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6h ago

Study Ties 2,000% Surge in Leucovorin Prescriptions for Autistic Children to Publicity

The rush to prescribe an unproven therapy reveals a gap between public claims and solid clinical data.

National health record data evaluated in the study demonstrates that public media features and endorsements from federal officials can instantly trigger a 2,000% surge in off-label prescription behaviors before broad clinical validation occurs. Credit: Neuroscience News
© Saul Loeb/AFP via Getty
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Overview

  • The UC San Diego–led analysis, published Monday in JAMA Network Open, found prescriptions for autistic children rose from about 34 to 835 per 100,000 visits by November 2025.
  • The study links the rise to a widely viewed media report in January 2025 and a September White House briefing featuring praise from President Trump and Robert F. Kennedy Jr., after which rates jumped again.
  • The FDA in March approved leucovorin for cerebral folate deficiency but declined to endorse it for autism due to insufficient evidence.
  • The evidence base remains thin after the largest positive autism trial was retracted in January for data errors and other concerns, with only small studies hinting at possible speech gains for some children.
  • Using the EPIC Cosmos database that spans more than 800,000 autistic children across the U.S., researchers call for large randomized trials and for tracking real‑world outcomes to assess benefits and long‑term safety.