What is Leucovorin?

Leucovorin, also called folinic acid, is a form of folate (vitamin B9). It is commonly used in medicine to reduce the side effects of certain chemotherapy drugs and to treat some folate-related conditions. Unlike standard folic acid supplements, leucovorin can bypass certain metabolic pathways and may more easily reach the brain.

Why is Leucovorin Being Studied in Autism?

Research has found that some children with autism spectrum disorder (ASD) have difficulties with folate metabolism or transport into the brain. In particular, a subset of children with autism test positive for folate receptor alpha autoantibodies (FRAA), which can interfere with folate transport.

A randomized, placebo-controlled clinical trial showed that children with autism and language impairment who received leucovorin experienced improvements in verbal communication, especially those who tested positive for FRAA. Other studies and reviews suggest potential benefits in areas such as speech and social interaction, though results vary and research is ongoing.

Potential Benefits

Leucovorin may support language development and communication in some children with autism and it may be more effective in children with documented folate metabolism or transport issues (e.g., FRAA-positive or cerebral folate deficiency). Generally, this medication is well tolerated in clinical studies when used under medical supervision.

Limitations

Please understand, this is NOT a cure for autism. Improvements, when seen, are usually partial and vary between children. Also, not every child responds. Benefits may be more likely in children with specific folate-related abnormalities. Our understanding of the medical evidence is still limited. While promising, more large-scale and long-term studies are needed. Leucovorin, at therapeutic doses, is prescription only and is not the same as over-the-counter folic acid supplements.

Safety Considerations

Medical supervision is essential. Leucovorin is a prescription medication. Dosing and monitoring should be managed by a qualified clinician. Side effects may include mild gastrointestinal upset, agitation, or sleep changes. Serious side effects are uncommon but possible. Leucovorin can interact with certain medications, so a doctor must review a child’s full medical history. Blood or antibody testing may be necessary before starting treatment to identify children most likely to benefit.

Key Takeaway

Leucovorin (folinic acid) is a promising therapy under investigation for certain children with autism, especially those with folate-related abnormalities. However, it is not appropriate for every child and should never be started without professional guidance.

Schedule An Appointment

If you are interested in whether leucovorin may help your child or to discuss whether a supervised trial of leucovorin might be appropriate contact us today about testing for folate receptor autoantibodies (FRAA) or other folate-related issues.