Beyond standard therapy: drugs under investigation for the treatment of gastrointestinal stromal tumor
Year: 2015
Type: review
Abstract: INTRODUCTION: Gastrointestinal stromal tumor (GIST) is the most common nonepithelial malignancy of the GI tract. With the discovery of KIT and later platelet-derived growth factor α (PDGFRA) gain-of-function mutations as factors in the pathogenesis of the disease, GIST was the quintessential model for targeted therapy. Despite the successful clinical use of imatinib mesylate, a selective receptor tyrosine kinase (RTK) inhibitor that targets KIT, PDGFRA and BCR-ABL, we still do not have treatment for the long-term control of advanced GIST. AREAS COVERED: This review summarizes the drugs that are under investigation or have been assessed in trials for GIST treatment. The article focuses on their mechanisms of actions, the preclinical evidence of efficacy, and the clinical trials concerning safety and efficacy in humans. EXPERT OPINION: It is known that KIT and PDGFRA mutations in GIST patients influence the response to treatment. This observation should be taken into consideration when investigating new drugs. RECIST was developed to help uniformly report efficacy trials in oncology. Despite the usefulness of this system, many questions are being addressed about its validity in evaluating the true efficacy of drugs knowing that new targeted therapies do not affect the tumor size as much as they halt progression and prolong survival.
Language: English
Subfield: Gastroenterology
Field: Medicine
Domain: Health Sciences
Open Access status: green
Awards: Exploiting Biological Networks to Improve Clinical Treatment of Ovarian Cancer, G3771814487, G4172824049, Fox Chase Clinical Epidemiology and Validation Center, Heartland Institute for Clinical and Translational Research, Integrated Microfluidic Exosome Profiling for Early Detection of Cancer, The Molecular Actions of Imatinib Mesylate in GISTs, San Antonio Center for Biomarkers of Risk for Prostate Cancer (SABOR), Heartland Institute for Clinical and Translational Research, The University of Kansas Cancer Center
OpenAlex ID: W1562782564