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

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 cancer and some forms of anemia.

Research suggests leucovorin may improve verbal communication in some children, although health leaders say that it is not a cure for autism.

“In the coming weeks, the FDA will approve prescription leucovorin as a treatment for children with cerebral folate deficiency and autistic symptoms,” FDA Commissioner Dr. Marty Makary, National Institutes of Health Director Dr. Jay Bhattacharya and Centers for Medicare & Medicaid Services head Dr. Mehmet Oz wrote in an opinion piece.

Once the FDA label change is made, Medicaid and the Children’s Health Insurance Program (CHIP) will be required to cover the drug nationwide, they added.

More than half of all U.S. children are insured through those programs.

Some scientists say leucovorin shows promise for children who are folate-deficient, but they warn that the data is limited.

“It is important to note that leucovorin is not a cure for autism but has demonstrated an improvement in speech-related deficits for autism,” Makary, Bhattacharya and Oz added in their commentary.

In the same piece, they also advised pregnant women to use Tylenol (acetaminophen) carefully and in consultation with their doctors. Some studies suggest a possible link between acetaminophen use during pregnancy and conditions like autism and ADHD in children.

“These studies are looking for associations, they don’t show causation,” Dr. Peter Bernstein, an obstetrician-gynecologist at Mount Sinai Hospital in New York City told Politico. “Not treating a fever may increase the risk of the pregnancy, certainly more than the Tylenol use.”

On Monday, health regulators in the U.K. also reaffirmed acetaminophen’s safety, saying there is no evidence it causes autism. Overseas, acetaminophen is called paracetamol.

“Patient safety is our top priority. There is no evidence that taking paracetamol during pregnancy causes autism in children,” said Alison Cave, chief safety officer of the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA). “Paracetamol remains the recommended pain relief option for pregnant women when used as directed.”

More information

The Mayo Clinic has more on leucovorin.

SOURCES: Politico, Sept. 22, 2025; Politico Magazine, opinion piece, Sept. 22, 2025