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Feasibility of a secretome factory to treat paediatric interstitial lung disease

Project description

A standardised treatment solution for childhood interstitial lung disease

Mesenchymal stem cell (MSC)-based therapies are successful in treating adult patients, yet they remain unapproved for paediatric use. This gap is particularly evident in childhood interstitial lung disease (ChILD), where preclinical studies suggest MSC therapy’s potential but face hurdles like cost, standardisation, and scalability. In this context, the ERC-funded NICHILD project has emerged with a mission to standardise and automate MSC secretome production for paediatric ChILD treatment. Drawing on advancements in isolation techniques, micro scaffolds, and bioreactors, the project aims to provide a reliable, scalable, and standardised solution for ChILD therapy. Specifically, it will develop comprehensive validation of production processes, establish standard operating procedures that are compliant with manufacturing practices, and implement rigorous quality control measures.

Objective

Despite their astonishing clinical success on adults, there are still no mesenchymal stem cell (MSC)-based products approved for paediatric use. Preclinical studies support a potential beneficial role of MSC therapy to prevent progression in diseases called childhood interstitial lung disease (ChILD). However, MSCs still suffer from the cost of culturing and storage facilities and a lack of standardization of cell populations, their quality and quantity, donor-related factors, protocols for isolation, in vitro expansion cell delivery and dosing. Our goal is to standardize and automatize the production of MSC secretome and validate it for its translation to paediatric use to treat ChILD. We will explore the feasibility of a secretome factory based on (1) isolation of MSCs from donor bone marrow, (2) expansion of MSCs in a micro scaffold called nichoid and (3) long-term culture of MSC for secretome production in an optically monitored bioreactor called MOAB-nichoid.
To achieve these goals, in this PoC we will validate the main key performance indicators of the production process and prepare a go-to-market strategy for the secretome biodrug. Our outcomes will be 1) a MOAB-nichoid production line, with total costs compatible with its industrial production and marketing by a pharmaceutical company; 2) standard operating procedures (SOP) for the production process in full compliance with the GMPs in the sector of cell and tissue therapy products (PTC); 3) secretome quality parameters obtained through a comprehensive characterization; and 4) demonstration in vitro of the safety and therapeutic efficacy in the pathologies studied. The outcome of this project will provide a national and international reference point for the industrial production of MSC secretome to treat ChILD and then extending this approach to other pathologies, also affecting adult patients, including chronic intestinal disease and osteoarticular diseases.

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Topic(s)

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HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants

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Call for proposal

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(opens in new window) ERC-2022-POC1

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Host institution

POLITECNICO DI MILANO
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.

€ 80 000,00
Address
PIAZZA LEONARDO DA VINCI 32
20133 Milano
Italy

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Region
Nord-Ovest Lombardia Milano
Activity type
Higher or Secondary Education Establishments
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Total cost

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Beneficiaries (2)

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