Posted: 17 October 2023
Australia has a new frontier technology centre that is set to revolutionise drug development for metastatic prostate cancer and childhood neuroblastoma.
The Australian Centre for Targeted Therapeutics (ACTT) has been awarded $15 million from the Medical Research Future Fund’s Frontier Health and Medical Research initiative to develop next-generation medicines to treat diseases in ways that have been impossible until now.
The majority of the 150,000 Australians diagnosed with cancer every year will be treated with drugs developed over 25 years ago that can cause severe side-effects.
While scientists have uncovered many of the drivers of cancer, about 80% of all disease-causing proteins have been considered “undruggable”.
With new targeted protein degrader technology, researchers can create next-generation cancer medicines as well as treatments for many illnesses that were previously thought to be untreatable.
The ACTT – a collaboration between experts from WEHI, the Children’s Cancer Institute and Monash University – is the first research centre of its kind in Australia, focusing on the development of targeted protein degrader medicines and technology.
WEHI acting director, Professor Alan Cowman AC, said the centre would help kickstart a new biotech industry for Australia, establishing the nation as a leader in this frontier technology.
“Targeted protein degrader technology is one of the most exciting advances in drug discovery and development, with the potential to make a transformative impact,” Prof Cowman said.
“By bringing together a team of leading scientists and clinicians, and establishing a highly skilled workforce, the ACTT will enable Australia to become a major player in this pioneering technology.
“The COVID-19 pandemic revealed a vulnerability in Australia due to our lack of sovereign capability in drug discovery and development.
“The ACTT will help to bridge the critical gap between discovery and translation, a vital investment that will strengthen the nation’s ability to develop innovative drugs against current and future health threats.”
With further investment, the centre has the potential to deliver significant revenue into Australia through co-development and licensing deals.
The MRFF Frontier Health and Medical Research initiative funding will enable research to validate promising drug development targets identified for neuroblastoma and prostate cancer treatments, positioning these research programs for progression towards clinical trials.
The ACTT platform will also be used to broaden the range of proteins – and diseases – that can be targeted by protein degrader technology.
Next-generation treatments for neuroblastoma and prostate cancer
Neuroblastoma is a childhood cancer that claims more lives of children under five than any other cancer. Children diagnosed with neuroblastoma face gruelling treatment, and less than half of those with high-risk disease will survive.
Children who respond to treatment often face life-long side effects including hearing loss, learning difficulties, heart conditions, growth and hormonal deficiencies, infertility and secondary cancers.
Prostate cancer is the most commonly diagnosed cancer among men, and the second most common cause of cancer death for men in Australia. Current treatments for prostate cancer are invasive and involve significant side effects that greatly affect quality of life, but do not eliminate the cancer.
The five-year survival rate for patients diagnosed with advanced disease is about 30%, with more than 3000 Australian men dying from advanced prostate cancer each year.
The ACTT will take advantage of top Australian cancer experts and research to progress targeted protein degrader treatments for neuroblastoma and prostate cancer towards clinical trials.
Professor Michelle Haber AM, Executive Director of Children’s Cancer Institute, said: “Currently there are extremely limited treatment options for children with aggressive neuroblastoma, with current treatment drugs highly toxic and often ineffective.
“Through this unique collaboration and new technology, we now have a very exciting opportunity to develop a different type of treatment for neuroblastoma − one that works by degrading a specific protein, which our research has shown is key to driving this cancer’s growth.
“By targeting and degrading this protein in cancer cells, this new treatment will be far less harmful to healthy cells, meaning that more children survive with fewer side-effects.
“For parents of children with aggressive neuroblastoma this offers hope, where previously survival rates for these children have been dismal.”
Frontier technology to deliver precision medicine
Targeted protein degrader technology is a cutting-edge approach to drug development that has many advantages over traditional methods.
Unlike conventional drugs that only inhibit the activity of disease-causing proteins, targeted protein degrader drugs completely destroy these proteins.
With the potential to target only disease-causing proteins and leaving healthy parts of the body alone, the pioneering technology can deliver precision treatments that do not cause debilitating side effects.
Although the initial focus of the centre will be cancer, the ACTT platform will broaden the range of proteins that can be targeted by protein degrader technology, with the ultimate goal of addressing currently untreatable inflammatory diseases like ulcerative colitis and Crohn’s disease, and neurological conditions such as Alzheimer’s disease, Huntington’s disease and Parkinson’s disease.
Chief investigators at the Australian Centre for Targeted Therapeutics: Professor John Silke (WEHI), Professor Guillaume Lessene (WEHI), Professor Michelle Haber (Children’s Cancer Institute), Professor David Komander (WEHI), Professor Susan Charman (Monash University), Dr Rebecca Feltham (WEHI), Dr Bernhard Lechtenberg (WEHI).
The chief investigators acknowledge the support and guidance of Professor Mark Dawson (Peter MacCallum Cancer Centre and Centre for Cancer Research, University of Melbourne) in the establishment of the ACTT.
