FDA Approves Leucovorin for Autism Use Amid Ongoing Questions About Effectiveness

Federal health officials have approved the use of leucovorin for children with autism, despite limited scientific evidence supporting its effectiveness, prompting both interest and caution among families and medical professionals in Southern Nevada.

Leucovorin is a generic medication derived from folic acid and has long been used to support cancer patients undergoing chemotherapy. In recent months, it gained widespread attention within autism communities after parents shared anecdotal reports suggesting some nonverbal children experienced improvements in speech shortly after starting the drug. Those reports led to increased demand and an accelerated approval process by federal regulators.

Local families, including those in Las Vegas, have begun exploring the treatment as part of broader care plans. Seven-year-old Christopher Wilson, who has autism and has been largely nonverbal since birth, is one such patient. His father said Christopher has made gradual progress through years of behavioral therapy but remains limited in expressive communication, prompting the family to try leucovorin under medical supervision.

Clinicians caution that the medication should not be viewed as a cure. Pediatric neurologists in Nevada say existing research shows mixed results and emphasizes that benefits, when observed, may apply only to a narrow group of patients, particularly those with certain folate-related deficiencies. Physicians stress the importance of continued therapy and careful monitoring while more data is collected.

Experts also raised concerns about access and supply. Because leucovorin is a generic medication produced by multiple manufacturers, consistency and availability may vary. Federal health agencies are currently preparing a large-scale clinical trial to more rigorously evaluate the drug’s potential role in autism treatment, with enrollment expected to begin later this year.

While families continue to seek new options, doctors emphasize that evidence-based therapies remain the cornerstone of autism care, and any new treatments should be approached with measured expectations and professional guidance.

Source: FOX5 Las Vegas