Leucovorin use for autism surged after public promotion campaigns

Researchers from the University of California San Diego found that prescriptions for leucovorin, a drug sometimes used off-label for autism spectrum disorder (ASD), rose sharply among children after widespread media attention and public statements from White House officials. The study, published May 18, 2026 in JAMA Network Open, analyzed national electronic health record data and found prescribing rates increased more than 2,000% compared with prior years.

Families of children with autism are often searching for therapies that might improve communication and quality of life, especially when treatment options are limited. What this study shows is 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 broad use."

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

Leucovorin, also known as folinic acid, is a 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 medication. However, researchers note that large-scale studies confirming the drug's effectiveness and long-term safety for children with ASD have not yet been completed.

To better understand prescribing trends, the researchers analyzed records from the Epic Cosmos database, which includes more than 300 million patient records from over 1,800 hospitals and 41,500 clinics across all 50 states and Washington, D.C. 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 roughly 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 in early 2025 before surging later that year. By August 2025, prescribing rates had risen 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 rise in prescribing coincided with a February 2025 national television news segment featuring a family who reported dramatic language improvements in their child after treatment with leucovorin. Interest in the medication expanded further after White House officials publicly promoted leucovorin in September 2025 as part of broader autism-related initiatives.

"The timing was striking," Rothman said. "The increases began after a widely viewed media story and accelerated again after federal officials publicly discussed the medication. It highlights how rapidly clinical practice can shift when a treatment captures public attention."

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

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

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

"We now have a real-world example of how public attention can accelerate adoption of a therapy before the evidence fully catches up," Rothman said. "The next step is making sure we generate the rigorous data needed to help families and clinicians make informed decisions."