The US Food and Drug Administration (FDA) has quietly approved leucovorin only for cerebral folate deficiency, walking back earlier claims by President Donald Trump and Health Secretary Robert F Kennedy Jr that the drug could treat autism. The move comes after a surge in outpatient prescriptions for children aged five to 17, which rose by 71% following a September press conference touting the vitamin B derivative as an autism treatment.
At the September event, FDA Commissioner Marty Makary described folinic acid as the first FDA-recognised treatment for autism, claiming it could help 50-60% of autistic children. Trump said the drug gave hope to parents, while Kennedy called it an exciting therapy. However, neurodevelopmental specialists expressed shock at the lack of evidence, with Professor William Graf of Connecticut Children's Medical Center noting the announcement caused a 'ruckus' among doctors.
The FDA's 10 March approval limits leucovorin to cerebral folate deficiency, a rare condition that can mimic autism. Makary's statement now refers to treating 'developmental delays with autistic features' rather than autism itself. Graf said the move appears to be a backtrack, adding that he has seen no patients respond to the drug in his clinic.
The largest study on leucovorin for autism, involving just 77 children, was retracted in January after re-analyses failed to replicate its results. The American Academy of Pediatrics does not recommend the drug for autism due to limited evidence. Even Dr Richard Frye, who suggested the drug to officials, was surprised by the lack of research before the announcement.
Critics argue the government promoted an unproven treatment while cutting funding for autism research and services. Leon Epstein, chief of neurology at Lurie Children's Hospital, stressed that physicians must rely on science-based therapies, not declarations.
