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Decoding and Targeting Treatment-Resistant Metastatic Neuroblastoma

Project description

Understanding treatment resistance in neuroblastoma metastasis

Neuroblastoma (NB) remains a major challenge in paediatric oncology, as relapsed cases often have poor prognoses. Existing models do not accurately replicate relapsed human NB in critical metastatic sites such as the bone and bone marrow. Recently developed patient-derived and humanised in vivo and 3D tumour organoid models have proven to be valuable tools for preclinical drug testing. The ERC-funded Decode Relapse project aims to better understand treatment resistance and relapse in NB metastasis while identifying innovative therapies for resistant and metastatic disease. Specifically, it will develop patient-derived in vivo and ex vivo models of relapsed NB within the metastatic bone marrow niche and expose them to chemotherapy to investigate the mechanisms of metastasis and treatment escape.

Objective

The childhood cancer neuroblastoma (NB) is a major challenge in pediatric oncology, and children with relapse have a very poor prognosis due to treatment-resistance at metastatic sites. There is an urgent need to better understand NB treatment resistance to inform the design of novel therapeutic strategies. However, current models do not mimic relapsed human NB in its most common metastatic niches in the bone and bone marrow. We recently developed advanced patient-derived and humanized NB in vivo models and 3D tumor organoid models which are excellent tools for preclinical drug testing. Here, we aim to further develop and exploit these models, investigate mechanisms of NB metastatic treatment resistance/relapse, and target relapsed NB with combination therapies.

First, we will develop patient-derived in vivo and ex vivo models of relapsed NB in the human metastatic bone marrow niche exposed to standard-of-care chemotherapy treatment. These models will be exploited to investigate mechanisms of metastasis and treatment escape upon therapy and at relapse. We will integrate NB and stromal cell lineages, cell states and molecular details with phenotype and drug response, and elucidate NB tumor cell plasticity and clonal evolution. The mechanistic data and our new models will help us to identify novel therapeutic targets and compounds targeting relapsing and resistant NB, which we will validate experimentally. The project will lead to a deeper understanding of NB metastatic treatment resistance and identification of novel cell state-directed treatments to target resistant and metastatic disease.

My combined background in clinical medicine/pathology, in vivo/ex vivo modeling, NB chemoresistance, and preclinical drug testing coupled with development and application of state-of the-art advanced assays will generate the next generation of patient-derived models, mechanistic insight and novel treatment against relapsed NB.

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Topic(s)

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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-2023-COG

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Host institution

LUNDS UNIVERSITET
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.

€ 2 000 000,00
Address
Paradisgatan 5c
22100 Lund
Sweden

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Region
Södra Sverige Sydsverige Skåne län
Activity type
Higher or Secondary Education Establishments
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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.

€ 2 000 000,00

Beneficiaries (1)

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