‘Vacumn cleaner’ drug could unlock new MND treatments

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Australian patients could soon be among the first in the world to trial a single-dose injection to halt and potentially reverse some symptoms of motor neurone disease and frontotemporal dementia, after researchers in Sydney discovered a way to clear blockages linked to both diseases in mice.

Abnormal clusters of a protein called TDP-43 are a common feature of many neurodegenerative diseases, including motor neurone disease (MND), frontotemporal dementia (FTD), and Alzheimer’s.

Dr Annika van Hummel, Professor Yazi Ke, and Professor Lars Ittner are part of the research team at Macquarie University who have been searching for ways to treat MND and frontotemporal dementia for 15 years.

Dr Annika van Hummel, Professor Yazi Ke, and Professor Lars Ittner are part of the research team at Macquarie University who have been searching for ways to treat MND and frontotemporal dementia for 15 years.Credit: Samantha Christensen

Patients with MND lose muscle strength in the early stages before gradually losing the ability to walk, talk, swallow and breathe. Most die within five years of diagnosis.

FTD is the name given to the group of disorders that occur when cells in the frontal and temporal lobes of the brain are lost. Although rare, it is the second-most common form of dementia in people younger than 65. Actor Bruce Willis was diagnosed with FTD in 2023, aged 68.

After 15 years of investigating ways to break down or prevent these clusters from forming, Professor Lars Ittner and his team of neuroscientists at Macquarie University have developed a single-dose genetic medicine that can halt the progress of both MND and FTD in mice.

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Ittner said the most surprising part of the research, published in the international neuroscience journal Neuron on Friday, was that some of the mice even regained strength and improved motor performance, suggesting that dissolving the protein clusters was helping return normal function to the affected neurons.

“The hope is that you’re not only stopping the disease, which sometimes has already progressed to really bad state, but actually, you can regain some functions,” he said. “It will not be a miracle, but … regaining some minor neurological function can make a big impact for patients.”

A start-up company spun out of the research, Celosia Therapeutics, will now seek funding and regulatory approval to take the research to human trials within two years.

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