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Updated: May 23, 2026

A Mouse Model of Incompletely Resected Soft Tissue Sarcoma for Testing (Neo)adjuvant Therapies
Published on: July 28, 2020
New therapeutic targets in soft tissue sarcoma
Elizabeth G Demicco1, Robert G Maki, Dina C Lev
1Department of Pathology, The University of Texas M.D. Anderson Cancer Center, Houston, 77030-4009, USA.
Abstract:
Soft tissue sarcomas are an uncommon and diverse group of more than 50 mesenchymal malignancies. The pathogenesis of many of these is poorly understood, but others have begun to reveal the secrets of their underlying mechanisms. With considerable effort over recent years, soft tissue sarcomas have increasingly been classified on the basis of underlying molecular alterations. In turn, this has allowed the development and application of targeted agents in several specific, molecularly defined, sarcoma subtypes. This review will focus on the rationale for targeted therapy in sarcoma, with emphasis on the relevance of specific molecular factors and pathways in both translocation-associated sarcomas and in genetically complex tumors. In addition, we will address some of the early successes in sarcoma-targeted therapy as well as a few challenges and disappointments in this field. Finally, we will discuss several possible opportunities represented by poorly understood, but potentially promising new therapeutic targets, as well as several novel biological agents currently in preclinical and early phase I/II trials. This will provide the reader with the context for understanding the current state of this field and a sense of where it may be headed in the coming years.
Insights
Targeted therapy for soft tissue sarcomas is advancing, driven by molecular classification. This review covers current successes, challenges, and future opportunities in developing novel agents for these rare cancers.
Area of Science:
- Oncology
- Molecular Pathology
- Pharmacology
Background:
- Soft tissue sarcomas (STS) are a heterogeneous group of over 50 mesenchymal malignancies with complex pathogenesis.
- Advances in molecular classification have identified specific alterations driving STS development.
- Understanding these molecular drivers is crucial for developing effective treatments.
Purpose of the Study:
- To review the rationale and progress of targeted therapy in soft tissue sarcomas.
- To emphasize the role of molecular factors and pathways in translocation-associated and genetically complex STS.
- To discuss current successes, challenges, and future directions in sarcoma-targeted therapy.
Main Methods:
- Literature review focusing on molecular alterations, targeted agents, and clinical trial data in soft tissue sarcomas.
- Analysis of translocation-associated sarcomas and genetically complex tumor subtypes.
- Evaluation of preclinical and early-phase clinical data for novel therapeutic targets and agents.
Main Results:
- Molecular classification has enabled the development of targeted agents for specific sarcoma subtypes.
- Early successes in targeted therapy have been observed, alongside notable challenges and disappointments.
- Several promising novel therapeutic targets and biological agents are under investigation.
Conclusions:
- Targeted therapy represents a significant advancement in the management of soft tissue sarcomas.
- Continued research into molecular mechanisms and novel agents is essential for improving patient outcomes.
- The field is evolving, with potential for new breakthroughs in the coming years.
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