What Science Says About Leucovorin, the Drug Trump Promotes for Autism

Leucovorin, a decades-old chemotherapy support drug, is drawing new attention after President Donald Trump touted it as a potential therapy for children with autism. Families and doctors say the drug, a high-dose form of vitamin B9, has helped improve communication skills in some children, but experts caution the science remains limited.

Four-year-old Jose Morales-Ortiz of Minnesota is one example. His guardian, Keith Joyce, says that before starting leucovorin, Jose struggled with basic speech. Within months of treatment, he began forming conversations and interacting more with others. Joyce credits the change to leucovorin, though he stresses the need for more scientific evidence.

Researchers believe the drug helps children with cerebral folate deficiency, a condition in which antibodies block folate from entering the brain. Small studies have suggested that leucovorin may improve communication and language skills in some children with autism. Pediatric neurologist Dr. Richard Frye has reported promising results in clinical trials, but he emphasizes that autism is complex and treatments must be individualized.

The FDA recently announced plans to update leucovorin’s label so it can be prescribed for children with low folate levels in the brain, making it more widely covered by Medicaid. Still, the Autism Science Foundation and other experts warn that large, placebo-controlled trials are needed to confirm its effectiveness. Some scientists also worry the approval sets a dangerous precedent by lowering the bar for drug approvals.

Leucovorin is generally considered safe, with side effects including nausea, diarrhea, and appetite loss. More serious risks include possible interactions with seizure medications. Parents are also being warned not to substitute over-the-counter folinic acid supplements, which contain far lower doses and untested additives.

Despite the uncertainty, interest in the drug has surged, with online support groups growing rapidly. For families like Jose’s, the improvements offer hope. But experts stress that while leucovorin may help some children, it is not a cure for autism — and much more research is still needed.

Credit: CNN Newsource

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