Demand for Leucovorin Surges Following FDA Comments on Autism Treatment
A decades-old drug, leucovorin, is experiencing a significant increase in demand as parents seek potential treatments for autism following recent statements from the Food and Drug Administration and endorsements from prominent figures.
Since FDA Commissioner Marty Makary suggested the drug could help “hundreds of thousands” of children with autism last month, doctors and researchers report being overwhelmed with inquiries. While approved for treating chemotherapy side effects, leucovorin is being explored for off-label use in autism, based on emerging research into folate deficiencies. The American Academy of Pediatrics does not recommend leucovorin for routine use in children with autism, citing a lack of conclusive evidence.
The surge in interest is fueled by online communities, including a now-suspended Facebook group called Leucovorin for Autism, which quickly grew to 84,000 members. Parents are sharing anecdotal reports of improvements in speech and social awareness, like Keith Joyce, legal guardian to a four-year-old boy named Jose, who credits the drug with positive changes. However, experts caution against interpreting these reports as definitive proof. “It puts physicians in a very tough position because they’re being asked to prescribe something that is not evidence-based,” said Dr. Shafali Jeste, an autism expert at UCLA. The increasing prevalence of autism, now affecting approximately 1 in 31 eight-year-olds according to the U.S. Centers for Disease Control and Prevention, underscores the urgent need for effective treatments.
The FDA’s initial focus is on expanded approval related to cerebral folate deficiency (CFD), a rare genetic condition linked to autism-like symptoms, but acknowledges the data are limited. The National Institutes of Health will support further research into leucovorin’s impact on CFD and its potential benefits for individuals with autism, with plans for post-market surveillance and safety studies. Health and Human Services officials stated the agency based its plan on an analysis of over 40 case studies showing 85% of patients experienced some benefit.
