Overview

• The FDA said it would update leucovorin’s label to include use for cerebral folate deficiency, a rare condition sometimes associated with autism-like symptoms.
• The Guardian reported the agency relied on a literature review rather than the customary large randomized trials and did not issue dosage or safety guidance.
• GSK has not yet submitted a label-change application for the brand-name product but says it will seek the cerebral folate deficiency indication.
• Researchers caution that findings to date are preliminary and warn against treating the decision as a therapy for autism broadly, noting uncertainty over how many autistic people have cerebral folate deficiency.
• Families have long tried leucovorin off label with mixed results, and a 2021 review found mostly mild side effects with occasional aggression, agitation, headaches, insomnia and increased tantrums.