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Thursday, March 12, 2026

Leucovorin Walkback

 number oposts discussed Trump's support for discredited notions about autism A September news conference was a firehose of lies.

Ariana Eunjung Cha and Rachel Roubein at WP:

Nearly six months ago, federal health officials gathered at the White House with President Donald Trump and vowed to “go bold” on autism. They sketched out plans for what they described as an “exciting treatment” for children with the condition — a decades-old drug, leucovorin, newly recast as a potential breakthrough.

“Hundreds of thousands of kids, in my opinion, will benefit,” Food and Drug Administration Commissioner Marty Makary said at the time.


But the administration is scaling back that vision. FDA announced Tuesday that it will expand approval for leucovorin, but only for a separate condition that some people with autism also have — not for autism itself.

...
“When we first started considering the ability to expand an indication for leucovorin, we did consider broadly whether there was data to support its use in a broader autism spectrum disorder population,” said a senior agency official.


But a systematic review of the literature led the agency to focus elsewhere. The “strongest data,” officials said, were concentrated among patients with the genetic form of cerebral folate deficiency, a condition they estimate affects “less than one in a million” people.
...


The initial press conference in September where Trump and his aides touted the prospect of expanding the drug’s use triggered immediate pushback. The president separately blamed Tylenol for causing autism — a claim without a proven link — and many in the medical establishment questioned the idea of prescribing leucovorin for a much broader population. Professional societies and individual physicians alike warned that the evidence base was thin.

A decades-old drug typically used alongside chemotherapy, leucovorin has been tested for autism-related symptoms in only a handful of small clinical trials in the United States and abroad. Some studies suggested modest gains in speech and behavior. But the data fell far short of the rigorous, large-scale evidence the FDA generally requires before granting approval for a new use.

The largest of the leucovorin-autism studies was retracted by the European Journal of Pediatrics on Jan. 29; after the authors acknowledged some errors, the editors wrote that the publication “no longer has confidence in the validity of the results and conclusions reported in this article.”