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Australian scientists developing breakthrough drug to treat a rare form of dementia in younger people

Australian scientists are developing a new drug to combat a form of dementia that causes horrifying cognitive damage and personality change in people aged in their 60s and under.

The drug, sodium selenate, has been shown to put the brakes on cognitive decline and neuro­degenerative damage in patients who are suffering from behavioural variant frontotemporal dementia. Picture: AP
The drug, sodium selenate, has been shown to put the brakes on cognitive decline and neuro­degenerative damage in patients who are suffering from behavioural variant frontotemporal dementia. Picture: AP

Australian scientists have developed a breakthrough drug which combats a form of dementia that causes horrifying cognitive damage and personality changes in people aged in their 60s and under.

The drug, sodium selenate, has been shown to put the brakes on cognitive decline and neuro­degenerative damage in patients who are suffering from behavioural variant frontotemporal dementia (bvFTD) in Phase 1 trials led by Monash University researchers.

BvFTD is the second most common type of dementia in people aged under 60 and affects about 50,000 Australians.

The disease is associated with behavioural disturbances and personality changes that are highly disruptive to the lives of patients and their loved ones, leading to marriage breakdowns and reckless financial decisions, and can occur in people as young as 35.

The typical survival is five to seven years after diagnosis.

Monash department of neuroscience research fellow Lucy Vivash said the drug works by reversing the damage to the neurons in the brain which is caused by the build-up of a protein called tau. The drug activates enzymes that disperse the build-up of the protein in the brain.

“The problem is there is no treatment for these people; currently the best sort of medications are antipsychotics and depressants that taper the behaviours (but) they don’t actually treat the underlying disease,” she said.

“This is why it’s really exciting. What we’re looking at is a drug we think, and hope, targets the underlying mechanism of the disease.”

Dr Vivash said that BvFTD had tragic implications for those that contract it, with the disease often going unrecognised for years and a typical diagnosis taking five years.

“It’s really horrible, you tell people about it and they don’t believe it,” she said.

“It occurs in people who are quite young compared to other dementias – they’re in their 50s or 60s or middle age.

“They tend to experience these bizarre types of behavioural change and become impulsive, rude in social situations and laugh inappropriately and tell terrible jokes.

“It’s really bizarre behaviour which their family and friends find really upsetting, and it can lead to people getting divorced and can be really disruptive because of these weird symptoms, and patients get dismissed by their doctor who tells them you’re depressed or having a midlife crisis.”

Dr Vivash said that it was not yet understood what caused BvFTD, with only five per cent of cases reporting that they have a family member with the disease.

“We don’t really know; it seems like this enzyme that this drug is activating is slightly underactive in these patients but we don’t know what the trigger mechanism of the build-up of this protein,” she said.

“It is very much unknown at this point.”

Yarra Valley resident Rob Forbes, whose wife Michelle, 57, was diagnosed with BvFTD three years ago, said that he had noticed real improvements in his wife’s condition since she joined the trial earlier this year.

Mr Forbes realised something was wrong with his bright and capable wife with whom he had run four businesses when he found all her papers in disarray.

Since Michelle started taking sodium selenate he has noticed she has started picking up after herself and taking out the bins, habits which she had dropped as her disease progressed.

“It started with a lot of little things changing, small stuff like folding washing, taking washing off the line and ironing her clothes,” he said.

Dr Vivash said despite the promising results, the drug was probably seven years away from being fully approved and that she was actively recruiting for 100 patients for Phase 2 trials.

The results have been published in the journal Alzheimer’s and Dementia: Translational Research and Clinical Interventions.

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Original URL: https://www.theaustralian.com.au/science/australian-scientists-developing-breakthrough-drug-to-treat-a-rare-form-of-dementia-in-younger-people/news-story/87f4a5de2aa6fb1a3c15078fc4771da0