The Florey Institute of Neuroscience & Mental Health is set to revolutionize drug discovery for Motor Neurone Disease (MND) with a world-first drug screening technology. A $5 million grant from the Australian Medical Research Future Fund (MRFF) will enable large-scale screening of potential MND treatments using a platform based on induced pluripotent stem cells (iPSCs).
iPSC-Based Drug Screening
Dr. Chris Bye, the project leader, explained that the technology utilizes iPSCs derived from over 100 MND patients, predominantly those with the sporadic form of the disease. These iPSCs can generate an unlimited supply of motor neurons exhibiting the key characteristic of MND: neuronal death. This allows researchers to test the efficacy of potential treatments by observing their ability to sustain motor neuron survival.
"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 stated.
Improving Clinical Trial Efficiency
The iPSC-based screening approach aims to enhance the quality of drug trials by identifying and eliminating ineffective drug candidates early in the development process. This could significantly reduce the number of failed clinical trials and accelerate the discovery of effective MND treatments.
"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," Dr. Bye added.
Addressing an Unmet Need
MND is a devastating neurodegenerative disease affecting over 2,000 Australians, with an average survival rate of just over two years. Despite nearly 200 drugs being tested in human trials over the past 25 years, effective treatments remain elusive. This new technology offers a potential disruption to the traditional drug discovery model.
Health Minister Mark Butler emphasized the government's support for this innovative research, stating, "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."
Collaborative Support
The Florey team's efforts have been supported by FightMND, the Victorian State Government, and Balcon Group, alongside a team of leading MND clinicians, scientific experts, and key industry leaders. This collaborative approach positions the Florey Institute to potentially deliver genuine therapeutics to clinical trials for MND patients.
