Key Highlights
- FDA approves leucovorin for cerebral folate deficiency, not autism symptoms.
- Trump administration touted the drug as a potential treatment for autism, but data is insufficient.
- Leucovorin mainly used to mitigate chemotherapy side effects or enhance cancer treatments.
- Patient prescriptions for leucovorin surged 71% following Trump’s announcement.
The FDA’s Decision on Leucovorin and Autism
Federal officials are celebrating a new approval from the Food and Drug Administration (FDA) concerning leucovorin, a synthetic form of vitamin B9. But the celebration is bittersweet for those hoping it might treat autism symptoms.
A Promise Unfulfilled
Remember when President Donald Trump and FDA Commissioner Marty Makary announced they were going to relabel leucovorin “so that it can be available to children with autism”? You might think this is new, but… not quite. The actual change the FDA proposed nearly six months ago did not say the medication would be approved for autism. Rather, it was initiating approval for patients with cerebral folate deficiency—a rare neurological condition.
Expert Doubts
The Autism Science Foundation’s Alycia Halladay weighed in: “The actual change is 1,000% different from the administration’s rhetoric.” Even though leucovorin isn’t approved for autism, doctors can prescribe it off-label. Some were doing so based on findings from a handful of small trials mostly outside the U.S., even before Trump’s announcement in September.
Data Shortfalls
“We don’t have sufficient data to say that we could establish efficacy for autism more broadly,” said a senior FDA official. Leucovorin is primarily used to help mitigate side effects of chemotherapy or enhance its effectiveness for cancer patients. But the promise made by Trump and Makary seems to have sparked a rush: prescriptions rose 71% in the two months following their announcement.
The Backlash
David Mandell, a psychiatry professor at the University of Pennsylvania, called it “terrible for families” with autistic children. This back-and-forth about what treats autism and what doesn’t is just not good enough. Families deserve more careful science and accurate information.
So there you have it: leucovorin gets the thumbs down for autism but a green light for cerebral folate deficiency, at least until the data backs up the claims.
