WASHINGTON (AP) — The recent move by the Trump administration to approve the old drug leucovorin as a potential treatment for autism has taken many experts by surprise, sparking a heated debate over its efficacy and the integrity of supporting evidence.
Dr. Richard Frye, the physician behind the proposal, had engaged with federal agencies about a tailored approach to utilizing leucovorin, anticipating further study would be required. “We were kinda surprised that they were just approving it right out of the gate without more studies,” Frye remarked.
The decision has been criticized as yet another example of the Trump administration’s controversial rollout strategies, raising concerns that the unproven solution requires much more rigorous investigation before gaining acceptance in the medical community. Prominent autism advocacy groups expressed their reservations, categorizing the evidence supporting leucovorin’s use as both weak and insufficient, stating, We have nothing resembling even moderate evidence that leucovorin is an effective treatment for autism symptoms, said David Mandell, a psychiatrist at Penn.
Despite the scrutiny, a surge in interest from parents is observable as online discussions proliferate. Brian Noonan of Phoenix describes how he learned of the drug via an AI chatbot while looking for potential treatment options for his son. After initiating treatment with leucovorin through a compounding pharmacy, Noonan reports tangible improvements in his child’s neurological function.
Experts in the autism field caution against hastily endorsing this option, noting a host of medications previously regarded as promising fell short upon extensive testing. “Small studies often find populations that are very motivated,” warns Dr. Lawrence Gray, urging a return to stringent clinical trials before any broad implementation is considered.”
Continuing, specialists emphasize that while the theory connecting folate deficiencies and autism exists, it remains unproven and requires much deeper investigation. Prominent advocates for a more cautious approach suggest that the complexities surrounding autism necessitate a well-founded, scientifically validated route before any drug can receive approval for widespread use.
The FDA’s recent announcement hinted at merely updating the existing generic drug label to include its use in autism, shifting initial expectations for new formulations. As the debate unfolds, researchers hope to clarify the implications of leucovorin while emphasizing the critical need for balanced perspectives and responsible practices within the medical community.
