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Therapeutics
Review
Miglustat as a therapeutic agent: prospects and caveats
  1. Rosemarie E Venier1,
  2. Suleiman A Igdoura1,2
  1. 1Department of Biology, McMaster University, Hamilton, Ontario, Canada
  2. 2Department of Pathology and Molecular Medicine, McMaster University, Hamilton, Ontario, Canada
  1. Correspondence to Professor Suleiman A Igdoura, McMaster University, 1280 Main St. W. LSB 335, Hamilton, Ontario L8S 4K1, Canada; igdoura{at}mcmaster.ca

Abstract

A viable treatment for lysosomal storage disease has been very difficult to attain. One option is pharmacological inhibition of synthetic pathways to reduce substrate accumulations. Miglustat N-butyldeoxynojirimycin (NBDNJ), an inhibitor of glucosylceramide synthase, has shown much promise in clinical trials for the treatment of Type I Gaucher disease. The molecular events invoked by NBDNJ in cell culture and in animal models have not been so definitive. This review discusses the biochemical and molecular impact of NBDNJ as it relates to its potential as a therapeutic drug.

  • Metabolic disorders
  • Lipid disorders
  • Neurosciences
  • Movement disorders (other than Parkinsons)
  • Molecular genetics

http://dx.doi.org/10.1136/jmedgenet-2012-101070

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    2012, Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.