In the News
Trials to start of daily pill for MND that could reverse condition
February 18, 2024 (HERALD SUN AUSTRALIA) By Sarah Booth—
Australian motor neurone disease patients will be the first in the world to trial a “novel” treatment that scientists hope could not only slow — but actually reverse — the condition.
If successful, the US biotech firm Spinogenix drug would be the first regenerative treatment for the devastating and rapid neurodegenerative disease — which kills two Australians a day — and may also help fight Alzheimer’s and schizophrenia.
The first MND human trial will begin next month, thanks to a successful safety trial in Melbourne, and patients could take the daily pill for up to a year after the regulator approved access for 11 months post-trial even if patients received a placebo initially.
The trial is open to 24 MND patients with Lou Gehrig’s disease (ALS).
The drug was effective in animal trials and, while these do no guarantee success in people, Spinogenix founder Dr Stella Sarraf told the Herald Sun she was “super excited”.
Neale Daniher walks through a guard of honour. Picture: Michael Klein
She said their first-of-a-kind drug targeted the connections between our neurons in a bid to regrow those lost to MND, Alzheimer's or schizophrenia.
“[It] restores synapses, these are connections between the neurons that enable communication,” she said. “We believe this will show benefits in motor, respiratory and cognition; so walk, breathe and remember.
“We really hope this improves the lives of Australian patients.” She said MND was complex, so needed multiple types of treatments, but it would mean everything if their drug, SPG302, worked.
“I know what it’s like to fight the battle (of a neurodegenerative disease),” she said.
“I’ve lost both parents.”
“If I make an impact with a drug that helps people win that battle … it’s my dream.”
“We’re hoping our drug will not only fight the disease, but destroy it.”
She said groups like MND Australia and Neale Daniher’s Fight MND had been instrumental in raising awareness, without which Spinogenix would not be to “move as quickly as we are” in Australia.
Neale Daniher’s Fight MND had been instrumental in raising awareness. Picture: Getty
Melbourne dad-of-two Todd Johnson, 48, said living with an incurable disease like MND was just as much of a mental fight as a physical one.
He said it was a “battle trying to stay positive” but clinical trials “give you hope” and “helps you feel part of something … a way to contribute to finding that cure”.
“The psychological impact of … who knows maybe this could be the drug,” he said.
Australia research executive director Dr Gethin Thomas said while this was early research, every new trial helped locals access new therapies and it would be “brilliant” if it progressed to stage two.
“We need as many different therapies as possible … an arsenal of weapons,” he said.
Any eligible Australian patient can apply for the trial — which will run in three hospitals in Sydney, Brisbane and Adelaide — at clinicaltrials.gov.
