White House endorsement spurs rise in unproven autism drug prescriptions for children

A new study published in the New England Journal of Medicine shows that a September 22, 2025 White House briefing in which President Donald Trump promoted leucovorin as a potential autism treatment coincided with a noticeable uptick in pediatric prescriptions. The weekly dispensing rate for children aged 0-17 grew from roughly 5 per 100,000 before the announcement to about 7 during the briefing week and climbed to 8 by the end of 2025, while adult rates remained flat.

The researchers, led by pediatrician Kao-Ping Chua of the University of Michigan Medical School, analyzed a pharmacy-claims database covering over 90 % of U.S. retail prescriptions, encompassing more than 150,000 fills for over 47,000 children. Although the FDA has approved leucovorin for certain genetic disorders and to mitigate chemotherapy side effects, the American Academy of Pediatrics advises against its use for autism because dosing, efficacy, and safety are unproven.

FDA officials later clarified that the agency lacks sufficient evidence to endorse leucovorin for autism, despite a separate approval for cerebral folate deficiency in March 2026. The findings raise concerns that political endorsements can influence clinical practice and expose children to untested therapies.