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Published : 31 Oct 2025, 09:43 PM
The American Academy of Pediatrics on Friday said it does not recommend the routine use of leucovorin - a form of vitamin B9 - in children with autism, citing a lack of evidence on the treatment's benefits and risks to support widespread use in this population.
The announcement follows a flood of demand from parents eager to try the treatment after US health officials touted the decades-old drug in a White House press conference on Sept 22 that focused on autism causes and treatments.
The generic drug, originally developed by GSK, is a form of folinic acid currently used in people undergoing chemotherapy but can be prescribed off-label for other uses.
The FDA said it has started the process of approving the drug for people with a rare genetic condition called cerebral folate deficiency that can cause autism-like symptoms, based on an analysis of published data on some 40 cases showing a benefit.
The agency also mentioned the potential use of the treatment in individuals with autism, but cautioned that available data for this population was limited and said additional studies were needed to assess its safety and efficacy.
Dr Kristin Sohl, an autism expert at the University of Missouri who worked on the guidance, said that while the FDA intends to approve leucovorin for a rare neurological condition, there is "very limited evidence" for its use in autism, leading the AAP to advise against a broad recommendation for its use.
Sohl said the AAP's goal is to help pediatricians speak with parents about the evidence behind the treatment and urged shared decision-making, but acknowledged that there are many unknowns.
"We don't know who this medicine is supposed to work for. We don't know whether it's safe. We don't know what dose to start with," she said.
"If this is something that can be supportive of an autistic child's developmental progress, then great, but we also don't want to just throw things at someone because we don't know what else to do," she said.
If a practitioner decides to prescribe leucovorin for autism, the group recommends that clinicians prioritise reducing the risk of harm and closely monitoring for harmful side effects.
Sohl said she has faced many requests for the treatment in her own practice since the announcement, and so far has only prescribed it once, and only in the context of a controlled study agreed upon by the parent and the patient.
