Health officials have highlighted the potential of the cancer medication leucovorin to offer significant benefits for children with autism spectrum disorder (ASD). During a press conference, federal health officials discussed research suggesting that leucovorin, a prescription vitamin used to treat cancer and anemia, could be effective in off-label use for children with autism. The FDA plans to approve leucovorin as a potential treatment for speech-related deficits associated with ASD, with the agency updating the drug’s label to indicate its use for cerebral folate deficiency, linked to autism. This change could lead to Medicaid coverage for the drug in states, providing more accessibility for families.
Studies have shown that leucovorin could help some non-verbal children gain the ability to speak. Leucovorin functions similarly to folic acid, helping to restore folate levels in patients who may have difficulty absorbing or using it. Experts like Katy Dubinsky, a pharmacist and CEO of Vitalize, have noted the drug’s potential for improving communication skills in children with autism. The NIH is also launching new research initiatives to study the drug’s safety and effectiveness. While leucovorin is FDA-approved for reducing the toxic effects of methotrexate in cancer treatment and for treating anemia, it is not yet prescribed for autism. However, some families have reported significant improvements in their children’s communication skills when using it off-label.
Richard E. Frye, a behavioral child neurologist in Arizona, has studied leucovorin in his research on experimental treatments for autism. He explained that leucovorin is a form of vitamin B9 and is water-soluble. Frye emphasized that while leucovorin can improve language and other skills in children with autism, it should be used in combination with behavioral and speech therapy. Dr. Marc Siegel, a clinical professor at NYU Langone Health and Fox News’ senior medical analyst, also spoke about the drug’s potential, noting that central folate deficiency may interfere with neurological function and that leucovorin can address this by converting to folate in the brain. He acknowledged that while small studies show improvements, larger, controlled trials are necessary to confirm its efficacy and safety.
Leucovorin’s long-term use in cancer treatment has made it a well-established medication, reducing associated risks. However, some children may experience hyperactivity or not respond to the drug. Experts advise families to work with medical professionals when considering leucovorin, as it is prescribed at much higher doses than regular vitamins. Some children may require a compounded form due to sensitivities to additives in commercial products. The goal, as stated by Frye and Dubinsky, is to get leucovorin approved for autism so it can be widely prescribed as one of the first treatments once diagnosed. FDA approval would provide stronger medical backing, leading to insurance coverage and broader acceptance in the medical community. While leucovorin is not a cure-all, it is seen as a potential tool to improve outcomes for children with autism when combined with other therapies.