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US pediatrician group recommends against routine use of drug for autism

The American Academy of Pediatrics highlights limited evidence for leucovorin's safety and efficacy in autism despite widespread parental demand and FDA label updates.

  • On Friday, the American Academy of Pediatrics said it does not recommend leucovorin for routine use in autistic children, citing limited evidence while acknowledging small studies suggest potential benefits for some with CFD.
  • Following the FDA and remarks, interest surged as the agency said it had 'initiated the approval' for cerebral folate deficiency, prompting increased parental demand.
  • Social media and limited studies reveal Keith Joyce's Leucovorin for Autism Facebook group grew from 5,000 to 84,000 members despite only three small studies and no large trials.
  • The AAP advised paediatric prescribing clinicians to engage in shared decision-making about leucovorin, noting major knowledge gaps and Dr. Shafali Jeste said, `It puts physicians in a very tough position because they're being asked to prescribe something that is not evidence-based.`
  • A Health and Human Services spokesperson said the FDA's plan relied on over 40 case studies, and the NIH will support further CFD research.
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SRN News broke the news in on Friday, October 31, 2025.
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