Researchers in this theme investigate the biological processes that drive MND development and progression. Using advanced ‘omics’ technologies at genetic, protein, and metabolite/lipid levels, we aim to identify novel druggable targets.
Drug discovery
Building on identified targets, we explore potential drug candidates through medicinal chemistry expertise and collaborations with global MND researchers, pharmaceutical, and biotech partners. Our approach includes discovering new compounds and repurposing existing drugs. Screening and validation ensure potency, safety, and optimal bio-distribution (pharmacokinetics (PK) and absorption, distribution, metabolism, and excretion (ADME)).
Drug development
Promising candidates undergo rigorous preclinical testing to assess their efficacy in laboratory models of MND. Our goal is to select the most viable drug for progression to human trials. We employ best-in-practice animal and ex vivo human models to test drugs, confirm target engagement, and evaluate biomarker sensitivity - critical steps in ensuring clinical readiness.
Translational research
We bridge laboratory discoveries to clinical applications by collaborating with neurologists, contract research organizations, and drug manufacturers. This includes conducting formal safety and toxicology studies, scaling up drug production, and preparing for clinical trials. Our ultimate aim is to bring new treatments to patients with MND as efficiently as possible.
Impact and outcomes
Despite the long and costly path from discovery to clinical application, our team remains committed to advancing therapies for MND. Our work has successfully translated laboratory findings into human clinical trials, contributing to the development of new treatment options. Recent success include:
- A drug called PMX205 targeting the immune system was discovered by Professor Woodruff at the University of Queensland in 2004 and has recently completed a clinical trial in people with MND at The Wesley Hospital, Brisbane.
- Associate Professor Ngo has repurposed Trimetazidine (a drug used to treat heart conditions in Europe), recently completing a Phase 2a trial in people with MND in Australia and The Netherlands.
Our successes in moving drugs from the laboratory into clinic trials bolsters our hope that we will one day identify a suite of treatments that have a significant impact on the lives of everyone living with MND.
Our team
- Professor Markus Barth
- Dr Fernanda Cardoso
- Dr Jean Giacomotto
- Dr John Lee
- Associate Professor Dominic Ng
- Associate Professor Shyuan Ngo
- Associate Professor Peter Noakes
- Dr Margreet Ridder
- Dr Daniel Schweitzer
- Associate Professor Frederik Steyn
- Associate Professor Adam Walker
- Associate Professor Anthony White
- Associate Professor Joy Wolfram
- Professor Trent Woodruff
- Dr Alain Wuethrich