Gastrointestinal stromal tumors (GIST) are sarcoma in the gastrointestinal tract which may be caused by somatic gain-of-function mutations in the KIT or PDGFRA gene. Imatinib mesylate has shown significant safety and great effectiveness for patients with KIT-positive unresectable, advanced, or metastatic GIST. The response rate (RR) was no less than 50%, disease control rate (DCR) of more than 85%, and the median progression-free survival (PFS) has been nearly two years. Also, imatinib has been shown to improve the prognosis of GIST patients. Meanwhile, sunitinib malate, used for patients with imatinib-resistant GIST or imatinib-intolerant patients, has also shown modest tolerability and fairly good outcomes. Sunitinib shows a less than 10% RR, a 30% to 40% DCR, and a median of nearly 8 months of PFS. Molecularly targeted therapy has prolonged overall survival of advanced GIST patients from 1. 5 years in the pre-imatinib era to 5 years after the introduction of imatinib. There remains, however, a great unmet medical need for new agents and/or multidisciplinary treatments for advanced GIST patients.