Research ArticleCancer

Dual inhibition of MDMX and MDM2 as a therapeutic strategy in leukemia

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Science Translational Medicine  11 Apr 2018:
Vol. 10, Issue 436, eaao3003
DOI: 10.1126/scitranslmed.aao3003

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A new staple of leukemia treatment?

As suggested by their name, tumor suppressor genes prevent tumorigenesis, and their expression or activity is often lost in cancer cells. One of the best known tumor suppressors is p53, which is inactivated in a variety of cancer types, often through up-regulation of its endogenous suppressors. Despite numerous attempts to reactivate p53 by a variety of approaches, none have successfully advanced beyond clinical trials thus far. Now, Carvajal et al. applied yet another tactic to restore p53 activity by using a stapled peptide to inactivate both of its endogenous inhibitors, for situations where the tumor suppressor is inactive but not completely lost. The authors demonstrated the effectiveness of this approach in human acute myeloid leukemia using in vitro and in vivo models, along with preliminary testing in a patient with leukemia.