How advanced drug discovery tech is fast-tracking new treatments in Australia

On the same day that the National Drug Discovery Centre was officially launched at WEHI in Melbourne, Australia, the World Health Organization declared COVID-19 a pandemic. 

Within weeks, the NDDC had tested over 400,000 compounds to find starting points for new drugs that could kill the virus. 

It was a revealing moment; before the NDDC, there was no high-powered screening facility in Australia that could work at this speed and scale. 

“At last, we could back the best biomedical breakthroughs from our most brilliant scientific minds – on our shores,” says centre co-head, Associate Professor Jeff Mitchell. 

“From day one, we’ve been empowering Australia’s world-class researchers to help turn their cutting-edge science into new treatments for patients, sooner.” 

Since launching in 2020 – with support from the Australian Government’s Medical Research Future Fund (MRFF), the Victorian Government, WEHI and philanthropic backers – the centre has analysed over 10 million compounds for more than 30 projects working towards new treatments for diseases ranging from COVID-19 and cancer to malaria, endometriosis and neurodevelopmental disorders. 

Funding support from the MRFF and Therapeutic Innovation Australia has been critical, subsidising the cost of screening and enabling researchers across the nation to access the centre’s unique tech and expertise. 

Advancing drug discovery at speed and scale 

Rapid high-capacity screening takes fundamental biological breakthroughs out of the lab and kick-starts the journey towards a new medicine. 

When a scientist identifies a disease target, like a protein that helps cancer spread, the NDDC can run millions of tests to find specific compounds that can block it. 

“It's like searching for – and actually spotting – the tiniest of needles in a massive haystack,” says centre co-head Associate Professor Kym Lowes.  

The centre’s state-of-the-art robotic technologies run at a speed and scale comparable to those used in leading global pharmaceutical companies. 

For WEHI director Professor Ken Smith, it’s a critical missing link. 

“We know the journey from a promising initial screen to the launch of a new, life-saving drug takes many years and significant investment,” he says. 

“But without this first vital step, our most important medical breakthroughs will likely never reach the clinic to help patients.” 

Five years since it launched, advances powered by the NDDC are helping transform medical research in Australia and fast-tracking development of new home-grown treatments. 

Targeting hard-to-treat cancers 

An NDDC-led screen advanced groundbreaking research towards a new drug for a range of difficult-to-treat cancers. 

Specialists from the Queensland Emory Drug Discovery Initiative (QEDDI) working with University of Queensland (UQ) researchers turned to the NDDC to find compounds that target an overactive enzyme present in advanced stages of many cancers. 

The centre uncovered starting points for the development of a first-of-its-kind drug for the treatment of cancers including metastatic prostate cancer, breast cancer, ovarian cancer and colon cancer.  

QEDDI has developed the program and has discovered lead compounds with class-leading potency.   

Hope for neurodevelopmental disorder    

Prader-Willi Syndrome (PWS) is a devastating neurodevelopmental disorder, affecting over 400,000 people globally.   

Hunting for a hit that could be the basis for a future drug for this debilitating condition, the NDDC analysed about 300,000 compounds for the WEHI-led research team. 

The project targets SMCHD1, a protein that controls the expression of genes that are missing or incorrectly switched off in PWS patients. 

Investment from WEHI’s strategic investment fund 66ten and the Prader-Willi Research Foundation of Australia is supporting further development of this potential new treatment.  

New treatment for pulmonary hypertension  

Pulmonary arterial hypertension (PAH) is a rare, incurable and life-threatening condition characterised by high blood pressure in the lungs, which disproportionately impacts women and remains a major global health challenge. 

A groundbreaking drug discovery effort led by researchers at the Monash Institute of Pharmaceutical Sciences (MIPS) is shedding light on potential new treatments for this devastating disease. A recent screen at the NDDC identified promising starting points for novel therapies, now being developed at the Australian Translational Medicinal Chemistry Facility. 

This cutting-edge research targets an inflammatory protein critical to the progression of PAH, a disease that affects about 1 in 100,000 people worldwide. 

The team has been awarded about $500,000 in investment from BioCurate’s Proof of Concept Fund to advance their work, with additional funding of nearly $1 million from the National Health and Medical Research Council and support from Therapeutic Innovation Australia further underscoring the transformative potential of this initiative. 

Next-gen cancer medicines 

Screening at the NDDC identified compounds that are now in development by WEHI spinout company Ternarx to produce a drug that can tag harmful, disease-causing proteins for destruction.  

The first Australian company of its kind, Ternarx is dedicated to developing targeted protein degrader medicines and technology, a powerful new tool for destroying disease-causing proteins that cannot be targeted by conventional drugs.  

The company has an initial focus on neuroblastoma, a childhood cancer that claims more lives of children under five than any other cancer, and prostate cancer, the most commonly diagnosed cancer among Australian men. 

Ternarx is funded through a $15 million grant from the MRFF Frontier Health and Medical Research initiative and is a collaboration between WEHI, the Children’s Cancer Institute and Monash University.   

Better COVID anti-virals 

With COVID-19 a leading cause of death in Australia, more effective antiviral treatments are keenly needed.  

Screening at the NDDC early in the pandemic led to the discovery of compounds that kill the virus, by targeting two proteins that are essential for SARS‑CoV‑2 to replicate. 

The WEHI-led program is now developing a combination antiviral therapy, backed by funding from the MRFF and the Wellcome Trust, with pre-clinical testing showing promising results. 

One of the targeted proteins is linked to the body’s immune response, offering hope of potential benefit for the treatment of long COVID. Funding from the Cumming Global Centre for Pandemic Therapeutics is supporting further development of this research, and its potential application in broad-spectrum antivirals for future pandemics.