Autism Families Are Demanding Leucovorin — But Most Doctors Aren't Ready to Prescribe It
A federally hyped autism drug has sparked fierce demand among parents, while physicians and major medical groups urge caution over insufficient evidence.
A Desperate Search for Answers
For Swathi Balantrapu, finding effective help for her 10-year-old son with autism has meant leaving no option unexplored. She started with applied behavior analysis therapy, a widely accepted evidence-based approach. When that felt insufficient, she enrolled her son in a $7,000 movement and stretching program. She even traveled to Germany for stem-cell therapy, spending $40,000 annually over three consecutive years.
Then, last fall, a federal press conference introduced her to something new — and reignited her hope.
"I would do anything for my child," Balantrapu said. "Maybe there is something to it. It just feels like one last stone unturned."
The treatment capturing her attention was leucovorin, a folinic acid compound derived from vitamin B9, most commonly used in cancer care. Federal health officials publicly suggested it could treat a rare neurological condition called cerebral folate deficiency and might benefit "hundreds of thousands" of children on the autism spectrum.
What Is Leucovorin — and Why Is It Controversial?
Leucovrin itself is not a new or mysterious drug. Physicians have safely used it for decades to treat cerebral folate deficiency, a rare brain disorder — and that application remains uncontested. The controversy stems from whether the drug has any meaningful benefit for the much broader population of children diagnosed with autism spectrum disorder.
Medical experts told NPR the federal government's messaging overstated the evidence considerably. In fact, the administration later walked back its initial claims, limiting expanded approval only to patients with FOLR1-related cerebral folate transport deficiency — an exceptionally rare condition with fewer than 50 documented cases globally.
"Right now we don't have sufficient data to say that we could establish efficacy for autism more broadly," a senior FDA official acknowledged in a follow-up briefing.
Despite this correction, the damage to public perception had already taken hold.
A Flood of Demand Doctors Weren't Prepared For
The original press conference last fall sent shockwaves through online autism communities. Social media groups emerged almost immediately, with tens of thousands of parents sharing physician referrals, prescription tips, and personal anecdotes about the drug. A Facebook group moderated by Keith Joyce, a father raising a 5-year-old with autism, attracted roughly 80,000 members before Facebook removed it for repeated violations of community standards.
Joyce said his child began speaking after starting leucovorin, a story he shared widely to encourage other families. "Parents were very frustrated," he recalled. "Many doctors were saying, 'I'm not going to prescribe it simply because there's not enough information.'" After Facebook pulled the group, Joyce attempted to rebuild the community on Telegram. Several other groups have since emerged on WhatsApp and other platforms, with dozens of messages circulating daily — sharing prescriber names, dosing questions, and administration tips.
Prescriptions for leucovorin surged by 71% in the final quarter of 2025, according to a recently published paper. The authors noted that the spike illustrated "the apparent power that public authority figures have to drive sudden changes in health care practices."
Clinicians Caught in the Middle
For many physicians, the leucovorin wave has created an uncomfortable and time-consuming dilemma. Dr. Shafali Jeste, an autism researcher at UCLA, described the situation as an overwhelming burden on clinical staff.
"All of a sudden, there's this onslaught of information being disseminated that we now have to explain, disentangle and interpret," she said. "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."
On a single Monday following the initial announcement, Jeste saw nine patients — and all but one family raised questions about leucovorin. She predicted that enthusiasm for the drug would take a "really long time" to subside, even following the FDA's more limited revised guidance.
Dr. Frederick Bassal, a child neurologist at UC Davis, noted that many parents left the original press conference believing leucovorin was already a proven autism treatment. Others came away assuming that a large share of autistic children have cerebral folate deficiency — a claim rooted in a small, underpowered study that experts say cannot support such a broad conclusion.
"The study was too small to conclude that a correlation exists," said Dr. Alice Kuo, a UCLA pediatrician.
Major Medical Groups Advise Against Routine Use
The American Academy of Pediatrics and the Coalition of Autism Scientists have both stated clearly that they do not recommend leucovorin as a routine autism treatment, citing a lack of sufficient evidence. Most major health systems have issued similar internal guidance. Kaiser Permanente and Oregon Health and Science University have explicitly stated that their providers will not prescribe the drug until more comprehensive safety and efficacy research is completed.
Dr. Sarah Mohiuddin, who directs the Multidisciplinary Autism Program at Michigan Medicine, acknowledged the difficult position many clinicians find themselves in. Leucovorin is generally considered a safe medication, and brief use likely poses minimal harm. However, the drug's safety profile has not been formally established in children with autism, and no long-term data exists for use beyond six months in this patient population.
Parents Persist Despite Limited Guidance
For families like Balantrapu's, the FDA's revised and narrowed approval has done little to quiet the search. When her son's pediatrician in Palo Alto declined to prescribe leucovorin, she turned to Facebook — as did tens of thousands of other parents.
Joyce called the FDA's updated guidance "maddening," saying it would make obtaining a prescription "even more difficult." For parents who fought publicly to promote the drug's potential, the reversal stings.
"It actually makes me angry," Joyce said. "I put him out there and all this to promote the effect. Now they are basically saying the opposite."
Whether the revised federal position will meaningfully reduce demand remains to be seen. For now, many families continue navigating treatment largely on their own — guided more by online communities than by clinical consensus.
