AAP Declines to Recommend Drug for Autism Supported by Trump Administration

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The American Academy of Pediatrics (AAP) is advising against the routine use of leucovorin for children with autism, despite rising demand from families following recent federal promotion of the drug. Although Trump administration officials highlighted leucovorin as a potential treatment in September and the FDA moved toward approving it for cerebral folate deficiency – a condition associated with autism features – the AAP says evidence of its benefit for autism is limited. Interest among families has surged, but the organization notes that it remains unclear which children might benefit, what appropriate dosing would be, and whether the drug is safe long-term. The AAP urges pediatricians to engage in shared decision-making, emphasize the current lack of strong evidence, and closely monitor any cases in which the drug is prescribed, while calling for larger, well-controlled clinical trials to better understand leucovorin’s effectiveness and safety.