Stem cells and drug design

Victorian College of Pharmacy researchers hope stem cells will help them better understand the action of existing drugs and aid in the discovery of new drugs for illnesses such as depression, obesity and anxiety.

While the role of stem cells in transplant technology has sparked much debate in the research and wider communities, stem cells also have unlimited potential as a tool in drug design. And it is a field that researchers from the Victorian College of Pharmacy and the Department of Physiology are keen to further explore.

Professor Colin Pouton, head of the Department of Pharmaceutical Biology and Pharmacology, is part of research team hoping to identify the actions of drugs on human nerve cells (neurons) and the production of neurons from embryonic stem cells. Professor Pouton says the potential of embryonic stem cells in the development of new drugs for conditions such as depression, anxiety and obesity should not be underestimated. This is one of the key areas on which the collaborative research team from Pharmacy and the Faculty of Medicine’s Department of Physiology will focus, with the benefit of $500,000 funding.

The bulk of the funding for the three-year project, titled ‘Neurons isolated from embryonic stem cells as functional models for drug discovery’, came from the Australian Research Council Linkage Project, with a smaller contribution from the research team’s industrial partner, Stem Cell Sciences. The research team also includes Pharmacy’s Dr John Haynes and Dr Rick Lang from Department of Physiology. The project is looking at a range of issues including different ways of reproducing human embryonic stem cells in significant numbers, the study of dopaminergic neurons from the mid-brain, and will attempt to determine how pure neurons react to certain drugs.

“We will be doing something that has never been done before - researching the controlled development of neurons from embryonic stem cells and studying the actions of drugs on the pure neurons,” Professor Pouton said.

The team is currently working with embryonic stem cells from mice, but is hoping to use human stem cells in their research in the near future.

“Working with human embryonic stem cells is technically very difficult – while we can reproduce mouse embryonic stem cells by bathing cells in a growth medium and allowing them to divide freely, we can’t reproduce or grow human cells in large numbers. Once science overcomes this difficulty and can produce embryonic stem cells in a pure form, we will be able to direct the production of human neurons. And that is the key to allowing us to better understand the action of existing drugs and discover new drugs which act on the brain, which is our ultimate aim.”

Professor Pouton says there are many drugs on the market – such as Prozac – that could be refined to work on specific areas of the brain.

“Obesity, schizophrenia, motivation, anxiety and depression are all conditions that could be treated with drugs which are designed to work on selective areas of the brain, so gaining a greater understanding of brain function is extremely valuable,” he said. He cites Parkinsons disease as another example. “If we can isolate, purify and reproduce dopaminergic neurons, which are the cells which degenerate in the disease, these cells could also be used for transplant purposes,” he says. “But to understand illnesses of the brain such as Parkinson’s disease, we first need to research the dopaminergic neurons and their reactions to certain drugs - because it is the degeneration of these neurons which give rise to symptoms of the disease.”