Washington, D.C., Sept. 23, 2025— In a landmark move, the U.S. Food and Drug Administration (F.D.A.) has announced that leucovorin, a drug traditionally prescribed to mitigate the side effects of chemotherapy, will now be relabeled to include autism treatment as a therapeutic use.
Background
Leucovorin, also known as folinic acid, has long been a staple in oncology for reducing the toxic impact of methotrexate. Over the last decade, however, researchers have investigated its effects on neurological development, particularly among children with autism spectrum disorder (ASD) who also present with folate receptor autoantibodies.
Clinical studies have suggested that leucovorin can improve communication, language acquisition, and social interaction in certain subgroups of children with autism. Still, the drug’s application in this field has remained off-label — until now.
Voices from the Community
Announcing the decision, Dr. Karen Mitchell, Director of Pediatric Neurology at the F.D.A., stated:
“The evidence to date shows leucovorin may significantly improve the quality of life for a subset of children with autism. Today’s relabeling ensures physicians can prescribe the drug with clearer guidance and parents can access it with greater confidence.”
Families and advocates have hailed the move as a breakthrough. Melissa Rivera, mother of a 7-year-old on the spectrum, shared:
“We’ve seen incredible progress with leucovorin in my son’s speech therapy. To know the FDA now formally recognizes it is validation for parents like us who fought for this.”
Not all voices are in agreement, however. Dr. Leonard Hughes, a child psychiatrist in Boston, cautioned:
“While the studies are promising, leucovorin is not a cure. It should be viewed as part of a broader therapy plan, not a stand-alone solution. We must avoid giving families false hope.”
What Comes Next
The FDA emphasized that post-market monitoring will continue, and further research is encouraged to refine patient eligibility criteria. Physicians are expected to begin prescribing leucovorin under the new label by early 2026.
This decision could also influence insurance coverage, making treatment more affordable for families. Advocacy groups are now urging state health agencies to accelerate reimbursement approvals.
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