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Updated: May 2, 2026

In Vivo Model for Testing Effect of Hypoxia on Tumor Metastasis
Published on: December 9, 2016
Targeted therapies for advanced Ewing sarcoma family of tumors
Yunyun Jiang1, Joseph Ludwig2, Filip Janku1
1Department of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Abstract:
The prognosis of adolescent and young adult patients battling metastatic Ewing sarcoma family of tumors (ESFT) remains less than 30% despite the development of systemic therapies. In the era of personalized medicine, novel molecular targets have been tested in preclinical or clinical settings in ESFT. In this review, we focus on early clinical and translational research that identified multiple molecular targets, including IGF-1R; mTOR; tyrosine kinase inhibitors; EWS-FLI1-related targets, and others. Overall, novel targeted therapies demonstrated modest efficacy; however pronounced and durable antineoplastic responses have been observed in small subsets of treated patients, for example with IGF-1R antibodies. Identifying outcome-predicting biomarkers and overcoming treatment resistance remain major challenges. Due to the rarity of ESFT, multi-institutional collaboration efforts of clinicians, basic and translational scientists are needed in order to understand biology of therapeutic response or resistance, which can lead to development of novel therapeutic methods and improved patient outcomes.
Insights
Novel targeted therapies show modest efficacy for metastatic Ewing sarcoma family of tumors (ESFT), with some patients experiencing durable responses. Biomarker identification and overcoming resistance are key challenges for improving outcomes in this rare cancer.
Area of Science:
- Oncology
- Translational Research
- Molecular Biology
Background:
- Metastatic Ewing sarcoma family of tumors (ESFT) has a poor prognosis, with survival rates below 30% despite systemic therapies.
- Personalized medicine approaches are exploring novel molecular targets for ESFT treatment.
Purpose of the Study:
- To review early clinical and translational research on novel molecular targets in ESFT.
- To summarize the efficacy and challenges of targeted therapies for ESFT.
Main Methods:
- Literature review of preclinical and clinical studies.
- Focus on identified molecular targets including IGF-1R, mTOR, tyrosine kinase inhibitors, and EWS-FLI1-related targets.
Main Results:
- Novel targeted therapies demonstrated modest overall efficacy in ESFT.
- A subset of patients showed pronounced and durable antineoplastic responses, notably with IGF-1R antibodies.
Conclusions:
- Identifying predictive biomarkers and overcoming treatment resistance are critical challenges.
- Multi-institutional collaboration is essential to advance understanding and develop improved therapies for rare ESFT.
- Further research is needed to enhance patient outcomes for ESFT.
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