FDA declines to approve drug touted by Trump as a treatment for autism

"The U.S. Food and Drug Administration on Tuesday approved leucovorin, a synthetic form of vitamin B9, as a treatment for a rare genetic condition that causes folate deficiencies in the brain. The decision comes just months after U.S. president Donald Trump, Secretary of Health and Human Services Robert F. Kennedy, Jr., and FDA chief Marty Makary lauded the drug as a treatment for autism."

"Autism is not caused by a folate deficiency, says David Mandell, a professor of psychiatry at the University of Pennsylvania, who studies autism. The data suggesting so are outdated and weak. Although it can sometimes be tied to specific genetic factors, many researchers believe autism has no single cause."

"Now the agency has approved its use for cerebral folate deficiency—a rare genetic condition that may affect fewer than one in a million people, though its true prevalence is unknown. It is caused by a mutation on the FOLR1 gene and can produce some similar symptoms to autism, such as communication issues. But autism is a separate condition, and a broad diagnosis."

The FDA approved leucovorin, a synthetic form of vitamin B9, as a treatment for cerebral folate deficiency, a rare genetic condition affecting fewer than one in a million people caused by mutations in the FOLR1 gene. This approval contradicts earlier statements by Trump administration officials who promoted the drug as an autism treatment. Following scientific review, the FDA determined insufficient data supported leucovorin's use for autism. Autism is not caused by folate deficiency, and researchers emphasize autism has multiple potential causes rather than a single etiology. Cerebral folate deficiency can produce symptoms superficially similar to autism, such as communication difficulties, but represents a distinct condition. Leucovorin is typically used to manage chemotherapy side effects in cancer patients.

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