Overview

• The FDA published a Federal Register notice approving a GlaxoSmithKline-made formulation of leucovorin after the company previously withdrew it when production stopped.
• In its review, the agency cited data from 40 patients with cerebral folate deficiency, a rare condition with neurological features that can overlap with autism.
• Leucovorin, also known as folinic acid, is long used to counter methotrexate toxicity, augment some chemotherapies, and treat certain anemias, and it is manufactured by multiple companies.
• Research from SUNY Downstate and others suggests many children with autism may have folate transport or processing abnormalities, and small studies have reported symptom improvements with leucovorin, leading to off-label prescribing.
• The Autism Science Foundation says the evidence is in very early stages, and University of Pennsylvania’s Dr. David Mandell describes current support as very weak, underscoring the need for larger, standardized trials.