"At a press conference in late 2025, federal officials made some big claims about leucovorin, a prescription drug usually reserved for people on cancer chemotherapy. "We're going to change the label to make it available [to children with autism spectrum disorder]," said Dr. Marty Makary, commissioner of the Food and Drug Administration. "Hundreds of thousands of kids, in my opinion, will benefit." The FDA still hasn't made that label change. Since Makary's remarks, though, more than 25,000 people have joined a Facebook group called Leucovorin for Autism. Most members appear to be parents seeking the drug for their autistic children."
"Offit says he realized years ago that leucovorin's popularity was far ahead of the science. "I saw it for what it was, which was yet the next magic medicine to treat autism, in a long line of magic medicines to treat autism that haven't worked," Offit says. Offit has chronicled the rise and fall of many of those products in his books and blog posts. "First it was secretin, an intestinal hormone," he says. "Then it was Lupron, chemical castration, antibiotics, megavitamins, nicotine patches, and my personal favorite, which is raw camel's milk.""
Federal officials publicly claimed plans to change leucovorin's label to allow use for children with autism, but the FDA has not made that change. Since the remarks, over 25,000 people joined a Facebook group called Leucovorin for Autism, mainly parents seeking the drug. Some physicians began prescribing leucovorin off-label for autistic children despite medical-group warnings. Increased demand caused a shortage, prompting the FDA to temporarily permit imports of tablets made in Spain and sold in Canada that are not U.S.-approved. Dr. Paul Offit warns popularity exceeds evidence and likens leucovorin to prior unproven autism remedies.
Read at www.npr.org
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