During a White House press conference on autism, Food and Drug Administration Commissioner Marty Makary shared that the agency would be filing a federal register notice to label the drug leucovorin as treatment for autism.

“Hundreds of thousands of kids, in my opinion, will benefit,” he claimed during the Sept. 22, 2025 event. 

Shortly after Makary’s announcement, prominent physician groups, including the American Academy of Pediatrics and the American Psychiatric Association, released statements debunking his dubious claims about the drug as treatment for autism.

“It will require many more years of research before we know if leucovorin is an appropriate treatment for individuals with autism,” the American Psychiatric Association’s same-day statement read. “Autism spectrum disorders exist on a spectrum of neurodiversity. The country must focus its resources on expanding access to care and building the evidence-base for future treatments.” 

Rewire News Group asked three autism experts what parents need to know about the risks and benefits of leucovorin. 

Leucovorin is folinic acid, an active form of folate (also known as B9) that doesn’t need to be broken down by enzymes to be used in the body. It is available in pill or IV form. While most individuals can obtain the folate their bodies need through beans and leafy greens, or via vitamin supplements, some cannot properly metabolize folate and need prescription medication to maintain proper cell functioning.

Autism or Autism Spectrum Disorder (ASD) is a neurological and developmental disorder that is characterized by differences in social communication; restrictive, repetitive behaviors that may manifest in motor movements, speech, or interests and challenges with transitioning from one activity or setting to another; and over- or under- reactivity to sensory input.

These characteristics occur on a spectrum, meaning that each autistic individual has different areas in which they may need support, which may include behavioral, occupational, physical, and speech language therapies, along with accommodations to help manage sensory sensitivities and other needs in daily life.   

David Mandell, a professor and the director of the Penn Center for Mental Health who conducts autism research, told RNG he was shocked by Makary’s announcement. 

“The evidence for leucovorin was the same as for hyperbaric oxygen or mega doses of vitamin D,” Mandell said. “There is really no evidence at all that these are meaningful treatments.”    

As a double board-certified child, adolescent, and adult psychiatrist who treats autism, Ritu Goel was especially concerned about the confusion and doubt this announcement caused for parents of autistic children, some of whom are desperate to find anything that may help their child connect and communicate with them. 

“Every … parent now has, suddenly, this hope that, ‘Oh, I give this tablet to my child, and they will be better,’” Goel said.

The reality is far more complicated.

“The major reason we prescribe leucovorin to people is because they’re in cancer treatment, usually chemotherapy, and the leucovorin helps protect healthy cells,” Mandell said. 

When taken with methotrexate, which........

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