FDA Backtracks on Autism Drug, Confusing Treasure Coast Families

Families across Martin, St. Lucie, and Indian River counties caring for children with autism are grappling with conflicting guidance after federal health officials touted a drug called leucovorin last fall — a derivative of vitamin B9, also known as folinic acid — as a potential autism treatment, only to sharply limit that claim weeks ago.

The U.S. Food and Drug Administration last week walked back its earlier, broader statements about the drug, expanding formal approval only for patients with FOLR1-related cerebral folate transport deficiency, an extremely rare condition with fewer than 50 confirmed cases identified worldwide. That reversal came after prescriptions for leucovorin had already surged 71% in the final quarter of 2025, according to a paper published this month. The authors attributed the rise to the influence of the original federal announcement.

The whipsaw has placed local pediatricians and child neurologists in a difficult position. Major medical organizations, including the American Academy of Pediatrics and the Coalition of Autism Scientists, have stated they do not recommend the drug for autism broadly, citing insufficient evidence. Families seeking prescriptions have been turning to social media groups — some with tens of thousands of members — to share doctors' names and tips for obtaining the medication.

Experts nationally have raised specific safety concerns parents should weigh. The drug's safety profile has not been formally established for children with autism, and no data exists on long-term use beyond six months in that population. A dosing protocol commonly followed dates to 2013 and has been replicated by only a handful of studies. It can call for doses up to 100 times the minimum recommended daily intake of folate, a related compound. Additionally, one of only two double-blinded, placebo-controlled trials testing the drug in autistic children was retracted by the European Journal of Pediatrics on Jan. 29 due to errors in results and concerns about statistical analyses.

Leucovorin is generally considered safe and has been used for decades in cancer care. Brief use in otherwise healthy children is considered unlikely to cause harm by some clinicians. A senior FDA official said patients should speak directly with their physicians about whether the treatment is appropriate for their individual circumstances.

Families in the Treasure Coast region seeking guidance should contact the St. Lucie County Health Department or speak with their child's pediatrician before pursuing the medication independently.

This article was generated with AI assistance using publicly available information. It was reviewed and approved by a human editor before publication. TC Sentinel uses AI writing tools in accordance with FTC guidelines.