Annals of Surgical Oncology

, Volume 9, Issue 9, pp 831–839

The GIST of targeted cancer therapy: A tumor (Gastrointestinal stromal tumor), a mutated gene (c-kit), and a molecular inhibitor (STI571)

Educational Review

DOI: 10.1007/BF02557518

Cite this article as:
DeMatteo, R.P. Annals of Surgical Oncology (2002) 9: 831. doi:10.1007/BF02557518


Although gastrointestinal stromal tumor (GIST) is the most frequent mesenchymal neoplasm of the gastrointestinal tract, until recently it has been an obscure disease. Now, there is widespread scientific and clinical interest in GIST because its principal pathogenetic defect has been identified and a specific molecular inhibitor of GIST has been developed. Most GISTs contain a gain-of-function mutation in thec-kit proto-oncogene. Mutation results in constitutive activation of the Kit receptor tyrosine kinase, which induces cellular proliferation. STI571 is an oral agent that selectively inhibits Kit. It is a landmark development in cancer treatment and marks a new era of targeted molecular therapy. Its efficacy proves that a specific inhibitor can counteract the effects of a genetic defect responsible for neoplasia. Althought ST1571 was first applied to GIST only 2 years ago, it has already revolutionized the treatment of patients with metastatic disease and is also currently being tested as an adjuvant therapy after the resection of primary GIST.

Key Words

Gastrointestinal stromal tumorGISTc-kit Proto-oncogeneSTI571

Copyright information

© The Society of Surgical Oncology, Inc 2002

Authors and Affiliations

  1. 1.Department of SurgeryMemorial Sloan-Kettering Cancer CenterNew York