Nikhil Prasad Fact checked by:Thailand Medical News Team Nov 02, 2025 7 hours, 22 minutes ago
Medical News: Medical Experts Urge Caution Amid Growing Debate
The American Academy of Pediatrics (AAP) has officially announced that it does not endorse the routine use of leucovorin—also known as folinic acid—for children diagnosed with autism spectrum disorder (ASD). The statement follows recent moves by U.S. health authorities and the Food and Drug Administration (FDA) to recognize leucovorin as a treatment for cerebral folate deficiency (CFD), a condition that can occur in some children with ASD.
Pediatricians Caution Against Routine Use of Leucovorin for Autism
Calls for clarity from the pediatric community intensified after the Department of Health and Human Services (HHS) said in September that the FDA’s collaboration with pharmaceutical giant GlaxoSmithKline (GSK) to bring back its discontinued leucovorin product, Wellcovorin, would “authorize treatment for children with ASD.” This announcement suggested that state Medicaid programs could soon cover leucovorin as an autism-related therapy. However, this
Medical News report highlights that the AAP’s latest position has put the brakes on this rapidly forming narrative.
AAP Says Evidence Is Still Too Limited
In its newly issued “interim guidance,” the AAP stated that current evidence is insufficient to support widespread leucovorin prescribing for autism. The organization emphasized that while initial studies show some potential benefits, there is a pressing need for larger, more rigorous clinical trials to determine both the drug’s efficacy and safety for broad ASD use. The AAP confirmed that it does not yet have formal prescribing guidelines for leucovorin in autism-related indications.
The HHS, meanwhile, announced that the National Institutes of Health (NIH) will launch confirmatory trials to further explore leucovorin’s impact on children with autism. This research will build upon smaller studies that have reported encouraging—though preliminary—findings.
Understanding How Leucovorin Works
In certain children with autism, autoantibodies against folate receptor alpha (FRα) can block folate, a crucial B vitamin, from entering the brain. This results in cerebral folate deficiency, which is associated with various neurological and behavioral issues. Leucovorin is believed to bypass this malfunctioning transport mechanism, directly supplying the brain with the needed folate. The FDA cited a review of scientific literature from 2009 to 2024, including mechanistic and patient-level data, in support of its renewed focus on Wellcovorin.
One notable study, a double-blind and placebo-controlled clinical trial involving 80 participants in India, reported improvements in the Childhood Autism Rating Scale (CARS) and Child Behavior Checklist scores for children treated with leucovorin compared to those on placebo. The most significant improvements were observed in children with elevated folate receptor autoantibody levels. However, given that approximately one in every 3
1 children in the U.S. was diagnosed with autism by age eight in 2022, the AAP noted that such small-scale studies are far from definitive.
Mixed Reactions and Growing Controversy
The AAP acknowledged that early results appear promising for select cases but stressed that guidance must balance scientific integrity with respect for neurodiversity. The organization also criticized federal officials for what it called “premature promotion” of leucovorin as a generalized autism treatment.
Controversy surrounding autism-related treatments escalated after public comments from President Donald Trump and Health Secretary Robert F. Kennedy Jr., who made speculative remarks linking Tylenol use during pregnancy to increased autism risk. Kennedy later clarified that the data was “suggestive but not conclusive,” while Tylenol’s manufacturer, Kenvue, strongly rejected the claims and opposed any labeling changes without stronger evidence.
Adding to the tension, Texas Attorney General Ken Paxton has filed a lawsuit against Kenvue, accusing the company of misleading pregnant women about potential risks associated with Tylenol exposure, including autism and developmental disorders.
A Call for Patience and Scientific Rigor
While families of children with autism are eager for effective treatment options, medical experts stress that evidence must guide any decision involving new or repurposed therapies. Leucovorin shows early promise in addressing specific metabolic forms of autism but remains far from a proven universal therapy. The AAP’s stance serves as a reminder that scientific validation and ethical responsibility must take precedence over political pressure or public demand.
References:
https://www.aap.org/en/patient-care/autism/use-of-leucovorin-in-autistic-pediatric-patients/
https://www.hhs.gov/press-room/hhs-trump-kennedy-autism-initiatives-leucovorin-tylenol-research-2025.html
https://pubmed.ncbi.nlm.nih.gov/39243316/
https://www.cdc.gov/mmwr/volumes/74/ss/ss7402a1.htm?s_cid=ss7402a1_w
https://www.reuters.com/business/healthcare-pharmaceuticals/us-health-chief-says-not-enough-data-show-tylenol-causes-autism-still-advises-2025-10-29/
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