FDA Plans Leucovorin as Autism Prescription Treatment for Children

The FDA has approved leucovorin, a form of folinic acid, as a prescription treatment for children with cerebral folate deficiency who exhibit autism-like symptoms, marking a significant development in personalized autism care. This approval, announced by FDA Commissioner Marty Makary alongside NIH Director Jay Bhattacharya and CMS Administrator Mehmet Oz during a White House event with President Trump, underscores the drug's potential to improve verbal communication in affected children, although it is not a cure for autism. The Trump administration is expanding access to leucovorin, requiring state Medicaid and CHIP programs to cover the medication, which may benefit hundreds of thousands of children, particularly those with chronic folate deficiency. Additionally, the administration issued a precautionary warning for pregnant women to limit acetaminophen use unless medically necessary, citing emerging research that links prenatal exposure to increased autism risk. The NIH is investing over $50 million in new research projects through the Autism Data Science Initiative to further understand autism spectrum disorder, aiming to accelerate breakthroughs in causes, diagnostics, and outcomes. While experts advise caution and emphasize the need for proper diagnostic testing before treatment, these steps collectively represent a major federal effort to address autism through both treatment and prevention strategies.