Leucovorin for Autism: Controversial Approval Faces Skepticism from Experts

Mon Oct 13 2025 14:56:46 GMT+0300 (Eastern European Summer Time)

The Trump administration's fast-tracking of leucovorin, an old drug repurposed for autism treatment, raises alarms among leading researchers, citing weak evidence and potential risks.

The FDA's recent approval of leucovorin as a treatment for autism came as a surprising move for experts, who argue that this unproven medication lacks solid backing and warrants further study. Critics emphasize that most autism cases appear to be genetically rooted, and caution against jumping into unfounded treatments. While some doctors are already prescribing the drug based on preliminary studies, leading psychiatrists urge careful clinical trials before widespread adoption.

WASHINGTON (AP) — The recent announcement from the Trump administration about repurposing leucovorin, a derivative of vitamin B, as a treatment for autism has drawn immediate criticism from experts in the field. Dr. Richard Frye, the Arizona-based pediatric neurologist who initially proposed the idea, expressed surprise at the lack of thorough studies backing the decision. We were kinda surprised that they were just approving it right out of the gate without more studies or anything, Frye stated.

This decision has raised alarms among the nation’s leading autism research groups, who argue that the available studies supporting the drug's efficacy are both minimal and inadequate. David Mandell, a psychiatrist at the University of Pennsylvania, noted, We have nothing resembling even moderate evidence that leucovorin is an effective treatment for autism symptoms. The consensus among experts indicates that autism is predominantly genetically driven, with various factors influencing its expression.

In contrast to the excitement surrounding leucovorin's approval, the current stance emphasizes the necessity for proper clinical trials to validate its potential benefits and address safety concerns. Dr. Lawrence Gray, a pediatric development specialist, warns that many treatments that seem promising in small trials often fail during larger scalability. Consequently, the medical community advocates for a cautious approach—advising that the medication be considered only in controlled environments until more conclusive evidence emerges.

With conflicting opinions circulating, varying reports of efficacy among parents are emerging, particularly in social media forums. Some parents have reported noticeable improvements in their children after starting treatment with compounding versions of leucovorin, suggesting that although anecdotal evidence exists, it cannot substitute for rigorous scientific validation.

As the situation evolves, experts urge stakeholders to proceed thoughtfully, highlighting that unsubstantiated treatments pose risks not only to patients but also to the integrity of autism treatment frameworks.