A growing number of United State (U.S.) parents of autistic children are urging doctors to prescribe leucovorin, a decades-old drug once used to ease chemotherapy side effects, amid a social media-fuelled belief it can improve speech and social interaction, despite limited scientific backing.
The demand follows public endorsements from former President Donald Trump and Food and Drug Administration (FDA) Commissioner Marty Makary, who recently described leucovorin as potentially beneficial for hundreds of thousands of autistic children.
In the month since Makary’s comments, paediatricians and autism specialists say they have faced a wave of requests from desperate families convinced the treatment could unlock their children’s abilities.
“My Facebook feed is flooded with parents swearing that leucovorin works,” said Dr David Mandell, an autism researcher and psychiatry professor at the University of Pennsylvania.
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A Facebook group, Leucovorin for Autism, created in May by Keith Joyce, guardian to four-year-old Jose, who is taking the drug, has ballooned to over 84,000 members. Joyce claims his child has shown marked improvements in speech and social awareness since starting leucovorin.
But many clinicians warn that enthusiasm has outpaced evidence.
“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, head of paediatrics at UCLA, who has resisted parental requests to use the drug.
Leucovorin, manufactured by GSK, is approved for managing side effects of chemotherapy and certain types of anaemia, but can legally be prescribed off-label for other conditions.
The FDA has proposed expanding its approval to include cerebral folate deficiency (CFD), a rare genetic disorder affecting roughly one in a million children, but not autism itself.
The agency’s proposal was based on an analysis of 40 published case studies, which suggested that 85 per cent of CFD patients showed some improvement in speech or communication.
However, the data remains limited, and no large, placebo-controlled trials in autistic children have been conducted.
“We don’t yet understand who benefits, by how much, or for how long,” said Dr Karam Radwan, director of the Neurodevelopmental Disorders Program at the University of Chicago. “So far, the benefit appears modest.”
Advocates argue that leucovorin may help autistic children with folate receptor autoantibodies, molecules believed to block folate, a vitamin vital for brain signalling, from entering the brain. Some studies claim these antibodies may be present in up to 75% of autistic individuals, though their clinical relevance remains uncertain.
The American Academy of Pediatrics has advised against the routine use of leucovorin in autism, citing insufficient evidence. Nonetheless, parents continue to turn to social media for advice on dosages and sourcing, prompting Facebook to repeatedly remove and reinstate Joyce’s group over misinformation concerns.
The Independent report noted that Joyce admits the science is thin but says the improvements in his son are enough to justify trying. “It’s not curing his autism,” he said, “but it improved his quality of life. I’m convinced it’s real.”
For doctors, the growing demand underscores a familiar dilemma: balancing parental hope against the slow pace of scientific validation.
