Stem Cells


in the beating of heart muscle made from iPSCs exposed to an anti-cancer compound and duplicat- ed the side-effect previously only seen in patients. The experiment showed that human tissue grown from stem cells can mimic the side-effects of medi- cine that are seen in people. Large pharmaceutical companies are using stem cells to test experimental drugs in an effort to dramatically reduce the cost and risk of the discovery process. More evidence that iPSCs can create heart cells for short-cut drug testing came when the Roche team used them to confirm cardiac toxicity from an antiviral medica- tion that it had been developing.


Umbilical cord blood is a potential vast source of primitive hematopoietic stem and progenitor cells available to reconstitute the hematopoietic system and/or restore immunological function when used as a source for bone marrow transplantation. There are more than 40 private family cord stem cell banks in the US and an estimated 120 addi- tional firms internationally offering these services. Cord Blood Banking is one way to preserve stem cells from diverse populations, which may offer research samples for new medical advancements and drug discovery. Broxmeyer, et al (May 2011 in Blood) evaluated recovery of functional hematopoietic progenitor cells cryopreserved for 20 years. Highly efficient recovery (80-100%) of multipotential hematopoi- etic progenitors was apparent and CD34(+) cells isolated from cryopreserved Cord Blood had engrafting capability in immunodeficient mice reflecting recovery of long-term self-renewal. In this study, functionally responsive CD4(+) and CD8(+) T lymphocytes, generated iPSCs with dif- ferentiation representing all three germ cell lineag- es in vitro and in vivo.


Thus, an additional source of cells that may be used for stem cell applications or converted to iPSCs for personalised medicine lay in the wealth of individual’s cells and tissue stored in both pub- lic and private cell banks. Since the drug discovery and diagnostic technologies that utilise human cells are rapidly evolving, these approaches may allow people to one day have their own cells available to be used in coupled diagnosis, drug screening and personalised treatment of their diseases. Companies, such as Next Healthcare Inc, focus on this leading edge of personalised healthcare by pro- viding tissue and cell storage solutions to individu- als through their physicians.


The outlook for regenerative medicine is bright. Companies are poised to replenish their technolo- gy pipelines with developing stem cell science from research universities and early stage biotech. The


Drug Discovery World Fall 2011


iPS potential


Patient cell


Neural cells


iPS cells


Cardiac cells


Beta cells Hepatocytes


iPS cell uses


Candidate drugs?


genetic screens


Drug or


Transplantation studies (disease in vivo)


Cellular studies (disease in vitro)


stem cell market was estimated to be $21.5 billion globally in 2010 and is forecast to reach $63.8 bil- lion by 2015 – including both tools and therapeu- tics. The knowledge gained in drug development has been integrated with new programmes for cell therapy and combination drug and cell therapies. Many large pharmaceutical companies are devel- oping internal and external regenerative medicine programmes, including Pfizer, Johnson & Johnson, Shire and GlaxoSmithKline. Some of this expan- sion is based on knowledge gained by employing stem cell technology in the drug discovery process; others, such as Shire, are acquiring commercial stage companies, like Advanced Cell Technologies. Stem cells are key to replacing cells lost in degen- erative diseases and for repairing cells in damaged tissue, similar to organ transplants of the past. Stem cells or their differentiated cell products offer a probable and manufacturable source of replace- ment cells to treat diseases including Parkinson’s, stroke, heart disease and diabetes. Many compa- nies are involved in regenerative medicine with more than 1,100 clinical studies under way involv- ing stem cell therapy in the US, Europe and Japan. These cell therapy companies include Aastrom Biosciences, Cellerant, Geron, Cytori, Osiris Therapeutics, StemCells, BioE and ViaCyte. There are more than 200 companies developing stem cell products for the biopharmaceutical industry. Companies that supply the products and services that support research and development, include Life Technologies, Sigma-Aldrich, Cellular Dynamics International, IPierian and ViaCord. Mesenchymal stem cells (MSCs, also known as bone marrow stromal cells or skeletal stem cells) are multipotent stem cells that can differentiate into chondrocytes (cartilage cells), osteoblasts


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