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Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor
Published on: May 2, 2022
New treatments in advanced gastrointestinal stromal tumor
1Department of Medical Oncology, Vall d'Hebron University Hospital.
Purpose Of Review:
The current article revisits the most recent advances that occurred in the field of gastrointestinal stromal tumor (GIST) therapeutics.
Recent Findings:
GIST is driven by the oncogenic activation of KIT or PDGFRA receptor tyrosine kinases, and agents targeting these receptors lead to substantial benefit throughout the entire course of the disease. Two new drugs were approved in 2020. On one hand, ripretinib obtained the regulatory approval for the treatment of GIST patients after progression to all standard treatments. On the other hand, avapritinib became the first agent ever displaying activity in GIST driven by the multiresistant PDGFRA D842V mutation. The addition of both drugs to GIST therapeutics constitutes a remarkable milestone, particularly considering that the last agent approved was back in 2012. Similarly, the recent identification of neurotrophic tyrosine receptor kinase (NTRK) fusions in a subset of KIT/PDGFRA wild-type GISTs led to an open window for tailored treatment using specific NTRK inhibitors. Finally, multiple efforts have been made toward the clinical implementation of circulating tumor DNA evaluation to guide clinical decisions in GIST.
Summary:
GIST has been consolidated over the years as a paradigmatic model in personalized medicine for the successful development of novel therapeutic strategies through targeted inhibition of oncogenic drivers.
Insights
Recent advances in gastrointestinal stromal tumor (GIST) therapeutics include new drugs like ripretinib and avapritinib, offering hope for patients with advanced or resistant GIST. Targeted therapies for NTRK fusions and circulating tumor DNA evaluation are also emerging.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Gastrointestinal stromal tumors (GIST) are driven by specific oncogenic mutations in KIT or PDGFRA receptor tyrosine kinases.
- Targeted therapies have revolutionized GIST treatment, offering substantial benefits throughout the disease course.
Purpose of the Study:
- To review recent therapeutic advances in gastrointestinal stromal tumor (GIST) treatment.
- To highlight novel drug approvals and emerging therapeutic strategies for GIST.
Main Methods:
- Review of recent clinical findings and drug approvals in GIST therapeutics.
- Analysis of targeted therapies for specific GIST mutations and fusions.
- Discussion of the role of circulating tumor DNA (ctDNA) in GIST management.
Main Results:
- Two new drugs, ripretinib and avapritinib, were approved in 2020 for GIST treatment.
- Avapritinib shows efficacy in GIST with the challenging PDGFRA D842V mutation.
- NTRK fusions identified in a subset of GISTs allow for tailored treatment with NTRK inhibitors.
- Clinical implementation of ctDNA evaluation is being explored to guide GIST treatment decisions.
Conclusions:
- GIST serves as a model for personalized medicine due to successful targeted therapies.
- Recent therapeutic advancements, including new drug approvals and identification of novel targets, mark significant progress in GIST management.
- The integration of ctDNA analysis holds promise for guiding personalized treatment strategies in GIST.
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