Motor Neuron Disease

New Motor Neuron Disease Study

New Hope for Motor Neuron Disease

Tailoring Treatment For Stroke Brain Damage

Repairing Brain Damage In Stroke

A Potential Therapy For Motor Neuron Disease

Motor Neuron Disease Study
First Published: Enews - November 2011
Updated:

Flinders researchers are embarking on a groundbreaking new study to test for a 'biomarker' in blood and urine samples of people with motor neuron disease (MND).

The research could one day lead to the use of new medications, and also aims to determine how advanced the disease is in people with MND. At present, the only way to establish stages of the condition is by visual assessment and some invasive techniques such as muscle biopsies.

MND is a fatal, non-curable disease which affects about 1,400 Australians at any one time. The disease impacts the motor neurons - the cells that control voluntary muscle activity including speaking, walking, breathing and swallowing.

In about 90 per cent of cases the patient has no family history of MND and the disease appears to have occurred without reason, while the remaining 10 per cent of cases are either hereditary or caused by an associated gene defect.

Led by Flinders University research fellow Dr Mary-Louise Rogers, the research team will analyse blood and urine samples for a specific molecule (called a biomarker) that may be present in people with MND.

The research, funded through a $16,000 grant from the FMC Foundation, will be used as 'proof of concept' in larger studies where the biomarker will be tested to see if it can measure the effectiveness of a range of clinical trial drugs in curing or delaying symptoms.

"Currently there is only one drug commercially available but it only improves quality of life for a few months," Dr Rogers said.

"We hope our research will be able to help pharmaceutical companies eliminate the drugs that are no good earlier in the process and continue investigating drugs that appear to be doing something."

While the project is still in its early stages, Dr Rogers said researchers would begin testing a small sample of people with MND in the coming months.

"Because it's such a horrible disease it's really important to find a cure, and if we have some way of knowing if drugs are working or not we can get those medications pushed through the clinical trial process a lot quicker."

New Hope for Motor Neuron Disease
First Published: Enews - August 2010
Updated:

Flinders Medical Centre researchers, in collaboration with two Canadian research teams, have developed a ground-breaking treatment which has the potential to significantly extend the healthy life of a Motor Neuron Disease sufferer.

Motor Neuron Disease, also called Amyotrophic Lateral Sclerosis, is a fatal neurodegenerative disease which causes the death of motor nerve cells (also called neurons) within the spinal cord and brain that are responsible for controlling muscle movement.

It results in creeping paralysis, and with no effective treatment available to reverse or halt the disease most patients only live an average of 2-3 years after diagnosis.

Emeritus Professor Robert Rush and his team  from the Department of Human Physiology at Flinders Medical Centre have successfully demonstrated a new antibody therapy that can extend the lives of mice with Motor Neuron Disease by up to 20 per cent.

This research has been supported in part by donations made to the Flinders Medical Centre Foundation.

"This new potential treatment has now been shown to work in two separate experiments using a mouse model that closely resembles the human disease," Professor Rush said.

"In human terms this could translate to an extra 10 healthy years for a Motor Neuron Disease sufferer if the treatment is given in the early stages of the disease." 

The treatment has been developed in collaboration with researchers from Canada's Magill University and the University of British Colombia. It is based on using a particular protein called an antibody to bind to a target on the motor nerves.  The antibody causes signalling within the cell that overrides the degeneration.

It has been shown in the mice to prevent the onset of a number of the symptoms of Motor Neuron Disease such as muscular weakness, and to prevent further degeneration of the motor neuron nerve cells in the spinal cord.

The research teams are currently seeking a suitable pharmaceutical company to help translate these findings into clinical trials next year.

Tailoring Treatment For Stroke Brain Damage
First Published: Investigator - December 2008
Updated:

Flinders researchers are investigating whether tailoring treatments to individual brain cell populations can help stroke victims recover more quickly.

The research team is working on new ways to selectively target different cell populations in the brain and to modify their function by introducing new genes.

‘We’ve already made good progress in this area by delivering genetic material via an uptake mechanism that is normally used by the cells themselves,’ Dr Hakan Muyderman, a researcher in the Centre for Neuroscience at Flinders said.

‘The next step is to see if we can use this technique on selected brain populations to treat the effects of stroke in animal models of this disease.’

Stroke is the second biggest killer after heart disease in Australia. More than 60,000 people suffer stroke or recurring stroke each year. A common effect of stroke is damage to the brain and changes in behaviour and personality.

Delivering treatment to specific cells of the brain has not been possible before now and researchers hope their work may create better treatments for stroke and other diseases of the central nervous system such as Alzheimer’s, Parkinson’s and motor neuron disease in the future.

The research will also help shine light on the role of glial cells, also known as astrocyctes. Glial cells greatly outnumber neurons in the brain and it is believed that they play many essential roles in normal brain function.

Brain insult or injury activates these cells which initiate responses that can have both protective and damaging effects on the surrounding neurons.

Hakan said understanding the role of these cells has been limited as there have been few specific approaches that have allowed the properties of the cells to be selectively targeted in an intact living brain.

‘A gene delivery system like the one we are creating that can selectively target these cells and produce short-term changes in gene expression will be of significant value in creating a better understanding of the role of these cells,’ said Hakan.

Repairing Brain Damage In Stroke
First Published: Investigator - August 2008
Updated:

Scientists at Flinders Medical Centre are fine-tuning new technology that could target and deliver treatment to specific groups of brain cells that have been damaged by stroke.

There are billions of different types of cells in the brain, each with their own function and response to injury. Targeting treatment to specific groups of these cells has not been possible before now.

Dr Håkan Muyderman from the Department of Medical Biochemistry and his colleagues have developed a technique that utilises a natural function of cells to deliver genetic material directly into specific brain cells to either repair them or alter their function so they are no longer damaging to the brain.

They have been making good progress and will soon see if this approach can be used to treat the brain damage and behavioural changes that develop in stroke.

Stroke has become the second biggest killer after heart disease in the developed world and is the leading cause of disability in Australia.

An attack can be caused either by a sudden disruption of blood flow to the brain or a haemorrhage that leaks blood into the brain, causing devastating damage to brain tissue.

Dr Muyderman’s research will also help shine light on the role of glial cells (also known as astrocytes), as they have not yet been well defined. Glial cells greatly outnumber nerve cells in the brain and are believed to play many essential roles in normal brain function.

Understanding the role of these cells has been limited as there have been few scientific approaches that have allowed the properties of the cells to be selectively targeted in an intact living brain.

“A gene delivery system capable of selectively targeting sub-populations of brain cells will be of significant value in creating a better understanding of the contribution of these cells to normal brain function and disease,” said Dr Muyderman.

This research could also contribute to better treatments for other diseases of the central nervous system such as Alzheimer’s, Parkinson’s and motor neuron disease.

A Potential Therapy For Motor Neuron Disease
First Published: Investigator - April 2007
Updated: New Hope for Motor Neuron Disease

Scientists at Flinders Medical Centre, led by Professor Robert Rush and Dr Mary-Louise Rogers from the Department of Human Physiology, have developed a potential therapeutic agent which could be used to treat the motor neuron disease Amyotrophic Lateral Sclerosis (ALS).

ALS is a fatal neurodegenerative disease marked by the death of motor neurons within the spinal and brain stem which are responsible for controlling muscle movement. With the progressive breakdown of these nerve cells within the central nervous system the body loses control of voluntary muscle movement.

“Amyotrophic Lateral Sclerosis is a devastating illness that results in creeping paralysis and death,” said Dr Mary-Louise Rogers. “There is currently no effective treatment to reverse or even halve the disease.”

The causes of ALS are not fully understood but include an impaired ability of the motor neuron to inactivate damaging compounds that accumulate in cells and result in damage. For example, a build up of a substance called glutamate has been linked to the death of motor neurons in ALS.

Glutamate is a natural chemical which acts as a neurotransmitter within the nervous system. Excess amounts are usually absorbed by surrounding cells, however in ALS it appears this absorption process fails, leading to a build up of glutamate which destroys the motor neurons.

Dr Rogers and a team of investigators have created a gene therapy which has demonstrated an ability in an animal model to reverse motor neuron death caused by traumatic injury. The treatment combines an antibody which targets the affected nerves and a drug component (the gene) which stimulates these nerves to start a repair process.

The team are now investigating this gene therapy further to see if it could prevent, reverse or slow the damage of motor neuron death in ALS.

While this investigation has provided positive results, further testing is required both in a mouse model of ALS and clinically before it can be used as a treatment.

“While we are still a way off, we are currently in a unique position to determine whether this treatment has a positive affect on diseased motor neurons,” said Dr Rogers. “We are hopeful that a successful outcome of our experiments may be to encourage clinical development of this treatment for patients with ALS.”