientific evidence, which only supported leucovorin's use for CFD-FOLR1. "Right now, we don’t have sufficient data to say that we could establish efficacy for autism more broadly," stated a senior FDA official. Autism experts welcomed the FDA's decision, emphasizing the importance of evidence-based treatment. Andy Shih, Chief Science Officer at Autism Speaks, noted, "Today’s decision by the FDA reflects what the science has indicated so far: that evidence for leucovorin as an effective treatment for autism remains limited."

MedscapeDisability ScoopFDA Rescinds Broad Autism Claims, Approves Leucovorin Only for Rare Genetic DisorderUSA TodayCNN

The reversal has drawn criticism from families who felt misled by the initial claims. Keith Joyce, guardian of a child with autism who was taking leucovorin, expressed feelings of betrayal, stating, "I kind of felt vindicated with what I was doing. But now they’re saying, ‘Oh, nope, we’re out, and we were wrong.’" Experts have also cautioned against the premature promotion of the drug, with David Mandell from the University of Pennsylvania warning that families are now experiencing "whiplash" regarding treatment options.

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While the FDA's approval is limited to a rare genetic condition, the surge in leucovorin prescriptions reflects a broader public interest fueled by the Trump administration's earlier claims. The American Academy of Pediatrics has previously stated that evidence supporting leucovorin's use in autistic children is "currently limited," a sentiment echoed by various medical professionals. Additionally, a study that initially supported leucovorin's efficacy for autism was retracted due to errors, further complicating the narrative surrounding the drug.

CNNUSA TodayDisability ScoopFDA Rescinds Broad Autism Claims, Approves Leucovorin Only for Rare Genetic Disorder

The FDA continues to encourage further research into leucovorin's potential applications, particularly regarding autism. Despite the lack of formal approval for this broader use, doctors may still prescribe the drug off-label. However, experts warn that parents should proceed with caution, as the drug's side effects can include irritability and aggression, and its long-term effects remain unknown.

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• “Today’s approval represents a significant milestone for patients living with cerebral folate transport deficiency due to the FOLR1 variant, a rare genetic condition that has had no FDA-approved treatment options until today,” — Marty Makary, FDA Commissioner

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• “There is no evidence to say that leucovorin will help most people with autism, and there’s certainly no evidence to say it’s safe,” — Dr. Alycia Halladay, Autism Science Foundation

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• “Families are always searching for ways to support their child, and early reports about the promise of leucovorin brought hope to many.” — Andy Shih, Autism Speaks Chief Science Officer

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• “I kind of felt vindicated with what I was doing. But now they’re saying, ‘Oh, nope, we’re out, and we were wrong.’” — Keith Joyce, Guardian of a child with autism

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The FDA's decision underscores the critical need for rigorous scientific validation before promoting treatments for complex conditions like autism, highlighting the challenges families face in navigating the healthcare landscape.

Scientific American