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Trump team backs an unproven drug for autism — but does it work?

Leucovorin would be available to only a minority of autistic people, and has not been tested for safety or effectiveness in a large trial. Some researchers are worried.
By
  1. Heidi Ledford

According to Martin Makary, head of the US Food and Drug Administration (FDA), the drug leucovorin will help “hundreds of thousands of kids” with autism. But a day after Makary praised leucovorin’s powers at a White House event, some specialists are warning that the science to warrant Makary’s enthusiasm is far from solid.

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doi: https://doi.org/10.1038/d41586-025-03103-7

With additional reporting by Helen Pearson

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