Millions of dollars raised in this year's "Big Freeze" will fund a world-first trial of a drug hoped to significantly extend the life of people with motor neurone disease.
The medication, called Tecfidera, has recently emerged as a treatment for multiple sclerosis, another debilitating disease which affects the central nervous system, and for which there is also no cure.
Up to 150 people from across Australia will be recruited to find out if Tecfidera could also be used to slow the progression of MND.
The trial has been made possible thanks to a $4.75 million research grant from not-for-profit group FightMND, though the vice-chairman and prominent figurehead, former AFL player and coach Neale Daniher, will not participate.
His disease is too advanced to meet the trial's criteria.
"We have a panel of experts who make recommendations on where the money should be invested," Daniher explained.
"We don't determine who goes on the trial."
The former Melbourne coach was first diagnosed with motor neurone disease in 2013, after he started noticing a weakness in his hand.
Since then his speech has become slurred. More recently, he said his upper-body strength had been deteriorating.
Even so, Daniher has defied the odds. He was diagnosed four years ago, when typical life expectancy for those with motor neurone disease is just over two years.
Each day in Australia, at least two people are diagnosed with the condition, and it's responsible for about 800 deaths a year.
The new clinical trial will be led by Professor Steve Vucic and Professor Matthew Kiernan from the University of Sydney and Westmead Hospital, but will involve centres around the country, including Barwon Health in Geelong.
Professor Vucic said Tecfidera had been shown to increase the "good part of the immune system".
Motor neurone disease occurs when nerve cells controlling muscles weaken and eventually die, meaning people progressively lose the availability to move, speak, swallow and breathe
"We don't know what causes motor neurone disease but the immune system seems to play a role in controlling how quickly the nerves die," Professor Vucic said.
"We are looking at a treatment that will hopefully slow down the disease's progression significantly.
"We have very few cures for anything in neurology but we have ways of modulating and slowing down and keeping the disease at bay.
"We know that MND patients survive three-to-five years and to increase that by any amount will be a huge improvement."
The researchers are looking for people to participate in the trial who have been diagnosed in the past two years, and who have "relatively preserved" breathing and swallowing.
"We are trying to get people who are earlier in their disease so we can protect what's there to be protected," Professor Vucic said.
The trial is due to begin early next year, with early results expected by August.
People interested in participating can contact steve.vucic@sydney.edu.au.
