Friday, 18th March, 2016
Ewing Theatre, Level 5, Clinical Sciences Building, Royal Melbourne Hospital
Abstract: There is an imperative to develop new cellular models for neurological diseases and conditions. Many large pharmaceutical companies have withdrawn from neuroscience research because of the failure to convert findings in animal models to drugs for human disease. Patient-derived stem cells make good cell models because they carry disease-causing mutations on a background of normal human variation. They can be reproducibly grown and maintained in the lab, in quantity, from patients and healthy controls. Multipotent neural stem cells are found in everyone's nose. Normally they regenerate the sensory neurons of the olfactory organ throughout life. Easily accessible by biopsy, we are using olfactory stem cells to understand the cellular basis of schizophrenia. Automated imaging and analysis of experiments in 96 well format has led us to new drug candidates for Hereditary Spastic Paraplegia.
Bio: Professor Mackay-Sim has had a long interest in regeneration and repair of the nervous system. For many years his passion has been to understand the regeneration and repair of the olfactory mucosa, the organ of the sense of smell in the nose, in which new sensory nerve cells are made throughout adult life. This is a fascinating biological question that has many direct applications to understanding human disease and repairing other parts of the nervous system. Olfactory tissue is easily accessible and provides the scientist and clinician with neural cells and the adult stem cells that give rise to them. In essence the nose provides a "window into the brain" to study cellular processes of disease and also provides adult neural stem cells with the potential to repair the nervous system. Professor Mackay-Sim and his research team are using olfactory stem cells to develop cellular models of diseases such as schizophrenia, Parkinson's disease and motor neurone disease. They have already identified differences in nerve cell regeneration in schizophrenia and bipolar disorder that help understand how these diseases develop. Professor Mackay-Sim is scientific director of a clinical trial in which cells from the nose (called "olfactory ensheathing cells") are taken from the nose of people with paraplegia after traumatic spinal cord injury, grown in the lab, and transplanted into their own injured spinal cord. This trial provides a precedent for future trials using adult stem cells.