Researchers exploring the underlying causes of autism have identified a surprising potential treatment—a widely used cancer drug. Leucovorin, a medication typically prescribed to reduce chemotherapy side effects, has demonstrated promising effects in improving autism symptoms. Autism spectrum disorder (ASD) is a neurodevelopmental condition that affects communication, social interactions, and behavior. Nonverbal Children Showed ImprovementOver the past 13 years, Dr Richard Frye, a pediatric neurologist from Arizona, has conducted multiple studies suggesting that leucovorin could help children with autism make significant developmental progress. Notably, some nonverbal children who received the drug have started speaking for the first time. Speaking to NYPost, Dr Frye said, "There isn't a single pill to cure autism, but leucovorin has helped many children. Rising Autism Rates and the Need for New Treatments."Autism Has Increased By 175% In USThe prevalence of autism diagnoses has surged in recent years, increasing by 175% between 2011 and 2022 in the United States. According to the Centers for Disease Control and Prevention (CDC), approximately 1 in 36 children in the U.S. is now on the spectrum.Experts believe this increase is due to greater awareness, improved diagnostic criteria, and expanded screening efforts. However, environmental factors and policy changes may also play a role, underscoring the need for innovative treatment options like leucovorin.How Leucovorin May HelpDr. Frye’s interest in leucovorin’s potential stemmed from research in the early 2000s that identified unusual neurochemical patterns in children with autism. Scientists discovered that many children with neurodevelopmental disorders had low levels of folate (vitamin B9) in their brains, a condition known as cerebral folate deficiency (CFD).Further studies revealed that over 75% of children with autism had folate receptor alpha autoantibodies, which hinder folate transport to the brain. In contrast, only 10% to 15% of children without autism exhibited these antibodies.To counteract this deficiency, Dr Frye began treating his patients with leucovorin, a form of folinic acid that bypasses the folate transport blockage caused by these receptors to counteract this deficiency. He finally concluded that the drug may help restore proper folate levels in the brain, leading to noticeable improvements in cognitive and social development.Promising Outcomes In Clinical StudiesMany children who received leucovorin have shown remarkable progress. In one case, a child who previously sat in a corner experiencing seizures began interacting with family members and playing with a sibling after starting treatment. While not a cure, the improvements were significant. Dr Frye and his team continue to investigate leucovorin’s broader effects, particularly on social interaction and comprehension. Preliminary findings suggest that older children receiving the drug exhibit a richer vocabulary, improved language skills, and enhanced understanding of speech.