Trump and RFK Jr's Autism Treatment Announcement Sparks Medical Controversy and FDA Reversal
In a significant development that has rocked the medical community, former President Donald Trump and Health and Human Services Secretary Robert F. Kennedy Jr promoted leucovorin as a treatment for autism during a White House press conference on September 22, 2025. The event, held in the Roosevelt Room, featured key health officials including FDA Commissioner Martin Makary, Medicare and Medicaid Administrator Mehmet Oz, and Dr. Dorothy Frank. This announcement triggered immediate consequences, with outpatient prescriptions for the drug surging by 71% among children aged five to seventeen in the following weeks, according to new research data.
FDA's Quiet Reversal and Limited Approval
However, on March 10, 2026, the Food and Drug Administration approved leucovorin only for cerebral folate deficiency, a very rare condition that can sometimes present with autistic-like features. This move represents an apparent walk-back from the earlier statements made by administration officials about the drug's broader application for autism spectrum disorders. FDA Commissioner Marty Makary clarified in a statement that the treatment seems to help with "developmental delays with autistic features," specifically referencing cerebral folate deficiency rather than autism itself.
This reversal stands in stark contrast to Makary's earlier, more explicit claims. During the September press conference, he declared folinic acid as the first FDA-recognized treatment for autism, stating that "hundreds of thousands of kids, in my opinion, will benefit" and that being autistic "may be entirely preventable." He further elaborated on various media platforms, claiming the drug "may help 50 to 60% of kids with autism" and that "two-thirds of kids can see a clinical improvement" in their symptoms.
Medical Community's Response and Evidence Concerns
The medical community responded with significant concern and skepticism. Dr. William Graf, a professor of pediatrics and neurology at Connecticut Children's Medical Center, noted that the announcement "predictably caused a ruckus" among neurodevelopmental specialists. He explained that many parents, seeking to be "good parents," immediately began requesting the medication despite limited evidence of its effectiveness for autism.
The scientific evidence supporting leucovorin for autism treatment remains thin. The largest study on the subject, which followed only 77 children, was retracted in January 2026 after re-analysis failed to replicate the original findings. Other studies have faced criticism for small sample sizes and methodological flaws. Consequently, the American Academy of Pediatrics stated in October 2025 that it does not recommend leucovorin for autistic children due to limited evidence.
Even Dr. Richard Frye, the Arizona physician who initially suggested leucovorin's potential to officials, expressed surprise that regulators didn't require more research before making their announcement. "We were kinda surprised that they were just approving it right out of the gate without more studies or anything," Frye told the Associated Press.
Broader Implications for Public Health Trust
Medical experts have raised serious concerns about the broader implications of this episode. Dr. Leon Epstein, chief of neurology at Lurie Children's Hospital of Chicago, emphasized that "science is a matter of facts and data. It's not a matter of just making something up and declaring it." He stressed that physicians must act with "science-based studies and science-based therapies" in their patients' best interests.
Dr. Graf highlighted the ethical concerns, stating that giving treatments without evidence is "just wrong. It's unethical." He pointed to a history of unproven autism treatments, including chelation and hyperbaric oxygen, some of which have proven dangerous. "If a B vitamin were a cure for neurodevelopmental disorders, everyone would already have known this," Graf noted, referencing previous unsuccessful attempts with vitamin-based approaches decades earlier.
Misinformation and Public Trust Consequences
The controversy extends beyond this specific treatment to broader issues of public health communication and trust. Dr. Epstein warned that such claims can "create disbelief in the system," adding that "the problem here is you cannot trust our public health officials because they are not following science."
Dr. Graf echoed these concerns, stating that "the role of government is not to put gasoline on the misinformation fire" already rampant regarding autism. He characterized the promotion of leucovorin as "selling false hope" and "almost like public deception." The consequences, experts warn, may undermine confidence in public health institutions and scientific guidance more broadly.
This episode occurs against a backdrop of broader policy challenges, including congressional cuts to Medicaid and other services for autistic individuals, as well as ongoing debates about healthcare funding and vaccine policies. The FDA's December 2025 decision to pull warnings against dangerous, unproven autism treatments further complicates the landscape of autism care and information.
