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.”