Enormous effort is being made to find an effective treatment for motor neurone disease (MND), a rare but devastating illness which causes weakness in the muscles, leading eventually to paralysis.
Current drugs benefit only a small proportion of patients and have only limited effects on survival.
Postdoctoral researcher Dr Oliver (Dunhui) Li (Perron Institute and the Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University) has been awarded a grant of $549,537 from FightMND to lead a drug development project testing a new strategy to repair and protect dying neurons and wasting muscles in MND using a gene therapy approach.
“The aim is to broaden our understanding in the quest for an effective therapeutic to slow disease progression and improve function and survival outcomes in MND patients,” project leader Dr Li said.
“Nerve fibre degeneration is an integral feature of MND, a neurodegenerative disease that affects nerve cells in the brain and spinal cord that control voluntary muscle movement.
“This neurodegeneration results in loss of communication and connectivity between neurons and muscles, leading to progressive muscle weakness and atrophy, so promoting nerve fibre regeneration is seen as a viable therapeutic intervention strategy.
“Our project focuses on a gene demonstrated to be associated with MND disease risk and disease progression, with a role in restricting nerve fibre regeneration.
“We have developed a novel approach to dial down the expression of this gene, hoping to progress this as a disease-modifying strategy to enhance nerve fibre growth and promote nerve cell survival for both sporadic and inherited forms of the disease.”
Co-Investigators are Professor Steve Wilton (CMMIT, Murdoch University and Perron Institute), Professor Anthony Akkari and Dr Loren Flynn (Co-Leads of the Motor Neuron Disease Genetics and Therapeutics group at the Perron Institute and CMMIT), Professor Bradley Turney (Florey Institute, Melbourne), Professor Jeroen Pasterkamp (University Medical Centre, UMC Utrecht Brain Centre, the Netherlands) and Professor Frank Mastaglia (Perron Institute).
“With the generous funding from FightMND and ongoing support from the Giumelli Family Foundation we can dive into a new biological aspect of the disease which could eventually bring promising clinical benefits to patients,” Dr Li said.
Photo caption: L-R Dr Oliver Li, Dr Loren Flynn and Professor Anthony Akkari in the Perron Institute lab.