The FDA recently declined to endorse leucovorin for autism, citing a lack of strong evidence for it.
President Donald Trump, alongside Secretary of Health and Human Services Robert F. Kennedy Jr. and Medicare and Medicaid Administrator Mehmet Oz, speaking about autism at a press conference held on September 22, 2025.
Last September, President Donald Trump, Robert F. Kennedy Jr, and other health officials declared they had uncovered a new, potentially groundbreaking treatment for autism spectrum disorder : the existing medication leucovorin. A study out today shows that plenty of families believed them. Researchers at the University of California, San Diego examined nationwide prescription trends of the drug last year. The prescription rate of leucovorin among children with autism skyrocketed by late 2025, particularly following, they found.
The results indicate that Trump’s endorsement had significant influence on these families, the researchers say, despite the lack of data supporting the drug’s effectiveness.
“Families of children with ASD are often searching for therapies that might improve quality of life, especially when treatment options are limited,” the study’s lead author Joshua Rothman, a clinical assistant professor of pediatrics at UCSD’s school of medicine, told Gizmodo. “While some small studies have shown promise, we don’t yet have convincing evidence to recommend this treatment to all children with ASD. ”The announcement last September largely focused on acetaminophen .
At that time, Trump and other officials proclaimed to have uncovered a link between women taking acetaminophen during pregnancy and a higher risk of autism in their children. Many expertsLeucovorin is a form of folate, or B9, a vitamin important to many functions, including the healthy development of a fetus.
Though traditionally used to offset the side effects of certain cancer treatments, it’s also become a standard treatment for cerebral folate deficiency—a condition where folate can’t be transported to the brain through normal means. Unlike other forms of folate, like folic acid, leucovorin can reach the brain, allowing it to reverse this deficiency.
Limited studies have suggested that children with autism are likely to have low levels of folate in their brain, and some of the symptoms of cerebral folate deficiency can also resemble severe autism. Based on these tenuous connections, RFK Jr. claimed in September that leucovorin was an exciting therapy that might benefit “large numbers of children” with autism. Former FDA Commissioner Marty Makary, whothat leucovorin could possibly help “hundreds of thousands of kids.
” At another time, he implied that leucovorin could treat up to “50% of kids with autism” who might have this deficiency. Given the widespread attention that the September announcement drew, Rothman and his team wanted to see how it might have changed the use of leucovorin.
They analyzed medical records from EPIC Cosmos, a database containing over 300 million patient records collected from health care systems across the U.S. They specifically tracked prescription rates of leucovorin among more than 800,000 children diagnosed with autism between January 2023 and January 2026. The team’s research showing how prescription rates of leucovorin among children with autism changed over time.
© Rothman et al/JAMA Network Open The Trump announcement wasn’t the only major thing in 2025 to potentially boost the popularity of leucovorin. A Fox News report in February 2025Rothman and his team saw a clear bump in the prescription rate of leucovorin in the wake of the Fox News report, followed by an even larger spike after the Trump announcement. The rate in 2023 and 2024 was around 34 prescriptions per every 100,000 outpatient visits among children with autism.
By November 2025, though, it had surged to 835 prescriptions per 100,000 visits.
“We found that leucovorin prescriptions for children with ASD increased over 2,000% after widespread media attention and public statements from White House officials,” Rothman said. The team’s results were Since the September announcement, the Trump administration has stepped back from its bombastic endorsement of the drug.to endorse it as a treatment for autism, with FDA officials explicitly citing a lack of strong evidence that would support the decision.and it’s gotten weaker lately.
This past January, a journal yanked a positive trial testing leucovorin supplements in children with autism after outside researchers discovered numerous “errors” and “concerns” with the data,to the retraction notice. It was the largest trial of the drug conducted so far, with 77 children, and one of only five trials total. The study by Rothman and his team isn’t intended to weigh in on the usefulness of leucovorin for autism.
But given how many families have now been prescribed the drug, there should be more research done on the topic, Rothman argues.
“Because leucovorin prescriptions have increased so significantly for children with ASD, it would be worthwhile to assess outcomes and share these results,” he said. So the team hopes that other researchers will be able to track the long-term outcomes of the families and children who have taken leucovorin for autism.
“It is our duty as scientists and clinicians to generate the rigorous data needed to help families and clinicians make informed decisions,” he added. Elon Musk Said He’d Stop Political Spending. He’s the Third-Largest Donor in the MidtermsThe Mess at the FDA Just Got Bigger as Chief Marty Makary Resigns
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