A new strand of research planned by WA researchers towards developing a drug for Amyotrophic Lateral Sclerosis (ALS) has attracted funding from the US Defense Department.
The Therapeutic Ideas Award is equivalent to approximately $760,000 in Australian dollars.
ALS is the most common form of motor neurone disease.
“Motor neurons are cells in the brain and spinal cord that allow us to move, speak, swallow and breathe by sending commands to the muscles that carry out these functions,” said Principal Investigator, Dr Sarah Rea, Senior Research Fellow at the Perron Institute and Murdoch University’s Centre for Molecular Medicine and Innovative Therapeutics (CMMIT).
“As motor neurons degenerate and die, they stop sending messages, which causes the muscles to weaken and waste away. Ultimately, the brain loses its ability to initiate and control voluntary movements.
“One of the hallmarks of ALS is the mislocalisation and abnormal aggregation of a protein called TDP-43 in surviving neurons,” Dr Rea said.
“This is a major molecular marker for ALS and a form of dementia that is caused by progressive damage and loss of nerve cells in the frontal and temporal lobes of the brain.
“It is also known that increased expression of an associated second protein, p62, a master regulator of both neuroinflammatory cell signalling and protein degradation pathways, induces these changes in TDP-43 expression leading to neuron death.
“Our planned research will look at this second protein to understand whether it may be an appropriate target for a future molecular-based therapy to tackle ALS.”
Co-investigators are Dr Loren Flynn (Perron Institute and CMMIT), Professor Anthony Akkari (Perron Institute and CMMIT), Professor Sue Fletcher (Murdoch University), Professor Brad Turner (Florey Institute) and Associate Professor Rakesh Veedu (Perron Institute and CMMIT).
“Delivery into the central nervous system remains a critical problem for the development of effective neurodegenerative disease therapeutics, and as a collective team we now have three major grants allowing us to attack this issue from multiple avenues.
“The ongoing support of the Perron institute, the Racing for MNDi Foundation and the WA Health Department has provided the support needed to progress the project and secure this substantial funding,” Dr Rea said.