FDA Reverses Course on Autism Drug After Trump and RFK Jr's Controversial Endorsement
In a significant development, the US Food and Drug Administration (FDA) has quietly walked back earlier statements promoting leucovorin as a treatment for autism, following a press conference where former President Donald Trump and Health Secretary Robert F. Kennedy Jr. touted the drug. The event, held in the Roosevelt Room of the White House on September 22, 2025, also featured FDA Commissioner Martin Makary, Medicare and Medicaid Administrator Mehmet Oz, and Dr. Dorothy Frank, but it has since led to confusion and a surge in prescriptions.
Surge in Prescriptions and Subsequent FDA Clarification
After the September announcement, outpatient prescriptions for leucovorin, a vitamin B derivative, increased by 71% for children aged five to 17, according to new research. However, on March 10, the FDA approved the drug only for cerebral folate deficiency, a very rare condition that can mimic autism symptoms. This move appears to backtrack from earlier claims, with Commissioner Makary now stating it helps treat "developmental delays with autistic features" rather than autism broadly.
At the press conference, Makary had been more explicit, calling folinic acid the first FDA-recognized treatment for autism and claiming it could benefit "hundreds of thousands of kids." Trump echoed this, saying it "gives hope to many parents," while Kennedy described it as "an exciting therapy." This rhetoric caused immediate concern among neurodevelopmental specialists, who noted a flood of parental inquiries despite limited evidence.
Medical Community's Reaction and Ethical Concerns
Doctors like William Graf, a professor at Connecticut Children's Medical Center, expressed shock at the claims, warning that they led to unnecessary spinal taps and experimental dosing without solid clinical trials. Graf stated, "I haven't seen anybody respond to it. This is not a panacea." He criticized the government for promoting unproven treatments while cutting funding for Medicaid and autism research.
The largest study on leucovorin for autism, involving only 77 children, was retracted in January after data re-analysis failed to support initial findings. Other studies have faced criticism for small sample sizes and methodological flaws. The American Academy of Pediatrics does not recommend leucovorin for autistic children due to insufficient evidence.
Broader Implications for Public Health Trust
Experts warn that such incidents erode trust in public health officials. Leon Epstein, chief of neurology at Lurie Children's Hospital, emphasized, "Science is a matter of facts and data. It's not a matter of just making something up." Graf added that promoting unproven therapies is "selling false hope" and contributes to misinformation, which is already rampant in autism discussions.
Historically, dangerous treatments like chelation and hyperbaric oxygen have been promoted for autism, and the FDA's recent withdrawal of warnings against such unproven methods has raised further alarms. Richard Frye, a physician involved in early discussions, expressed surprise that regulators didn't require more research before the announcement.
This episode highlights ongoing challenges in autism treatment and the critical need for evidence-based approaches to maintain public confidence in healthcare systems.
