Trump & Kennedy’s Autism Drug Claims Backtrack: FDA Limits Leucovorin Use

The recent push by President Donald Trump and Health and Human Services Secretary Robert F. Kennedy Jr. To promote leucovorin as a treatment for autism has sparked concern among neurodevelopmental specialists, followed by a quiet recalibration from the Food and Drug Administration (FDA). Although initial pronouncements suggested a breakthrough in autism treatment, the FDA has now clarified that leucovorin’s approval is specifically for cerebral folate deficiency, a rare genetic condition that can sometimes present with symptoms similar to autism.

The shift comes after a surge in prescriptions for the vitamin B derivative following the Trump-Kennedy announcement in September 2025. New research, detailed in The Lancet, shows outpatient leucovorin prescriptions for children aged five to 17 in the US rose by 71% in the weeks following the initial claims. This increase was driven, in part, by parents seeking any potential intervention for their children, fueled by what some doctors describe as a sense of obligation to “try this” based on the administration’s statements.

A Narrowed Focus: Cerebral Folate Deficiency

The FDA’s approval on March 10th, 2026, specifies leucovorin’s leverage for cerebral folate deficiency, a condition where the brain is unable to properly process folate. Marty Makary, commissioner of the FDA, explained that the treatment can help address “developmental delays with autistic features” that sometimes accompany this rare disorder. This is a significant narrowing of scope from earlier statements. Just months prior, Makary had explicitly described folinic acid as the first FDA-recognized treatment for autism itself, suggesting it “may be entirely preventable” and potentially benefit “hundreds of thousands of kids.” He even claimed on Becker’s Healthcare Podcast that two-thirds of children with autism could see clinical improvement with the drug.

President Trump echoed this optimism, stating the treatment “gives hope to the many parents with autistic children,” while Secretary Kennedy called it “an exciting therapy that may benefit large numbers of children.” These pronouncements, however, were made even as HHS data indicated cuts to research grants focused on autism, a seeming contradiction noted by several observers.

Limited Evidence and Retracted Research

The basis for the initial enthusiasm surrounding leucovorin remains shaky. William Graf, a professor of pediatrics and neurology at Connecticut Children’s Medical Center, notes that the rhetoric “caused a ruckus” within the medical community, prompting debate about whether families should attempt the medication and whether widespread testing for folate deficiency was warranted. However, Graf emphasizes that there was and continues to be, a lack of robust evidence to support these interventions.

The largest study on leucovorin for autism, involving only 77 children, was retracted in January 2026 after re-analysis failed to replicate the original findings. Other studies have been criticized for modest sample sizes and inadequate blinding. The American Academy of Pediatrics (AAP) issued a statement in October 2025 explicitly stating it does not recommend leucovorin for autistic children due to the limited evidence.

Even Richard Frye, the Arizona physician who initially alerted officials to the potential of leucovorin, expressed surprise that the FDA didn’t require more research before making its initial announcement. “So 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.

A History of Unproven Autism “Cures”

The promotion of leucovorin echoes a long history of unproven and sometimes dangerous “cures” for autism. Leon Epstein, chief of neurology at Lurie Children’s hospital of Chicago, stresses the importance of relying on scientific evidence. “Science is a matter of facts and data. It’s not a matter of just making something up and declaring it.” He warns that such pronouncements can erode trust in public health officials and science-based medicine.

Past interventions, such as chelation therapy (to remove heavy metals) and hyperbaric oxygen therapy, have been shown to be ineffective and potentially harmful. The FDA recently removed a warning against these dangerous, unproven treatments, highlighting the ongoing need for vigilance.

The Broader Context: Funding Cuts and Misinformation

The situation is further complicated by ongoing cuts to Medicaid and other services for autistic individuals, as reported by Autism Speaks. This, coupled with the spread of misinformation about autism, raises concerns about the potential for harm. Graf argues that the administration’s actions amount to “public deception” and contribute to a climate of false hope.

The FDA’s revised stance, focusing leucovorin’s application on the specific case of cerebral folate deficiency, appears to be an attempt to course-correct. However, the initial pronouncements have already had a tangible effect, driving up prescriptions and potentially diverting resources from evidence-based interventions. The incident underscores the critical need for public health officials to base their recommendations on rigorous scientific evidence and to avoid making unsubstantiated claims that could mislead the public and harm vulnerable individuals.

Looking ahead, the focus will likely return to supporting research into the complex causes of autism and developing effective, evidence-based interventions. Continued surveillance of prescription patterns and ongoing evaluation of emerging research will be crucial to ensuring that individuals with autism and their families receive the best possible care. The incident also highlights the importance of critical thinking and consulting with qualified healthcare professionals before pursuing any unproven treatment.