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Gene Expression Signatures Identify Novel Therapeutics for Metastatic Pancreatic Neuroendocrine Tumors
Journal article   Open access   Peer reviewed

Gene Expression Signatures Identify Novel Therapeutics for Metastatic Pancreatic Neuroendocrine Tumors

Aaron T Scott, Michelle Weitz, Patrick J Breheny, Po Hien Ear, Benjamin Darbro, Bart J Brown, Terry A Braun, Guiying Li, Shaikamjad Umesalma, Courtney A Kaemmer, …
Clinical cancer research, Vol.26(8), pp.2011-2021
04/15/2020
DOI: 10.1158/1078-0432.CCR-19-2884
PMCID: PMC7165057
PMID: 31937620
url
https://doi.org/10.1158/1078-0432.CCR-19-2884View
Published (Version of record) Open Access

Abstract

Pancreatic neuroendocrine tumors (pNETs) are uncommon malignancies noted for their propensity to metastasize and comparatively favorable prognosis. Although both the treatment options and clinical outcomes have improved in the past decades, most patients will die of metastatic disease. New systemic therapies are needed. Tissues were obtained from 43 patients with well-differentiated pNETs undergoing surgery. Gene expression was compared between primary tumors versus liver and lymph node metastases using RNA-Seq. Genes that were selectively elevated at only one metastatic site were filtered out to reduce tissue-specific effects. Ingenuity pathway analysis (IPA) and the Connectivity Map (CMap) identified drugs likely to antagonize metastasis-specific targets. The biological activity of top identified agents was tested using two pNET cell lines (BON-1 and QGP-1). A total of 902 genes were differentially expressed in pNET metastases compared with primary tumors, 626 of which remained in the common metastatic profile after filtering. Analysis with IPA and CMap revealed altered activity of factors involved in survival and proliferation, and identified drugs targeting those pathways, including inhibitors of mTOR, PI3K, MEK, TOP2A, protein kinase C, NF-kB, cyclin-dependent kinase, and histone deacetylase. Inhibitors of MEK and TOP2A were consistently the most active compounds. We employed a complementary bioinformatics approach to identify novel therapeutics for pNETs by analyzing gene expression in metastatic tumors. The potential utility of these drugs was confirmed by cytotoxicity assays, suggesting drugs targeting MEK and TOP2A may be highly efficacious against metastatic pNETs. This is a promising strategy for discovering more effective treatments for patients with pNETs.
Adult Aged Antineoplastic Agents - pharmacology Biomarkers, Tumor - genetics Cell Line, Tumor Computational Biology - methods Drug Evaluation, Preclinical - methods Female Gene Expression Regulation, Neoplastic Humans Male Middle Aged Molecular Targeted Therapy Neoplasm Metastasis Neuroendocrine Tumors - drug therapy Neuroendocrine Tumors - genetics Neuroendocrine Tumors - pathology Pancreatic Neoplasms - drug therapy Pancreatic Neoplasms - genetics Pancreatic Neoplasms - pathology Prognosis RNA-Seq - methods

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