Controversy Surrounds FDA Approval of Leucovorin for Autism Treatment

In September 2026, former President Donald Trump and Robert F. Kennedy Jr., Secretary of the U.S. Department of Health and Human Services (HHS), announced leucovorin, a vitamin B derivative, as a potential treatment for autism. This announcement led to a significant increase in outpatient prescriptions for leucovorin among children aged five to 17, despite a lack of substantial evidence supporting its efficacy for autism.

The Guardian

Following the press conference, outpatient prescriptions for leucovorin surged by 71%, as many parents sought to explore this new treatment option. William Graf, a professor of pediatrics and neurology at Connecticut Children’s Medical Center, noted that parents felt compelled to try the medication in hopes of benefiting their children. However, the FDA later clarified that leucovorin was approved solely for cerebral folate deficiency, a rare condition that can exhibit symptoms similar to autism. This apparent retraction raised concerns among medical professionals regarding the initial claims made by Trump and Kennedy.

The Guardian

Experts have expressed significant skepticism about the use of leucovorin for autism. Graf criticized the lack of solid evidence regarding appropriate dosages and the absence of major clinical trials. He stated, “This is not a panacea. This is not a wonder drug.” The American Academy of Pediatrics (AAP) has also advised against recommending leucovorin for autistic children due to insufficient evidence. Furthermore, the largest study on leucovorin for autism, which involved only 77 children, was retracted after failing to replicate its initial findings.

The Guardian

Richard Frye, an Arizona physician who initially suggested leucovorin's potential, expressed surprise that regulators approved the treatment without more rigorous research. Graf emphasized the ethical implications of administering treatments without evidence, stating, “Giving anyone a treatment without evidence is just wrong. It’s unethical.”

The Guardian

The controversy surrounding leucovorin's approval has raised alarms about the potential for public deception and the erosion of trust in public health officials. Experts warn that such claims can contribute to widespread misinformation about autism treatments. Graf remarked, “The role of government is not to put gasoline on the misinformation fire,” highlighting the dangers of promoting unproven therapies.

The Guardian

Marty Makary, FDA commissioner, initially claimed that leucovorin could benefit “hundreds of thousands of kids” with autism, suggesting that it might help 50 to 60% of affected children. However, following the FDA's approval for cerebral folate deficiency only, he stated that the treatment could assist with “developmental delays with autistic features.” Trump and Kennedy echoed sentiments of hope for families, with Kennedy calling it “an exciting therapy.”

The Guardian

There is a notable discrepancy between the initial enthusiasm surrounding leucovorin's potential benefits for autism and the subsequent FDA approval, which limited its use. Critics argue that the lack of comprehensive clinical trials and the retraction of previous studies undermine the credibility of the claims made by Trump and Kennedy.

The Guardian

• “Hundreds of thousands of kids, in my opinion, will benefit,” — Marty Makary, FDA Commissioner

The Guardian

• “Giving anyone a treatment without evidence is “just wrong.” — William Graf, Professor of Pediatrics and Neurology

The Guardian

• “The role of government is not to put gasoline on the misinformation fire,” — William Graf, Professor of Pediatrics and Neurology

The Guardian