Cell Therapy

Cell therapy describes the process of introducing new cells into a tissue in order to treat a disease. Cell therapies often focus on the treatment of hereditary diseases, with or without the addition of gene therapy. Cell therapy is a sub-type of Regenerative Medicine.

Cell therapy, and other regenerative medicines such as tissue engineering, comprise a separate therapeutic platform technology to that of the current three pillars of healthcare: pharma, biologics and medical devices. It is certainly a disruptive technology; however, it is not new. The cell therapy initiative has its origins rooted in blood transfusion, bone marrow and organ transplantation, tissue banking and reproductive in vitro fertilization. Modern cell-based therapies have progressed from the first recorded human–human blood transfusion by James Blundell (Guy’s Hospital, London, UK) through to the advanced cellular therapies of today. This 200 year journey, based initially on clinical trial and error and more recently on laboratory science, has culminated in the necessary critical mass and unique challenges to justify being a distinct industry in its own right. Thus, today cell therapy is the fourth and most recent therapeutic pillar of global healthcare.

Other indicators, including declining market volatility, suggest that a distinct healthcare industry is emerging with strong prospects for growth.

There are many potential forms of cell therapy, reviewed in more detail throughout this article:

• The transplantation of stem cells or progenitor cells that are autologous (from the patient) or allogeneic (from another donor).
• The transplantation of mature, functional cells (Cell Replacement Therapy).
• The application of modified human cells that are used to produce a needed substance (cell-based gene therapy).

These forms of cell therapy are not currently known to exist, but may be possible in future depending on both research outcomes and ethical concerns:

• The xenotransplantation of non-human cells that are used to produce a needed substance. For example, treating diabetic patients by introducing insulin-producing pig cells directly into their muscle.
• The transplantation of transdifferentiated cells derived from the patient's own differentiated cells. For example, the use of insulin-producing beta cells transdifferentiated from isolated hepatocytes as a treatment for diabetes.