New Approach Aims to Address a Root Cause of Autism in Specific Patient Groups
In a move to expand treatment options for individuals with autism-related symptoms, the U.S. Food and Drug Administration (FDA) announced today it has initiated the process to approve leucovorin calcium tablets for a specific neurological condition: cerebral folate deficiency (CFD).
This action aims to make a targeted treatment available for patients whose symptoms, including autistic features, seizures, and developmental delays, are linked to this rare disorder.
This decision follows a comprehensive FDA review of scientific literature from 2009 to 2024, which concluded that leucovorin calcium can effectively aid individuals with CFD. CFD impairs the brain’s ability to transport folate, a crucial vitamin for brain function. The FDA’s new approach is based on the premise of repurposing an existing drug to address a root cause of certain autism symptoms.
“We have witnessed a tragic four-fold increase in autism over two decades,” said FDA Commissioner Dr. Marty Makary. “Children are suffering and deserve access to potential treatments that have shown promise. We are using gold standard science and common sense to deliver for the American people.”
The FDA is collaborating with GSK, the manufacturer of Wellcovorin (leucovorin calcium), to update the drug’s label to include its new use for adults and children with CFD. Dr. George Tidmarsh, Director of the FDA’s Center for Drug Evaluation and Research, highlighted this as part of the agency’s broader commitment to repurpose existing drugs for chronic diseases.
While the current approval focuses on patients with confirmed CFD, the FDA acknowledged that some individuals with autistic features and related autoantibodies to the folate receptor alpha may also benefit.
However, the agency stressed that more research is needed to determine the drug’s safety and effectiveness in this broader patient group. The FDA’s action reflects a growing focus on personalized medicine and treating the underlying biological causes of complex neurological conditions like autism.
READ: FDA Launches Major Crackdown On Deceptive Drug Advertising
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