Some children with autism may have trouble getting enough folate (a B vitamin) into their brains, even when they eat foods with folate. Researchers have found that a medicine called leucovorin might help bypass this problem and improve communication in some nonverbal children with autism. However, not all studies show the same results, and major medical organizations haven’t officially recommended it yet. This review helps doctors understand what we know and don’t know about using leucovorin for autism.
The Quick Take
- What they studied: Whether a medicine called leucovorin can help children with autism by getting more folate (a B vitamin) into their brains
- Who participated: This is a review article that looked at research on children with autism spectrum disorder, some of whom may have a condition where their brains don’t absorb folate properly
- Key finding: Some studies show leucovorin may help nonverbal children with autism communicate better, especially those with specific immune system markers. However, other studies found no improvement, and major pediatric organizations don’t currently recommend it
- What it means for you: If your child has autism and you’re considering leucovorin, talk with your doctor first. While it may help some children, the evidence is mixed, and it’s not an official treatment recommendation yet
The Research Details
This is a review article, which means doctors and researchers looked at all the existing studies about leucovorin and autism to summarize what we know. Rather than doing a new experiment, the authors gathered information from previous research to help other doctors understand the topic better.
The review focuses on a specific idea: some children with autism might have trouble getting folate (a B vitamin) into their brains. This happens because their immune systems make proteins called autoantibodies that block folate from entering brain cells. Leucovorin is a man-made form of folate that can sometimes get into the brain even when these blocking proteins are present.
The authors looked at studies showing that about 71% of children with autism have these blocking proteins, and they reviewed evidence about whether leucovorin actually helps these children.
Review articles are important because they help doctors make sense of lots of different studies that sometimes show different results. By looking at all the evidence together, doctors can better understand what treatments might work and which ones need more research. This is especially important for autism, where families are often looking for new treatments and may see information on social media that isn’t always accurate.
This review was published in a respected medical journal for pediatricians, which means it was checked by other experts. However, because it’s a review rather than a new study, it depends on the quality of the studies it examined. The authors honestly point out that some studies show benefits while others don’t, which shows they’re being fair about the mixed evidence.
What the Results Show
The research shows that many children with autism (about 71%) have immune system proteins that block folate from getting into their brains. This is called cerebral folate deficiency, and it’s a real biological problem that scientists can measure.
When researchers gave leucovorin to some children with autism, particularly nonverbal children who had these blocking proteins, some studies found improvements in communication and language skills. These improvements were noticeable enough that families and doctors started asking for the treatment more often.
However, other studies found that leucovorin didn’t help, even in children who had the blocking proteins. This means the results are mixed—it helps some children but not others, and scientists aren’t sure yet why some respond and others don’t.
Because of this mixed evidence, the American Academy of Pediatrics (the main organization of children’s doctors in the United States) has not officially recommended leucovorin as a treatment for autism.
The review highlights that leucovorin has become popular on social media and among policymakers, which has led many families to ask their doctors for it. This shows how information spreads quickly online, sometimes faster than scientific evidence can catch up. The review also notes that doctors need better ways to identify which children might benefit from leucovorin before starting treatment.
This research builds on earlier discoveries that folate problems might be connected to autism. The finding that autoantibodies block folate in most children with autism is relatively recent and has changed how scientists think about the condition. However, the fact that leucovorin doesn’t help all children with these antibodies suggests the problem is more complicated than originally thought.
The biggest limitation is that the studies reviewed show mixed results, so it’s hard to say for certain whether leucovorin works. We don’t know yet which children are most likely to benefit. The review also doesn’t include information about long-term safety or side effects of using leucovorin in children. Additionally, many studies have been small, so larger research is needed to be more confident about the results.
The Bottom Line
Current medical evidence suggests that leucovorin may help some children with autism improve communication, particularly nonverbal children who have the specific immune system markers. However, the evidence is mixed (some studies show benefits, others don’t), so this is not an official recommendation. If you’re interested in trying leucovorin, discuss it with your child’s doctor who can evaluate whether it might be appropriate for your child. The confidence level is moderate—there’s enough evidence to study further, but not enough to recommend it as a standard treatment.
Parents and doctors of children with autism who are nonverbal or have significant communication challenges might want to discuss this with their pediatrician. Children who have been tested and found to have the specific immune system markers blocking folate might be better candidates. This is less relevant for children with autism who communicate well or whose families aren’t interested in medication-based treatments.
If leucovorin does help a child, improvements in communication might appear over weeks to months of consistent use. However, some children show no improvement even after several months, which is why it’s important to have realistic expectations and work closely with your doctor to monitor progress.
Want to Apply This Research?
- If your child starts leucovorin, track specific communication behaviors weekly: number of new words spoken, attempts to communicate, or responses to questions. Use a simple 1-10 scale to rate communication effort and clarity each week to see if there are changes over time.
- Work with your doctor to establish a baseline of your child’s current communication abilities before starting leucovorin. Then use the app to log observations about speech, gestures, and attempts to communicate daily. This creates a clear record to share with your pediatrician at follow-up visits.
- Set monthly check-in reminders to review communication progress with your child’s doctor. Track not just speech but also non-verbal communication like pointing, eye contact, and social engagement. If no improvement is seen after 2-3 months, discuss with your doctor whether to continue or try a different approach.
This review summarizes current research on leucovorin for autism but does not constitute medical advice. Leucovorin is not officially recommended by the American Academy of Pediatrics for treating autism. Before starting any new treatment, including leucovorin, consult with your child’s pediatrician or autism specialist. They can evaluate your child’s specific situation, discuss potential benefits and risks, and monitor for any side effects. The evidence for leucovorin is mixed, and it may help some children but not others. Do not start, stop, or change any autism treatments without professional medical guidance.