Fresh hope for motor neurone disease

professor-robert-rush-and-dr-mary-louise-rogersScientists from Flinders University have just received a prestigious $100,000 grant to continue their groundbreaking research into motor neurone disease.

Awarded by the Motor Neurone Disease Research Institute of Australia, the Roth Foundation MND Research Grant will assist researchers Dr Mary-Louise Rogers (pictured) and Emeritus Professor Robert Rush (pictured) to test a novel treatment that could eventually lead to a potential therapy for the disease.

Motor neurone disease, also called amyotrophic lateral sclerosis, is a neurodegenerative disease which causes the death of motor nerve cells within the spinal cord and brain that are responsible for controlling muscle movement.

In Australia one person dies from the condition each day while another is diagnosed, and with no effective treatment available to reverse or halt the disease most patients only live an average of three years after diagnosis.

Professor Rush said the novel therapy developed by the researchers, called immunogenes, may enable antibodies to shoot genes, missile-like, into the nerves to stop the production of toxic proteins which are a known cause of motor neurone disease in about 10-15 per cent of cases.

“About 90 per cent of cases arise spontaneously but we know that in a small number of patients, motor neuron disease occurs when there is an overproduction of bad proteins which causes the nerve cells to die,” Professor Rush, from the Human Physiology department, said.

“But we’ve altered the antibody that targets the motor neuron so that it can carry a gene, and this antibody is like a missile in that it shoots directly into the neuron and acts as an inhibiting agent that prevents toxic protein from being produced,” he said.

Professor Rush said the funding, which follows an initial Roth Foundation grant of $90,000 in 2011, would allow the research team to trial the therapy in animals and, eventually, humans.

In another breakthrough, Dr Rogers and Professor Rush are embarking on a groundbreaking new study to test for a “biomarker” in blood and urine samples of people with motor neurone disease.

At present, the only way to establish stages of the condition is by visual assessment and some invasive techniques such as muscle biopsies.

“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, including our gene therapy, is working or not we can get those medications pushed through the clinical trial process a lot quicker,” Dr Rogers said.

“And if we can tell from the biomarker that someone has the disease before the symptoms show up we also might be able to treat them earlier.”

The research, Dr Rogers said, is being supported by a $16,000 Flinders Medical Centre Foundation Seeding Grant and involves PhD candidate Stephanie Shepheard, the first and only Flinders student to receive an Australian Rotary Health Neville and Jeanne York PhD Scholarship for Motor Neuron Disease.

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