AINEWS
The drug, a folic acid form approved for genetic disorders and chemo side effects, saw pediatric prescriptions rise from 5 to 8 per 100,000 after a White House autism briefing.
A White House autism briefing on Sept. 22, 2025 produced no new clinical evidence, no FDA label change, and no new pediatric guideline. Within a week, prescriptions of a drug that the American Academy of Pediatrics says should not be used for autism had begun to climb. By the end of 2025, they were running roughly 60 percent above where they had been the week before the briefing.
The drug is leucovorin, a form of folic acid. The FDA has approved it for specific inborn errors of folate metabolism and as a rescue agent for patients on folate-antagonist chemotherapy. Its use in children with autism is off-label, meaning prescribed for a condition the drug was not approved to treat, and unproven. The AAP does not recommend it, citing a lack of evidence on dosing, efficacy, and safety.
A new analysis published Aug. 12 in the New England Journal of Medicine, reported by Science News, puts a hard number on the post-briefing change. Pediatric leucovorin dispensing rose from about 5 children per 100,000 the week before the Sept. 22 announcement, to roughly 7 per 100,000 the week of the announcement, to about 8 per 100,000 by the end of 2025. Between late 2024 and the end of 2025, more than 150,000 prescriptions were dispensed to over 47,000 children, according to a database that captures more than 90 percent of U.S. retail pharmacy prescriptions.
The pediatric curve was not mirrored in adults. That detail matters: it ties the prescription spike to a specific population, and to a specific federal message about that population, rather than to a broader trend in leucovorin use.
"Our study demonstrates the power of the federal government to influence clinical practice," said Kao-Ping Chua, a pediatrician and health policy researcher at the University of Michigan Medical School and the study's lead author. Chua warned that non-evidence-based federal communications can produce concrete harms, including "false hope" and exposure to side effects of ineffective treatments.
A separate analysis using emergency-department and outpatient-clinic data, published in the Lancet, found a similar post-briefing rise in pediatric leucovorin prescribing. Two independent datasets, two different time windows, the same direction.
What the numbers describe is not a policy change. There was no new guideline, no new approval, and no new evidence on leucovorin's effect on autism symptoms. What changed was a federal signal, delivered from a White House podium, followed by a measurable change in what families asked for and what pediatricians wrote. The AAP's recommendation, on the record before and after the briefing, did not move the same needle.
For pediatricians, the practical question is now concrete: how to respond when a family arrives with a prescription request shaped by a federal announcement, a treatment that has no proven benefit for the diagnosis in question, and a professional society that has said not to use it.