Researchers report possible breakthrough in hunt for Motor Neurone Disease cure

Australian scientists are planning clinical trials on humans for a variant of a drug that successfully stopped the progression of Motor Neurone Disease (MND) in mice.

Cross-section of a human brain. (AAP)

Cross-section of a human brain. (AAP) Source: AAP

Peter Simko lost his wife, Jen, to Motor Neuron Disease (MND) in November 2015.

The vibrant mother-of-three had been diagnosed with the debilitating disease just 15 months earlier.

Before she died, Ms Simko began raising funds for MND research in the hopes of learning more about the disease, which has no known cause or cure.

MND, also known as ALS or Lou Gehrig's disease, is a terminal neurological disease that progressively restricts a person's use of their limbs and ability to speak, swallow and breathe.

It is also suffered by physicist Stephen Hawking and caught the world's attention with the viral social media campaign, The Ice-bucket Challenge.
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Breakthrough?

Now, scientists believe they are one step closer to finding a cure.

Researchers at Oregon State University have been able to stop the progression of MND for nearly two years in a specific type of mouse they used to study the disease.
 
This allowed the mice to approach their normal lifespan.

The scientists said these findings were some of the most compelling ever produced because no other treatment had been discovered that could prolong human survival for more than a month. 

The treatment was based on six years of research and involved delivering a copper-ATSM compound to damaged cells in the spinal cord and mitochondria - linked to the death of motor neurons in MND patients.   

But experts cautioned this approach was not as simple as taking copper supplements, which can be toxic even in moderate doses.
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Clinical trials

University of Melbourne School of Biomedical Sciences researcher Dr Peter Crouch told SBS phase-one clinical trials of a drug from the treatment would begin later this year.

"The drug that will be tested has been modified so that it can safely be given to humans," Dr Crouch said.

"Dates for stage-one trials and participant expressions of interest will be announced in the coming months."  

But Dr Crouch warned they wouldn't know if the treatment was effective in humans until further testing. 

"We are optimistic that this drug will be effective, however it's unlikely to be a single definitive cure," he said.

While the treatment is unlikely to repair neuronal loss caused by MND, if successful, it could slow further progression of the disease. 

A recent increase in public donations accelerated MND research worldwide and facilitated much of the research behind this breakthrough. 

"Public fundraising for MND research over the last few years has been an absolute game changer," Dr Crouch said.

Peter Simko is continuing his wife's legacy by ensuring this funding continues.  
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Mr Simko and his family will host the second annual "Rock off MND" charity concert in Geelong on March 5.

Last year's event was attended by more than 600 people and raised $63,000 for MND research.

"This amount enabled us to establish the Jenny Simko MND Research Grant which was recently awarded by the MND Research Institute of Australia," Mr Simko told SBS. 

A range of musicians, including ARIA Hall of Fame inductee Ross Wilson and Indigenous singer Benny Walker, will perform this year, with all proceeds going to MND research.


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By Michelle Rimmer
Source: SBS News


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