Principles of stem cell biology and cancer: future applications and therapeutics. Edited by T. Regad, T. J. Sayers and R. C. Rees. John Wiley & Sons (2015)
Part I. Stem Cells
Autoimmune diseases, which affect 3 – 5% of the general population (Rabusin et al., 2008), are characterized by the recognition and destruction of specific tissues by the immune system. Standard therapy consists of immunosuppressants, which control but do not cure; to achieve a cure, the immune system needs to be eradicated and replaced using an HSCT (Sullivan et al., 2010; Tyndall, 2012). Autologous cells have fewer complications for this purpose, but if a predisposition to a specific autoimmune disease is present, the disease may return. Nevertheless, in some instances, autologous cells can work (Reiff et al., 2011).
To remove the underlying susceptibility to a disease, allogeneic HSCT is necessary. This replaces the susceptible immune system with a nonsusceptible one. However, the morbidity and mortality associated with allogeneic HSCT are higher than for autologous HSCT. Treatment-related complications that are potentially beneficial in cancer treatment, such as low-degree GVHD, are problematic in the treatment of autoimmune disease. It is not easy to justify the benefits of treatment modalities with 2 – 4% short-term mortality, such as HSCT, for diseases with mostly long-term associated mortality. With the advent of next-generation sequencing, it seems increasingly likely that autologous HSCT combined with gene therapy to remove underlying susceptibility from transplanted cells may become a realistic option in the future (Coleman and Steptoe, 2012).