Trump's Touting of an Unproven Autism Drug Surprised Many
The Trump administration backed updating leucovorin's label to treat autism based on small studies showing improved language in some children, despite expert caution.
- President Donald Trump's administration announced on Monday it would repurpose the generic drug leucovorin as a treatment for autism.
- The announcement followed earlier studies, including one in 2018 by Richard Frye involving 48 children showing language improvements with leucovorin.
- Many specialists caution that these studies are small, use varied methods, and lack moderate evidence, urging careful, controlled trials before approval.
- Critics highlight that initial small studies with positive results frequently fail to be confirmed in larger trials, with psychiatrist David Mandell emphasizing the lack of sufficient evidence to support leucovorin as an effective treatment for autism symptoms.
- The decision has sparked investor interest and online discussion but also raised concerns about a haphazard rollout and the need for further research on folate-related autism factors.
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Can Leucovorin Treat Autism? What the Research Shows
After President Donald Trump on Sept. 22 endorsed the use of the cancer drug leucovorin as an autism treatment, the Food and Drug Administration said that it will approve its use for cerebral folate deficiency, which is seen in some people with autism.
Trump's Touting of an Unproven Autism Drug Surprised Many, Including the Doctor Who Proposed It
WASHINGTON (AP) — When President Donald Trump’s administration announced it would repurpose an old, generic drug as a new treatment for autism, it came as a surprise to many experts — including the physician who suggested the idea to the nation’s top health officials. Dr. Richard Frye told The Associated Press that he’d been talking […]
Trump’s touting of an unproven autism drug surprised many, including the doctor who proposed it - The Boston Globe
Many researchers agree the drug warrants additional study, particularly for patients with a deficiency of folate, or vitamin B9, in the brain that may play a role in autism.

Touting of unproven autism drug surprised many, including the doctor who proposed it
The nation’s leading autism groups and researchers quickly distanced themselves from the decision on leucovorin, a derivative of vitamin B, calling the studies supporting its use “very weak" and ”very small."
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