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Developmentally programmed pediatric sarcomas: a versatile platform for drug discovery and molecular precision medicine

Project description

Mapping to transform paediatric sarcoma research

Paediatric sarcomas represent approximately 20 % of childhood cancers. Despite medical advancements, their survival rates have remained stagnant for the past three decades. This underscores the need for novel approaches in understanding and treating these devastating tumours. With this in mind, the ERC-funded SARCOMAkids project will develop a new approach. Specifically, it will focus on Ewing sarcoma, a developmental cancer driven by fusion oncogenes. The project will construct innovative cellular models from human pluripotent stem cells. By mimicking the developmental origins of these tumours, researchers hope to accelerate drug discovery and precision medicine. Through mapping of oncogene-competent cell states and crafting supportive tumour microenvironments, SARCOMAkids promises to revolutionise our understanding of paediatric sarcomas.

Objective

Pediatric sarcomas account for ~20% of childhood cancers. They have disappointing survival rates, with very little therapeutic progress over the last three decades. We clearly have to rethink the science and find new ways to tackle these devastating tumors. I propose that new cellular models are needed that account for the developmental origins of pediatric sarcoma, in order to accelerate drug discovery and molecular precision medicine.

Focusing on Ewing sarcoma, which is a developmental cancer caused by a (known) fusion oncogene expressed in (unknown) cells-of-origin, we will pursue a “build it to understand it” approach and construct in vitro and in vivo tumor models starting from human pluripotent stem cells (hPSCs). We will validate these models against our single-cell and spatial maps of Ewing sarcoma tumors, and we will pursue initial applications in academic drug discovery – targeting the regulatory programs of Ewing sarcoma cells, metabolic dependencies of the tumor microenvironment and developmentally programmed tumors in their in vivo context.

To build Ewing sarcoma models in a molecular defined manner, we will map oncogene-competent cell states by inducing EWS-FLI1 expression in hPSC-based models of human development (Aim 1). We will create supportive tumor microenvironments by 3D differentiation and CRISPR screening in stromal cells (Aim 2). Finally, we will evaluate the ability of in vitro models to form tumors in mice, and we will pursue full in vivo modeling of Ewing sarcoma using genetically engineered teratomas (Aim 3).

Compared to patient-derived xenografts, organoids or cell lines, our approach captures early events of tumorigenesis, providing complementary in vitro and in vivo models of Ewing sarcoma for biomedical and translational research. This approach will generalize to other tumor types, as it takes the concept of developmental cancers seriously and operationalizes it using cellular programming and high-throughput functional biology.

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HORIZON-ERC - HORIZON ERC Grants

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Call for proposal

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(opens in new window) ERC-2022-COG

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Host institution

ST. ANNA KINDERKREBSFORSCHUNG GMBH
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 1 999 124,00
Address
ZIMMERMANNPLATZ 10
1090 Wien
Austria

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Region
Ostösterreich Wien Wien
Activity type
Research Organisations
Links
Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

€ 1 999 124,00

Beneficiaries (1)

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