Can the prescription drug leucovorin treat autism? History says, probably not

Published: Thursday, January 22, 2026 at 6:04 pm

Leucovorin for Autism: A Look at the Science and Controversy

The potential use of the prescription drug leucovorin to treat autism has sparked debate and raised questions about its efficacy. In late 2025, the Food and Drug Administration (FDA) commissioner suggested a label change to make leucovorin available to children with autism spectrum disorder, anticipating significant benefits for a large number of children. However, the FDA has yet to implement this change.

Following the initial announcement, a Facebook group dedicated to leucovorin for autism gained over 25,000 members, primarily parents seeking the drug for their children. Concurrently, some physicians began prescribing leucovorin off-label, despite warnings from medical organizations. This surge in demand has led to a drug shortage, prompting the FDA to temporarily allow imports of leucovorin tablets not approved for use in the United States.

The rationale behind leucovorin's potential use stems from its connection to folate, a critical nutrient for brain development, and cerebral folate deficiency. This deficiency can lead to various neurological issues, including autism. Studies have suggested a link between cerebral folate deficiency and autism, with some research indicating that leucovorin could potentially correct the deficiency and improve symptoms.

However, the scientific evidence supporting leucovorin's effectiveness is limited and controversial. While some studies have shown improvements in certain autistic children, the results are preliminary and not conclusive. Critics point to the lack of rigorous clinical trials and the potential for misleading interpretations of the data. Some experts caution that the drug is not a cure-all and that its benefits may be limited to a specific subset of autistic individuals.

The history of leucovorin's use in treating Fragile X syndrome, a condition often associated with autism, provides a cautionary tale. Initial enthusiasm for the drug waned after clinical trials revealed it was no more effective than a placebo.

BNN's Perspective:

While the potential of leucovorin to help some individuals with autism is intriguing, it's crucial to approach this with caution. The current evidence is not strong enough to warrant widespread use, and more rigorous research is needed. Parents should consult with their physicians and carefully weigh the potential benefits and risks before considering leucovorin for their children.

Keywords: leucovorin, autism, cerebral folate deficiency, FDA, folate, autism treatment, off-label prescriptions, clinical trials, Fragile X syndrome, autism spectrum disorder

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