Dr Anthony Cook researches age-related neurodegenerative diseases such as Alzheimer’s disease and motor neurone disease. The unifying theme of his research is the study of human cell-based models.

Many researchers use animal models such as fruit flies or mice to investigate human diseases. ‘One of the advantages of studying human cells,’ explains Dr Cook, ‘is that they possess an inherent genetic variability between them that reflects human populations. Animal models of disease don’t capture this variation quite as well.’

‘At the Wicking Centre, I’ve been developing human cell-based models that complement animal-based research. The reason that we need these models is so we can see whether the findings from animal-based research are transferable to humans. If the same responses occur in human cells, then it’s a good sign that the animal model findings could be useful for developing potential therapies.’ Dr Cook notes that his research is a ‘key component of translating bench research to useful new therapies’.

Dr Cook’s laboratory uses induced pluripotent stem (iPS) cell technology. ‘It was developed in Japan,’ he says, ‘and has revolutionised health and medical research.’ Research using iPS cell technology begins with a live person – cells, typically skin or blood cells, are obtained from a donor using ethically approved protocols, and these are ‘reprogrammed’ in the laboratory. ‘This reprogramming creates a cell that we call an induced pluripotent stem cell, which is highly similar to an embryonic stem cell.’

iPS cell technology allows researchers to study brain cells from people living with neurodegenerative diseases. ‘Once we have the iPS cells, we mimic the normal developmental processes of a human cell by introducing biochemical cues in a certain order and timing, to turn them into the cell type we’re interested in. So, if we’re studying the brain, we can turn iPS cells into neurons that we can use to model disease processes.’ Dr Cook explains that iPS cell technology is so powerful because it makes it possible to study nerve cells from large numbers of people with different genetic risk profiles.

Another exciting aspect of his research, says Dr Cook, is another technological advance known as CRISPR/Cas gene editing. ‘We can take the iPS cells from people with gene variants that cause a disease and use CRISPR/Cas technology to correct the gene sequence to that found in people without the disease.’ This produces isogenic cell lines – a pair of cell lines that are genetically identical except for the disease-causing variant. ‘These can be turned into neurons or whatever cell type is needed, and we can study how the gene variant changes the function of those cells.’

Dr Cook entered the field by following opportunities that interested him, many of which had the potential to make a difference to human health. ‘It started with a little bit of serendipity. I enrolled in engineering and found that it wasn’t for me, so I went and did a science degree. Then, having never studied biology at high school, I did first year biochemistry, and remember clearly one of the lecturers saying we can make transgenic animals. This intrigued me and set in motion my whole undergraduate degree in which I studied as much molecular and cell biology as I could, and became involved in a research group through an undergrad lab placement in my summer holidays.

‘UTAS is a great place to be doing biomedical research,’ says Dr Cook. ‘We have a highly engaged population who are genuinely interested in what we do and who are willing to participate in our research. And because the Wicking Centre is part of the College of Health and Medicine, and we are in the one building with both the Menzies Institute for Medical Research and the School of Medicine, there are many opportunities for collaboration.’

Where might this research lead? ‘There is potential for iPS cell technology to provide a pathway to precision medicine,’ says Dr Cook. ‘For example, iPS cells make it possible to conduct drug discovery experiments using nerve cells from many people, to identify drugs that are effective, and to identify those cells that respond versus those that do not. Both the development of new treatments and the possibility of tailoring those treatments on an individual basis mean that iPS cell technology has the potential to improve health outcomes for a range of conditions.’