A revolutionary drug that can potentially reverse the effects of motor neurone disease (MND) has entered its first human trials anywhere in the world in Melbourne.
Known for the time being as SPG302, the drug is the first of its kind and looks to slow, or perhaps even reverse, the devastating effects of MND.
Manufactured by US pharmaceutical company Spinogenix, SPG302 has had positive preliminary results in mouse subjects and the company is now looking to move into human trials. The drug is taken as a daily pill.
Specifically, the trial will be looking at the most common form of MND, amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease after a famous American baseball player who had the disease.
Spinogenix is working with Melbourne’s Nucleus Network – a coalition of medical researchers that describes itself as “Australia’s largest Phase 1 clinical research organisation”.
The researchers are looking for 112 patients with ALS. The Nucleus Network will be working with Melbourne’s Alfred Hospital, Sydney’s Brain and Mind Centre, the Royal Brisbane and Women’s Hospital and Flinders Medical Centre in Adelaide to find subjects.
The first part of the trial, which involves a healthy control group with no symptoms of ALS or MND, is already underway.
Dr Stella Sarraf, Spinogenix founder, told the Herald Sun the drug takes a novel approach to MND, focusing on restoring connections between neurons in the brain called synapses.
“It’s the first synaptic regenerative therapy to be tested in ALS or MND,” she said.
“It could actually help it get better. These synapses are really just these connections between the brain.
“In [neurodegenerative] diseases, we lose these connections.”
In addition to MND, SPG302 has shown promising results in mice reversing the cognitive effects of Alzheimer’s disease, showing SPG302 has the potential to be something of a wonder drug for neurodegenerative disorders.
MND is a broad term used to describe a group of diseases that affect nerve cells called “motor neurones”.
Motor neurones carry messages from your brain to the muscles via the spinal cord. These messages allow you to make voluntary movements such as walking, swallowing, talking and breathing.
But with MND, the nerves become damaged and start to die and the muscles gradually get weaker and waste away. The speed at which MND progresses and survival times vary for each person.
Currently, there is no cure for MND and it significantly shortens your life, with the average person dying two to three years after being diagnosed. But this varies greatly from person to person.
Anybody who lives in an AFL-dominated state will most likely be aware of former player and MND-sufferer Neale Daniher and his tireless efforts to raise money and awareness for the disease. The most recent ‘Big Freeze’ in June boosted the tally to almost $16 million.
Mr Daniher was first diagnosed with MND in 2013 and is still with us today.
Dr Sarraf said she hopes SPG302 could one day lead to extended lifespans for MND-sufferers – like Mr Daniher’s – becoming the norm.
“It was beyond our expectations to see SPG302 improve the course of motor symptoms and prolong life,” she said.
“So we’re actually very hopeful, and have evidence, that it should help at various stages.”
Were you aware of motor neurone disease? Do you think this drug is likely to change how the disease is treated? Let us know in the comments section below.
Also read: New Alzheimer’s drug can drastically slow disease progression
How does one become available for the MND human trials