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Updated: Dec 23, 2025
Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Overview of « druggable » alterations by histological subtypes of sarcomas and connective tissue intermediate
Nicolas Penel1, Loïc Lebellec2, Jean-Yves Blay3
1Department of Medical Oncology, Centre Oscar Lambret, Lille, France; Lille University, Medical School, Lille, France.
Abstract:
We summarize herein the literature data about molecular targeted therapies in sarcomas and conjunctive tissue intermediate malignancies. For each clinical setting, the level of evidence, the mechanism of action and the target are described. The two major axes include (i) identification of subgroups of tumors with druggable alteration irrespective of the histological diagnosis (e.g. NTRK), and (ii) druggable target of pathway related to the physiopathology of the tumor: denosumab and bone giant cell tumor, imatinib and soft tissue giant cell tumor, mTOR inhibitor and PECOMA.
Insights
Molecular targeted therapies show promise for sarcomas and connective tissue tumors. Identifying specific genetic alterations (like NTRK) and targeting tumor pathways offers new treatment strategies.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Sarcomas and connective tissue malignancies are rare cancers.
- Treatment options for these tumors are often limited.
- Molecular targeted therapies represent a promising area of research.
Purpose of the Study:
- To review and summarize existing literature on molecular targeted therapies for sarcomas and connective tissue intermediate malignancies.
- To describe the level of evidence, mechanism of action, and targets for these therapies in various clinical settings.
Main Methods:
- Literature review of studies on molecular targeted therapies.
- Analysis of data based on clinical setting, evidence level, mechanism of action, and molecular targets.
- Categorization of therapies based on tumor subgroups with specific alterations and pathway-related targets.
Main Results:
- Identification of actionable molecular alterations (e.g., NTRK fusions) across different histological types.
- Specific targeted therapies discussed include denosumab for giant cell tumor of bone, imatinib for soft tissue giant cell tumor, and mTOR inhibitors for PECOMA.
- Evidence levels and mechanisms of action for each therapeutic approach are detailed.
Conclusions:
- Molecular targeted therapies offer new avenues for treating sarcomas and connective tissue tumors.
- Stratifying patients based on molecular alterations and targeting specific pathways can improve treatment efficacy.
- Further research is needed to expand the application of these targeted approaches.
