In 1998 stem cell research hit the headlines when the first paper was published in Science, describing the characteristics of human embryonic stem cell (hESC) line.
Australia has been at the forefront of stem cell science since the discovery of hESCs. Through the pioneering work of Professor Alan Trounson, the establishment of the Australian Stem Cell Centre and more recently the creation of Stem Cells Australia, we have continued to play a leading role in the research into this exciting field.
There are currently many groups around the country carrying out research in three broad areas:
- Research into the biology of stem cells, how they change from embryonic phase through to being specific cell types, what causes them to change and how scientists can be sure that a population of cells is one type of cell or another, which will ultimately be important if the cells are to be used as transplantation material.
- Research into the tools necessary to carry out stem cell research, the scale up of stem cells into large numbers of cells and the creation of quality standards for stem cell creation and care
- Research into disease specific cures using stem cells.
There are two basic types of stem cell: Adult stem cells and embryonic stem cells. More information on these cell types can be found in the About stem cells section.
These lines have been blurred in recent years with the invention of induced pluripotent stem cells (iPS cells). Scientists have discovered that any cell from the body can be placed into an evolutionary ‘reverse gear’ using clever genetic engineering. The result is that any cell in the body can be taken backwards to being an embryonic-like stem cell. In theory, these cells can then be manipulated to form any other cell in the body. This has created a number of exciting opportunities.
Firstly carriers of genetic diseases or afflictions can donate cells that can then be used to model their genetic disease. Work has commenced on many diseases; from well known diseases such as Parkinson’s and Macular Degeneration, through to rare diseases that affect small numbers of patients such as Friedreich’s Ataxia and LOHN.
Secondly, drugs that show promise for treating such diseases can be quickly tested against these cell lines for adverse reactions. Normally this process would take much longer and cost considerably more. This should speed up the pathway of drugs from discovery to the clinic.
Finally the prospect of transplant material sits in the distant future. What is remarkable about iPS cells is that are the patients’ own genetic material and as a result organ or cellular rejection risk is thought to be low.
All of this is achieved without the ethical concerns that use of stem cells created from embryos brings. It is small wonder that this remarkable discovery won its inventor, Shinya Yamanaka, the joint Nobel prize in Physiology or Medicine in 2012.
Research Supported by the Foundation
The first project supported by the Foundation is directed towards a disease specifc research aim using iPS cells.
Centre for Eye Research Australia (CERA)
The NSCFA has recently agreed a two-year program of support with the Centre for Eye Research Australia (CERA). Our support is targeted at the Neuroregeneration Research Unit headed by Dr Alice Pébay and specifically at research on stem cells being undertaken their targeting stem cell treatments for two genetic diseases: Leber’s Hereditary Ocular Neuropathy (LOHN); and Macular Degeneration. CERA are aiming to generate induced pluripotent stem cell lines with material derived form sufferers of those two diseases. These lines can then be used initially to study the diseases and later to screen potential drugs for harmful side effects and ultimately for potential transplant material for sufferers with the long-term hope of reversing the condition.
Dr Pébay’s laboratory will use our support funding to the initial derivation of those lines. The foundation has signed an initial two-year support agreement with CERA with the potential for extension for a further two years.