Overview

• The agency’s action is confined to cerebral folate deficiency, though President Trump and senior officials suggested it could help certain autism-related symptoms, prompting criticism from researchers.
• HHS cited an internal review of 23 publications reporting an 85% rate of some clinical benefit in CFD, while experts stressed the small, heterogeneous studies do not justify broad claims about autism.
• FDA officials described approving a new use tied to CFD as GSK said it will submit a labeling request for Wellcovorin, and the company made no reference to autism in its statement.
• Clinicians flagged unresolved basics such as who to test, how to diagnose CFD reliably, and what dose to use, noting current assessments may involve spinal taps or a folate receptor antibody test not cleared for pediatric diagnosis.
• Families and some clinicians have used leucovorin off label for years, reporting outcomes that range from modest gains to notable improvements in speech, sleep, toileting and behavior, while scientists call for larger controlled trials.