Surge in Prescriptions for Leukovorin Amid Autism Treatment Debate

Prescriptions for leukovorin, also known as folinic acid, have surged dramatically in the United States, according to a new study published in JAMA Network Open. This increase coincides with widespread media attention and endorsements from prominent political figures promoting the drug as a hopeful therapy for autism spectrum disorder.

Leukovorin is typically prescribed to address folate deficiency in the brain, a rare condition linked to low levels of vitamin B9, and to mitigate side effects from certain chemotherapy treatments. However, it has not received approval from the U.S. Food and Drug Administration (FDA) for autism treatment.

Impact of Media and Political Endorsements on Prescription Rates

The study analyzed over 300 million medical records across the U.S. and found that leukovorin prescriptions were initially stable at approximately 34 prescriptions per 100,000 medical visits. Following a high-profile media report in January 2025 highlighting a child’s notable improvement after treatment, the rate climbed to 225 per 100,000 visits by August.

Subsequent public statements by political leaders further accelerated this trend, pushing prescriptions to exceed 835 per 100,000 visits by November 2025. Researchers attribute this rapid increase to the influence of media narratives and political advocacy on physicians’ prescribing behaviors, often preceding robust scientific validation.

Families’ Search for Autism Therapies and Scientific Uncertainty

Joshua Rothman, lead author and researcher at the University of California, San Diego, explained that families of children with autism frequently seek any available options to enhance communication skills and quality of life, especially given the limited treatment choices currently accessible.

Individual success stories have circulated widely, including one from Missouri where a child with severe speech difficulties reportedly began forming complete sentences and expressing emotions for the first time after starting leukovorin therapy. Despite such accounts, the researchers emphasize that current scientific evidence remains insufficient to confirm the drug’s effectiveness in treating autism.

Calls for Rigorous Clinical Trials and Evidence-Based Decisions

The study’s authors stress the need for comprehensive clinical trials to evaluate leukovorin’s safety and therapeutic value for autism. They warn that media and popular enthusiasm can hasten the adoption of treatments before conclusive scientific data is available.

Ultimately, the researchers advocate for clearer data to guide families and healthcare providers in making informed, evidence-based treatment decisions regarding autism therapies.