Parents of children with autism are increasingly pursuing a controversial treatment known as leucovorin, a derivative of vitamin B9, following a recent announcement by federal health officials suggesting its potential benefits. This surge of interest reflects deep desperation among families seeking effective therapies for their children, as many have already explored various alternatives.
Swathi Balantrapu has been searching for effective therapies for her 10-year-old son diagnosed with autism. Initially, she tried applied behavior analysis (ABA) therapy, an evidence-based approach. Subsequently, she invested in a $7,000 movement and stretching program, and even pursued stem cell therapy in Germany, costing approximately $40,000 per year over the past three years. After a press conference last fall, where federal officials highlighted leucovorin as a promising treatment, Balantrapu felt renewed hope. “I would do anything for my child,” she expressed. “Maybe there is something to it. It just feels like one last stone unturned.”
The recent endorsement of leucovorin by federal health officials claimed it could aid in treating a rare brain condition known as cerebral folate deficiency, which could potentially benefit “hundreds of thousands” of children with autism. This announcement ignited considerable interest among parents, prompting social media groups to form where thousands exchanged information about doctors willing to prescribe the medication.
Despite the enthusiasm, major medical groups cautioned against the routine prescription of leucovorin for autism. The contrasting views have created tension between healthcare providers and concerned parents. “All of a sudden, there’s this onslaught of information being disseminated that we now have to explain, disentangle, and interpret,” said Dr. Shafali Jeste, an autism researcher at the University of California, Los Angeles. “It’s a big burden, and we’re all willing to bear it… but I think that a lot of it ends up becoming a distraction.”
While using leucovorin to treat cerebral folate deficiency is well-established, experts indicated that the messaging from the Trump administration was misleading. Last week, the administration clarified its position, narrowing the approval for the drug to a specific subset of patients with an extremely rare condition, FOLR1-related cerebral folate transport deficiency, which has fewer than 50 known cases worldwide. A senior official from the FDA stated, “Right now we don’t have sufficient data to say that we could establish efficacy for autism more broadly. It’ll be up to patients to talk with their physicians to see if that might be right for them.”
The fallout from the initial announcement has already impacted public perception, with many families still eager to explore this new avenue of treatment. Last week, Dr. Frederick Bassal, a child neurologist at the University of California, Davis, noted that many parents left the press conference believing that leucovorin was a proven treatment for autism. Some assumed a significant number of autistic children also suffered from cerebral folate deficiency, a claim lacking substantial evidence according to experts like Dr. Alice Kuo.
As demand for leucovorin soared, Balantrapu turned to social media platforms like Facebook to connect with others seeking the drug. She joined a Facebook group moderated by Keith Joyce, which gained around 80,000 members sharing experiences and doctor referrals. Joyce, who cares for a 5-year-old boy with autism, reported that many parents believe leucovorin could help nonverbal children start speaking. “Parents were very frustrated,” he recalled, describing the situation as akin to “opening Pandora’s box.”
Despite the apparent interest, many doctors remain hesitant to prescribe leucovorin, citing the lack of sufficient evidence. Dr. Sarah Mohiuddin, who leads the Multidisciplinary Autism Program at Michigan Medicine, explained that uncertainty lingers after the announcement. Major medical organizations, including the American Academy of Pediatrics, have issued statements advising against its use for autism until more research is conducted.
Leucovorin is generally considered safe, but concerns about its long-term effects on children with autism remain. No data exists on the drug’s long-term use beyond six months in this demographic. Current prescribing protocols may involve dosages significantly higher than the recommended daily intake of folate, raising further questions.
Recent studies have indicated a 71% increase in leucovorin prescriptions during the last quarter of 2025, attributing this surge to the influence of public figures and government messaging. Joyce described the FDA’s recent announcements as “maddening,” expressing frustration over the new limitations on its use.
While some healthcare providers refuse to prescribe leucovorin, others in private practice, such as Dr. Richard Frye, actively advocate for its use, believing it can significantly benefit many children. Frye, who has faced skepticism from mainstream medicine, serves a patient population where approximately 80% receive prescriptions for leucovorin.
“Refusing to acknowledge [the benefits of leucovorin] and refusing to at least try it is really very confusing to me,” Frye asserted. He has founded a pharmaceutical company to develop a new formulation of the drug and expressed hope for broader approval in the future.
Parents seeking this treatment often face significant financial barriers. Balantrapu encountered steep fees at Frye’s clinic, amounting to nearly $1,896 for an initial visit, with follow-up consultations costing $948. Many practitioners who prescribe leucovorin operate outside of major health systems, leading to cost variations and accessibility challenges.
As the debate continues, healthcare professionals must navigate the complicated landscape of patient demand and scientific evidence. The delicate balance between maintaining trust and adhering to evidence-based practices remains crucial, as many families search for effective therapies for their children.
The complexity of prescribing decisions underscores the need for more research into treatments for autism. Dr. Mohiuddin emphasized the importance of focusing on evidence-based interventions to provide better support for families. “They need more funding and support for day-to-day services and supports that already exist,” she stated.
With the ongoing discussions around leucovorin, the path forward for families and healthcare providers alike remains uncertain, as they grapple with the intersection of hope, science, and patient care.