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Cancer Cell. 2014 Jan 13;25(1):9-11. doi: 10.1016/j.ccr.2013.12.019.

From anecdote to targeted therapy: the curious case of thalidomide in multiple myeloma.

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Division of Hematology/Oncology, Robert H. Lurie Comprehensive Cancer Center, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA. Electronic address:
Gene Regulation Laboratory, Peter MacCallum Cancer Centre, Melbourne, VIC 3002, Australia; Sir Peter MacCallum Department of Oncology, University of Melbourne, Parkville, VIC 3052, Australia.


Thalidomide and related drugs are key drugs for the treatment of multiple myeloma (MM). These agents bind to cereblon, a component of a ubiquitin ligase complex, altering the specificity of the complex to induce the ubiquitylation and degradation of Ikaros (IKZF1) and Aiolos (IKZF3), transcription factors essential for MM growth.

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