[PDF][PDF] Small-molecule antagonists of apoptosis suppressor XIAP exhibit broad antitumor activity

AD Schimmer, K Welsh, C Pinilla, Z Wang… - Cancer cell, 2004 - cell.com
AD Schimmer, K Welsh, C Pinilla, Z Wang, M Krajewska, MJ Bonneau, IM Pedersen…
Cancer cell, 2004cell.com
Apoptosis resistance commonly occurs in cancers, preventing activation of Caspase family
cell death proteases. XIAP is an endogenous inhibitor of Caspases overexpressed in many
cancers. We developed an enzyme derepression assay, based on overcoming XIAP-
mediated suppression of Caspase-3, and screened mixture-based combinatorial chemical
libraries for compounds that reversed XIAP-mediated inhibition of Caspase-3, identifying a
class of polyphenylureas with XIAP-inhibitory activity. These compounds, but not inactive …
Abstract
Apoptosis resistance commonly occurs in cancers, preventing activation of Caspase family cell death proteases. XIAP is an endogenous inhibitor of Caspases overexpressed in many cancers. We developed an enzyme derepression assay, based on overcoming XIAP-mediated suppression of Caspase-3, and screened mixture-based combinatorial chemical libraries for compounds that reversed XIAP-mediated inhibition of Caspase-3, identifying a class of polyphenylureas with XIAP-inhibitory activity. These compounds, but not inactive structural analogs, stimulated increases in Caspase activity, directly induced apoptosis of many types of tumor cell lines in culture, and sensitized cancer cells to chemotherapeutic drugs. Active compounds also suppressed growth of established tumors in xenograft models in mice, while displaying little toxicity to normal tissues. These findings validate IAPs as targets for cancer drug discovery.
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