Federal officials announced in late 2025 potential label changes for leucovorin, a prescription drug typically used for cancer chemotherapy patients, to include treatment for children with autism spectrum disorder, but experts caution that the science may not support such widespread use. Dr. Marty Makary, commissioner of the Food and Drug Administration, stated at a press conference that the label change would make the drug available to children with autism, predicting that "hundreds of thousands of kids, in my opinion, will benefit."

The announcement sparked considerable interest within the autism community, with many parents eager to explore new treatment options. Leucovorin, once a relatively obscure medication, has seen a surge in popularity among those seeking interventions for their children's autism symptoms.

However, the enthusiasm surrounding leucovorin is not universally shared. Dr. Paul Offit, a researcher closely following the issue, expressed concern that the drug's popularity has outpaced the scientific evidence supporting its effectiveness in treating autism. "Interest in leucovorin has been on the rise among some parents of children with autism," Offit stated, "But researchers like [me] say that the drug's popularity is far ahead of the science."

Leucovorin is a medication primarily used to counteract the toxic effects of certain chemotherapy drugs. Its potential application in autism stems from theories about folate deficiencies or metabolic issues in some individuals with autism. While some small studies have suggested potential benefits, larger, more rigorous trials are needed to confirm these findings and establish appropriate dosage guidelines and safety protocols.

The FDA's consideration of a label change reflects a growing awareness of the diverse needs within the autism community and a willingness to explore potential therapies. However, experts emphasize the importance of relying on evidence-based medicine and caution against adopting treatments without sufficient scientific validation. The debate surrounding leucovorin highlights the challenges of balancing hope and scientific rigor in the search for effective interventions for autism spectrum disorder. The FDA's decision regarding the label change remains pending, and further research is expected to play a crucial role in determining the drug's future role in autism treatment.