On September 22, 2025, President Donald J. Trump and U.S. Health and Human Services (HHS) Secretary Robert F. Kennedy announced new actions to address the autism epidemic. The Food and Drug Administration (FDA) is updating the label for leucovorin, a medication for cerebral folate deficiency linked to autism, to authorize its use for children with autism spectrum disorder (ASD) who show language, social, or adaptive gains. This makes leucovorin the first FDA-recognized therapeutic for children with cerebral folate deficiency and autistic symptoms, potentially allowing state Medicaid programs to cover it for ASD. However, it is noted that leucovorin is not a cure and may only improve speech deficits in a subset of children, requiring close medical supervision and behavioral therapy.
Secondly, HHS will address acetaminophen (Tylenol and similar products) by initiating a safety label change and launching a nationwide public service campaign. While promising, the FDA encourages clinicians to use acetaminophen at the lowest effective dose for the shortest duration during pregnancy due to conflicting literature on its link to autism, while acknowledging it's often the only option for fevers and pain given the adverse effects of alternatives like NSAIDs. The FDA is also partnering with manufacturers to update labeling and conduct new research.
Finally, the National Institutes of Health (NIH) announced over $50 million in funding for 13 projects as part of the Autism Data Science Initiative (ADSI). These projects will integrate large-scale biological, clinical, and behavioral data with an exposomics approach, examining environmental, nutritional, medical, and social factors alongside genetics. The research will employ advanced methods like machine learning and organoid models, cover all age groups, and include community engagement to align with the needs of autistic individuals, families, and clinicians. Confirmatory trials and new research into leucovorin's impact, including safety studies, will also be launched by NIH.
