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Drug breakthrough could substantially slow progression of motor neurone disease, say scientists

  • On November 10, University of Wollongong scientists published a Nature Communications proof-of-concept led by Dr. Christen Chisholm unveiling MisfoldUbL, a molecule targeting toxic misfolded SOD1 proteins.
  • Proteins that misfold in cells can accumulate into aggregates that damage and eventually kill motor neurones, and about 35% of people with inherited MND in Australia carry SOD1 mutations that increase misfolding.
  • MisfoldUbL acts like a protein recycling tag that attaches to misfolded SOD1 and directs the cell's waste-disposal system; in mouse models, it slowed symptoms and protected motor neurones.
  • Researchers are now hopeful the drug can be tested on humans, as preclinical studies showed improved movement, gait, nerve function, and motor neurone protection in mice with no current cure for MND.
  • The project builds on the late Professor Justin Yerbury's idea, funded by a $1 million FightMND grant in 2020, while University of Wollongong researchers develop a spin-off diagnostic tool for earlier genetic diagnosis.
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ALS News Today broke the news in on Monday, November 10, 2025.
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