Clinical collaboration for Parkinson's research
A collaboration between the , , the University of Sydney, , and is part of an Australian-led, international clinical trial to fast-track Parkinson's disease research.
With the combined depth of expertise across eight clinical trial sites around Australia including the Brain and Mind Centre's Parkinson's disease Research Clinic, the Australian Parkinson's Mission (APM) collaboration combines a revolutionary clinical trial design using repurposed drugs with genomics research to target disease-causing pathways.
Parkinson鈥檚 disease currently affects around 100,000 Australians, with an additional 14,000 being diagnosed with the disease each year. There is no known cure yet and currently, treatments address symptoms.
With the support of a federal government grant, the most significant investment in Australian Parkinson鈥檚 research to date, the Australian Parkinson鈥檚 Mission (APM) is a significant step towards developing disease-modifying treatments for Parkinson鈥檚 patients.
Over five years, the APM will combine clinical trials with genomics research, biomarker analysis and induced pluripotent stem cell testing, to test the effectiveness of repurposed drugs that have demonstrated potential for slowing or stopping Parkinson鈥檚 disease progression.
Bringing together global experts in the Parkinson鈥檚 arena
Professor Simon Lewis, Director of the ForeFront Parkinson's Disease Research Clinic at the Brain & Mind Centre is the Clinical Lead for the program, bringing critical expertise in clinical trial design to the balance of skills.
He says the clinical chemistry between the Brain and Mind Centre and the Garvan Institute makes for a perfect partnership.
鈥淔rom the very beginning of these discussions, I was very keen on the novel concept of targeting different pathways in the same trial鈥� said Professor Lewis.
鈥淥n the clinical side, we measure the traditional indicators of disease progression, whilst some of our leading scientists at the University of Sydney, Prof Glenda Halliday and Dr Nic Dzamko have developed blood tests evaluating target and disease engagement to look at those important pathways underpinning the disease. Finally, the genomics arm led by the Garvan allows us to progress the field of precision medicine by identifying key genetic signatures.鈥�
The first clinical trial is being conducted across eight sites across Australia, integrating clinical, biomarker and genomic information to identify the patients who respond to treatments that target specific changes taking place in the brain.
鈥淲e鈥檙e hoping that the outcomes of our clinical trials will provide evidence to suggest that a targeted treatment for treating patients with particular genetic signatures is possible鈥�, said Professor Lewis.
Repurposing drugs
The first clinical trial is a three-arm randomised rial of repurposed drugs that have been proposed by an international committee of leading Parkinson鈥檚 experts.
Participants will undergo genetic and biomarker analysis to classify variations specific to the disease and the particular response to treatment. Over 48 weeks, they will receive one of the three trial medications and continue on their usual Parkinson鈥檚 medication. At the end of the 48 week treatment period, the study medication will be stopped, and 12 weeks later they will be reassessed.
鈥淭he reason for that is that if just by chance any of those drugs happen to improve the symptoms of Parkinson's disease, but not modify the progression of it, you'd expect there to be a washout at the end of that last 12 weeks鈥�, said Professor Lewis.
Despite the impact of COVID-19, recruitment has been going strong and recruitment for APM Clinical Trial Two is expected to commence while the first trial is still running. 鈥淚n clinical trial two we will test different agents and target different pathways, in an effort to explore new ways beating the disease鈥�, Lewis explains.
New hope for Parkinson鈥檚 patients
Developing better patient care and family support for people with Parkinson鈥檚 Disease and related conditions is the key mission of the Brain and Mind Centre鈥檚 ForeFront Parkinson鈥檚 Disease Research Clinic.
鈥淲e鈥檙e currently running a symptomatic trial for Parkinson鈥檚 patients who have depression and we鈥檙e also recruiting for patients with Parkinson鈥檚 Disease, Dementia and Dementia Lewy Bodies, for a trial to try and improve their concentration鈥�, said Professor Lewis.
Also on the horizon is a trial that aims to treat Parkinson鈥檚 patients experiencing freezing of gait episodes 鈥� a particular research passion for Professor Lewis. His team is also working on another disease slowing trial for treating another rare movement disorder, known as Progressive Supranuclear Palsy.
With all clinical trials, there is a vital balance required for maximum results. Both the need for research funding, combined with the importance of patient involvement are the foundational building blocks for a successful outcome, working towards the successful development of patient care and treatment in the future.