Dementia ultrasound treatment clinical trial

Thank you for your interest in the dementia research at QBI’s Clem Jones Centre for Ageing Dementia Research.   

The Clem Jones Centre for Ageing Dementia Research (CJCADR) is driven by the insight that fundamental, basic research is required to provide a solution to neurodegenerative diseases, in particular those affecting older people, including Alzheimer's disease. These approaches converge around four priorities: 

  • to understand the pathomechanisms of dementia; 
  • to develop new technologies and tools that help us understand pathological processes;
  • to develop novel diagnostics and biomarkers; and 
  • to design new treatments and preventive approaches. 

Primarily established to support fundamental brain research at a cellular and molecular level, CJCADR discoveries are moving toward clinical studies and trials, including:

  • Ultrasound as a potential non-invasive therapy for Alzheimer’s disease – phase I clinical trial in planning.
  • Determining the ideal exercise intensity and duration for older people to maintain and improve cognitive function - current clinical study.
  • Exploring the links between sleep apnoea and increased risk of dementia - current clinical study.

CJCADR researchers are developing a pioneering ultrasound technique that could delay the effects of dementia. A phase I clinical safety trial involving a small number of patients, which is planned for commencement late in 2019, will explore whether the technique discovered at UQ’s Queensland Brain Institute (QBI) in 2015 is safe to use in the fight against Alzheimer’s disease.

The technology, which temporarily opens the blood-brain barrier enabling the clearance of toxic protein plaques from the brain, has successfully reversed Alzheimer’s symptoms, and restored memory function in animal models. The phase I clinical safety trial planned for late 2019 is the next step in the development pathway to bring this exciting technology from initial discovery to application.

FAQs

What stage is QBI at with dementia research?

Since the Clem Jones Centre for Ageing Dementia Research was established in 2013, researchers have made a number of scientific discoveries that have progressed development of the ultrasound technology from the initial discovery through to our current point. These include:

  • The breakthrough discovery, published in 2015 that ultrasound can clear the toxic amyloid-beta plaque build ups that are the hallmark of Alzheimer’s disease,
  • That repeated treatments with ultrasound in mice are effective in clearing toxic protein plaques from the brain and restoring memory function, without the need for additional therapeutic drugs.
  • That the non-invasive ultrasound technique can be used safely on older rodents
  • The research has progressed to a large animal model that has more similarities with humans and the ultrasound technique has been found to be safe in a small number of sheep 
  • The research team now includes engineers who are designing the ultrasound equipment.
  • The team is developing immunotherapies with potential to enhance the effectiveness of the ultrasound technology.

Current CJCADR research also includes: 

  • A clinical trial to determine the ideal intensity and duration of exercise for older people to maintain cognitive function
  • A clinical study to investigate the link between sleep apnoea and dementia.
  • Research to understand what happens inside cells as toxic proteins begin to accumulate, resulting in potential treatment targets.
  • Research into biomarkers that may signpost the initial inflammatory response in the brain associated with Alzheimer’s disease, which may lead to earlier diagnosis and new treatments targets.
     

When will the treatment be available for those living with dementia?

It is too early to give a definitive answer. 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.

 

What is a clinical trial?

Clinical trials are research investigations in which people volunteer to test new treatments, interventions or tests as a means to prevent, detect, treat or manage various diseases or medical conditions. Clinical trials are run over three phases. The first stage of a clinical trial is phase I which determines safety but not whether the treatment is effective. Once the safety trial concludes then later stage clinical trials investigate whether the treatment works.

 

When will the ultrasound clinical trial commence?

The University of Queensland, through the Queensland Brain Institute, will work with dementia clinicians to commence recruiting patients for a small clinical safety trial in late 2019. The University has partnered with senior dementia specialists to conduct the trials, which will be run in Brisbane, Australia.The exact commencement date for recruitment and the specific location of the trials is yet to be determined.

 

What will the trials involve?

Trials for the use of ultrasound for the treatment of dementia have been successful in mouse models. Initial tests in sheep indicate that ultrasound is safe to use in a larger brain. The next stage of the process is to test the technology in humans. Phase I of the clinical trial is designed to establish that scanning ultrasound is safe for human use. Once the technology has been deemed safe, phase two of the trial is designed to determine whether ultrasound is an effective treatment for dementia.

 

What is the overarching aim of the trial?

The initial trial aims to confirm that scanning ultrasound is a safe, non-invasive treatment for dementia. Subsequent trials aim to establish the use of scanning ultrasound an effective treatment for Alzheimer’s disease.  In parallel, the researchers will investigate scanning ultrasound as a drug delivery tool in order to increase the effectiveness of drugs designed for dementia and, potentially, a range of other neurological disorders.  

How will we recruit for the trial?

The University of Queensland, through the Queensland Brain Institute, will work with a senior specialist clinician to recruit for the safety trial. There will be rigorous criteria - subject to ethical approval - to validate candidates for the trial. We estimate up to 30 potential candidates in total will be screened, in order to identify approximately 10 suitable participants for the safety trial.

 

Is QBI keeping a list of people interested in participating in the clinical trial?

No, QBI is unable to keep a list of interested parties as recruitment will be through specialist clinicians. 

 

If you have already received the funds required to commence the clinical trials, then why are you asking for more donations?

These clinical trials are testing just one of a number of possible methods for treating dementia – learning more about the disease and its causes remains a primary point of focus for our scientists and clinicians. Researchers across the University are approaching this issue with different strategies and donor funding will help these projects significantly. The more support received, the more likely we will reach positive outcomes, treatments and understandings sooner.

 

How much of my donation actually goes to the cause?

When you give through The University of Queensland, 100 per cent of donor contributions go to your nominated cause.

 

How can I find out more?

Information on the project’s progress will be made available periodically through QBI's website. You can also subscribe to the CJCADR newsletter below: