Media release

From: Macquarie University

A world-first clinical trial has begun at Macquarie University of an experimental drug that has the potential to improve survival time and quality of life for people with motor neuron disease (MND).

About 2,500 Australians currently live with MND (also known as amyotrophic lateral sclerosis, or ALS), with 800 new cases diagnosed each year. While most people die within two to five years of diagnosis, some may live only months. A small percentage, like celebrated physicist Stephen Hawking, may live for decades.

Macquarie University’s Centre for Motor Neuron Disease Research is the largest MND research centre in Australia and is part-funded by research grants and by public donations amounting to more than $10 million over the past five years.

Neurologist Professor Dominic Rowe, a director at the Centre, has been treating people with MND for more than 20 years and has great hope for the latest trial.

“The trial uses the drug 3K3A-APC, which we hope will reduce inflammation in the brain and spinal cord for patients with MND,” he said.

“It has already been trialled in the US for patients who have had severe strokes, and it has shown potent anti-inflammatory effects as well as helping repair the blood-brain barrier, both of which are challenges in MND.”

Produced by US company ZZ Biotech, which is working closely with Macquarie University on the trial, 3K3A‑APC is a genetically modified version of ‘activated protein C’, a substance our bodies produce naturally to protect cells.

“We are very excited to be working with Professor Rowe and the amazing team at Macquarie University on this important clinical study,” said Kent Pryor, chief executive officer of ZZ Biotech.

“We have high hopes that 3K3A-APC, with its multiple beneficial effects in the brain, will one day benefit patients with MND, stroke, and other diseases.”

A research team testing the molecule in mouse models of MND found it dramatically improved survival in certain cases.

This is the first time the drug will be trialled on human subjects with MND, with 16 people to take part.

“Because of the work that’s already been done with this drug on stroke patients, we know how this molecule behaves in people and we know it’s safe,” Professor Rowe said.

“Positron emission tomography (PET) scans carried out before and after treatment will allow us to measure whether this molecule actually gets into people's brains.

“We’ll be able to assess whether it can help repair the blood-brain barrier, and if it can change the baseline level of brain inflammation that occurs in MND.”

The trial has been made possible thanks to a million-dollar donation by the charity Firies Climb for MND. NSW firefighters raise funds for the cause by being sponsored to make the gruelling 1,504-step climb up the Sydney Tower while carrying 20kg of gear.

Professor Rowe said his team had been applying for grants to initiate the trial for three years, but without luck.

“As an early-phase study, and one that was relatively expensive, we had trouble securing funding,” he said.

“This wonderful donation from Firies Climb for MND has made possible an exciting trial that has real potential to potential to improve people’s lives dramatically.”

The 3K3A-APC trial is one of five MND clinical trials set to begin at Macquarie University before the end of 2022. The others will assess the efficacy of two different gene therapies, an antiretroviral drug, and a cancer drug.

“Currently, we can’t cure MND, and we can’t reverse it,” Professor Rowe said. “But with early diagnosis, and if we can dramatically slow the progression of disease, I firmly believe we can turn this disease around.

“Two people with MND are dying in Australia in every day. If we could improve people's average survival from three years to 30 years, that, to me, would constitute a cure.”

Learn more about the work of the Macquarie University Centre for MND Research