FDA to Approve New Use of Cancer Drug for Autism Symptoms in Kids

Key Takeaways

The U.S. Food and Drug Administration will soon approve leucovorin for children with cerebral folate deficiency and autism symptoms

Research suggests the drug may improve verbal skills, but it is not a cure for autism

Medicaid and CHIP will be required to cover the treatment once approved

TUESDAY, Sept. 23, 2025 (HealthDay News) — A decades-old cancer drug may soon be approved to help kids with certain autism-related symptoms linked to folate deficiency.

The U.S. Food and Drug Administration (FDA) is preparing to clear leucovorin — a generic drug also known as folinic acid — for use in kids with cerebral folate deficiency and symptoms of autism.

The move, expected in the coming weeks, would mark a new use for the drug, which is already prescribed for patients with can