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Wednesday, October 22, 2025

“FDA Considers Leucovorin for Autism Treatment”

The U.S. government has supported an unfamiliar drug as a potential remedy for autism symptoms, which medical experts claim lacks strong evidence. During a White House briefing, Dr. Marty Makary, the U.S. Food and Drug Administration (FDA) commissioner, stated that leucovorin could benefit “hundreds of thousands” of children with autism.

According to the FDA, leucovorin, a form of the vitamin folate, will be categorized as a treatment for patients with cerebral folate deficiency (CFD), a neurological disorder. This classification marks the initial step towards potentially approving leucovorin as a treatment option for certain individuals with autism in the United States.

Folate, a B vitamin present in foods like leafy greens, legumes, and citrus fruits, is crucial for various bodily functions. The synthetic form of folate, known as folic acid, is commonly found in vitamin supplements and fortified grains. Folic acid is especially important for the normal development of a fetus’s spine, brain, and skull.

Leucovorin, similar to folic acid, is recognized in Canada for reducing the adverse effects of methotrexate, a cancer medication. The FDA has approved leucovorin tablets for individuals with cerebral folate deficiency, based on a limited number of studies indicating improved verbal communication. Although some U.S. doctors have been using the drug off-label, it is now officially recognized for this specific condition.

Leucovorin is believed to aid children with autism who have a receptor preventing folate from entering the brain. However, experts emphasize that leucovorin is not a definitive cure and requires further research.

Autism, a neurodevelopmental disorder primarily influenced by genetics, is also associated with environmental factors such as advanced parental age and exposure to air pollutants. While leucovorin shows promise, more extensive studies are needed to validate its efficacy as an autism treatment. Scientists and medical professionals stress the importance of thorough research and controlled clinical trials before endorsing leucovorin as a standard therapy for autism.

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