The U.S. Food and Drug Administration is considering approval of the drug leucovorin to treat children with cerebral folate deficiency and symptoms of autism.
Kevin Pelphrey is the Harrison-Wood Jefferson Scholars Foundation Professor of Neurology at the University of Virginia School of Medicine. At UVA’s Child Health Research Center, he researches biological markers for autism and develops tools to better detect and treat those on the autism spectrum.
Dr. Beth Ellen Davis is a UVA Health developmental behavioral pediatrician specializing in autism. She works with families affected by the disorder.

Kevin Pelphrey researches tools to better detect and treat people with autism spectrum disorder. (Contributed photo)
Both spoke with UVA Today about leucovorin and what both patients and parents should know about the drug.
Q. What can you tell us about the drug, leucovorin, that has been making headlines?
Pelphrey: Leucovorin is a medication that was originally developed to be helpful in the administration of chemotherapy and all of the terrible side effects. It’s basically folinic acid, a member of the folate family commonly known as vitamin B9, a naturally occurring nutrient that you find in foods. One of the key elements of the medication known as leucovorin is that it gets into cells very easily, including into the brain.
Folate is really important for many aspects of brain function and if you have a deficiency in folate, you can have neurological and psychiatric symptoms, including fatigue, depression, anxiety and, in severe cases, seizures and abnormal brain development.
Neurologists discovered that some babies born with folate deficiency ended up having severe seizures that could be treated with folate replacement if started early. Those babies have folate receptor auto antibodies that can keep folate from getting into the brain, resulting in severe cerebral folate deficiencies.
Q. How has this news resonated with patients with autism, and parents of children with autism? Is this reason to be optimistic, or cautious?
Davis: Because there are so few developmental behavioral pediatricians in Virginia and the USA, we see thousands of children with autism and their families at UVA Health’s Neurodevelopmental and Behavioral Pediatrics from all over the state, every year. Caregivers have been asking us about the use of folinic acid for their child with an autism spectrum disorder for quite a few months. Parents are seeking medical guidance about the risks and benefits of using prescription dose folinic acid to improve autistic symptoms. Currently, there is no medication or supplements to cure autism, nor is it our clinical protocol to prescribe leucovorin for improvement or treatment of autism symptoms at this time.
Q. How does folinic acid, or a deficiency of it, relate to children with autism?
Pelphrey: It was discovered that about half of children with autism have more folate receptor auto antibodies than you see in children not on the autism spectrum, and we don’t yet know why. As a result, scientists thought to try leucovorin, because that drug gets folinic acid and folate into the cells of the brain because it bypasses those antibodies.