FDA Approves Leucovorin for Autism Treatment Amid Expert Concerns

The U.S. Food and Drug Administration (FDA) has approved leucovorin, a derivative of vitamin B, for treating autism spectrum disorder (ASD). This decision has surprised many experts, including Dr. Richard Frye, an Arizona-based child neurologist, who had been discussing developing a customized version of the drug for children with autism, assuming more research would be required. Dr. Frye expressed his surprise at the approval without further studies.

Leading autism groups and researchers have distanced themselves from the decision, citing the supporting studies as "very weak" and "very small." Dr. David Mandell, a psychiatrist at the University of Pennsylvania, stated, "We have nothing resembling even moderate evidence that leucovorin is an effective treatment for autism symptoms."

Despite the limited evidence, some doctors are prescribing leucovorin, repurposing versions used for chemotherapy or ordering new formulations from compounding pharmacies. Researchers agree that the drug warrants additional study, particularly for patients with a deficiency of folate in the brain that may play a role in autism. However, they emphasize that it should only be taken in carefully controlled clinical trials.

The FDA's approval has raised concerns about the hasty rollout of the announcement, which critics say has elevated an unproven drug that needs far more study before being approved as a credible treatment for the complex brain disorder.