Immune Response and Immunotherapy in GIST

  • Gerardo A. Vitiello
  • Benjamin D. Medina
  • Ronald P. DeMatteoEmail author


Imatinib mesylate, a small molecule inhibitor of KIT and PDGFRA, dramatically improves the prognosis of patients with metastatic gastrointestinal stromal tumor (GIST) and serves as a paradigm for targeted molecular therapy. However, imatinib is rarely curative, and recurrent or resistant disease often develops. Immune-checkpoint blockade is a promising new approach to cancer therapy. Correlative research in human GIST specimens has suggested that certain characteristics of the immune infiltrate affect GIST prognosis. Using a genetically engineered mouse model of GIST, we have demonstrated that the therapeutic efficacy of imatinib is partially dependent on the immune system, and imatinib synergizes with multiple immunotherapies. In this chapter, we review the basics of current GIST therapy, detail the macrophage and T cell immune responses in GIST, summarize relevant preclinical GIST immunotherapy research, highlight ongoing and completed clinical trials with immunotherapeutic agents, and suggest future areas for immune therapy in GIST.


Gastrointestinal stromal tumor Immunotherapy Imatinib CTLA-4 PD-1 PD-L1 CD40 


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Copyright information

© Springer International Publishing AG, part of Springer Nature 2019

Authors and Affiliations

  • Gerardo A. Vitiello
    • 1
  • Benjamin D. Medina
    • 1
  • Ronald P. DeMatteo
    • 2
    Email author
  1. 1.Department of SurgeryNew York University Langone Health SystemNew YorkUSA
  2. 2.Department of SurgeryHospital of the University of PennsylvaniaPhiladelphiaUSA

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