Children with autism may see speech improvement with off-label prescription drug
An off-label prescription drug, leucovorin, has shown promising results in helping some non-verbal children with autism discover the ability to speak. This prescription vitamin, similar to folic acid, works to restore folate levels in patients who may struggle with absorption or utilization. While leucovorin is FDA-approved for reducing toxic effects of methotrexate in cancer treatment and treating certain types of anemia caused by folate deficiency, it is not typically prescribed for autism.
However, families have reported significant improvements in communication skills among autistic children who have used leucovorin off-label. Dr. Richard E. Frye, a behavioral child neurologist, has conducted research on experimental treatments for autism, including leucovorin. He notes that the main benefit of leucovorin for children with autism is language improvement, along with enhancements in social function, reduced repetitive behaviors, and improved attention skills.
Dr. Marc Siegel, a clinical professor of medicine, explains that leucovorin helps address central folate deficiency in the brain, which can impact neurological function in children with autism spectrum disorder. While small studies have shown positive outcomes in terms of neurological function and communication skills, larger clinical trials are needed to further validate these findings.
It is important for families to work closely with medical professionals when considering leucovorin for autism treatment, as the drug is typically given at higher doses than regular vitamins. Some children may require a compounded form of leucovorin due to sensitivities to additives in commercial products. While leucovorin is currently available in pharmacies, FDA approval for autism treatment would provide stronger medical backing, potentially leading to insurance coverage, standardized dosing guidelines, and broader acceptance in the medical community.
While leucovorin may not be a standalone solution for autism, it has the potential to enhance outcomes when used in conjunction with behavioral and speech therapies. Dr. Andy Shih, the chief science officer of Autism Speaks, emphasizes the importance of larger controlled clinical trials to determine the efficacy and safety of leucovorin for autism treatment. If future studies confirm its effectiveness, leucovorin could become a more accessible option for children with autism who may benefit from it.
