AAP Declines to Recommend Leucovorin for Routine Autism Treatment Amid Ongoing Controversy

The American Academy of Pediatrics (AAP) has issued interim guidance stating that it does not recommend the routine use of leucovorin for children with autism, citing limited evidence. This announcement comes amid a complex and contentious debate surrounding autism treatments and potential links to common medications.

AAP's Stance on Leucovorin

The AAP's decision, released on Friday, emphasizes that "the current evidence base remains too limited to support" recommendations for leucovorin as a treatment for autism spectrum disorder (ASD). The organization calls for large clinical trials to determine the drug's efficacy and safety profile for the broader autism population.

This guidance follows a September announcement by the U.S. Department of Health and Human Services (HHS) that positioned leucovorin, a decades-old drug, as a treatment for cerebral folate deficiency (CFD), which is present in some children with ASD. The FDA is working with GSK to allow the company's previously discontinued leucovorin product, Wellcovorin, to treat CFD.

While the AAP acknowledges that small studies have shown "potential benefit in carefully selected cases," it maintains that these results, though promising, require further investigation. The organization stresses that any future guidance must be "grounded in both scientific rigor and respect for neurodiversity."

Controversial Claims and Political Tensions

The leucovorin debate is set against a backdrop of controversial claims about autism causes and treatments. The AAP has criticized the Trump administration for linking autism to childhood vaccines and the use of acetaminophen during pregnancy without sufficient evidence.

Following a September 22 White House press conference on autism, AAP President Susan Kressly, M.D., stated that the event was "filled with dangerous claims and misleading information that sends a confusing message to parents and expecting parents and does a disservice to autistic individuals."

During the press conference, President Donald Trump repeatedly advised against taking Tylenol, referring to the Kenvue (formerly Johnson & Johnson) pain reliever. However, U.S. Health Secretary Robert R. Kennedy Jr. later clarified that there is insufficient evidence to definitively link Tylenol to autism, though he suggested a cautious approach.

Legal and Regulatory Implications

The controversy has spilled into the legal arena, with Texas Attorney General Ken Paxton filing a lawsuit against Kenvue. The suit alleges that the company "deceptively" marketed Tylenol to pregnant women despite knowing about a potential increased risk of autism and other disorders.

Kenvue has pushed back against these claims, urging the FDA not to change the label of Tylenol over the unsubstantiated link between its use during pregnancy and autism.

As the debate continues, the National Institutes of Health plans to launch confirmatory trials and new research into the effects of leucovorin, potentially providing more definitive answers in the future.