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Autism Leucovorin Prescriptions Jump 2,000% After Media and White House Promotion

Researchers say off-label use now outpaces evidence.

Overview

  • The surge was documented Monday in JAMA Network Open by a UCSD team using the Epic Cosmos database of nationwide electronic health records.
  • Prescribing climbed from a baseline near 34 per 100,000 encounters to more than 835 per 100,000 by November 2025 and then plateaued at an elevated level in December and January.
  • Rates began rising after a widely viewed February 2025 TV story about a child who improved on the drug and jumped again following a September 22, 2025 White House promotion featuring President Trump and Robert F. Kennedy Jr.
  • Evidence for autism benefits remains limited, and the largest positive trial was retracted in January 2026 over data errors and concerns.
  • The FDA in March 2026 approved leucovorin for an ultra-rare cerebral folate transport deficiency but not for autism, and experts urge outcome tracking and large randomized trials before broader use.