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I saw news that leucovorin might be used to treat autism in kids. Is there actually good evidence it helps, or is this more hype than reality?
—Unsure About a Cure
In a September 2025 press conference, President Trump and his medical advisors discussed what they framed as a new “exciting treatment” for treating autism in children: leucovorin. They indicated that they would be starting the process of making this drug more widely available, but provided relatively few additional details about the evidence behind the drug’s efficacy.
Leucovorin, also known as folinic acid, is actually not “new” at all; it’s a decades-old medication that is primarily used in cancer treatment. It treats side effects from a particular chemotherapy drug called methotrexate and is used to enhance the effectiveness of other chemotherapy drugs, for colon cancer in particular. There are a number of other related uses, mostly in cancer.
There has been speculation that this medication could help treat autism because individuals with autism are more likely to have cerebral folate deficiency, a condition this medication treats. The fact that this condition is more common in autistic children than in nonautistic children does not necessarily mean that treating it would help, but it suggests a hypothesis to test this.
A number of small randomized controlled trials have provided early results suggesting that, indeed, treatment could improve some symptoms of autism. One of the larger examples is this 2024 study in which 80 children with autism were randomized into a treatment group that received folinic acid and a control group that did not. The treatment group had larger improvements on a scale measure of autism symptoms, which include speech and communication, as well as in behavioral symptoms, relative to the control group. These results were statistically significant.
Another small study found improvement in language skills, concentrated in children with a particular folate status. A 2021 meta-analysis pointed to four randomized studies at that time, along with a number of observational studies, which provided suggestive positive results. In total, fewer than 300 children have been involved in randomized trials for this condition.
Overall, these are interesting and exciting findings and certainly worthy of more research. However, in the spring of 2026, the FDA sounded a much more cautionary note. They suggested that leucovorin might be useful only in a very, very limited set of cases with a cerebral folate deficiency, which can cause autism-like symptoms. They do not, at this stage, see leucovorin as a widespread treatment for autism in general. This doesn’t mean that we will not continue to see prescriptions and more research, but for now, the FDA has not indicated optimism.
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