FDA Approves Controversial Autism Drug Amidst Skepticism

Sun Sep 28 2025 23:22:54 GMT+0300 (Eastern European Summer Time)

In a surprising decision, the FDA approved leucovorin, a generic drug, as a treatment for autism, sparking debates among experts about its efficacy.

The FDA's recent approval of leucovorin, a derivative of vitamin B, as a treatment for autism has been met with surprise and skepticism from leading autism researchers and organizations. Many experts argue that the supporting studies are weak and insufficient, cautioning against widespread use without more rigorous trials. Despite the concerns, interest in the drug is growing among parents who claim to see improvements in their children. This contentious decision highlights the challenges of finding effective treatments for autism in an often polarized landscape.

WASHINGTON (AP) — In a surprising move, the FDA announced the approval of leucovorin, an established generic drug, as a treatment for autism, raising eyebrows among experts in the field. Dr. Richard Frye, a key advocate for adapting the medication for autistic children, expressed his astonishment at the immediate approval without further extensive studies. While leucovorin is known to aid in boosting levels of folate in the brain, critics argue that the scientific studies backing this decision are both limited and inconclusive.

Leading autism groups swiftly distanced themselves from the FDA's decision, deeming the evidence for leucovorin's efficacy as 'very weak.' Dr. David Mandell from the University of Pennsylvania emphasized that there is simply no substantial evidence that leucovorin can significantly alleviate autism symptoms. Despite this skepticism, reports indicate a growing number of doctors prescribing the drug, citing anecdotal evidence of improved symptoms among some patients.

The origins of the leucovorin theory date back two decades when it was suggested that low folate levels might contribute to autism. However, continuing studies have failed to establish a concrete link, and many researchers maintain that autism's roots are predominantly genetic, influenced by various factors including paternal age at conception.

Nevertheless, a growing chorus of parents is embracing leucovorin, often guided by online discussions and social media. Some share success stories of marked improvements in their children's social and communication skills after beginning treatment. One parent noted their child's newfound ability to make eye contact and speak in sentences after starting the medication.

Experts advise caution, stressing the crucial need for rigorous clinical trials to substantiate any claims regarding the drug’s effectiveness. Dr. Lawrence Gray, a pediatric developmental specialist, highlighted the importance of adhering to established guidelines, warning against straying into unverified treatments for autism.

As the debate continues, it underscores the ongoing challenges within the medical community regarding effective treatments for autism and the need for more thorough research to ensure the safety and efficacy of emerging therapies.