FAQ FREQUENTLY ASKED QUESTIONS ABOUT STEM CELL RESEARCH Can Nephrogenix do anything for me now? The research and development into cellular therapies for renal disease being performed by the Renal Regeneration Consortium is currently very early stage. With the rate of chronic kidney disease increasing by 6-8% per annum, there is a great need for alternative treatment options to be developed. However, there are currently only two treatment options for patient with end stage renal disease - dialysis and transplantation. In the long term, we hope to be able to repair or completely regenerate damaged kidneys using stem cells. What is a stem cell and what might we be able to do with them? A stem cell is a cell which has the ability to not only replicate itself many times, but can also generate daughter cells which turn into other more specialised cell types. Our bodies contain many stem cells and without stem cells we would not be able to live day to day. While very rare, there are stem cells in our bone marrow continually making new red and white blood cells; stem cells in our skin constantly replacing the surface of our bodies; stem cells in the liver that can respond to liver damage; and stem cells in our intestine turning over the lining of our guts. These adult stem cells persist throughout our lives but are limited in the types of specialised cells they can turn into. For example, a stem cell in the crypt of your gut can only turn into epithelial cells of the gut, while dermal stem cells can only replace skin. Such stem cells are referred to as multipotent. In constrast, during development there exist stem cells with an ability to turn into any cell type. Embryonic stem cells are referred to as 'pluripotent'. One type of embryonic cell type that is pluripotent is the cells of the inner cell mass of the embryo before it implants into the uterus wall and begins its life as a fetus. Cells harvested from this timepoint are referred to as embryonic stem cells and it is these cells that researchers are hoping may be able to be used to repair or regenerate damaged organs. The advantage of these cells is the wide variety of cell types that can be made from these cells and the ability to grow them up to very large numbers in culture for delivery to the patient. However, it is not legal to use the cells of the patient to derive new embryonic stem cells - the process called therapeutic cloning - and therefore a therapy using ES cell lines would require careful matching and immunosuppression. It has been stated that 'adult' stem cells, while very rare and often not easily identified or isolated, will be a better alternative given that they could be harvested from the patient requiring treatment. There is also evidence that the number and variety of cell types that an adult stem cell can become may be greater than originally thought, suggesting that we may be able to harvest more readily accessible adult stem cells and turn them into the cell type we need replaced. Each of these alternatives has advantages and disadvantages. To date, it is not possible to say which of these approaches is going to be feasible. What sort of stem cells will be used by Nephrogenix for the treatment of kidney disease? Researchers at Nephrogenix will be investigating both embryonic and adult stem cells for use in cellular therapy. We believe that it is not yet possible to be confident that one or other approach will be successful, and hence both need to be researched. Our current studies using embryonic stem (ES) cells employ ES cell lines approved for use by the National Institutes of Health, USA. None of these cell lines can be used for the treatment of humans. They are purely research tools to evaluate the feasibilty of the approach. Nephrogenix does not intend to derive new ES cell lines, nor do we advocate the use of therapeutic cloning. Should we be using embryonic stem cells to develop cures for diseases? Some groups are opposed to the derivation of embryonic stem cell lines from discarded embryos originally harvested for in vitro fertilisation. Recent surveys of the Australia population conducted for Biotechnology Australia found that 82% of the population support stem cell science but, perhaps unrealistically, expect it to improve our way of life in the next five to ten years. In addition, three quarters of Australians (73%) agree that 'Embryonic stem cells left over from IVF treatments should be made available for research purposes'. The ES cell research being performed by scientists within the Renal Regeneration Consortium is being performed in accordance with national and international ethical guidelines.