A prescription drug typically used for seizures and behavioral issues is showing potential in helping some non-verbal autistic children develop language skills. Leucovorin, a form of vitamin B9 similar to folic acid, helps restore folate levels in individuals who struggle to absorb or utilize it effectively, according to pharmacist Katy Dubinsky, CEO of Vitalize. While not currently FDA-approved for autism treatment, some families report significant communication improvements in their children after using leucovorin off-label.
Dr. Richard E. Frye, a behavioral child neurologist, has researched leucovorin as an experimental treatment for autism. He emphasizes the vitamin's long-term safety record and highlights its potential benefits beyond language development, such as improved social interaction, reduced repetitive behaviors, and increased attention span. "Leucovorin is a special type of vitamin B9 that is water-soluble and doesn't accumulate in the body," Frye explained. He noted that folates have a history of safe long-term use, which is why they're commonly added to food.
Dr. Marc Siegel, a clinical professor of medicine, suggests leucovorin's positive impact might be linked to addressing central folate deficiency in the brain, a condition potentially affecting a significant portion of children with autism. He explains that leucovorin converts to folate through a unique pathway in the brain and preliminary studies indicate improvements in neurological function and communication. However, he stresses the need for more extensive research.
While leucovorin's long history of use provides some reassurance regarding its safety profile, experts urge caution. Dr. Frye advises families to work closely with medical professionals due to the higher doses used compared to standard vitamins and the potential need for compounded forms for children sensitive to certain additives. He also emphasizes the importance of testing for folate pathway abnormalities to identify suitable candidates.
Experts acknowledge that leucovorin is not a standalone cure for autism and likely works best in conjunction with other therapies. Dr. Frye believes it can enhance the effectiveness of behavioral and speech therapies. Dr. Andy Shih of Autism Speaks emphasizes the individual nature of autism and cautions against generalizing findings from small studies. He underscores the need for larger clinical trials to validate leucovorin's efficacy and safety. While Dubinsky acknowledges the potential for leucovorin to become a more widely accessible treatment option, she, like other experts, stresses the importance of further research and consultation with healthcare providers.
