An Integrative Approach to Precision Cancer Medicine Using Patient-Derived Xenografts

Mol Cells. 2016 Feb;39(2):77-86. doi: 10.14348/molcells.2016.2350. Epub 2016 Feb 2.


Cancer is a heterogeneous disease caused by diverse genomic alterations in oncogenes and tumor suppressor genes. Despite recent advances in high-throughput sequencing technologies and development of targeted therapies, novel cancer drug development is limited due to the high attrition rate from clinical studies. Patient-derived xenografts (PDX), which are established by the transfer of patient tumors into immunodeficient mice, serve as a platform for co-clinical trials by enabling the integration of clinical data, genomic profiles, and drug responsiveness data to determine precisely targeted therapies. PDX models retain many of the key characteristics of patients' tumors including histology, genomic signature, cellular heterogeneity, and drug responsiveness. These models can also be applied to the development of biomarkers for drug responsiveness and personalized drug selection. This review summarizes our current knowledge of this field, including methodologic aspects, applications in drug development, challenges and limitations, and utilization for precision cancer medicine.

Keywords: cancer drug development; cancer genomics; patient-derived xenografts; precision medicine.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology
  • Biomarkers, Pharmacological / metabolism
  • Biphenyl Compounds / pharmacology
  • Camptothecin / analogs & derivatives
  • Camptothecin / pharmacology
  • Drug Synergism
  • Graft Survival*
  • Humans
  • Irinotecan
  • Mice
  • Mice, Nude
  • Mice, SCID
  • Molecular Targeted Therapy / methods*
  • Nitrophenols / pharmacology
  • Piperazines / pharmacology
  • Precision Medicine
  • Stomach Neoplasms / drug therapy
  • Stomach Neoplasms / genetics
  • Stomach Neoplasms / immunology*
  • Stomach Neoplasms / pathology
  • Sulfonamides / pharmacology
  • Tumor Burden / drug effects
  • Xenograft Model Antitumor Assays / methods*


  • ABT-737
  • Antineoplastic Agents
  • Biomarkers, Pharmacological
  • Biphenyl Compounds
  • Nitrophenols
  • Piperazines
  • Sulfonamides
  • Irinotecan
  • Camptothecin