Targeted therapy for cancer: the gastrointestinal stromal tumor model.

Gastrointestinal stromal tumors (GISTs) are unique tumors, arising largely due to oncogenic mutations in KIT or PDGFRA tyrosine kinases. Although surgery remains the most effective treatment, the remarkable clinical success achieved with kinase inhibition has made GIST one of the most successful examples of targeted therapy for the treatment of cancer. The insight gained from this approach has allowed a deeper understanding of the molecular biology driving kinase dependent cancers, and the adaptations to kinase inhibition, linking genotype to phenotype. Mutation tailored kinase inhibition with second generation TKI's, and combination immunotherapy to harness the effects of TKIs remain exciting areas of investigation.