Motor Neurone Disease and dementia breakthrough discovered in Macquarie Park innovation district

A single-dose genetic treatment is being developed to halt the progression of Motor Neurone Disease (MND) and Frontotemporal Dementia (FTD) - and it is happening here in Macquarie Park!

A team of neuroscientists at Macquarie University led by Professor Lars Ittner has focused on clarifying the causes of pathological TDP-43 build-up—a critical factor in the progression of MND and FTD. Celosia Therapeutics - a Macquarie University spin-out company – has catalysed on this research, leading to a significant breakthrough.

Motor Neurone Disease (MND) is a fatal condition affecting 2,100 Australians and 400,000 globally. It encompasses a group of diseases characterised by the progressive loss of neurons crucial for brain-to-muscle communication. It impacts voluntary movements like walking, swallowing, and speech.

Similarly Frontotemporal Dementia affects 5-15% of MND-diagnosed individuals. Although less common, it ranks as the second most common type of dementia among those under the age of 65, underscoring the demand for effective treatments.

Celosia Therapeutics and the research team’s pioneering efforts in our innovation district has resulted in the development of “CTx1000”, a genetic treatment aimed at targeting specific Motor Neurone Disease variants.

CTx1000 targets the pathological variant of TDP-43 while allowing the healthy protein to function normally. This ensures safety and efficacy, potentially offering a one-shot treatment even in advanced stages of disease. Preliminary trials on mice have yielded positive results, paving the way for human clinical trials.

Fellow researcher Professor Dominic Rowe, one of Australia’s leading MND clinicians and researchers said:

“I am currently treating more than 200 people with MND, but only five of them with a specific type of familial MND are currently able to access genomic therapy. Treatment like that doesn’t exist for anyone else. For most patients, all we can do is provide good quality, multidisciplinary care.

This new research is incredibly promising in slowing the progression of MND and FTD for the vast majority of our patients.”

While human clinical trials are still some way off, the promising results from preliminary trials signal a potentially long-term remedy for MND and FTD patients.

With clinical trial operators, medtech and pharmaceutical companies and a cluster of pathology leaders based in our district, this latest breakthrough underscores the continuous innovation within Macquarie Park's biosciences and health ecosystem.