A new therapy targeting misfolded proteins shows promise in slowing motor neurone disease in mice.

A team at the University of Wollongong, led by Dr. Christen Chisholm, has developed a promising new therapy for a genetic form of motor neurone disease (MND) using a molecule called Misfold UbL. The treatment targets and tags misfolded SOD1 proteins—linked to MND—enabling the body’s natural waste system to destroy them before they form harmful clumps. In mouse models, the therapy slowed symptom onset, protected motor neurons, and preserved muscle connections. The research, inspired by the late Dr. Justin Yerbury’s work and supported by Fight MND, offers potential for earlier diagnosis and could apply to other neurodegenerative diseases like Alzheimer’s and Parkinson’s.