Establishment and characterization of a human gastrointestinal stromal tumour (GIST) xenograft in athymic nude mice

Anticancer Res. 2009 Nov;29(11):4331-6.


Background: The majority of gastrointestinal stromal tumours (GISTs) contain oncogenic KIT (v-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog) or platelet-derived growth factor-alpha (PDGFRA) receptor tyrosine kinase (TK) mutations and are initially, but only temporarily sensitive to TK inhibitors. The aim of this study was to establish and characterize a human GIST xenograft that could be used for evaluating various molecularly targeted therapies.

Materials and methods: GIST tissue from four patients was implanted under the skin of athymic nude mice. In one case a tumour line was established.

Results: The xenograft showed characteristic GIST morphology and exhibited the same mutation profile as that of the patient.

Conclusion: A human GIST xenograft with mutation in KIT exons 11 and 17 has been established and maintained in nude mice for 3 years (13 passages). This model will enable further studies on mechanisms of resistance, combination therapies and allow testing of novel targeted therapies.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Animals
  • Benzamides
  • Drug Resistance, Neoplasm
  • Female
  • Gastrointestinal Stromal Tumors / genetics
  • Gastrointestinal Stromal Tumors / pathology*
  • Gastrointestinal Stromal Tumors / therapy
  • Humans
  • Imatinib Mesylate
  • Immunohistochemistry
  • Karyotyping
  • Male
  • Mice
  • Mice, Nude
  • Middle Aged
  • Mutation
  • Piperazines / pharmacology
  • Proto-Oncogene Proteins c-kit / genetics
  • Pyrimidines / pharmacology
  • Receptor, Platelet-Derived Growth Factor alpha / genetics
  • Xenograft Model Antitumor Assays / methods*


  • Benzamides
  • Piperazines
  • Pyrimidines
  • Imatinib Mesylate
  • Proto-Oncogene Proteins c-kit
  • Receptor, Platelet-Derived Growth Factor alpha