FDA leucovorin approval sparks autism supplement debate

FDA leucovorin approval sparks autism supplement debate


Tara Bannow covers hospitals, providers, and insurers. You can reach Tara on Signal at tarabannow.70.

Parents may flock to a dietary supplement with the same key ingredient as leucovorin, which the FDA just approved for children with autism. But there are drawbacks. 

The Food and Drug Administration has reapproved GSK’s branded version of leucovorin to treat a neurological condition called cerebral folate deficiency that affects many kids with autism. While leucovorin has been used for decades to ease the side effects of chemotherapy, some doctors also prescribe it off label to patients with autism, even though its effectiveness has only been tested in a handful of small studies.

That’s because research has shown that kids with autism often have an antibody that blocks folate from being transported into the brain, said Richard Frye, chief scientific officer of the Autism Discovery and Treatment Foundation, an autism research nonprofit in Phoenix, Ariz. 

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