A preliminary study suggests that prescribed doses of folinic acid, a reduced form of Vitamin B known as folate, could help improve the language and communication skills of children with autism spectrum disorder (ASD).
The study also identifies specific biomarkers that can predict treatment response in children with autism and verbal communication problems.
The findings stem from a placebo-controlled trial in which children were randomized to receive either high-dose folinic acid or a placebo.
Up to two percent of American children are said to experience symptoms that place them on the autism spectrum. Many of these children have difficulty communicating and interacting with others, especially within a social setting.
Researchers do not yet fully understand all the reasons behind the development of ASD and, importantly, there are currently no approved treatments that address the core symptoms of this disorder.
“The only currently approved medications for autism are both antipsychotic medications that address non-core symptoms and can lead to unwanted side effects,” said John Slattery, clinical research program manager at Arkansas Children’s Research Institute and a co-author of the study.
Scientific research has linked this disorder to abnormalities in the metabolism of folate as well as genes that are involved in folate metabolism. Certain studies have also shown that the offspring of women who took folate supplements before conception and during pregnancy had a lower risk of having a child with ASD.
About 10 years ago a condition known as cerebral folate deficiency (CFD) was described in which the concentration of folate is below normal in the central nervous system but not in the blood. Many children with CFD had ASD symptoms and responded well to treatment with high-dose folinic acid.
Lead author Dr. Richard Frye of and his team had previously shown that folate receptor autoantibodies were found with a high prevalence in children with ASD.
In the current study, researchers found that participants with folate receptor autoantibodies had a more favorable response to the folinic acid treatment. This leads the way to a test that might be useful for clinicians to determine if high-dose folinic acid might be a treatment for a particular child with ASD.
Related research on laboratory rat models have confirmed the deleterious effects of folate receptor antibodies on brain development and function.
“Improvement in verbal communication was significantly greater in participants receiving folinic acid as compared with those receiving the placebo,” Frye said. He added that the findings should be considered preliminary until the treatment has been assessed further in larger long-term studies.
The study appears in the journal Molecular Psychiatry.