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Updated: Aug 3, 2025

Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor
Published on: May 2, 2022
Molecular Tailored Therapeutic Options for Advanced Gastrointestinal Stromal Tumors (GISTs): Current Practice and
Fabio Catalano1, Malvina Cremante1, Bruna Dalmasso2
1Medical Oncology Unit 1, IRCCS Ospedale Policlinico San Martino, 16132 Genoa, Italy.
Abstract:
Gastrointestinal stromal tumors (GISTs) are one of the most common mesenchymal tumors characterized by different molecular alterations that lead to specific clinical presentations and behaviors. In the last twenty years, thanks to the discovery of these mutations, several new treatment options have emerged. This review provides an extensive overview of GISTs' molecular pathways and their respective tailored therapeutic strategies. Furthermore, current treatment strategies under investigation and future perspectives are analyzed and discussed.
Insights
Gastrointestinal stromal tumors (GISTs) are mesenchymal tumors with diverse molecular changes. Understanding these mutations has led to new treatments, with this review detailing pathways, therapies, and future directions.
Area of Science:
- Oncology
- Molecular Biology
- Gastroenterology
Background:
- Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal neoplasms of the gastrointestinal tract.
- GISTs exhibit significant molecular heterogeneity, influencing clinical behavior and treatment response.
- Recent decades have seen advancements in understanding GIST-specific molecular alterations.
Purpose of the Study:
- To provide a comprehensive review of the molecular pathways implicated in GIST development.
- To correlate specific GIST mutations with tailored therapeutic strategies.
- To discuss current and emerging treatment options, including future perspectives for GIST management.
Main Methods:
- Literature review of scientific articles and clinical studies on GISTs.
- Analysis of molecular alterations and their impact on GISTs.
- Synthesis of information on targeted therapies and ongoing clinical investigations.
Main Results:
- Identification of key molecular drivers in GISTs, such as KIT and PDGFRA mutations.
- Association of specific mutations with differential responses to tyrosine kinase inhibitors (TKIs).
- Overview of approved and investigational targeted therapies, including imatinib, sunitinib, and ripretinib.
Conclusions:
- Molecular profiling is crucial for personalized GIST treatment.
- Targeted therapies have significantly improved outcomes for GIST patients.
- Continued research into novel therapeutic strategies holds promise for further advancing GIST care.
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