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National Centre for Adult Stem Cell Research Stem cell biology from the lab to the clinic Stem cells are at the forefront of biomedical innovation, promising new ways to repair and replace damaged cells and tissues, and new ways to uncover the causes of diseases, leading to new diagnostics and new drugs. Our goal is to understand the biology of adult stem cells for their diagnostic and clinical application in neurological disorders and diseases such as schizophrenia, Parkinson’s disease, motor neuron disease and multiple sclerosis. With more than 40 research staff and students the Centre is part of Griffith University’s Eskitis Institute for Cell and Molecular Therapies, drawing on expertise across the disciplines from stem cell biology to drug discovery. Our research focuses on applications of adult neural stem cell biology in understanding disease, in cell therapy and in drug discovery, drawing on techniques in molecular and cellular neurobiology, systems biology, and bioinformatics. With a strong clinical focus, we use stem cells from people with brain disease to better understand disease aetiology for new diagnostics and new therapies. With expertise in neuroanatomy, neurophysiology and behavioural neuroscience, we use stem cells to discover new cell transplantation therapies to repair the brain and spinal cord. With experience in cell therapy clinical trials our goal is regenerative medicine using the patient’s own stem cells for autologous therapies, avoiding technical and ethical complications of other stem cells sources, including immune rejection. The goal of the National Centre for Adult Stem Cell Research is to advance adult stem cell research by: • Increased understanding of adult stem cell biology; • Developing technologies for adult stem cell production; • Applying adult stem cells to understanding the cause of brain and nervous system diseases; • Applying adult stem cells to preclinical models of nervous system diseases; • Applying adult stem cells to drug discovery and development. stem cells the possibilities Stem cells from the adult nervous system stem cells the science Our research uses the stem cells that continually regenerate the sensory nerve cells of the olfactory mucosa, the organ of the sense of smell in the nose. These cells naturally repair the nervous system and are a source of stem cells to repair the brain and spinal cord and a source of stem cells for investigating diseases of the nervous system. Olfactory stem cells represent a true “breakthrough” in adult stem cell research. They have the ability to develop into many cell types of the body including cells from brain, heart, muscle, liver and kidney. They are easily accessed via the nose using a local anaesthetic, easily grown in the laboratory making them ideal for largescale investigations, transplantations and commercial applications. With an accessible source of adult stem cells and the know-how to find, grow, manipulate, and preserve them, we are at the forefront of adult stem cell research world-wide. Diseases and disorders of the brain and spinal cord Cellular models of disease The National Centre for Adult Stem Cell Research centres on five Clinical Themes: Parkinson’s disease, schizophrenia, motor neuron disease, multiple sclerosis and spinal cord injury which, combined, cost Australia around $4 billion each year in health and social costs, requiring continuing medical treatment and a significant burden on families and carers. These conditions are our focus in applying adult stem cell biology to discover new diagnostics and new treatments. In the same way that breast cancer cells are used to learn about breast cancer, it is possible to use stem cells from people with brain disease to learn about brain disease. The National Centre for Adult Stem Cell Research has over 50 adult stem cell lines from people with Parkinson’s disease, schizophrenia, motor neuron disease, and other diseases. The advantage of adult stem cells is that they can be grown in quantity for multiple assays and can be differentiated into cells of interest to investigate the effect of disease on the different cells of the brain. These patient-derived, disease-specific cell lines can be compared to identify the gene networks and biochemical pathways that contribute to disease and to investigate geneenvironment interactions, such as the role of toxins in the development of brain diseases. Cell transplantation therapies Each year about 250 Australians suffer from spinal cord injury. People with spinal cord injuries usually require continuing highlevel care for the rest of their lives, and suffer complications that shorten their lives by an average of 22 years. There are no effective treatments to repair the injured spinal cord. A specialised cell from the olfactory mucosa, the olfactory ensheathing cell, has proven therapeutic after transplantation in pre-clinical experiments. With its clinical partners, the National Centre for Adult Stem Cell Research has undertaken the first clinical trial of olfactory ensheathing cell transplantation in human paraplegia. In addition to spinal cord injury we are undertaking preclinical experiments of transplantation in Parkinson’s disease, multiple sclerosis and motor neuron disease patients. Images courtesy of Adrian Meedeniya and Brenton Cavanagh New drugs and diagnostics Adult stem cells will provide new cellular models of disease. Stem cells from different patients with the same disease can be compared to identify abnormalities common to all patients, including those with known genetic causes and those with no known cause. Cellular pathways that are commonly dysregulated in disease-specific stem cells, or brain cells derived from them, will provide new biomarkers for disease diagnosis and for new targets for drug therapies. Disease-specific stem cells can also be used in the drug development process for assessing the toxicity of new drug candidates and for assessing their efficacy. stem cells the research Research teams Research training, support and scholarships The research of the National Centre for Adult Stem Cell Research is organised in four Research Groups: Stem Cell Biology, Systems Biology and Bioinformatics, Developmental Biology and Cell Therapies. These teams bring a multidisciplinary and multi-team approach to our basic research on stem cell biology and the clinical research themes. Students are an integral part of research at the National Centre for Adult Stem Cell Research, as part of its program to encourage, educate and train young scientists. Scholarships are offered annually to outstanding PhD and Honours candidates, and vacation scholarships are offered to undergraduate students to work on the Centre’s core research programs. Students have access to all staff and facilities of the Centre and receive support to attend national and international meetings. Collaborations of scientists and clinicians in the Centre keep students immersed in the potential applications of their research in diagnosis and treatment of human disease. Research facilities Our extensive cell culture facility includes Australia’s only robotic system for high throughput cell culture and a unique closed system for stem cell culture. Other cell-based facilities include fluorescence-activated cell sorting, digital microscopy and live-cell imaging. Gene-based facilities include gene expression microarray and genotype analysis. Workflow, patient information and experimental data are strictly managed with a laboratory information management system that feeds into databases linking cell-based and gene-based analyses with patient-derived and quality-control information. The National Centre for Adult Stem Cell Research is part of Griffith University’s Eskitis Institute for Cell and Molecular Therapies, located in a $30 million purpose-built facility funded by the Australian Government and Queensland’s Smart State initiative. The Institute brings together cell and molecular biologists, structural and chemical biologists, and pharmaceutical and medicinal chemists with the goal of discovering new therapies. The Institute is unique in Australia with facilities and expertise specialising in high throughput biology and chemistry for drug discovery and systems biology. Institute facilities include a confocal plate reader for high-throughput cell assays, a high-throughput calcium imaging system, and several robotic cell-free assay systems. The Institute is home to the Queensland Compound Library, Australia’s largest collection of bioactive compounds, and the Queensland node of the Cooperative Research Centre for Cancer Therapeutics. National Centre for Adult Stem Cell Research Eskitis Institute for Cell and Molecular Therapies Griffith University Brisbane Innovation Park Don Young Road Nathan QLD 4111 Australia Telephone: +61 (0)7 3735 4233 Facsimile: +61 (0)7 3735 4255 Email: stemcells@griffith.edu.au www.griffith.edu.au/stem-cells