Although neurodegenerative diseases manifest with markedly different symptoms, they all share the fundamental underlying defect of the degeneration and death of nerve cells (neurons).
The human nervous system is incredibly complex. In neurodegenerative disease, combinations of genetic predisposing factors and/or environmental factors (for instance, diet or exposure to toxins or infectious agents) can trigger harmful processes in the nerves and supporting cells of the brain and spinal cord, and cause the nerves to die.
Great progress has been made in understanding some of the cellular mechanisms behind neurodegeneration, but these advances have yet to be translated into useful therapies. A particular focus of our laboratory research—aiming to bridge this gap between bench and bedside—is in the use of stem cells to model human disease "in a dish".
The promise of stem cells
The brain is composed of two cell types: nerve cells (neurons) that transmit signals and glia that insulate and support neurons as well as enable their function.
Stem cells, whether derived from embryonic or adult cells, can now be grown ("cultured") and manipulated in the lab ("in a dish") and converted into functioning neurons and glia. These provide an inexhaustible supply of cells for imaging, biochemistry, electrophysiology, drug discovery and testing, with the ultimate goal of using them to discover new treatments (cells or drugs) to repair damage. Moreover, the ability to generate neurons from individual patients brings within reach the promise of "stratified medicine", enabling treatment tailored to the stage and subtype of disease.
Into the clinic
Only some of our research takes place at the lab bench. Those undertaking clinical research collate and analyse demographic and clinical data from participating patients (and often their family members too, for use as controls) to gain a deeper insight into the clinical characteristics of the disease, such as the age of onset, symptom range and prognosis. They may also undertake neurological or psychological tests, and importantly, test potential new therapies in structured clinical trials.
Shared mechanisms, shared effort
Because of the common mechanisms shared by many neurodegenerative diseases, research at the Anne Rowling Clinic takes an integrative, multidisciplinary and collaborative approach.
Our work is intimately linked with the world-class research taking place at the various Centres that physically surround the Clinic: the Centre for Neuroregeneration, the Centre for Clinical Brain Sciences, MRC Centre for Regenerative Medicine, MRC Centre for Inflammation Research, Centre for Cognitive Ageing and Cognitive Epidemiology, Euan MacDonald Centre for Motor Neurone Disease Research and Centre for Multiple Sclerosis Research.