Leucovorin use for autism increased after public promotion campaigns

Researchers at the University of California San Diego found that prescriptions for leucovorin, a drug sometimes used off-label for autism spectrum disorder (ASD), increased sharply in children after widespread media attention and public statements from White House officials. The study, published on May 18, 2026 jama network openanalyzed national electronic health record data and found that prescribing rates had increased by more than 2,000% compared to previous years.

Families of children with autism are often looking for treatments that can improve communication and quality of life, especially when treatment options are limited. This study shows how quickly information shared through news coverage, social media, and public figures can influence real-world prescribing patterns, even before large clinical trials establish whether a treatment is truly safe and effective for widespread use.”

Joshua Rothman, MD, clinical assistant professor of pediatrics at UC San Diego School of Medicine and first author of the study

Leucovorin, also known as folinic acid, is the biologically active form of folic acid. Small clinical trials have suggested that some children with autism and folate-related deficiencies may experience improvements in verbal communication after taking the drug. However, researchers say large-scale studies confirming the drug’s effectiveness and long-term safety for children with ASD have not yet been completed.

To better understand the trends determined, researchers analyzed records from the Epic Cosmos database, which contains more than 300 million patient records from more than 1,800 hospitals and 41,500 clinics in all 50 states and Washington, DC. The study focused on 838,801 children with autism who accounted for more than 11.9 million outpatient encounters between January 2023 and January 2026.

For about two years, leucovorin prescribing rates remained relatively stable, averaging about 34 prescriptions per 100,000 outpatient encounters among children with autism. Rates then began climbing steadily through early 2025 and bounced back later that year. By August 2025, prescription rates had increased to 335 prescriptions per 100,000 encounters. In November 2025, rates climbed again to more than 835 prescriptions per 100,000 encounters.

The researchers observed that the initial increase in prescribing coincided with a February 2025 national television news segment that featured a family who reported dramatic improvement in their child’s language after treatment with leucovorin. Interest in the drug increased further after White House officials publicly promoted leucovorin as part of a broader autism-related initiative in September 2025.

“The timing was amazing,” Rothman said. “The increase began after a widely seen media story and accelerated again after federal officials publicly discussed the drug. It highlights how rapidly clinical practice can change when a treatment attracts public attention.”

The study does not determine whether leucovorin improves autism symptoms, nor does it evaluate patient outcomes after treatment. The researchers also cautioned that prescriptions recorded in the database may not always be linked to a confirmed medical indication.

Still, the authors say the rapid increase in use raises important questions for physicians, policymakers, and families. In March 2026, the US Food and Drug Administration approved leucovorin for cerebral folate transport deficiency, an extremely rare genetic neurological disease associated with specific genetic alterations, but the drug was not approved for autism spectrum disorder.

The researchers say the findings underscore the need for continued monitoring of prescribing trends and the need for larger randomized clinical trials to evaluate whether leucovorin is beneficial for specific groups of children with autism.

“We now have a real-world example of how public attention can accelerate the adoption of a therapy before the evidence has fully taken hold,” Rothman said. “The next step is to ensure we generate the rigorous data needed to help families and physicians make informed decisions.”