Article Summary:
The Food and Drug Administration (FDA) has announced that it will be modifying the label of the drug leucovorin, or folinic acid, to make it available for children with autism. Leucovorin is a modified version of vitamin B9, or folate, which is essential for cell growth and development.
Studies suggest that some individuals with autism may have antibodies that interfere with how folate is transported in the body, leading to a condition called cerebral folate deficiency. This can result in symptoms like speech difficulties, intellectual disabilities, and seizures, typically appearing around age 2.
The FDA’s decision is based on a review of studies from 2009 to 2024, which found that leucovorin can “improve certain symptoms” of cerebral folate deficiency. One study compared 40 people on the medication to 40 on a placebo, with those taking leucovorin showing “substantial improvement” in their symptoms.
While not a cure, some children with autism who have received folinic acid have seen significant improvements in their receptive and expressive language skills, particularly those with the folate transport-blocking antibodies. However, the drug does not work for all children with autism.
Experts caution that larger, placebo-controlled studies are needed to determine who would benefit from leucovorin and at what dosage. They also warn against viewing the drug as a potential cure or preventative measure for autism, as the data is still limited.
Leucovorin is currently approved by the FDA for treating the side effects of certain chemotherapy drugs and megaloblastic anemia. Its side effects are generally minimal, as it is derived from a water-soluble vitamin.