The Trump administration on Monday, Sept. 22, announced that the Food and Drug Administration will approve leucovorin, a folinic acid supplement, as a treatment for a subset of children with autism. The announcement was made during a press conference featuring President Donald Trump and Health and Human Services Secretary Robert F. Kennedy Jr., who described the drug as an “exciting therapy that may benefit large numbers of children who suffer from autism.”
Leucovorin, a form of vitamin B9 first used in the 1950s to counteract chemotherapy side effects, will be prescribed specifically for children diagnosed with “cerebral folate deficiency,” a condition in which the body cannot properly use folic acid in the brain. According to an FDA notice, the newly approved tablet form is intended to help with certain symptoms associated with this subset of autism cases.
“Some children with autism have genetic variations that prevent them from fully utilizing folic acid or vitamin B9,” explained Dr. Karam Radwan, Director of the UChicago Medicine Neurodevelopmental Clinic. “This leucovorin, or folinic acid, bypasses some of the steps the body normally takes to convert folic acid into a form that can cross the brain barrier.” Early studies suggest it may improve communication in affected children, though the evidence remains limited. “The benefits were often modest,” Radwan said, noting that most studies involved small sample sizes, with only 40 to 50 patients per trial.
Richard Frye, a pediatric neurologist, told the Washington Post that clinical trials are still in the early stages. “It’s something that we think might be able to help a lot of children,” he said. “We’re still on the 10-yard line.”
Dr. Radwan emphasized that leucovorin is not a cure for autism and is not appropriate for all patients. “You’re not going to see people improving in days,” he said. The medication can also trigger irritability or aggression in some children if not properly matched to their genetic profile. “We have to test for folate receptor alpha autoantibodies and identify the right patients. Giving leucovorin universally to anyone with autism would be the wrong message,” he cautioned.
Currently, testing for folate receptor alpha autoantibodies is not widely approved or covered by insurance, a gap Radwan hopes will be addressed alongside coverage for leucovorin itself.
The announcement has also drawn scrutiny for potential conflicts of interest. Leucovorin is sold by iHerb, a company affiliated with Dr. Mehmet Oz, who holds a government role as head of the Centers for Medicare and Medicaid Services. Critics have voiced concern that the policy could financially benefit government insiders. “This isn’t public health. It’s corruption dressed up as wellness,” tweeted Dr. Simon Maechling, an organic chemist, highlighting the ethical concerns raised by the announcement.
While leucovorin may provide some benefits for children with specific metabolic needs, experts stress that careful evaluation and oversight are essential to ensure the treatment is safe, appropriate, and evidence-based.
