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FDA Moves to Authorize Leucovorin for Cerebral Folate Deficiency Linked to an Autism Subgroup

Experts urge targeted testing pending larger trials before any broad use.

**HANDOUT**SINGLE USE** Robert Hendren
The FDA recently began the process of approving leucovorin as a treatment for autism, despite a lack of any large, phase 3 clinical trials. (Thinkstock)
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Overview

  • The FDA has asked GSK to update leucovorin’s label to include treatment of cerebral folate deficiency, a rare condition seen in only a subset of autistic individuals.
  • NIH oversight is ramping up as the agency’s review cites speech gains in small studies, though officials emphasize the evidence is not yet definitive.
  • Autism Science Foundation and the American Psychiatric Association caution that current trials are small and varied, urging more rigorous research before routine prescribing.
  • Clinicians advise confirming folate-receptor autoantibodies or cerebrospinal fluid folate abnormalities, tests that are specialized and not always accessible or covered.
  • Public interest and off-label use are rising after a viral TikTok anecdote and White House promotion, while supply variability pushes many families to compounding pharmacies for pediatric formulations.