Wednesday, 24 September 2025

Folinic acid (leucovorin) and autism: what kept you?

A Closer Look at the Drug Trump Is Touting for Autism: https://www.wsj.com/health/wellness/autism-leucovorin-science-research-0a666a8d

As the dust settles from what has been quite a monumental week for autism policy over on the US side of the Pond, particularly the new guidance on the use of leucovorin (folinic acid) for some autisms, some important questions emerge... 

Science has known about the potential connections between some autisms and cerebral folate deficiency (CFD), those folate receptor autoantibodies and the usefulness of folinic acid for some autisms for a while. I talked about them back in 2012: Don't panic! Folate receptor autoantibodies and autism https://questioning-answers.blogspot.com/2012/01/dont-panic-folate-receptor.html So why did it take so long for such research to be government evaluated and considered worthy of such a move from 'bench to bedside'? What were previous administrations doing and what stopped this 'bench to bedside' move happening years ago?

I'm sure the answer is going to be complicated. Not least to include certain attitudes to autism (the autisms) from some certain quarters, what sorts of medicines are potentially indicated for said autisms (and whether there is monetary value attached to their usage) and the important need for as-near-as-possible solid science (particularly in the context of safety).

But it now appears there is more a 'pipeline' moving forwards, whereby similarly important biological mechanisms and related interventions can seemingly be quickly evaluated across the autisms. Indeed it again brings to mind the work on other medicines such as suramin and also the very promising results for CM-AT e.g. Pancreatic Replacement Therapy for Maladaptive Behaviors in Preschool Children With Autism Spectrum Disorder https://pmc.ncbi.nlm.nih.gov/articles/PMC10690476/ Also too, potentially important are the writings from the US FDA not so long ago about other intervention options for some autisms and the need for more controlled study e.g. Biological therapies need definitive randomized controlled clinical trials https://www.science.org/doi/10.1126/scitranslmed.adg2970 something that also seems to be a priority for the current US administration as older, less fruitful avenues of study will inevitably be replaced.

I think we have turned a corner in our understanding of the autisms and indeed, the desire to see safe and effective intervention options that help to remove at least some of the disability associated with the autisms. And where the US leads in policy around autism, other countries are sure to follow.