Our Strategic Programs
While Garvan's research has already had impact, we now seek to expand and scale that impact profoundly – so that Garvan is the source of life-changing impacts for patients across Australia and around the world.
Our Strategic Programs are our flagship strategies for impact. The Programs bring together multiple Labs across Garvan, along with partners in other organisations. They focus on an area of science where Garvan has distinctive strengths and critical mass. The Programs are actively focused on taking research discoveries all the way through to clinical impact, to improve health and change lives.
Using genomics to accelerate diagnosis, treatment and management of rare diseases
Rare diseases are individually rare but collectively common, and directly impact around 300 million people worldwide, including two million Australians. Rare diseases can affect any of the body’s systems, and can have severe and devastating impacts on health and quality of life.
One major challenge for individuals living with a rare disease is obtaining a timely and accurate diagnosis. For some, this is straightforward – but for most, clinical genetic testing is a dead end. Hundreds of thousands of Australians wait 5 years or more for a diagnosis, and most are never diagnosed at all. Without a diagnosis, these individuals are unable to access effective treatments, or understand how their disease will progress, or even know whether other family members are at risk.
A second big challenge is treatment. Even with a genetic diagnosis, only a tiny fraction (5%) of patients currently have an effective targeted treatment. So there is also an urgent need to develop new rare disease therapies, and to ensure that as many patients as possible can benefit.
Led by Associate Professors Owen Siggs and Jodie Ingles, the Garvan Rare Disease Program is tackling the twin challenges of diagnosis and treatment, to make a genuine difference to the lives of rare disease patients and their families. To do this, we’re building on Garvan’s deep expertise in genomic research. Our researchers and clinicians include leading experts in diseases of the immune system, heart, eyes, kidney, and brain. We work with clinical and technical experts, at Garvan and beyond, all to help realise our vision of quickly and accurately diagnosing, and effectively treating, every patient with a rare genetic disease.
Unpacking disease through the cell and the genome
Cancers, autoimmune diseases, neurological conditions, respiratory conditions, most cardiovascular conditions, and more: these are all ‘complex diseases’, with many contributing genetic and non-genetic factors. Together, complex diseases are by far the biggest contributor to disease burden worldwide – yet they are immensely challenging to study at the level of our genes and our cells.
The vision of the Genomics-led Drug Discovery Program is to understand how our genetic risk for complex diseases acts at the level of our individual cells – and to use this information to develop next-generation therapies that improve the health of millions living with complex disease.
Powering up the immune system’s cells
Immune-related diseases – including Infectious disease, allergy and autoimmunity – pose a significant global health burden. The COVID pandemic has killed millions worldwide; allergies affect 1 in 5 Australians; and autoimmune diseases are chronic health problems that can cause disability and lost income not just for patients but also their families.
The vision of the Precision Immunology Program is to transform the treatment of diseases affecting the immune system through precision immunology. We will reframe understanding of immune disorders, with the goal of developing new and effective precision treatment and prevention approaches.
Targeting the dynamic cellular and molecular environment that supports cancer cells
A cancer’s development, growth, spread and drug response is strongly influenced by its local surroundings. The Cancer Ecosystems Program translates state-of-the-art discoveries in the cancer microenvironment into tomorrow’s cancer drugs and diagnostics.
The Cancer Ecosystems Program understands that cancer doesn't develop in isolation. It interacts closely with its microenvironment to create a dynamic ecosystem of tumour, immune, and other cells. This ecosystem has the power to make or break a cancer by sending signals that support tumour growth and spread, by protecting the cancer from therapeutic agents or by suppressing the local immune response. That's why our Program is taking a new view on tumour targeting, using the most advanced methods to transform our understanding of where tumours live and how they grow.
Our team is finding new biomarkers and new targets, allowing us to develop and implement new tests and treatments that can make a difference for cancer patients. Our focus areas are breast, prostate, and pancreatic cancer, and we have large numbers of uniquely valuable samples from patients with those types of cancer. For example, Program members lead the Avner Australian Pancreatic Matrix Atlas (APMA ), the Garvan-St Vincent's Prostate Cancer group bio- and data bank with over 15,800 patients, and the Garvan Breast Cancer Atlas cohorts of curated high-value samples. These cohorts include untreated and treated patients, as well as longitudinal samples from the same patients.
We draw on the international leadership of our members in the fields of single-cell and spatial transcriptomics, matrix and spatial proteomics, epigenetics, computational modelling and immunology to discover new targets for cancer treatment. We use state-of-the-art intravital imaging approaches to visualise disease processes dynamically. This live-imaging, along with study of our longitudinal samples, illuminates the effects of time and treatment on cancer. Potential therapeutic targets are validated using our team expertise in a range of preclinical biological tests including in vivo disease models , patient-derived breast tumour xenografts and models of drug resistance.
We partner with the Garvan Centre for Targeted Therapy and with industry to develop new tests and treatments, which can be clinically implemented via our partnerships with St Vincent’s Hospital, The Chris O'Brien Lifehouse, New South Wales Early Phase Clinical Trials Alliance, and others. Our goal is to develop new, more effective ways to target cancer and make a real difference for patients.
Precision medicine for cancer
There are currently few, if any, treatment options for cancers that are resistant to chemotherapy and immunotherapy. For patients with these cancers, lifespan and quality of life are dramatically decreased.
The vision of the Dynamics of Cancer Resistance Program is to drive the bench-to-bedside translation of new precision medicine therapies. Anchored in Garvan’s outstanding discovery research in treatment-resistant cancer, we will identify the next wave of cancer drug targets – and we’ll facilitate and enable rapid clinical translation of cutting-edge research to provide patients with access to new treatment strategies for difficult to treat cancers.
Preventing fractures in individuals with osteoporosis and cancers that grow in bone
Bone fragility fractures are a major contributor to the burden of musculoskeletal disease worldwide. Fractures have devastating consequences in patients with osteoporosis and common cancers that have spread to bone, including a heightened risk of premature death. Leveraging Garvan’s unique and world-leading expertise in bone pharmacology, epidemiology, and bone and cancer biology, we will tackle specific challenges that, when overcome, will have a global impact in reducing the risk of fractures and lessening their adverse outcomes.
Bone fragility fractures affect over one quarter of older people, and worldwide, musculoskeletal diseases represent the fourth greatest burden of disease behind only heart disease, cancer and mental health disorders.
Fractures are also a major complication in patients with common cancers, which spread to bone and then proliferate in the bone microenvironment. The skeleton is also a site of tumour cell dormancy, where cancer cells hide in a long-term ‘dormant’ state and resist current therapies. When they are ‘woken up’, they cause cancer relapse, at which point prognosis is poor.
Currently, awareness of skeletal health, particularly osteoporosis and cancer-associated bone disease, is poor and we have little understanding of who will develop disease and when, and treatments are still limited.
The Skeletal Diseases Program will tackle these clinical challenges and fundamentally alter the landscape of bone health in Australia and worldwide. Our research seeks to develop personalised approaches to improve outcomes following osteoporotic fractures, identify and develop new therapeutic ways to improve bone quality and rebuild new bone, overcome barriers to the uptake of existing anti-osteoporosis medications, and prevent cancers from growing in the skeleton.
We will take a multi-pronged approach – encompassing cellular, genomic, implementation and clinical sciences – and engage a variety of internal and external resources, technical facilities, scientific and strategic expertise (including health economics, consumers and policy makers) to develop personalised approaches to the clinical management of bone disease.