Summary: A new national study reveals how rapidly public attention can reshape real-world medical practices before large-scale clinical trials prove a drug’s efficacy. Analyzing electronic health records of over 800,000 children with autism spectrum disorder (ASD), researchers found that prescriptions for leucovorin, a drug used off-label for autism, skyrocketed by more than 2,000%.
This unprecedented spike directly coincided with a national television news segment and subsequent public promotional statements by White House officials, highlighting the profound impact of media and political influence on clinical prescribing habits.
Key Facts
- Massive Prescribing Surge: Leucovorin prescriptions exploded from a baseline average of 34 per 100,000 outpatient encounters to over 835 per 100,000 encounters, marking an increase of over 2,000%.
- The Media Catalyst: Prescribing rates began climbing in February 2025 immediately following a national television news segment that showcased a family reporting dramatic language improvements in their child after using the drug.
- The White House Acceleration: The surge exponentially accelerated after September 2025, when White House officials publicly promoted leucovorin as part of federal autism-related initiatives.
- Big Data Scope: Researchers utilized the Epic Cosmos database, capturing a massive sample size of 838,801 children with autism representing more than 11.9 million outpatient encounters across the United States.
- FDA Approval Contrast: In March 2026, the U.S. Food and Drug Administration (FDA) officially approved leucovorin for an ultra-rare genetic neurological disease called cerebral folate transport deficiency, but the drug remains completely unapproved for ASD.
Source: UCSD
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,” said Joshua Rothman, MD, clinical assistant professor of pediatrics at the UC San Diego School of Medicine and first author of the study.
“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.”
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.”
Additional co-authors on the study include: Brian Kwan, MD, and Christopher Longhurst, MD, from UC San Diego; Anupam Jena, MD, PhD, from Harvard.
Study received no outside funding.
Jena reported receiving personal fees from Analysis Group, Los Angeles Times, New York Times, Wall Street Journal, Freakonomics MD, Doubleday Books, and Harry Walker Agency; and serving as an unpaid board member of the United Network for Organ Sharing (UNOS) outside the submitted work. No other disclosures were reported.
Key Questions Answered:
A: Leucovorin, also widely known as folinic acid, is a biologically active form of folic acid. It caught the public’s eye because a handful of small clinical trials suggested that a very specific sub-group of children, those with autism combined with folate-related deficiencies, showed improvements in verbal communication after taking it. However, large-scale studies confirming that it works safely for children with autism across the board have simply not been done yet.
A: No, and that is a crucial distinction. This specific study did not track clinical patient outcomes, nor did it evaluate whether the drug improved or worsened any symptoms of autism. It was strictly designed to function as a mirror for medical ethics and public health, proving how incredibly fast public hype and political messaging can dictate real-world medical actions before regulatory agencies or scientific consensus can catch up.
A: It is not illegal; prescribing a drug for a condition outside its officially approved mandate is a common medical practice known as “off-label” use. In fact, the FDA officially approved leucovorin in March 2026, but only for an ultra-rare genetic neurological disease called cerebral folate transport deficiency. Prescribing it for autism isn’t illegal, but because the extensive, rigorous trials required to establish its safety profile in children with ASD are incomplete, clinicians are essentially flying blind regarding its long-term effects.
Editorial Notes:
- This article was edited by a Neuroscience News editor.
- Journal paper reviewed in full.
- Additional context added by our staff.
About this Autism and pharmacology research news
Author: Lizelda Lopez
Source: UCSD
Contact: Lizelda Lopez – UCSD
Image: The image is credited to Neuroscience News
Original Research: Open access.
“Rates of Leucovorin Prescriptions for Children With Autism” by Joshua M. Rothman, Brian Kwan, Christopher A. Longhurst, and Anupam B. Jena. JAMA Network Open
DOI:10.1001/jamanetworkopen.2026.13286
Abstract
Rates of Leucovorin Prescriptions for Children With Autism
On April 10, 2025, US Health and Human Services Secretary Robert F. Kennedy, Jr, stated during a White House Cabinet meeting that “in September, we will know what has caused the autism epidemic.”
On September 22, 2025, President Trump and Secretary Kennedy unexpectedly promoted leucovorin as a treatment for speech-related deficits associated with autism spectrum disorder (ASD).
