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Professor Mackay-Sim is a neuroscientist using stem cells for two purposes: for understanding the biological causes of neurological and psychiatric diseases and for drug discovery. There is a great need for new treatments for brain diseases because the few drugs available treat only symptoms, not causes. In his novel research Professor Mackay-Sim uses patient-derived stem cells, initially, to investigate the cellular and genetic causes of brain diseases, and then, to look for potential drugs that reverse the disease deficits in patient cells. Drugs that treat the “disease-in-a-dish” are then candidates for clinical trials in patients. Professor Mackay-Sim developed a unique cell model: growing adult stem cells from the olfactory mucosa, the organ of smell in the nose. These stem cells normally regenerate the olfactory sensory neurons which get damaged throughout life. Professor Mackay-Sim, as Director of the National Centre for Adult Stem Cell Research, established NeuroBank, a biobank of olfactory stem cells from more than 300 people with various neurological diseases. This resource is unique internationally and includes stem cells from healthy controls and from people with neurological and psychiatric brain diseases including: schizophrenia, Parkinson’s disease, motor neuron disease, ataxia telangiectasia, Hereditary Spastic Paraplegia, mitochondrial mutation disorders, and Alzheimer’s disease. NeuroBank is the foundation for collaborative investigations into all these diseases. This research has led to identification of several drug leads for Parkinson’s disease and a new drug candidate for clinical trials in Hereditary Spastic Paraplegia. Professor Mackay-Sim was scientific director of a clinical trial in which other cells from the nose (called "olfactory ensheathing cells") were transplanted into the injured spinal cord to test their safety in three paraplegic patients. This trial provided a world-first precedent for a recent clinical trial in Poland in which a patient was able to walk after being transplanted with his own olfactory ensheathing cells. Work is progressing to another clinical trial in the Griffith Spinal Injury Project directed by Associate Professor James St John.


  • Member
    Griffith Institute for Drug Discovery, Australia2015


  • Doctor Of Philosophy
    Macquarie University (Sydney)
  • Bachelor of Arts
    Macquarie University (Sydney)