Temporal activation of p53 by a specific MDM2 inhibitor is selectively toxic to tumors and leads to complete tumor growth inhibition.

We have designed MI-219 as a potent, highly selective and orally active small-molecule inhibitor of the MDM2-p53 interaction. MI-219 binds to human MDM2 with a K(i) value of 5 nM and is 10,000-fold selective for MDM2 over MDMX. It disrupts the MDM2-p53 interaction and activates the p53 pathway in cells ...
with wild-type p53, which leads to induction of cell cycle arrest in all cells and selective apoptosis in tumor cells. MI-219 stimulates rapid but transient p53 activation in established tumor xenograft tissues, resulting in inhibition of cell proliferation, induction of apoptosis, and complete tumor growth inhibition. MI-219 activates p53 in normal tissues with minimal p53 accumulation and is not toxic to animals. MI-219 warrants clinical investigation as a new agent for cancer treatment.
Mesh Terms:
Administration, Oral, Animals, Antineoplastic Agents, Apoptosis, Cell Cycle, Cell Line, Tumor, Cell Proliferation, Humans, Indoles, Mice, Models, Molecular, Neoplasms, Protein Binding, Proto-Oncogene Proteins c-mdm2, Spiro Compounds, Time Factors, Tumor Suppressor Protein p53, Xenograft Model Antitumor Assays
Proc. Natl. Acad. Sci. U.S.A.
Date: Mar. 11, 2008
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