Dementia Reversed in Mice, Human Trials Set to Start After $10 Million Grant

By Mimi Nguyen Ly

Treatment for dementia is in sight after the Australian federal government announced $10 million funding for a study that has been able to fully reverse dementia in mice, with clinical trials on humans set to begin late 2019.

The pioneering technique was developed in 2015 at the Queensland Brain Institute (QBI), said a research body at the University of Queensland (UQ). Researchers used a noninvasive technique that employs scanning ultrasound to clear out toxic buildups in the brain that are the hallmark of Alzheimer’s disease, a common form of dementia.

Professor Jurgen Gotz said the results were completely unexpected.

“Cognition was restored. So the mice were perfectly fine afterward, which was a surprise to us, but obviously was extremely encouraging,” he told the Australian Broadcasting Network.

There is currently no cure for dementia, a degenerative condition.

Gotz noted that the degenerative condition reaches a “point of no return” at a certain stage, and so the earlier the intervention, the greater the chance of being able to reverse dementia.

“Ideally we would treat a patient at the pre-symptomatic stage and that’s obviously where more funding is required to be able to achieve that,” Gotz told the ABC.

“Inevitably there is a point of no return. So we want to treat as early as possible before all the damage has occurred,” he added.

Human Clinical Trials Set to Start

Some tests in a small number of sheep suggest that the scanning ultrasound technique is safe to use in larger brains.

With the recent funding, UQ researchers hope to start human trials in late next year to check whether the treatment is safe for human use. The trial will enlist up to 10 people with early-onset dementia.

“Once the technology has been deemed safe, phase two of the trial is designed to determine whether ultrasound is an effective treatment for dementia,” the UQ website states.

“The goal, long-term, is to come up with an affordable, portable device, which would help the millions of Alzheimer’s patients in our country and worldwide,” Gotz told the ABC.

The government grant, alongside philanthropic donations, have brought the research institute closer to its $30 million goal.

Professor Pankaj Sah, the director at the University of Queensland’s Brain Institute said the funding helps to fast-track scientists’ understanding of how scanning ultrasound can be used to more effectively treat dementia.

“The technology temporarily opens the blood-brain-barrier to remove toxic plaques from the brain and has successfully reversed Alzheimer’s symptoms and restored memory function in animal models,” Sah said, according to a UQ release.

“The human safety trials late next year are the next step, representing an investment in research that is already underway. Funding is essential if we are to continue to move closer to producing a non-invasive treatment for dementia.”

The funding will support the team of 90 researchers at QBI’s Clem Jones Centre for Ageing Dementia Research (CJCADR) over the next five years.

“As an indication, the average timeframe from the point of discovery to a treatment being available is 15 years. The initial ultrasound discovery was made in 2015,” the QBI website said.

Without a medical breakthrough, the number of Australians living with dementia is expected to increase to 1.1 million by 2056, the ABC reported.

Dementia is estimated to affect about 50 million people worldwide, 400,000 of whom are in Australia.

Those who may be interested in joining the trials can find out more information about whether they are eligible at www.anzctr.org.au, once the eligibility criteria are made available.