FDA adds leucovorin for speech deficit in cerebral folate deficiency

On Monday federal health officials announced that the U.S. Food and Drug Administration has added an indication for leucovorin, a form of folate, to treat speech related deficits in children with cerebral folate deficiency, a condition that can overlap with autism. The decision, unveiled by the Trump administration and promoted by Health Secretary Robert F. Kennedy Jr., came alongside renewed warnings about acetaminophen use during pregnancy, drawing immediate praise from some families and sharp caution from many researchers and advocacy groups.

Leucovorin, also called folinic acid, has been used for decades to reduce the toxic effects of certain chemotherapy drugs and to treat folate related anemias. Federal officials said the agency reviewed research published between 2009 and 2024 and concluded the drug can restore brain folate in children whose transport of folate into the brain is blocked, a problem often linked to folate receptor alpha autoantibodies. The agency and administration noted that reauthorizing a branded form of the drug will allow state Medicaid plans to cover treatment for cerebral folate deficiency and related speech deficits in autism.