The history of autism "cures" is a history of dashed hopes and frequent danger.
A drug endorsed by the Donald Trump administration which allegedly treats against a rare disorder that causes autism-like symptoms has triggered a surge in demand from parents, despite a lack of data supporting its use.
More parents in the U.S. are asking for leucovorin, believing it could unlock speech and social connection in their autistic children.
Pediatricians and specialists caution the science on leucovorin in autistic people as the data is limited and does not support widespread use.
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“My Facebook feed is flooded with parents swearing that leucovorin works,” said Dr. David Mandell, a professor of psychiatry and autism researcher at the University of Pennsylvania.
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Mandell and other scientists and doctors say Trump’s endorsement, without requiring large, randomized clinical trials, leaves practitioners facing emotional pleas from families while lacking data, guidance or confidence to prescribe the drug responsibly.
Leucovorin is approved to treat chemotherapy side effects but can be prescribed off-label for autism symptoms.
"It puts physicians in a very tough position because they're being asked to prescribe something that is not evidence-based," said Dr. Shafali Jeste, an autism expert and head of pediatrics at UCLA, who does not prescribe leucovorin despite repeated requests.
From the American Academy of Pediatrics:
Autistic children, adolescents, and young adults are valued members of our communities, and they deserve access to evidence-based care supported by high-quality research. Autism is a complex neurodevelopmental condition with diverse presentations and no single known cause. Current research points to a combination of genetic and environmental influences. For decades, autistic individuals have been subjected to treatments based on unproven theories—some of which caused harm with little to no benefit. As such, any new intervention must be carefully evaluated for both its potential benefits and risks before being widely adopted.
At this time, the American Academy of Pediatrics (AAP) does not recommend the routine use of leucovorin (folinic acid) for autistic children.
Early, small-scale studies have explored its use—particularly among children with documented cerebral folate deficiency—and some findings suggest potential benefit in carefully selected cases. These preliminary results are promising and have laid the groundwork for further investigation.
However, the current evidence base remains too limited to support specific clinical recommendations. Key questions about who may benefit, what dosing and monitoring are appropriate, and what the long-term safety profile looks like have not yet been adequately answered. Larger, well-controlled clinical trials are needed to determine whether leucovorin is a safe and effective option for the broader autistic pediatric population.
The AAP supports research and innovation that improve the quality of life for autistic children and their families. We also recognize the need for any emerging guidance to be grounded in both scientific rigor and respect for neurodiversity.
Pediatricians and prescribing pediatric care providers, including other physicians, physician assistants, and nurse practitioners, are encouraged to engage in shared decision-making with families who inquire about or request leucovorin, providing clear information about current evidence and potential risks. Autistic children, adolescents, and young adults benefit from access to robust supports and services based on their individual needs. Pediatricians and other pediatric care providers should work with families to optimize recommended services and emphasize continuation of well-established supports and therapies that are beneficial to the well-being of the child. The AAP does not have prescribing guidelines for leucovorin for the indication of autism. If a pediatrician or other physician, physician assistant, or nurse practitioner prescribes leucovorin for a pediatric patient, they should prioritize harm mitigation, closely monitor for adverse effects, and support families in navigating complex therapeutic decisions grounded in compassion, transparency, and scientific integrity.
As the evidence base continues to evolve, the AAP remains committed to reviewing new data and updating this guidance accordingly. The AAP supports continued research into all promising therapies that may improve health and developmental outcomes for autistic children, adolescents, and young adults.
