Thursday, November 21, 2013
This week in therapeutics
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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
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.
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
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
Patent and licensing status
J.-M. et al. Proc. Natl. Acad. Sci. USA; published online Oct. 21,
Contact: Emmanuel Richard, University of Bordeaux Segalen, Bordeaux,
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