Related Experiment Video
Updated: May 16, 2025
Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor
Published on: May 2, 2022
Gastrointestinal Stromal Tumor: Current Approaches and Future Directions in the Treatment of Advanced Disease
Carlo M Cicala1, Sebastian Bauer2, Michael C Heinrich3
1Sarcoma Translational Research Group, Vall d'Hebron Institute of Oncology (VHIO), Carrer de Natzaret 115-117, 08035 Barcelona, Spain.
Abstract:
This review discusses the current and future landscape of advanced gastrointestinal stromal tumor (GIST) treatment. It covers the role of tyrosine kinase inhibitors (TKIs), specifically imatinib, and further treatment options, such as sunitinib, regorafenib, and ripretinib, as well as avapritinib for platelet-derived growth factor receptor alpha D842V mutations. In addition, this review emphasizes individualized treatment strategies within multidisciplinary expert teams, including surgery and other locoregional therapies, together with the importance of mutation-guided approaches, particularly for wild-type GISTs. Finally, it explores the potential of next-generation KIT inhibitors, combination therapies, and other investigational approaches.
Insights
Advanced gastrointestinal stromal tumor (GIST) treatment involves tyrosine kinase inhibitors (TKIs) and personalized, mutation-guided strategies. Future approaches include next-generation KIT inhibitors and combination therapies for improved outcomes.
Area of Science:
- Oncology
- Gastrointestinal Oncology
- Molecular Targeted Therapy
Background:
- Gastrointestinal stromal tumors (GIST) represent a significant challenge in oncology.
- Current treatment paradigms for advanced GIST primarily rely on tyrosine kinase inhibitors (TKIs).
- Understanding the molecular basis of GIST, including specific mutations, is crucial for effective therapy.
Purpose of the Study:
- To review the current and future treatment landscape for advanced gastrointestinal stromal tumors (GIST).
- To highlight the role of various tyrosine kinase inhibitors (TKIs) and mutation-specific therapies.
- To emphasize the importance of individualized and multidisciplinary treatment approaches for GIST.
Main Methods:
- Comprehensive literature review of advanced GIST treatment strategies.
- Analysis of the efficacy of approved and investigational TKIs.
- Discussion of mutation-guided therapy, including for wild-type GIST.
- Exploration of multidisciplinary and personalized treatment planning.
Main Results:
- Imatinib remains a cornerstone TKI therapy for advanced GIST.
- Second- and third-line TKIs like sunitinib, regorafenib, and ripretinib offer options for refractory disease.
- Avapritinib demonstrates efficacy for specific mutations (PDGFRA D842V).
- Mutation status significantly influences treatment selection, especially in wild-type GIST.
Conclusions:
- Personalized, mutation-guided treatment strategies are essential for advanced GIST.
- Multidisciplinary teams are vital for optimizing surgical and locoregional interventions.
- Next-generation KIT inhibitors, combination therapies, and novel investigational approaches hold promise for future GIST management.
