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Folinic acid improves communication, eases autism symptoms in small study

Benefit greatest in kids who have antibody that might block vitamin’s transport into brain cells – a potential indicator of who will respond to treatment
October 18, 2016

In a small pilot study funded by Autism Speaks, treatment with folinic acid – a naturally occurring form of folate – improved communication and eased autism symptoms in language-impaired children who have autism. The gains were greatest in a subgroup of children who tested positive for an autoantibody that may partially block this vitamin from entering brain cells.

Folinic acid is a more metabolically active form of folate (also known as B9) than is folic acid, the synthetic form of folate found in many prenatal and children’s vitamins.

The findings appear today in the scientific journal Molecular Psychiatry.

“Though caution is needed with any small study, we hope these results will lead to the kind of large trial needed to fully validate a promising experimental treatment for a subset of individuals with autism,” comments Autism Speaks Interim Chief Science Officer Mathew Pletcher. “If confirmed by further research, this would exemplify the promise of personalizing treatments for autism’s many causes and subtypes.”

In the study, the researchers randomly selected 48 nonverbal or minimally verbal children with autism (average age 7 years) to take either two daily doses of folinic acid (up to 50 mg per day depending on body weight) or a look-alike dummy pill. Folinic acid occurs naturally in many foods (lentils, liver and many green vegetables), but at much lower levels.

Neither the researchers, the children nor their families knew who received the actual treatment (23 children) and who received the placebo (25 children) until after the 12-week trial concluded. Before and after the trial, the researchers administered standardized tests to measure verbal communication and behavioral symptoms of autism.

As a group, the children who took the folinic acid showed greater improvements in both verbal communication and behavioral symptom severity. However, the improvements were significantly greater among a subset of ten children in the folinic acid group who tested positive for an autoantibody. According to the researchers' theory, the antibody may interfere with the transport of the nutrient into brain cells.

The researchers propose that high doses of folinic acid helped improve brain function by overcoming this blockade and restoring normal folate levels inside brain cells. The theory is based on previous research by the group, which detected this folate-blocking autoantibody in 60 percent of children who have autism, but just 3 percent of children with developmental delays that do not involve autism.

The findings are also in line with a large body of research, by other scientists, showing that women who take prenatal vitamins containing folate have a lower risk of having children affected by autism.

“These observations suggest that identifying autism and the presence of folate receptor autoantibodies early in life may present an opportunity to prevent at least some of autism’s developmental deficits with folinic acid treatment,” says senior researcher Edward Quadros, of the State University of New York Downstate Medical Center. Before such treatment can be recommended, larger studies are needed to confirm safety and effectiveness.