Compassionate Use of Simvastatin for a Patient with Juvenile Metachromatic Leukodystrophy

Abstract

Background/Purpose: Juvenile metachromatic leukodystrophy (MLD) is a severe genetic neurodegenerative disease caused by deficiency of arylsulfatase A. Between the 3rd and the 16th year of life, demyelination causes motoric and cognitive disabilities. This leads to helplessness and early death. In early stages of the disease, hematopoietic stem cell transplant may positively influence the course of the disease. In later stages, only symptomatic treatment is possible. In 2015, it was reported that MLD mice receiving Simvastatin had less inflammatory cytokines and showed a better motor performance and less demyelination.