Finding treatments for motor neurone disease is the goal of WA neuroscientists who have received a one million dollar grant from Australian MND foundation FightMND.This is the first drug development grant for Western Australia provided by FightMND.
Researchers at the Perron Institute and Murdoch University are aiming to test their lead molecule for familial and sporadic forms of MND, also known as amyotrophic lateral sclerosis (ALS).
In most cases, the cause of this neurodegenerative disease is not known, but evidence from scientific studies suggests that both genetics and environment play a role in its development.
Although therapies are available to help control symptoms, there is no cure.
The FightMND grant will support current work towards developing a potential treatment, applying the molecular pharmacology of antisense oligonucleotide (AO) technology to alter genetic processes.
Former AFL footballer Neale Daniher is living with MND and is the co-founder and patron of FightMND. The foundation’s crusade to beat MND has gained significant momentum through its high profile awareness and fundraising campaign, ‘The Big Freeze’.
Perron Institute Director Professor Steve Wilton, who is also Director of the Centre for Molecular Medicine and Therapeutics at Murdoch University, is the principal investigator.
He is working with Professor Anthony Akkari, head of Motor Neurone Disease Genetics and Therapeutics Research and postdoctoral researcher Dr Loren Flynn at the Perron Institute and Murdoch University.
The project is a collaboration with Associate Professor Brad Turner from Melbourne’s Florey Institute, Professor Sue Fletcher and Dr Ianthe Pitout from Murdoch University, and Dr Craig Metz from US biotech company Black Swan Pharmaceuticals which has formed a partnership with the Perron Institute.
AO therapy has been successful in the development of treatments for Duchenne muscular dystrophy, receiving accelerated approval by the US Food and Drug Administration since 2016.
A novel AO therapeutic now being researched for MND has shown positive results in preclinical studies.Professor Akkari returned to Perth after several years in the United States to work with Professors Wilton and Sue Fletcher and their team and to build his own research stream to expedite drug development. As well as his Perron Institute/Murdoch University roles, he is the Chief Scientific Officer at Black Swan Pharmaceuticals which operates as a translational research engine, fast-tracking the development and clinical availability of novel therapeutic molecules.
“We are working with an excellent international multidisciplinary team who has extensive experience in working with regulatory agencies to quickly make drugs available for Phase 1 clinical trials,” Professor Akkari said.
It’s a matter of urgency that we deliver treatments for one of the world’s most devastating neurodegenerative conditions.”The team hopes to share preliminary results in six to 12 months.