Leucovorin saga, and extra | The Transmitter: Neuroscience Information and Views
Folate fuss: Internet searches and prescriptions for leucovorin have risen significantly since a White House announcement last September touting it as a treatment for autism, according to new investigations. That publicity led to 1 million additional internet searches for leucovorin in the two weeks following the announcement, one paper detailed, and a 14-fold increase in leucovorin prescriptions for children up to age 10 in the United States in the ensuing months, according to another paper. The White House’s comments were a misleading overstatement of the U.S. Food and Drug Administration’s decision to approve the drug’s use for cerebral folate deficiency, a rare condition that includes autism-like traits. At the time, The Transmitter reported on the evidence used to inform the agency’s decision, autism researchers’ concern about off-label use, and the questionable record of a leucovorin-promoting researcher. Then, in February 2026, a paper that reported benefits of leucovorin in autistic children was retracted because of data inconsistencies.
- “Restoration of axon initial segment plasticity via chemogenetic activation rescues autism-related behaviors” Cell Death & Disease
- “Modeling prenatal immune activation in human brain organoids uncovers IL-6–dependent interneuron dysmaturation” bioRxiv
