Abstract
A third of patients with triple negative breast cancer (TNBC) have relapsed disease within 2-5 years from initial diagnosis, leaving an unmet need for therapeutic targets. TNBC frequently harbors alterations of the PI3K/AKT/mTOR pathway, but single agent PI3K/AKT/mTOR inhibitors have not shown marked efficacy. In this study, we investigated a strategy to improve efficacy of PI3K-α inhibitor BYL719 (alpelisib) in TNBC. While BYL719 is effective at inhibiting cell proliferation in T47D, a triple positive cell line, it had limited activity in TNBC. This may be partially due to persistent phosphorylation of RB, and incomplete inhibition of p-S6 in TNBC, since the inhibitory effect of BYL719 on p-RB and p-S6 was significantly reduced in TNBC compared to T47D cells. Addition of the CDK4/6 inhibitor LEE011 to BYL719 caused a simultaneous reduction of p-RB and p-S6, and a more complete inhibition of p-S6, leading to decreased expression of the pro-survival protein MCL-1, an induction of apoptosis, and an enhanced reduction of tumor growth in a PDX model of TNBC. These findings suggest that inhibition of p-RB and p-S6 is important for an effective response to the treatment of TNBC, and provides a strong rationale for clinical development of combination therapy with BYL719 and LEE011 for treatment of metastatic TNBC with intact RB.Presentation: This study was presented in part as an abstract at the 2016 San Antonio Breast Cancer Symposium (P3-03-15) and the 2018 Cancer Research and Targeted Therapy in London.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
MeSH terms
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Aminopyridines / administration & dosage*
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Animals
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Antineoplastic Combined Chemotherapy Protocols / administration & dosage*
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Apoptosis / drug effects
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Cell Line, Tumor
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Cell Survival / drug effects
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Class I Phosphatidylinositol 3-Kinases / antagonists & inhibitors
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Cyclin-Dependent Kinase 4 / antagonists & inhibitors
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Cyclin-Dependent Kinase 6 / antagonists & inhibitors
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Drug Synergism
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Enzyme Inhibitors / administration & dosage
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Female
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Humans
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Mice
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Phosphorylation
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Protein Kinase Inhibitors / administration & dosage
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Proto-Oncogene Proteins c-akt / metabolism
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Purines / administration & dosage*
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Retinoblastoma Protein / antagonists & inhibitors
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Retinoblastoma Protein / metabolism
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Ribosomal Protein S6 Kinases / antagonists & inhibitors*
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Ribosomal Protein S6 Kinases / metabolism
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Signal Transduction / drug effects
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TOR Serine-Threonine Kinases / metabolism
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Thiazoles / administration & dosage*
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Triple Negative Breast Neoplasms / drug therapy*
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Triple Negative Breast Neoplasms / metabolism
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Triple Negative Breast Neoplasms / pathology
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Xenograft Model Antitumor Assays
Substances
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Aminopyridines
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Enzyme Inhibitors
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Protein Kinase Inhibitors
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Purines
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Retinoblastoma Protein
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Thiazoles
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Alpelisib
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MTOR protein, human
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Class I Phosphatidylinositol 3-Kinases
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PIK3CA protein, human
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Proto-Oncogene Proteins c-akt
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Ribosomal Protein S6 Kinases
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TOR Serine-Threonine Kinases
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CDK4 protein, human
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CDK6 protein, human
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Cyclin-Dependent Kinase 4
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Cyclin-Dependent Kinase 6
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ribociclib