Project description
Stem cell technology for the treatment of osteoarthritis
Osteoarthritis is a common type of arthritis associated with a breakdown of cartilage in joints. It is the most common chronic joint condition, affecting more than 40 million people in Europe alone. As there are limited treatment options available, osteoarthritis imposes a huge burden on healthcare systems. The goal of the EU-funded AutoCRAT project is to develop novel sustainable cell and cell-derived therapies for osteoarthritis. The project will use human-induced pluripotent stem cells for generation of articular chondrocytes for cartilage repair and human-induced mesenchymal stem cells for disease prevention and for the treatment of established disease. As an extended goal, the project will investigate potential application of the mesenchymal stem cell secretome as a next-generation therapy and will produce the therapeutics identified in the project using cost-effective robot-enabled processes in a novel manufacturing platform to expedite translation to patients.
Objective
AutoCRAT will develop novel sustainable cell and cell-derived therapies for osteoarthritis (OA). Osteoarthritis, a major disease, results in degradation of cartilage. It is the most common chronic joint condition, currently affecting >40 million Europeans. As no effective treatments for OA are available, it imposes a huge burden on the individual patient and European health care systems. Adult mesenchymal stem/stromal cells (MSCs) show early, promising clinical treatment efficacy in OA. However, costs of MSC production prevents widespread use for patients. Using human induced pluripotent stem cells (hiPSC), AutoCRAT will generate articular chondrocytes (hiCHOs) for cartilage repair to prevent development of OA and hiMSCs to treat established disease as sustainable therapeutic cell sources for therapies. Discovery of the osteoarthritis-specific hiMSC secretome will enable further refinement of these next-generation therapies; this will be achieved by molecular analysis of:
1) hiMSCs treated with synovial fluid from OA patients
2) EVs derived from these therapeutically-activated cells to develop the therapeutic osteoarthritis secretome.
To ensure sustainable supply of high quality, affordable products, AutoCRAT will also develop a manufacturing pipeline composed of automated, regulatory-compliant robotic systems for production of the developed hiCHOs and hiMSCs and cell-secreted extracellular vesicle (EVs). The manufacturing pipeline can be extended to many other major diseases. The AutoCRAT consortium is composed of leading European scientific teams in stem cell biology, OA, preclinical efficacy and safety demonstration, GMP- and GAMP-compliant manufacturing of MSCs, regulatory affairs and health economics analysis. All aspects of AutoCRAT discovery and translation innovations will be guided by the imperative to deliver economically viable and regulatory-compliant therapies to OA-patients, the European health care system at large, and benefits to the European economy.
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Funding Scheme
RIA - Research and Innovation actionCoordinator
H91 Galway
Ireland