World-first trials aim to slow motor neurone disease
Two world-first clinical trials conducted by scientists at Sydneyās Macquarie University are aiming to slow down the symptoms of motor neurone disease (MND).
MND destroys the nerve cells controlling the muscles that enable people to move, speak, breathe and swallow ā there is no cure.
One clinical trial will repurpose a HIV aids drug named Triumeq to target sporadic MND, which can strike anyone of any age and affects about 90 per cent of MND sufferers.
The other trial will use an Australian-made drug Copper-ATSM on patients with inherited MND, which affects 10 per cent of sufferers.
Professor Dominic Rowe from Macquarie University told the ABC 40 Australians are taking part in each trial.
āThis is the first time in the world that this multiple active retroviral therapy, Triumeq, has been tried in motor neurone disease,ā Professor Rowe said.
āSo weāre very excited and hopeful that weāre going to see some good things out of that trial.
āFor the second trial with Copper-ATSM ⦠weāre the only site in the world that has approval to use this therapy, so again weāre very hopeful weāre going to see some good things out of this.ā
The trials have been possible due to 60 scientists at Macquarie University who are solely working on MND research.
They are divided into several teams, including one team that uses thousands of tiny zebra fish to understand the biology of the neurodegenerative disease.
Firefighters to tackle āgruellingā fundraiser for MND
The teamās work, laboratory and the on-site clinic for treating patients are funded in large part by generous donations from the community ā and one group that has contributed significantly is NSW firefighters.
On Sunday October 23, they will climb 1504 stairs over 98 floors to the top of Sydney Tower Eye in their full firefighting kit to raise money for MND.
One-hundred-and-sixty participants collected donations worth $184,000 at the inaugural event in 2015, and 450 participants have already raised $235,000 this year.
Firefighters from other states, as well as New Zealand and Chile are also taking part.
This extraordinary challenge was the idea of Sydney firefighter Mathew Pridham, whose best friend Adam Regal was diagnosed with MND three years ago.
āAdam got given his diagnosis and you know 50 per cent of patients lose their battle within 18 months,ā Mr Pridham said.
āSo it just became a race to see what we could do to slow the disease down and what we could do to support people who were working so hard to try to end MND.
āProfessor Dominic Rowe and his team at Macquarie Uni were the absolute inspiration for this event āĀ I heard about Dom and then I met him and thought āwe need to do something to help that guyā.ā
Newcastle firefighter James Rumble will be doing the āgruellingā stair climb for the second time in memory of his fiancee Nicolaās mother Lorraine, who died from MND in 2014.
āIt really gets you motivated to think āwell Iām lucky enough to be able to do this still physically, whereas there are people with motor neurone disease who canātā,ā Mr Rumble said.
āDom Rowe was actually Lorraineās treating professor at Macquarie University so I hope they can find a cure ā thatās what Lorraine wanted as her final wish.ā
MND as cause of death rising in Australia
āThis is a disease that affects mothers, fathers, brothers, sisters, husbands, wives, sons and daughters,ā Professor Rowe said.
āIt kills people and we have to develop therapies that stop this disease now.
āThe scary thing is that over the last 30 years, motor neurone disease as a cause of death in Australia has increased by more than 250 per cent.
āIn 1986 it was the cause of death of 1 in 500 Australians, in 2014 it was just under 1 in 200.ā
The Triumeq trial started this month and the Copper-ATSM trial starts next month.
-ABC








