Five months after the Trump administration announced with fanfare that it had approved an unproven treatment for certain forms of autism, the U.S. Food and Drug Administration (FDA) has issued a sharp reversal. The agency confirmed today that leucovorin — a form of folinic acid — remains unapproved for treating autism spectrum disorder, a neurodevelopmental condition affecting communication and social interaction.
What the FDA Actually Approved
The FDA has authorized leucovorin for two specific, narrow uses: managing certain side effects of chemotherapy and treating cerebral folate deficiency — a rare, genetic disorder. It has not approved the drug for autism, contradicting earlier claims made by the administration.
Trump’s September 2025 Announcement
Then-President Donald Trump announced the so-called ‘new therapy’ during a White House press briefing in September 2025, calling it a source of hope for ‘many parents of autistic children’ and suggesting it could improve their quality of life. At the time, the announcement drew swift criticism from the U.S. medical and scientific community, which had already condemned the administration’s broader skepticism toward vaccines and acetaminophen. Experts called the autism-related approval claim ‘premature’ and unsupported by rigorous evidence.
Limited Evidence, Significant Concerns
While some small-scale studies — involving very few participants — suggested folinic acid might ease certain autism-related communication or interaction challenges, researchers stress that far more robust clinical trials are needed to confirm any benefit. Dozens of autism specialists issued a joint letter warning that approving leucovorin for autism at this stage could offer ‘false hope’ to families.
FDA Cites Insufficient Data
An FDA official told NBC News the agency stepped back due to ‘insufficient data’ — underscoring its commitment to evidence-based decision-making. That said, clinicians may still prescribe leucovorin off-label for autistic individuals diagnosed with cerebral folate deficiency or based on individual medical judgment.