Explainer: What is the rationale behind considering leucovorin as a treatment for autism?
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Sep 22 2025
0mins
Source: Reuters
FDA Approval: The U.S. FDA approved a version of leucovorin by GlaxoSmithKline, which had previously been withdrawn from consideration, following a review related to its use in patients with cerebral folate deficiency, a condition linked to neurological symptoms and autism.
Leucovorin and Autism: While leucovorin is used off-label for autism treatment, studies suggest a potential link between low maternal folate levels and autism risk; however, the evidence supporting leucovorin's effectiveness in treating autism is considered weak and requires further research.
About the author

Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.








