Monash University researchers are assessing a new drug that could be a game changer for the dementia that has struck US actor Bruce Willis, and countless others as young as 35: behavioural variant frontotemporal dementia (bvFTD).
Unlike other types of dementia, such as Alzheimer's Disease, there are no treatments; but a new drug — sodium selenate — was shown to be safe and well tolerated in people living with bvFTD, as outlined in the team's 2022 study.
Now, the team is announcing the next step: measuring the drug's impact on brain functioning.
While relatively rare, FTD causes progressive damage and shrinkage to either or both the frontal or temporal lobes of the brain, along with behavioural changes such as impulsivity, inappropriate behaviour and emotional indifference, and loss of language.
Neuroscientists Professor Terence O'Brien and Dr Lucy Vivash, from the School of Translational Medicine, are leading a phase 2b clinical trial in which half the participants will receive 52 weeks of treatment with sodium selenate, and the other half a placebo.
"This is an internationally unique clinical trial that, if positive, would bring to patients the first proven disease-modifying treatment for this currently untreatable and devastating progressive neurodegenerative disease," Professor O'Brien said.
"It's also an inexpensive drug, which is important, as we've seen recently that new promising treatments for dementias can cost much more than what governments and ordinary people can afford. Sodium selenate is not quite as cheap as aspirin, but it is unlikely to cost tens of thousands of dollars either."
Colleague Professor Amy Brodtmann, from the School of Translational Medicine, said while it is generally difficult to recruit patients with degenerative neurological conditions, FTD posed even more challenges. "Fronto-temporal dementia is often misdiagnosed as depression, anxiety or Alzheimer's Disease," she said.
"Carers or partners are often exhausted from navigating the health system for a correct diagnosis, as GPs tend not to think of dementia when the person in front of them is in their 30s, 40s or 50s."
Over the 52 weeks of the trial, the team will compare changes to brain volume in the treatment and placebo groups. They'll also look at the levels of tau, a protein involved in the development of FTD in the cerebrospinal fluid, the rate of cognitive decline, and behavioural changes.
The 12-month trial needs 120 participants, aged 35 years and older with a diagnosis of possible or probable bvFTD. Participation will involve cognitive tests, brain scans and regular phone check-ins.
