Targeting the XIAP/caspase-7 complex selectively kills caspase-3-deficient malignancies.

Caspase-3 downregulation (CASP3/DR) in tumors frequently confers resistance to cancer therapy and is significantly correlated with a poor prognosis in cancer patients. Because CASP3/DR cancer cells rely heavily on the activity of caspase-7 (CASP7) to initiate apoptosis, inhibition of activated CASP7 (p19/p12-CASP7) by X-linked inhibitor of apoptosis protein (XIAP) is ...
a potential mechanism by which apoptosis is prevented in those cancer cells. Here, we identify the pocket surrounding the Cys246 residue of p19/p12-CASP7 as a target for the development of a protein-protein interaction (PPI) inhibitor of the XIAP:p19/p12-CASP7 complex. Interrupting this PPI directly triggered CASP7-dependent apoptotic signaling that bypassed the activation of the apical caspases and selectively killed CASP3/DR malignancies in vitro and in vivo without adverse side effects in nontumor cells. Importantly, CASP3/DR combined with p19/p12-CASP7 accumulation correlated with the aggressive evolution of clinical malignancies and a poor prognosis in cancer patients. Moreover, targeting of this PPI effectively killed cancer cells with multidrug resistance due to microRNA let-7a-1-mediated CASP3/DR and resensitized cancer cells to chemotherapy-induced apoptosis. These findings not only provide an opportunity to treat CASP3/DR malignancies by targeting the XIAP:p19/p12-CASP7 complex, but also elucidate the molecular mechanism underlying CASP3/DR in cancers.
Mesh Terms:
Alkylation, Amino Acid Sequence, Animals, Antineoplastic Agents, Apoptosis, Breast Neoplasms, Caspase 3, Caspase 7, Colonic Neoplasms, Drug Resistance, Neoplasm, Enzyme Activation, Female, Humans, Kaplan-Meier Estimate, Lung Neoplasms, Lysine, MCF-7 Cells, Mice, Mice, Inbred NOD, Mice, SCID, Protein Interaction Maps, Signal Transduction, X-Linked Inhibitor of Apoptosis Protein, Xenograft Model Antitumor Assays
J. Clin. Invest.
Date: Sep. 03, 2013
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