The US government has issued controversial guidance recommending leucovorin, a form of folic acid, to treat speech difficulties in children with autism. The new health recommendations also discourage pregnant women from taking paracetamol to prevent autism. Leucovorin is primarily a cancer medication used alongside chemotherapy drugs like fluorouracil and methotrexate. It works by providing an active form of folate that healthy cells need to make DNA. The recommendation appears linked to a theory connecting low brain folate levels to cerebral folate deficiency, which shares symptoms with autism. However, while some small studies show promising results, experts caution that current evidence is insufficient to change medical practice. Larger clinical trials are needed before doctors can properly recommend leucovorin for autism treatment. The drug carries side effects including severe allergic reactions, seizures, nausea and vomiting.
Limited Evidence Supports Autism Treatment Claims
A 2021 review analysed 21 studies using leucovorin for autism or cerebral folate deficiency, finding general improvement in autism symptoms but calling for more research. A 2024 study of approximately 80 children aged two to 10 years showed those taking daily 50mg of folinic acid displayed more pronounced improvement compared to placebo groups. A similar 2025 study with Chinese children found greater improvement in social reciprocity skills. Despite these promising findings, none reach the standard required to change medical practice. Larger studies are essential before proper recommendations can be made. Like all medications, leucovorin has side effects including severe allergic reactions, seizures and nausea. Australian authorities should maintain current evidence-based practices until more robust clinical trial data emerges.
Source: Link