Recent advances and novel agents for gastrointestinal stromal tumor (GIST)

Gurpreet Lamba, Samir Ambrale, Byung Lee, Ridhi Gupta, Shamudheen M Rafiyath, Delong Liu, Gurpreet Lamba, Samir Ambrale, Byung Lee, Ridhi Gupta, Shamudheen M Rafiyath, Delong Liu

Abstract

The discovery of CD117 mutation in almost all gastrointestinal stromal tumors (GISTs) marked a milestone. Other spindle cell neoplasms arising from the GI tract including lipoma, schwannoma, hemangioma, leiomyoma, and leiomyosarcoma are typically CD117-negative. GIST research and clinical care now represent a paradigm of translating discoveries in the molecular pathogenesis of cancer into highly effective targeted therapies that selectively inhibit etiologic "driver" pathways, leading to dramatically improved clinical outcomes. A series of investigations and trials are underway to develop novel and effective ways to treat patients with GIST. In this review, we discuss the highlights of recent advances and novel agents for GIST therapy.

Figures

Figure 1
Figure 1
KIT (CD117) receptor tyrosine kinase structure and common mutations found in gastrointestinal stromal tumor. Arrows indicate the corresponding mutations in the exons.
Figure 2
Figure 2
KIT (CD117) gene structure and common mutations in gastrointestinal stromal tumor. Arrows indicate the positions of common mutations in the KIT gene.

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