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.
Paediatricians and specialists caution the science on leucovorin in autistic people as the data is limited and does not support widespread use.
In the month since Food and Drug Administration Commissioner Marty Makary promoted the decades-old drug from GSK, saying it could help hundreds of thousands of autistic children, doctors and researchers say they have been inundated by parents seeking information.
“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.
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.
Source: The Guardian, 1 November 2025
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