There’s been much in the news and on social media about a new “miracle drug” that purports to “cure autism.” So, what’s the truth behind leucovorin? What’s the Cope Center’s position on this controversial treatment?
Since March 2025, high-dose folinic acid, also known as leucovorin, has been increasingly used to treat children with autism. Dr. Cope explains, “folinic acid is a reduced form of Vitamin B9 that bypasses the need for the enzyme dihydrofolate reductase to enter the folate cycle.”
Leucovorin is lifesaving in children with a very rare condition, called cerebral folate deficiency, and early reports about nonverbal children starting to speak occurred in the context of this rare genetic disorder. Some researchers believe that many autistic individuals have difficulty absorbing Vitamin B naturally, which impacts brain development and could lead to autism and language delays. However, the evidence is mixed about whether this vitamin supplement is appropriate for all children with autism.
Several initial studies showed positive results, but these studies only examined a small group of subjects, which may limit how the findings apply to the broader population. Recently, a study including more participants was retracted due to data inconsistencies, highlighting the need for more research to understand the effects of leucovorin better.
Dr. Cope and our medical staff feel that the risks of leucovorin are generally mild, and there is anecdotal evidence that some patients increased their communication abilities and reduced negative behaviors after treatment.
In certain cases, leucovorin is prescribed for patients. There may also be some evidence that patients with antibodies to Vitamin B would have greater benefits. The Folate Receptor Autoantibody Test measures levels of blocking and binding antibodies to Vitamin B. The test is currently offered by only one lab and is not FDA-approved. We are not requiring or even recommending testing at this time.
The Cope medical team approaches the need for any testing and treatment on a case-by-case basis.