Overview

• President Trump and FDA Commissioner Marty Makary highlighted leucovorin as helpful for autism, but the FDA’s action is limited to treating cerebral folate deficiency (CFD).
• Scientists say evidence for improving autism symptoms is based on small, heterogeneous studies and urge large, controlled trials before broad use.
• GlaxoSmithKline said it has not yet filed a label-change application but plans to seek the CFD indication for Wellcovorin, and officials suggested coverage could expand under Medicaid and CHIP.
• Families and some clinicians have used leucovorin off label with mixed reports of gains in speech, behavior, and sleep, alongside side effects such as agitation or hyperactivity.
• CFD is rare and diagnosed with imperfect tools, including lumbar punctures and unapproved antibody tests, and experts note the FDA announcement lacked dosing guidance for pediatric use.