Leucovorin prescriptions for children rise 46% after federal officials' autism claims
Leucovorin prescriptions for children spiked after federal officials claimed the drug was an effective treatment for autism during a September 2025 press conference, according to a new Michigan Medicine-led study published in The New England Journal of Medicine.
A new study led by Michigan Medicine reveals a 46% surge in prescriptions for Leucovorin, a drug commonly used to counteract side effects of methotrexate, among U.S. children. This increase occurred after federal officials highlighted its potential as an autism treatment during a September 2025 press conference, despite limited evidence supporting its efficacy.
The U.S. Department of Health and Human Services proposed updating leucovorin's label to address cerebral folate deficiency, a rare genetic condition sometimes linked to autism. Following the press conference, the number of children aged 0 to 17 with filled leucovorin prescriptions rose from 4.6 per 100,000 to 6.7 per 100,000 weekly.
Lead researcher Dr. Kao Ping Chua warns that the effectiveness and long-term safety of leucovorin for children with autism remain uncertain, expressing concern about this rise in off-label prescribing.
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