The grants, totalling AU$1.5 million (about $1.1 million), came from FightMND, an Australian nonprofit patient advocacy group. Lezanne Ooi, PhD, an associate professor of biological sciences at the University of Wollongong and Illawarra Health and Medical Research Institute, was awarded a two-year, $250,000 Impact Grant to investigate whether electric signals from motor nerve cells might be used to protect these cells in people with motor neuron disease (MND).
MNDs are progressive neurological disorders that destroy the motor neurons controlling skeletal muscles, which are essential for walking, breathing, and swallowing. ALS is a quickly progressing form of MND; other forms include spinal muscular atrophy and primary lateral sclerosis.
Ooi’s previous work includes efforts to create nerve cells in the lab, using induced pluripotent stem cells. Taken from skin or blood of ALS patients, these stem cells can be programmed to generate nerve cells that aid in disease understanding and possibly the discovery of treatments.
Justin Yerbury, PhD, a professor of molecular biology at both the university and the institute, received an AU$1 million Drug Development Grant to further work into a combination treatment for both familial and sporadic forms of MND. Combination therapies contain multiple medicinal compounds that act on different facets of the same disorder.
“We believe that a three-pronged approach targeting distinct and complementary aspects of the underlying molecular causes of MND will be a more effective approach,” said Yerbury, who was diagnosed with a genetic form of this disease in 2016 and is now paralyzed.
The grant will enable him to bring a post-doctoral researcher and assistant researcher into the project.
Yerbury turned from professional basketball to scientific research following the diagnosis and death of several family members from MND, a decade before his own disorder became evident.
This grant will fund the use of new technology to examine, at the single-protein level, the proteins that form the toxic deposits in neurons underlying MND.
“We want to define how the shape of the proteins may give rise to MND and characterize differences between patients,” Yerbury said. “By knowing the shape that these proteins take, we can identify specific parts of the proteins to target with therapies.”
“We not only want to examine the potential causes, but also the potential treatments for MND.
In the next phase of the investigation, we hope to see improvements in our research output and also in our capability to examine drugs for translation into patients,” McAlary added.
“FightMND has done an amazing job raising awareness and funding during a period of decline in government funding,” Yerbury said.
“It shows that Australians are passionate about finding a cure for this devastating disease.”