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Facilitating the development of treatments for people with Huntington’s disease

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20 July 2018

Dementia became Australia’s second leading cause of death in 2013, according to data from the Australian Bureau of Statistics. Clinical trials in dementia are ongoing, but with no success to date.

As part of the work undertaken through MICCN’s Dementia, Aging and Neurodegeneration Network, a project is underway to facilitate clinical trials for Huntington’s disease (HD), for which there is a known genetic cause. Since the discovery of the HD gene in 1993, efforts have been directed to find treatments that will delay the onset or slow disease progression.

Preclinical animal studies are the essential basis informing clinical human trials. To date, drug development for HD has had some successes in animal models, but has failed to translate to human disease. A major roadblock is the different cognitive testing methodologies used in animals versus those used in humans.

MICCN researcher and neuropsychologist, Dr Yifat Glikmann-Johnston, is trying to bridge the gap between animal and human cognitive testing by finding tasks that work in the preclinical program in animal models, and which span all the way to the clinical phases in humans.

“Cognitive impairment in HD is a key symptom, and the decline in function is a primary concern for patients,” Dr Glikmann-Johnston said. “We assessed cognition by looking at participants’ spatial memory, which translates across species, and which is the cognitive domain of choice in preclinical animal testing. We asked our HD participants to navigate through a virtual on-screen environment, for example, a house, and then determined if they could remember things like the outline of the house, and the things they encountered in the house. We also asked them to undergo a brain MRI so that we could link spatial memory performance to the brain structures that degenerate in HD. We’re now analysing the data we’ve collected in the hope of identifying a mechanistically-driven cognitive outcome measure for HD clinical trials.”

The Australian Government announced as part of the 2014 Budget an additional $200 million over five years to boost Australia’s dementia research capacity. Dr Glikmann-Johnston’s study was made possible by the award of one of the NHMRC-ARC Dementia Research Development Fellowships in 2016, with Yifat being one of just two people selected to investigate HD.

“I’m very honoured to have received this pivotal Fellowship for dementia research. I’m optimistic about my findings facilitating the development of effective treatments for people with HD and similar neurodegenerative diseases.”

For more information on Dr Glikmann-Johnston’s work in HD, please contact her at t: 03 9902 0238, e: yifat.glikmann-johnston@monash.edu.

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