Generation and molecular characterization of pancreatic cancer patient-derived xenografts reveals their heterologous nature

Oncotarget. 2016 Sep 20;7(38):62533-62546. doi: 10.18632/oncotarget.11530.

Abstract

Pancreatic ductal adenocarcinoma (PDAC) is the most challenging type of cancer to treat, with a 5-year survival rate of <10%. Furthermore, because of the large portion of the inoperable cases, it is difficult to obtain specimens to study the biology of the tumors. Therefore, a patient-derived xenograft (PDX) model is an attractive option for preserving and expanding these tumors for translational research. Here we report the generation and characterization of 20 PDX models of PDAC. The success rate of the initial graft was 74% and most tumors were re-transplantable. Histological analysis of the PDXs and primary tumors revealed a conserved expression pattern of p53 and SMAD4; an exome single nucleotide polymorphism (SNP) array and Comprehensive Cancer Panel showed that PDXs retained over 94% of cancer-associated variants. In addition, Polyphen2 and the Sorting Intolerant from Tolerant (SIFT) prediction identified 623 variants among the functional SNPs, highlighting the heterologous nature of pancreatic PDXs; an analysis of 409 tumor suppressor genes and oncogenes in Comprehensive Cancer Panel revealed heterologous cancer gene mutation profiles for each PDX-primary tumor pair. Altogether, we expect these PDX models are a promising platform for screening novel therapeutic agents and diagnostic markers for the detection and eradication of PDAC.

Keywords: cancer panel; heterogeneity; pancreatic cancer; patient-derived xenograft; single nucleotide polymorphism.

MeSH terms

  • Animals
  • DNA Mutational Analysis
  • Disease Models, Animal
  • Exome
  • Gene Expression Profiling
  • Gene Expression Regulation, Neoplastic
  • Genetic Variation
  • Humans
  • Male
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Multivariate Analysis
  • Mutation
  • Neoplasm Transplantation*
  • Pancreatic Neoplasms / genetics*
  • Pancreatic Neoplasms / pathology
  • Polymorphism, Single Nucleotide*
  • Republic of Korea
  • Signal Transduction
  • Smad4 Protein / genetics
  • Tumor Suppressor Protein p53 / genetics

Substances

  • SMAD4 protein, human
  • Smad4 Protein
  • TP53 protein, human
  • Tumor Suppressor Protein p53