Improvement of lesions shown on MRI and CT scan by administration of dichloroacetate in patients with Leigh syndrome.

Brain lesions exhibited on MRI and CT scan in 2 patients with mitochondrial encephalomyelopathy representing Leigh syndrome were improved by administration of dichloroacetate (DCA). One patient had pyruvic acid dehydrogenase complex (PDHC) deficiency, the other had complex I deficiency. The efficacy of DCA was transient in the patient with the PDHC deficiency, lasting for about 2.5 months. The patient died at the age of 6, about 2 years after the initiation of DCA treatment. DCA administration was started in the patient with complex I deficiency when he was 15 months old and it is still effective at his present age of 24 months. His motor ability is developing, and he could walk without support at the age of 19 months. DCA administration should be tried in patients with mitochondrial diseases.