WASHINGTON, D.C. — The U.S. Department of Health and Human Services, joined by the FDA and NIH, announced a series of new actions aimed at addressing the rising rates of autism spectrum disorder (ASD) in the United States, which now affects an estimated 1 in 31 children.
The U.S. Department of Health and Human Services (HHS) and the Food and Drug Administration (FDA) announced that leucovorin, a treatment for cerebral folate deficiency, will be authorized as the first FDA-recognized therapeutic option for children with ASD who show speech and language improvements while taking the medication. State Medicaid programs will be allowed to cover leucovorin for autism-related treatment, and the National Institutes of Health (NIH) will conduct confirmatory trials and expanded safety studies.
Officials emphasized that leucovorin is not a cure for autism but may help reduce speech-related deficits in some children when combined with behavioral therapies.
In addition, HHS directed the FDA to begin updating safety labeling for acetaminophen (Tylenol and similar products), citing studies that have raised questions about possible links between prenatal use and neurodevelopmental outcomes. The FDA noted that research findings remain conflicting and underscored that acetaminophen remains an important treatment option for fevers during pregnancy, when alternatives may pose greater risks. Clinicians are being advised to prescribe the lowest effective dose for the shortest necessary duration.
A third measure announced was the launch of the Autism Data Science Initiative (ADSI), a $50 million NIH program supporting 13 projects nationwide. The initiative will integrate biological, clinical, and behavioral data with environmental and genetic factors, using advanced tools such as machine learning and organoid models. Projects will include community engagement to ensure alignment with the needs of families and individuals living with autism.
Health officials stated that these combined steps mark a significant shift in how federal agencies approach autism—through targeted treatments, updated safety guidance, and expanded scientific research.
