White House Backing Linked to Rise in Off-Label Autism Drug Use Despite Lack of New Evidence
A drug that lacks robust evidence for treating autism spectrum disorder saw a notable increase in pediatric prescriptions after receiving attention from the Trump administration in fall 2025. Leucovorin, which is typically used to manage folate deficiency, has been discussed as an off-label treatment for autism, though clinical research has not established its efficacy for this purpose.
The administration reportedly promoted leucovorin during a White House briefing without presenting new scientific evidence supporting its use for autism. Following this high-profile endorsement, dispensing of the drug to children rose, according to data referenced by Science News.
Medical experts have long emphasized the importance of evidence-based treatments for autism, a complex neurodevelopmental condition affecting communication and behavior. While some researchers continue to study various pharmacological approaches, the scientific consensus holds that existing evidence does not support leucovorin as a standard autism therapy.
The episode underscores ongoing debates about the role of political endorsements in shaping medical prescribing patterns and the challenges in communicating scientific uncertainty to the public.