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Do sugar-coated proteins feed Ewing sarcoma?

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A zebrafish EWSR1-FLI1 model produced CD99+ small-round-cell tumors with MAPK/ERK on; blocking heparan sulfate with Surfen reduced ERK1/2 signaling and tumorigenicity in vitro and in vivo.

Source

Dysregulated heparan sulfate proteoglycan metabolism promotes Ewing sarcoma tumor growth

Vasileva E, Warren M, Triche TJ, et al. · eLife · 2022

doi.org/10.7554/elife.69734Read the full paper ↗40 citationscc by

Study at a glance

Design
Animal / in-vitro — Cre-inducible human EWSR1-FLI1 zebrafish model of Ewing sarcoma plus in-vitro/in-vivo tests of the heparan sulfate antagonist Surfen
N
N=524 · Survival analysis in 524 EWSR1-FLI1 animals; controls included uninjected N=528, GFP N=328, Cre N=304; tumor incidence also scored in n=77 Cre-injected fish
Population
Wild-type zebrafish embryos/larvae expressing human EWSR1-FLI1, with supporting Ewing sarcoma cell assays
Outcome
SRBCT formation, MAPK/ERK activity, and whether Surfen lowers ERK signaling and tumorigenicity

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What they did

Built Cre-inducible human EWSR1-FLI1 expression in wild-type zebrafish, characterized SRBCTs and ECM/heparan sulfate metabolism, then treated with the heparan sulfate antagonist Surfen in cells and fish.

What they found

EWSR1-FLI1 caused rapid, high-penetrance SRBCTs that expressed canonical targets and CD99. Tumor growth tracked MAPK/ERK activation linked to dysregulated ECM and HSPG catabolism. Surfen reduced ERK1/2 signaling and decreased tumorigenicity. Survival analysis used 524 EWSR1-FLI1 animals vs large control groups.

The limits

What it doesn't show

A zebrafish/cell study cannot yet show that Surfen is a safe human therapy; Ewing remains without an approved molecularly targeted drug from this paper alone.

Key terms

EWSR1-FLI1
Most common Ewing sarcoma fusion oncogene, inducibly expressed here in zebrafish.
SRBCT
Small round blue cell tumor histology characteristic of Ewing sarcoma.
Heparan sulfate proteoglycans
ECM/cell-surface glycoproteins whose catabolism was linked to ERK-driven tumor growth.
Surfen
Small-molecule heparan sulfate antagonist that lowered ERK1/2 and tumorigenicity.
MAPK/ERK
Growth signaling pathway activated in the EWSR1-FLI1 tumors.
CD99
Diagnostic Ewing sarcoma surface marker expressed by the zebrafish tumors.

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The fusion oncogene was:

Common questions

What animal model?

Cre-inducible human EWSR1-FLI1 in wild-type zebrafish.

What tumors formed?

High-penetrance SRBCTs expressing EWSR1-FLI1 targets and CD99.

Which pathway was on?

MAPK/ERK, tied to HSPG/ECM metabolism.

What did Surfen do?

Reduced ERK1/2 signaling and tumorigenicity in vitro and in vivo.

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