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Endocrine/metabolic disease


Proteasome; uroporphyrinogen III synthase (UROS)

Mouse and cell culture studies suggest proteasome inhibitors could be useful for treating congenital erythropoietic porphyria, which is caused by destabilizing mutations in UROS. In human erythrocyte cell lines carrying mutations in UROS that destabilize the protein, the proteasome inhibitor Velcade bortezomib partially protected the synthase from premature degradation. In mice carrying analogous Uros mutations, Velcade decreased disease severity compared with saline. Next steps could include evaluating other proteasome inhibitors in the preclinical models and testing various dosing protocols.
Takeda Pharmaceutical Co. Ltd. and Johnson & Johnson market Velcade to treat multiple myeloma (MM) and mantle cell lymphoma (MCL).
Amgen Inc. and Ono Pharmaceutical Co. Ltd. market the selective proteasome inhibitor Kyprolis carfilzomib to treat MM.
At least 10 companies have proteasome inhibitors in Phase III testing or earlier to treat various cancers.

SciBX 6(45); doi:10.1038/scibx.2013.1292
Published online Nov. 21, 2013

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Blouin, J.-M. et al. Proc. Natl. Acad. Sci. USA; published online Oct. 21, 2013;
Contact: Emmanuel Richard, University of Bordeaux Segalen, Bordeaux, France