World-first Florey research aims to rapidly and accurately screen drugs to treat motor neurone disease

The Florey

Australian Government Medical Research Future Fund supports ground-breaking Florey project

Florey MND researchers are poised to begin large-scale drug screening of potential treatments for MND thanks to a $5 million grant from the Australian Medical Research Future Fund (MRFF).

Project leader Dr Chris Bye said the work centred on world-first drug screening technology developed at The Florey.

“To develop an alternative drug discovery pathway for MND, we built a library of ‘induced pluripotent stem cells’, or iPSCs, from more than 100 MND patients, mostly with the predominant ‘sporadic’ form of the disease.”

He said the iPSCs can be crafted to produce an unlimited source of cells that exhibit the disease’s key trait: dying motor neurons.

“We are then able to test whether potential treatments are able to keep motor neurons alive. We have already screened all the drugs tested in MND patients, and our results matched what was found in trials – 95 per cent did not work.”

Dr Bye said the project would improve the quality of drug trials by screening out drugs that showed promise in conventional research methods, but which the iPSC-based approach indicated may not be effective in people.

“Our breakthrough technology has the potential to revolutionise drug discovery for sporadic MND. We will be using it to conduct an unprecedented large-scale program of drug screening across high-value disease targets in MND.

“Motor neurone disease is a devastating, rapid and fatal neurodegenerative disease. Every day in Australia 2 people are diagnosed with MND, and 2 people die from the disease,” Dr Bye said.

“Despite almost 200 drugs reaching human trials in the past 25 years, we still have no effective treatment. Our technology aims to disrupt the usual model of drug discovery to find treatments for people with MND.”

Dr Bye said most potential MND treatments that showed promise in preclinical development had failed to work in people.

The Florey team is now uniquely positioned to deliver genuine therapeutics to clinical trial for people with MND.

“As well as this MRFF funding, we’ve been generously supported by FightMND, the Victorian State Government and Balcon Group - who all provided early funding to create the platform - as well as a team of leading MND clinicians, scientific experts and key industry leaders,” Dr Bye said. 

Health Minister Mark Butler described MND as an insidious disease that was rightly considered a “beast”.

“MND affects more than 2000 Australians, who tragically have an average survival rate of just over 2 years,” Minister Butler said.

“The Government is proud to support this innovative Australian research, which may unlock the potential for stem cell technologies to find an effective treatment for MND, by allowing therapies to be lab-tested with much greater speed and accuracy.”

Media contact:

Kathryn Powley, Media and Communications Manager

kathryn.powley@florey.edu.au | 0456 666 271