Cellular Therapy of Autoimmune Disease

Autologous bone marrow derived MSCs have been shown to be potently antiproliferative to stimulated T cells from normal subjects and autoimmune (e.g., RA, SSc, Sjögren’s, SLE) patients; in SSc patients, these MSCs were normal in respect to proliferation, clonogenicity, and differentiation to bone and fat.^14,15

Currently, few peer-reviewed publications concerning the results of using of MSCs in human autoimmune disease are available. There are several ongoing phase I/II clinical trials in autoimmune disease including MS, with discussions underway concerning other trials for other autoimmune disease such as type 1 diabetes mellitus, SSc, vasculitis, and SLE.

Important is the setting of clear therapeutic targets and harmonization of cell products, especially MSC source and type (autologous or allogeneic), cell expansion conditions, and trial protocols. In addition, long-term safety data collection across disciplines is required, and an international interdisciplinary registry of MSC-treated patients has been launched.

Summary

Cellular therapy using hematopoietic stem cells to support the ablated normal blood cells has enabled crossing a threshold of immunoablation, and early results suggest that a “resetting” of the immune system in patients with autoimmune disease beyond just immunosuppression is possible. Only phase III randomized trials will establish the true value of this approach. In contrast to the goals of treatment with HSC, MSCs are being used as “homing and healing” cells in various severe inflammatory settings including autoimmune disease, with phase I/II studies beginning. Importantly, no patient conditioning is required. Certainly, for diseases such as SSc where existing immunosuppressive has not been highly effective, stem-cell therapy may offer a fundamentally new approach to correct immune abnormalities and initiate repair. Although much work on stem cells needs to be performed in both the clinical and laboratory, there is strong optimism that a potential new era of therapy is now at hand.

Dr. Tyndall is professor and head of the department of rheumatology at the University of Basel, Felix Platter Hospital, in Basel, Switzerland.

References

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