Feasibility of a secretome factory to treat paediatric interstitial lung disease

Introduction

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).

Challenges in MSC Therapy

However, MSCs still suffer from several challenges, including:

• The cost of culturing and storage facilities
• A lack of standardization of cell populations
• Variability in their quality and quantity
• Donor-related factors
• Protocols for isolation and in vitro expansion
• Cell delivery and dosing

Project Goals

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
3. Long-term culture of MSC for secretome production in an optically monitored bioreactor called MOAB-nichoid

Validation and Strategy

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
4. Demonstration in vitro of the safety and therapeutic efficacy in the pathologies studied

Expected Outcomes

The outcome of this project will provide a national and international reference point for the industrial production of MSC secretome to treat ChILD. Additionally, this approach may be extended to other pathologies, also affecting adult patients, including chronic intestinal disease and osteoarticular diseases.