Expert Reaction
These comments have been collated by the Science Media Centre to provide a variety of expert perspectives on this issue. Feel free to use these quotes in your stories. Views expressed are the personal opinions of the experts named. They do not represent the views of the SMC or any other organisation unless specifically stated.
Professor Andrew Whitehouse is the Angela Wright Bennett Professor of Autism Research at The Kids Research Institute Australia
"Leucovorin (folinic acid) has been promoted as a 'treatment' for autism, but the evidence is weak. A few small trials suggest possible small improvements in behavioural outcomes, yet these studies are limited by small numbers, inconsistent findings, and a lack of independent replication.
Right now, the science does not come close to the standard needed to recommend leucovorin in the clinical management of autism. The field lacks large, multisite, rigorously controlled trials that could confirm whether benefits are real, reproducible, and meaningful in everyday life.
In short, leucovorin remains experimental. Current research is too narrow and preliminary to draw strong conclusions, and the enthusiasm far outstrips the scientific evidence. Families and clinicians should be cautious, and also know that there are already many support programs with strong scientific backing that reliably support development in autistic children. Compared to these proven approaches, leucovorin is still very much unproven."
Professor Adam Guastella is the Michael Crouch Chair in Child and Youth Mental Health at Children’s Hospital Westmead Clinical School and the University of Sydney
"The evidence about the use of leucovorin as a treatment for autism, either provided in pregnancy or to children with autism, is preliminary and requires stronger scientific studies to ensure safety and effectiveness. There are some promising studies suggesting possible benefits, but these studies have many limitations that suggest that further research is needed. They are not at the quality that is needed for widespread use.
There are also some studies suggesting negative side effects. More work is needed to be certain about the correct dose and timing, particularly when given in pregnancy and early childhood, before being taken up as a treatment for the community.
At a time when scientific research has been severely underfunded and challenged internationally, this work further highlights the importance and need for high quality scientific studies that can provide data to governments, policy makers, health practitioners and families about evidence-based practice."
Professor Dawn Adams is Endowed Chair in Autism Research in the Olga Tennison Autism Research Centre at La Trobe University
"The suggestion of using leucovorin to 'treat' autism is worrying. Firstly, because the research in this area is poor quality and not at the level that we would expect when making recommendations for approvals. It is also worrying because such statements impact how people understand and perceive autism.
Autism is not something that needs treating or to be cured, it’s a neurodevelopmental difference. When we frame it as a condition that needs 'fixing', we risk adding to the stigma autistic people already face.
What really makes a difference for autistic people is the right supports: inclusive classrooms, workplaces that adapt to needs, communities that understand communication and sensory differences, and policies shaped by good-quality research. These are the things that help autistic people thrive and the things that governments have the opportunity to create.
Autistic people don’t need treatments or cures. They need a world that accepts and values them for who they are."
Associate Professor Alex Polyakov is a Clinical Associate Professor at the Faculty of Medicine, Dentistry and Health Sciences, University of Melbourne and is a Medical Director of Genea Fertility Melbourne
"President Trump’s announcement that leucovorin (folinic acid) will be made available in the United States for the treatment of autism is an important development, but it needs to be put into perspective. Leucovorin is an active form of folate that has shown promise in some children with autism, particularly those with evidence of cerebral folate deficiency or antibodies that block folate transport to the brain. Early studies suggest that it may improve communication and social interaction in a subset of patients.
However, it is important to stress that this is still an emerging area of research. Larger, well-designed clinical trials are needed to confirm how effective leucovorin really is, which children are most likely to benefit, and what the long-term outcomes might be. Autism is a complex condition, with most cases strongly influenced by genetic factors and a range of environmental influences. This means that while leucovorin may help with certain symptoms in some individuals, it is unlikely to be a broadly effective treatment for all.
The availability of leucovorin for autism is therefore a step forward in expanding potential options, but families and clinicians should remain cautious and recognise that it represents a promising but still limited therapy, best suited to specific patients within the broader autism spectrum."
Professor Andrew Cashin is a researcher in Autism and Intellectual Disability at Southern Cross University
"On the basis of limited evidence in the US, Trump and Kennedy announced a plan to combat what they describe as an autism epidemic. Autism is framed as part of an explosion in chronic childhood disease. While it is clear the evidence related to the role played by Tylenol and Folate in the causation of autism is tentative at best, perhaps the more significant impact internationally is the shift in the narrative related to autism.
There is a marked difference from the generally held view of autism as a neurotype that forms part of human diversity to one of chronic disease and/or disability. For those with Autism Spectrum Disorder the importance of a mix of not only individual supports, but also reasonable adjustments, to promote wellbeing and functioning, is changed to a discussion of autism as a disease and disability that needs treatment.
When this happens, the lifelong nature of autism is lost, as autism is framed as part of an epidemic of chronic childhood illness. Further, the vision of acceptance of autism as part of neurodiversity is flipped to a vision of autism of an epidemic – and that once causation is established treatment and prevention can follow."