Trump and RFK Jr touted leucovorin as a treatment for autism. The FDA quietly walked it back

Outpatient prescriptions for the drug increased 71% after news, but FDA later approved it only for folate deficiency

When Donald Trump and Robert F Kennedy Jr stood up at the press conference in September to tout leucovorin, a vitamin B derivative, as a treatment for autism, some neurodevelopmental doctors were shocked - and they braced themselves. There was little evidence to suggest the folinic acid helps with autism, yet there was an immediate flood of parents calling and scheduling visits to talk about the medication.

"The average parent who maybe wasn't getting the right information said, 'Well, to be good parents, we need to try this,'" said William Graf, a professor of pediatrics and neurology at Connecticut Children's Medical Center. Outpatient leucovorin prescriptions for children ages five to 17 in the US rose by 71% in the weeks following the announcement, new research shows.

But on 10 March, the FDA approved leucovorin only for cerebral folate deficiency, in an apparent walk-back from officials' statements about autism. The treatment seems to help treat "developmental delays with autistic features", Marty Makary, commissioner of the US Food and Drug Administration (FDA), said in a statement - a reference to the ways cerebral folate deficiency, a very rare condition, can appear similar to autism.

Only months ago at the September press conference, Makary was much more explicit about folinic acid as the first FDA-recognized treatment for autism.

"Hundreds of thousands of kids, in my opinion, will benefit," he said, stating that being autistic "may be entirely preventable". The drug "may help 50 to 60% of kids with autism", Makary claimed on C-SPAN. He also said on Becker's Healthcare Podcast that "in the right population of children with autism, two-thirds of kids can see a clinical improvement and some a dramatic improvement in their autism symptoms".

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Graf noted that "predictably," this rhetoric "caused a ruckus".