Stem cell therapy — rheumatoid arthritis

RA is an inflammatory condition that affects 1% of the general population worldwide. Current management measures involve the use of synthetic or biological disease modifying anti-rheumatic drugs (DMARDs).

Preclinical work performed by the consortium demonstrated the potential of using allogeneic mesenchymal expanded adult stem cells from adipose tissue (eASCs) as a treatment for RA. The EU-funded REGENER-AR project translated this work to the clinic by conducting a multi-centre clinical trial.

MSCs are multipotent stem cells found in most tissues such as bone marrow and fat. The consortium has undertaken further investigation into the mechanism of eASC action in RA. Particular attention to the immune-modulating properties of MSCs has validated that certain functions of eASCs can be protective against RA.

Studies to determine how these allogeneic cells behave once administered were performed in a mouse model of RA. Scientists have also investigated the in vivo biodistribution and the efficacy effects of these cells, alongside alternative routes of administration (i.e. the intralymphatic route).

In the phase IIa clinical trial – which has been successfully completed – 53 refractory RA patients have been treated with different doses of eASCs and monitored for six months. The results of this first-in-man treatment of RA with eASC showed the safety of the treatment and gave a first positive indication of the potential efficacy of cell therapy with eASC in RA.

For the clinical application of this therapy, the eASC manufacturing process needs to be scaled up. For this purpose, partners have been working to find the optimal conditions of eASC expansion in bioreactors and to automate the process.

Project outcomes have demonstrated the safety and preliminary efficacy of eASC therapy on RA patients. This should pave the way for the further clinical development of this innovative treatment. Given the immunomodulatory properties of eASCs, this stem cell therapy could find also application in other immune-related disorders.