The concept of using cells for therapy is about as old as the first successful human blood transfusions. However, it is only in the last 10 years that the reality of using cells for therapy and regenerative medicine has come about.
The origins of pharmacy and pharmaceuticals were largely embedded in small molecules and their relatively simple application to treating disease. In fact, the more we explore how the body works, the more we realise something that is really quite obvious: life does not operate just at a simple chemical or molecular level, but also at rather a very complex macromolecular level. So to think that disease could always be treated by simple molecules is, in retrospect, quite naive. Of course, we always had a deep-seated realisation of this, but, until very recently, we did not have the knowledge to apply the power of larger molecules and cells.
The rise of biotechnology over 30 years ago brought an exciting change to medicine. Genetic engineering and gene expression allowed macromolecules such as human growth hormone and insulin to be produced in large quantities, and antibody engineering has opened up the commercial use of larger molecules as therapeutics in areas of unmet clinical need. Furthermore, these breakthroughs fitted the standard business model of the pharmaceutical industry: that is, to discover, develop and sell drugs to treat diseases, either by eliminating their symptoms or by actually curing the ailment.
The rise of stem cell research is about to change medicine forever. Technological breakthroughs, and our understanding of cellular mechanisms across a range of organs, mean that we are truly poised for some exciting times ahead, as witnessed by the recent report of a successful transplant of a tracheal section clothed in cartilage specially cultured from the recipient's own bone marrow stem cells. However, there is one big difference between advances in stem cell research and the rise of biotechnology: most of the funding for stem cell research has not come from the standard pharmaceutical or biotechnology business model sources, i.e. venture capital or the internal budgets of the R&D departments of pharmaceutical companies.
In fact, the biggest funding initiative to date is the establishment of the California Institute for Regenerative Medicine (CIRM) in early 2005, following the passage in late 2004 of Proposition 71 – the California Stem Cell Research and Cures Initiative. This initiative, which provides US$3 B in funding for stem cell research at California universities and research institutions, called for the establishment of a new state agency to make grants and provide loans for stem cell research, research facilities and other vital research opportunities. To date, the CIRM has approved 229 research grants of more than US$614 M, making it the largest source of funding for embryonic and pluripotent stem cell research in the world.
So stem cell research does not fit easily into the standard pharmaceutical or biotechnology business model, and yet it has the greatest potential for curing disease. Funding from traditional sources is unlikely to happen, so who are the funders likely to be? Certainly governments and not-for-profit institutions will continue to fund this research, but other likely sources of funds are the payers of private and national health insurance. Some pharmaceutical companies have already started to make moves in this area, and we await their further involvement with interest.