The Food and Drug Administration recently approved a drug to treat a rare brain disorder that resembles autism. The medication, leucovorin, is now available for children and adults with cerebral folate deficiency, a condition that limits the delivery of folate, a type of vitamin B, to the brain. This approval was granted to GSK, the original manufacturer of Wellcovorin, a branded version of leucovorin that was discontinued in 1997. However, GSK has stated that they do not intend to resume manufacturing or marketing Wellcovorin.
Cerebral folate deficiency affects approximately one in a million Americans and is a rare genetic condition. While the approval of leucovorin is a positive development for individuals with this condition, it also marks a shift from previous statements made by top health officials. In a press conference in September, President Trump and others suggested that leucovorin could benefit “hundreds of thousands” of children with autism. However, the FDA has since narrowed its review of the medication to focus on uses supported by more robust evidence.
It’s important to note that cerebral folate deficiency is not autism, but both conditions share similarities in terms of developmental delays. Folinic acid, a dietary supplement, has shown promise in improving symptoms in individuals with folate deficiency. Some parents of autistic children have turned to leucovorin, which contains the same ingredient, in an attempt to improve their children’s communication abilities. While initial studies showed promising results, the largest study demonstrating its efficacy was retracted in January.
Despite the scientific evidence casting doubt on the effectiveness of leucovorin for autism, there has been a surge in demand for the drug. Outpatient prescriptions of leucovorin increased by 71% in the weeks following the press conference where its potential benefits were discussed. This trend highlights the complex landscape of treatments for developmental disorders and the importance of evidence-based medicine.
STAT’s coverage of disability issues is made possible by grants from the Robert Wood Johnson Foundation and The Commonwealth Fund. The publication’s financial supporters do not influence its journalism decisions.
