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Review
. 2016 Aug 25:5:F1000 Faculty Rev-2077.
doi: 10.12688/f1000research.8631.1. eCollection 2016.

Recent advances in targeted therapy for Ewing sarcoma

Kathleen I Pishas  1 , Stephen L Lessnick  2
Affiliations
Review

Recent advances in targeted therapy for Ewing sarcoma

Kathleen I Pishas et al. F1000Res. .

Abstract

Ewing sarcoma is an aggressive, poorly differentiated neoplasm of solid bone that disproportionally afflicts the young. Despite intensive multi-modal therapy and valiant efforts, 70% of patients with relapsed and metastatic Ewing sarcoma will succumb to their disease. The persistent failure to improve overall survival for this subset of patients highlights the urgent need for rapid translation of novel therapeutic strategies. As Ewing sarcoma is associated with a paucity of mutations in readily targetable signal transduction pathways, targeting the key genetic aberration and master regulator of Ewing sarcoma, the EWS/ETS fusion, remains an important goal.

Keywords: EWS/FLI; Ewing sarcoma; ganitumab; sarcoma.

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Conflict of interest statement

KIP declares that she has no competing interests. SLL is the Acting Chief Medical Officer for Salarius Pharmaceuticals, LLC, (Salt Lake City, UT). No competing interests were disclosed. No competing interests were disclosed.

Figures

Figure 1.
Figure 1.. Chemical structures of Ewing sarcoma investigational agents.
Figure 2.
Figure 2.. PARP-1 is highly expressed in Ewing sarcoma tumors and cell lines.
( a) Broad Institute PARP-1 expression across a panel of 1,036 cell lines. ( b) PARP-1 expression (microarray) in Ewing sarcoma tumors and cell lines. ( c) PARP-1 expression is not correlated with overall or event-free survival in Ewing sarcoma (n = 20). Survival data sourced from Ohali et al. (2004). PARP-1, poly ADP ribose polymerase 1.
See this image and copyright information in PMC

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