{"id":634685,"date":"2025-12-16T13:00:51","date_gmt":"2025-12-16T18:00:51","guid":{"rendered":"https:\/\/www.singlecare.com\/blog\/?p=634685"},"modified":"2025-12-16T13:26:25","modified_gmt":"2025-12-16T18:26:25","slug":"leucovorin-for-kids-with-autism","status":"publish","type":"post","link":"https:\/\/www.singlecare.com\/blog\/leucovorin-for-kids-with-autism\/","title":{"rendered":"Leucovorin for kids with autism"},"content":{"rendered":"

Leucovorin<\/span><\/a> is a type of medication called a folic acid analog. It works by increasing folate levels (vitamin B9) in the body and protecting cells from the side effects of certain drugs. <\/span>Leucovorin<\/span> is currently approved by the Food and Drug Administration (FDA) to manage the side effects of large doses or overdoses of medications like methotrexate. Although managing drug side effects is leucovorin\u2019s FDA-approved indication, it is also used off-label for several conditions, including cancer treatments and ectopic pregnancy treatments. In September 2025, the FDA announced that it would soon be approving Leucovorin for kids with autism. Leucovorin has already been used off-label for autism, and this move by the FDA could make the medication a more standard treatment option for autism in kids.<\/span><\/p>\n

Leucovorin as a treatment for autism in kids<\/span><\/h2>\n

Autism, also known as autism spectrum disorder, is a neurodevelopmental disorder impacting the way people view the world, communicate and socialize with others, and behave. Symptoms are usually first noticed in early childhood, and it\u2019s estimated to impact<\/span> 1 in every 31 children<\/span><\/a>.<\/span><\/p>\n

There is a range of autism functionality, with some people needing very little support, some needing daily care, and some being non-verbal. Many people view autism not as a disease that\u2019s meant to be cured, but as a different way of being.<\/span><\/p>\n

What the research says<\/span><\/h3>\n

As the<\/span> FDA mentioned<\/span><\/a> in its September press release, there is<\/span> some research<\/span><\/a> showing positive use of leucovorin in people with cerebral folate deficiency (CFD), which is a condition that impacts the transport of folate to the brain. The FDA notes that people with cerebral folate deficiency share symptoms with people who have autism.<\/span><\/p>\n

Other studies have looked specifically at the use of leucovorin supplementation for the treatment of some symptoms of autism. A<\/span> 2020 review published in <\/span>Seminars in Pediatric Neurology<\/span><\/i><\/a> concluded that leucovorin may be able to \u201csubstantially improve\u201d autism symptoms, including language and verbal communication skills, especially in those who test positive for folate receptor alpha autoantibodies.<\/span><\/p>\n

While these studies show promise, experts note that it may be too soon to recommend leucovorin as a <\/span>universal<\/span><\/i> autism treatment for kids. Instead, what the research shows is that leucovorin may be a helpful potential treatment for certain autistic children. \u201c<\/span>It\u2019s not a cure for autism, but it may help a specific group of children whose brains aren\u2019t getting enough folate on their own,\u201d <\/span>says<\/span>\u00a0Nona Kocher<\/span><\/a>, MD, a board-certified psychiatrist based in Miami.<\/span><\/p>\n

\u201cThe strengths of these studies are that they are revealing a specific biological\/mechanistic link between folinic acid therapy and ASD-symptom improvements for those individuals who have a genetic or other biological deficiency that leads to reduced folate availability in the brain,\u201d says<\/span> Jared Schwartzer<\/span><\/a>, Ph.D., professor of psychology, neuroscience, behavior, and education at Mount Holyoke College.<\/span><\/p>\n

On the other hand, the limitations of these studies are a relatively low sample size and a limited number of experiments. \u201cThis makes it difficult to generalize to a larger population or identify potential contraindications (populations for which this treatment could be harmful and should be avoided),\u201d Dr. Schwartzer says.<\/span><\/p>\n

Also, <\/span>Dr. Kocher <\/span>points out that even if leucovorin proves helpful for certain children, it is not a behavioral medication. \u201c<\/span>It\u2019s targeting a specific metabolic pathway,\u201d she explains. \u201cThat makes it very different from the usual tools we rely on, like stimulants or antipsychotics.\u201d<\/span><\/p>\n

Dr. Schwartzer notes that in these studies, leucovorin hasn\u2019t shown significant adverse effects on kids. But that doesn\u2019t mean it\u2019s necessarily time to recommend it as an autism treatment. \u201cThe concern is not that Leucovorin is a dangerous or harmful medication,\u201d he explains. \u201cThe concern is that there are claims being made about its effectiveness beyond what the data are presently able to support.\u201d<\/span><\/p>\n

On Oct. 31, 2025, the<\/span> Academy of American Pediatrics (AAP) issued guidance<\/span><\/a> about leucovorin and children. It states that it currently doesn\u2019t recommend the routine use of leucovorin for autism in kids. The AAP acknowledges that small studies have found benefits for selected cases, but that more research is needed for a wider recommendation to be made.\u00a0<\/span><\/p>\n

Leucovorin dosage for kids with autism\u00a0<\/span><\/h2>\n

As the AAP notes in its recent guidance, questions about a leucovorin dose for kids with autism <\/span>still remain unanswered due to a lack of robust clinical trials. The FDA also has not yet released dosing guidance about leucovorin for autism in kids.<\/span><\/p>\n

Dr. Schwartzer predicts that the dosing used in research reports will likely be incorporated in the FDA decision. \u201cBut I also want to express caution about using research doses as a means to self-medicate,\u201d Dr. Schwartzer emphasizes. \u201cJust because these doses are published in the literature does not mean they alone represent the safe and effective dose.\u201d<\/span><\/p>\n

\u00a0<\/span>The typical dose mentioned in research ranges from \u201c2 mg\/kg per day, or 25 mg twice a day (50 mg maximum), and higher doses have been used for clinical evaluation purposes at a concentration of 50 mg twice a day,\u201d Dr. Schwartzer says.<\/span><\/p>\n

Leucovorin side effects and warnings<\/span><\/h2>\n

Like any medication,<\/span> leucovorin can cause side effects<\/span><\/a>. According to Dr. Kocher, the side effects most often seen in kids are <\/span>restlessness, irritability, trouble sleeping, stomach upset, or headaches. But these are typically mild and can usually be managed by adjusting the dose.<\/span><\/p>\n

\u201c<\/span>The studies that have looked at leucovorin and folinic acid supplementation for ASD symptoms report that the medication is well tolerated with few adverse effects,\u201d Dr. Schwartzer says. \u201cThis is certainly encouraging. However, there have not been enough large-scale trials (for example, a Phase III clinical trial) that can scale the medication use up across a large enough population to identify any other potential side effects or contraindications.\u201d<\/span><\/p>\n

Who should not take leucovorin for autism?<\/span><\/h2>\n

Although leucovorin is largely safe, there are some people who should avoid it. Here are tips from Dr. Kocher:<\/span><\/p>\n