Enzyme replacement therapy and substrate reduction therapy have had an incredible impact on the treatment of many lysosomal storage disorders (LSDs). However, in some LSDs the disorder continues to progress, in part due to downstream pathologic processes instigated by the storage material.
Williams et al. Neurobiol Dis (2014) March (Epub) report that combination therapy with miglustat, curcumin and ibuprofen provide a neuroprotective benefit compared with single therapy in a mouse model for NPC1. Mice treated with the combination therapy maintained body weight and motor function and had delayed onset of Purkinje cell loss compared with those treated with single therapy. This group concludes that targeting multiple unique in the pathogenic cascade of NPC1 maximises the clinical benefit in their mouse model.
Hilary Vernon, MD PhD