In a monumental achievement for Australian medical research, scientists at the University of Wollongong have published a world-first study that could fundamentally transform how motor neurone disease is treated. The breakthrough fulfills the visionary work of late Professor Justin Yerbury, who dedicated his life to finding a cure for the devastating condition that ultimately claimed his life.
The Legacy of a Visionary Scientist
Professor Yerbury, whose own family has been deeply affected by MND across generations, first conceived the radical idea years ago. He envisioned creating a treatment that could seek out and destroy the problematic proteins that mutate inside nerve cells of people with the fatal progressive condition. These mutations eventually cause muscle weakness, paralysis and death.
In 2020, the Fight MND charity granted Professor Yerbury $1 million to pursue this ambitious research at his world-leading University of Wollongong laboratory. Tragically, the scientist passed away from the disease before seeing his project come to fruition, leaving behind a legacy that his colleagues were determined to honour.
A World-First Therapeutic Breakthrough
Today, Professor Yerbury's friend and colleague Dr Christen Chisholm has led the team to publish their groundbreaking proof-of-concept study in the prestigious academic journal Nature Communications. The research demonstrates their innovative tool, called Misfold UbL, which can systematically locate, tag and eliminate the toxic proteins that overwhelm nerve cell defences and kill motor neurones.
Dr Chisholm described the publication as "massive on many fronts", noting that the therapy could not only transform MND treatment but might also have applications for other neurodegenerative diseases like Alzheimer's and Parkinson's.
"The therapy itself is very, very innovative, very new and has lots of potential," Dr Chisholm said. "It is going to be a breakthrough in terms of the way that we can treat proteins that are problematic."
How the Revolutionary Tool Works
The research focuses on the SOD1 protein (superoxide dismutase 1), which is mutated in approximately 35 percent of Australians with inherited MND, including Professor Yerbury's family. When these proteins misfold, they form insoluble clumps that damage and eventually kill motor neurons, leading to disease symptoms.
The Misfold UbL molecule operates like a sophisticated "search and destroy" mission at the cellular level. It first identifies the misfolded SOD1 proteins among the billions of proteins in a cell, specifically targeting only the problematic versions while leaving healthy proteins untouched.
Once identified, the tool attaches a tiny recycling tag that directs the cell's natural waste-disposal system to degrade the harmful protein. "What we designed searches out the misfolded SOD1 protein, and then tags it so that the cell knows this is a problem, this is waste, let's get rid of it," Dr Chisholm explained.
In animal testing, the treatment demonstrated significant benefits, including:
- Slowing symptom development
- Protecting motor neurones in the spinal cord
- Preserving muscle connections compared to untreated animals
Continuing the Fight Against MND
Dr Chisholm emphasized that the publication represents years of dedicated work by numerous scientists, all inspired by Professor Yerbury's original vision. The achievement is particularly meaningful given the challenges the team faced, including COVID-19 lockdowns that restricted laboratory access and the profound loss of their leader.
"For us to get this therapy to this level of recognition... that's really meaningful for us because we feel like we're paying something back to Justin and continuing his work," Dr Chisholm said.
The research also highlights the University of Wollongong's growing reputation in MND research. "This publication shows that our university and our university staff and the brilliant minds that are here at the university are capable of creating this cutting-edge, world-leading research," Dr Chisholm noted.
Community support through donations has been crucial to this breakthrough. The research was funded entirely by donations to FightMND and direct contributions to the Yerbury Lab at UOW, demonstrating how public generosity can drive meaningful scientific progress against devastating diseases.
As the work continues, University of Wollongong researchers are already developing spin-off tools aimed at earlier diagnosis, recognizing that treatment is most effective when administered before symptoms appear. This world-first achievement stands as a testament to one scientist's vision and a team's determination to see it through, offering new hope in the fight against motor neurone disease.
