Vitamin B Drug for Autism: Hope or Hype?

Leucovorin, a medication derived from vitamin B, is gaining attention as a potential treatment for autism spectrum disorder. The Trump administration has promoted it, leading to increased interest. Some parents, like Kathy Williams from New Jersey, have observed significant positive changes in their children, reporting enhanced communication and social interaction after starting the drug.

This drug, an inexpensive generic originally prescribed to mitigate chemotherapy side effects, works by bypassing a blockage that can prevent folic acid from reaching the brain. While a 2024 study indicated improvement in symptoms for children with cerebral folate deficiency, medical professionals caution that not all individuals with autism have this condition, and more research is deemed necessary.

Despite enthusiastic testimonials and claims of a "profound" upside with a "great safety profile," major medical associations, including the American Academy of Pediatrics, state there is "limited evidence" for leucovorin's effectiveness and safety in treating autism. They do not recommend its routine use, underscoring the ongoing debate between anecdotal successes and scientific validation.