L-arginine therapy on MELAS

MELAS is the most dominant clinical features among mitochondrial disorders. However the natural course of MELAS has not been clarified yet. In order to elucidate the natural course of MELAS, we have done the Japanese Cohort study on MELAS. By the age of onset, we divided MELAS into two subgroups, a juvenile form (onset is less than 18 years of age) and an adult form (onset is more than 18 years of age). Juvenile form is significantly different from adult form not only in the mean age of onset, but the mean age of death and survival rate (juvenile has 3.2 times higher chance of death than adult). Our date indicate that juvenile form of MELAS is more severe and poor prognosis than those seen in adult form. Based on the hypothesis that MELAS is caused by impaired vasodilation in an intracerebral artery, we evaluated the effects of administering L-arginine, a nitric oxide precursor. Patients were administered L-arginine intravenously at acute phase, or per orally at interictal phase. L-arginine infusions significantly improved all stroke-like symptoms suggesting stroke within 30 min, and oral administration significantly decreased frequency and severity of stroke-like episodes. L-arginine therapy showed promise in treating stroke-like episodes in MELAS.