The small trial is set to open to eligible Australians with ALS, the most common type of MND, by the start of next year, with work for the first stage — involving healthy volunteers — already underway.
The first-of-its-kind drug, SPG302, can be taken daily as a pill and has shown promise in preclinical trials for ALS and Alzheimers, another neurodegenerative disease.
Successful mice trials are no guarantee a treatment will work in humans, but Spinogenix founder Dr Stella Sarraf – whose US Biotech company developed the drug – said they were excited by the results.
“We really hope this is a story of hope for MND sufferers,” she said.
MND has no cure or effective treatment, meaning patients tend to quickly lose the ability to walk, talk and, eventually, breathe as their neurons fail.
Dr Sarraf said their “novel approach” was unlike anything else, designed to restore connections between neurons – known as synapses – that have been lost.
“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 diseases, we lose these connections.”
Footage of two mice with MND from an SPG302 trial show a mouse who did not get the drug shaking badly, while the treated mouse was able to run around with no visible tremors.
Dr Sarraf said they were “astounded” by the results, which were shared at an Annual ALS ONE Research Symposium, because the mice had a “very rapidly progressing” ALS that was “very advanced”.
“It was beyond our expectations to see SPG-302 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.”
Dr Sarraf said the drug’s small molecule size allowed it to pass the blood brain barrier – a common obstacle in drug development – “to get into the brain … where the synapses are”.
“If we didn’t get there, it was impossible.”
Melbourne’s Nucleus Network – part of the Alfred’s Research Alliance – will recruit up to 112 patients for the trial, working with Sydney’s Brain and Mind Centre, the Royal Brisbane and Women’s Hospital, and Adelaide’s Flinders Medical Centre.
Researchers hope the drug could be applied to other neurodegenerative conditions and taken alongside other emerging treatments.
Dr Sarraf told the Herald Sun she became determined to find treatments for such conditions after her father Parkinson’s diagnosis.
“(To start human trials) is a humbling feeling and I’m honoured,’ she said.
She said awareness of ALS and the need for treatments had grown, paying tribute to patients like Neale Daniher and his work to establish FightMND.
“He’s an inspiration not only to Australians but to MND sufferers worldwide,” she said.
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