Effect of Leucovorin on Mitochondrial Respiration and One-Carbon Metabolism in Autism Spectrum Disorder: A Double-Blind Placebo-Controlled Substudy.

What was studied

This was a side study of a placebo-controlled trial in children with autism. Children got leucovorin or placebo for 12 weeks. Leucovorin is a reduced folate. The dose was 2 mg/kg/day, up to 50 mg/day. Blood cell energy use was tested in 38 children. Blood chemical markers were tested in 42 children.

What they found

Leucovorin raised several measures of cell respiration compared with placebo. These included basal and maximal respiration. Folate receptor alpha autoantibody status changed the effect on some measures. The biggest gains were in children who tested positive. The SAM/SAH ratio rose and S-adenosylhomocysteine fell on leucovorin. After correction for chance, those changes were no longer significant.

What it means for patients

The authors say the results support the folate-mitochondrial-redox axis as a possible treatment target. They say larger groups must repeat the work.

Why it is not certain yet

The groups were small, with 17 to 24 children per arm. The moderator analyses were exploratory. Some blood marker changes did not survive correction. The abstract does not give results on autism symptoms.

Source

Effect of Leucovorin on Mitochondrial Respiration and One-Carbon Metabolism in Autism Spectrum Disorder: A Double-Blind Placebo-Controlled Substudy.

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