FDA Approves Drug Trump Touted for Autism to Treat Other Neurological Disorder

By Nicholas G. Miller

The Food and Drug Administration approved leucovorin, a drug the Trump administration has touted as a potential therapy for autism, to treat cerebral folate transport deficiency, a neurological disorder with some symptoms similar to autism.

FDA Commissioner Marty Makary said the approval could help those with cerebral folate transport deficiency "who have developmental delays with autistic features."

The FDA granted the approval based on a review of already published literature, rather than a clinical trial.

Last year, the Trump administration said that acetaminophen, the main ingredient in Tylenol, is a potential cause of autism and recommended pregnant women stop taking the over-the-counter-pain medication. Meanwhile, it advocated for leucovorin as a potential therapy to ease autism symptoms.

Leucovorin is a form of vitamin B9 referred to as folinic acid. It is given to some cancer patients receiving treatments that kill cancer cells by blocking folate pathways. Leucovorin helps the healthy cells recover and reduces side effects.

Doctors and scientists have said that leucovorin has shown promise in some small studies in improving verbal and social skills of children with autism. But they also have warned that its benefits might be overstated and more research is needed, The Wall Street Journal previously reported.

Meanwhile professional societies representing obstetricians continue to say pregnant women should use acetaminophen to treat pain and fever. Some previous studies have indicated risks of acetaminophen to fetal development, but others have found no link, The Journal has reported.

Autism is a neurodevelopment disorder that affects roughly one in 31 eight-year-olds in the U.S. The Trump administration has highlighted the rising number of autism diagnoses, but scientists say much of the growth is due to better screening and broader diagnostic criteria.

Write to Nicholas G. Miller at nicholas.miller@wsj.com.

(END) Dow Jones Newswires

March 10, 2026 10:41 ET (14:41 GMT)