New professional guidance to the nation’s pediatricians advises against using a drug in children with autism that many families are clamoring for after it was hyped by federal officials.
The American Academy of Pediatrics said late last week that it does not recommend routine use of leucovorin in kids who are on the spectrum because there is limited evidence to support the practice.
In September, Trump administration officials touted leucovorin as a possible treatment during a press conference about autism. The Food and Drug Administration said it would work to approve the decades-old drug for cerebral folate deficiency, a neurological condition associated with autism features.
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“This action establishes the first FDA-recognized therapeutic for children with cerebral folate deficiency and autistic symptoms,” officials with the U.S. Department of Health and Human Services said at the time. “The change will authorize treatment for children with ASD, with continued use if children show language, social, or adaptive gains.”
Since then, interest in leucovorin among families of children with autism has soared, said David Mandell, a professor of psychiatry and an autism researcher at the University of Pennsylvania.
“So many of my clinician colleagues in pediatrics, psychiatry, psychology and neurology tell me that their patients’ families are asking about it or for it,” Mandell said, adding that Facebook groups devoted to the topic have proliferated. “This is a dramatic shift from eight weeks ago.”
While small studies have suggested “potential benefit” particularly in treating children with cerebral folate deficiency, the evidence is insufficient to support routine use in kids with autism, according to interim guidance from the American Academy of Pediatrics.
It remains unclear who might benefit from leucovorin, what dosing and monitoring would look like and whether the drug is safe in the long-term, the guidance indicates. The pediatrics group said that larger, well-controlled clinical trials are needed to evaluate whether leucovorin is appropriate for children with autism more broadly.
“Medical decisions including emerging therapies like leucovorin must be thoroughly examined before we begin implementing them widely to ensure that children remain safe and healthy. It is important that we take the necessary time to understand the evidence and equip pediatricians with accurate information to navigate these conversations in support of their patients,” said Dr. Kristin Sohl, a member of the American Academy of Pediatrics Council on Children with Disabilities Executive Committee and immediate past chair of its Autism Subcommittee.
Leucovorin is a prescription form of folinic acid that’s generally used to treat side effects from chemotherapy as well as a specific kind of anemia, the pediatrics group said.
Pediatricians and other providers are being encouraged to “engage in shared decision-making” with families asking about leucovorin and to educate them about the existing evidence and possible risks. In cases where providers decide to prescribe leucovorin, “they should prioritize harm mitigation, closely monitor for adverse effects, and support families in navigating complex therapeutic decisions grounded in compassion, transparency, and scientific integrity,” the guidance indicates.
The American Academy of Pediatrics said its guidance will be updated as new data becomes available.
