Inhibiting the oncogenic translation program is an effective therapeutic strategy in multiple myeloma

S Manier, D Huynh, YJ Shen, J Zhou… - Science translational …, 2017 - science.org
S Manier, D Huynh, YJ Shen, J Zhou, T Yusufzai, KZ Salem, RY Ebright, J Shi, J Park…
Science translational medicine, 2017science.org
Multiple myeloma (MM) is a frequently incurable hematological cancer in which overactivity
of MYC plays a central role, notably through up-regulation of ribosome biogenesis and
translation. To better understand the oncogenic program driven by MYC and investigate its
potential as a therapeutic target, we screened a chemically diverse small-molecule library
for anti-MM activity. The most potent hits identified were rocaglate scaffold inhibitors of
translation initiation. Expression profiling of MM cells revealed reversion of the oncogenic …
Multiple myeloma (MM) is a frequently incurable hematological cancer in which overactivity of MYC plays a central role, notably through up-regulation of ribosome biogenesis and translation. To better understand the oncogenic program driven by MYC and investigate its potential as a therapeutic target, we screened a chemically diverse small-molecule library for anti-MM activity. The most potent hits identified were rocaglate scaffold inhibitors of translation initiation. Expression profiling of MM cells revealed reversion of the oncogenic MYC-driven transcriptional program by CMLD010509, the most promising rocaglate. Proteome-wide reversion correlated with selective depletion of short-lived proteins that are key to MM growth and survival, most notably MYC, MDM2, CCND1, MAF, and MCL-1. The efficacy of CMLD010509 in mouse models of MM confirmed the therapeutic relevance of these findings in vivo and supports the feasibility of targeting the oncogenic MYC-driven translation program in MM with rocaglates.
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