Leucovorin's Role in Autism Treatment: Scientific Insights and Regulatory Developments

Leucovorin, a drug approved for chemotherapy side effects, is being explored as a potential treatment for autism related to cerebral folate deficiency. While some small studies and anecdotal reports suggest improvements in communication and behavior, there is not enough rigorous scientific evidence to confirm its safety and efficacy for autism, raising concerns about premature approval and unregulated use. The FDA plans to update its labeling to reflect potential benefits, but experts warn against overestimating the current evidence.