Prescriptions rose 24-fold after a viral TV segment and presidential endorsement
STUDY: Rothman JM et al, JAMA Network Open. 2026;9(5)
STUDY TYPE: Cohort study
FUNDING: Independent
Background
On September 22, 2025, President Trump and HHS Secretary Robert F. Kennedy Jr. publicly endorsed leucovorin as a treatment for speech deficits in children with autism spectrum disorder. Leucovorin (folinic acid) is a biologically active form of folic acid, FDA-approved for use in cancer chemotherapy and, as of March 2026, for cerebral folate transport deficiency due to confirmed FOLR1 gene variants.
Small trials suggest it may improve verbal communication in folate-deficient children with autism, but one of those trials was retracted and large-scale data is missing, leading the FDA to pass on it in 2026.
The Study
- 838,801 US children under 18 with a confirmed autism, drawn from the Epic Cosmos EHR.
- Monthly leucovorin prescription rates per 100,000 outpatient encounters from Jan 2023 to Jan 2026.
- No comparator group; this is a prescription-trend analysis.
Leucovorin prescriptions held steady for two years, averaging 34.1 per 100,000 encounters per month from January 2023 through January 2025. Then two events shifted the curve. Rates climbed after:
- Feb 2025: A CBS Evening News segment in (Feb 2025) featuring a family whose nonverbal child reportedly spoke his first word within days of starting leucovorin, driving rates to 335 per 100,000 encounters.
- Sept 2025: The White House announcement, driving rates to 835 per 100,000 encounters.
Prescriptions plateaued but stayed elevated through January 2026. Most prescriptions were in children aged 5 to 11, and 77% were for boys.
Practice Implications
- The study tells us how publicity drives prescriptions
- The science is not perfect, but leucovorin (folinic acid) is a low-risk intervention in a population with limited treatment options.
- Although it is generic, some insurers are denying it and it is expensive.
— Chris Aiken, MD
Director, Psych Partners
Editor in Chief, Carlat Psychiatry Report
