4.3. Emerging and experimental therapeutic uses for HSCs

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


4.3.1. Autoimmunity

4.3.2. Metabolic diseases

4.3.3. Gene therapy

4.3.4. Tolerance

4.3.5. Graft engineering

4.3.6. Future developments

While therapeutic use of HSCs has mostly, and successfully, focused on cancer – and continues to do so – these cells have other applications, too (Figure 4.2). Some diseases, such as severe anaemias, have been treated with HSCT for decades. Treatment options for autoimmune disease and metabolic diseases increasingly include HSCs. Other uses, such as tolerance induction for solid organ transplantation and combination with gene therapy, are under active investigation, but not in routine use. Emerging improvements in the treatment of cancer may focus on more highly purified preparations, especially for autologous transplantation and specific adjuvants, such as T cells aimed at the tumour cells. We will briefly discuss some of these indications and the role of HSC in this section, as many of the future developments of HSC use will focus here.

Principles of Stem Cell Biology and Cancer 4.2

Figure 4.2. Overview of routine and novel indications for the clinical use of HSC transplantation. Current use is heavily focused on the treatment of haematopoietic and other malignancies (left). Grey boxes depict the ratio of transplants performed in each of the three categories. The middle column lists various indications for which HSCT has been used successfully for a longer period of time, but these still represent only a small proportion of the HSCT performed annually: slightly less than 2600 out of more than 50 000 transplants performed worldwide in 2006 (Gratwohl et al., 2010). The right column shows emerging potential uses, for which clinical experience is very limited – sometimes to only a few patients. Similar data have been reported for transplants in the United States in 2010; approximately 5% of the almost 18 000 HSCT performed have been for aplastic anaemia and other nonmalignant diseases (Pasquini and Wang, 2012).

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