Stem cell treatment for patients with autoimmune disease by systemic infusion of culture-expanded autologous adipose tissue derived mesenchymal stem cells

Journal of Translational Medicine


Ra et al. Journal of Translational Medicine 2011, 9:181

Jeong Chan Ra1*, Sung Keun Kang1, Il Seob Shin1, Hyeong Geun Park1, Sang Aun Joo1, Jeong Geun Kim2, Byeong-Cheol Kang3, Yong Soon Lee4, Ken Nakama5, Min Piao6, Bertram Sohl7 and Andras Kurtz8*



Prolonged life expectancy, life style and environmental changes have caused a changing disease pattern in developed countries towards an increase of degenerative and autoimmune diseases. Stem cells have become a promising tool for their treatment by promoting tissue repair and protection from immune-attack associated damage. Patient-derived autologous stem cells present a safe option for this treatment since these will not induce immune rejection and thus multiple treatments are possible without any risk for allogenic sensitization, which may arise from allogenic stem cell transplantations. Here we report the outcome of treatments with culture expanded human adipose-derived mesenchymal stem cells (hAdMSCs) of 10 patients with autoimmune associated tissue damage and exhausted therapeutic options, including autoimmune hearing loss, multiple sclerosis, polymyotitis, atopic dermatitis and rheumatoid arthritis. For treatment, we developed a standardized culture-expansion protocol for hAdMSCs from minimal amounts of fat tissue, providing sufficient number of cells for repetitive injections. High expansion efficiencies were routinely achieved from autoimmune patients and from elderly donors without measurable loss in safety profile, genetic stability, vitality and differentiation potency, migration and homing characteristics. Although the conclusions that can be drawn from the compassionate use treatments in terms of therapeutic efficacy are only preliminary, the data provide convincing evidence for safety and therapeutic properties of systemically administered AdMSC in human patients with no other treatment options. The authors believe that ex- vivo-expanded autologous AdMSCs provide a promising alternative for treating autoimmune diseases. Further clinical studies are needed that take into account the results obtained from case studies as those presented here.



Human AdMSC can be isolated from small amounts of adipose tissue, efficiently expanded to achieve more than 109 cells after 3 to 4 passages independent on donor age and disease status. The sustained potency and genetic stability of the cells make adipose tissue a very attractive source for multipotent cells. Their immun-modulatory function, homing and migratory patterns as well as previous clinical trials suggest that these cells are efficient for treatment for several classes of autoimmune diseases and their application is safe. Here, we demonstrated considerable therapeutic effects of culture- expanded autologous AdMSCs in a variety of autoimmune diseases in the frame of an ethically justified compassionate use application for patients with exhausted therapeutic options. Multiple intravenous infusions of cells resulted in clinical benefit in all treated patients in the follow up period. No adverse events were observed. The data provide first evidence for clinical benefit in autoimmune diseases, yet further scrutiny in controlled clinical trials with a sufficient numbers of patients are needed to draw a definitive conclusion on therapeutic efficacy and long term benefit. Importantly, the data show that multiple AdMSC infusion of up to 1 × 109cells in a period of less than one month is safe, corroborating data from preclinical and clinical trials using BMMSC and AdMSC. Furthermore, within this small sample size, no evidence of donor age-dependent efficacy, or age dependent in vitro cell expansion rate was found. The autologous stem cell application described.

here is based on the current state of the art and provides an outlook into treatments for patients suffering from a variety of incurable autoimmune related diseases with no remaining treatment options. While it is shown here that the technology for treatment of autoimmune using autologous AdMSC is in place and the expectations derived from preclinical studies can be confirmed, there is still a limited understanding of the modes of action. In conclusion, the systemic infusion of autologous stem cells described here offers promise for better management of a wide spectrum of autoimmune diseases, independent on patient’s age.

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