Drug discovery

Drug discovery

Scientist using screening technology
Drug discovery research seeks to understand how disease develops at a molecular level, identifying ‘targets’ for analysis. Drug discovery uses the identified molecular target to test drug-like chemicals, and realise disease impacts. The testing and realisation is the initial step in the drug discovery process. 

Rational design and scientific validation improve the properties of these drug-like ‘hits’ to create therapeutic drugs ready to treat disease in patients. The path of drug discovery, from initial understanding, through testing and development of a drug is referred to as the drug discovery pipeline.

What is high throughput screening?

High throughput screening is a gold standard for discovering ‘hits’ during the early stages of drug discovery. The technology uses automation to test hundreds of thousands of drug-like chemicals against molecular targets. The ‘hits’ discovered using high throughput screening provide a starting point for the development of new drugs.

Collaboration is key

The journey from scientific discovery to drug treatment is gradual. The development of anti-cancer drug venetoclax and clinical trials were preceded by two decades of research, involving a number of researchers and organisations, including Genetech, a member of the Roche Group, and AbbVie.

Early drug discovery requires advanced robotics and expert scientists to convert breakthroughs in biology to new medicines for disease treatment. We are working in collaboration with Australia’s medical research organisations to capitalise on our scientific discoveries and build our capability for delivering early drug discovery.

Case study: a new compound to kill malaria

In 2014, Institute scientists published a compound discovery that blocked a key ‘gatekeeper’ enzyme, Plasmepsin V, essential for malaria parasite survival. This compound, known as WEHI-916 was identified through high throughput screening.

The compound was developed to understand the effect the enzyme restriction would have on malaria. Our scientists found that Plasmepsin V is an ideal drug target because its inhibition halts malaria parasites from evading the host’s immune system. 


Professor Alan Cowman

Alan Cowman standing in a laboratory
Deputy Director and Joint Division Head

Dr Joanna Groom

Dr Joanna Groom at a microscope
Laboratory Head

Dr Hélène Jousset Sabroux

Hélène Jousset Sabroux
Jousset Sabroux
Head of Screening Laboratory

Dr Gary Pitt

Dr Gary Pitt
Program Manager, Drug Discovery Initiative

Dr Brad Sleebs

Dr Brad Sleebs
Laboratory Head
Two researchers smiling at the camera

Institute researchers have developed a compound that may be the first step toward a new class of antimalarial drugs.

Venetoclax trial participants

Professor Andrew Roberts and collaborators have shown that patients with an advanced form of leukaemia can achieve complete remission with a novel tablet treatment.