One step closer

Not having control over your body may seem like an alien notion to the vast majority of us, but this is the reality for 10 million people living globally with Parkinson’s disease. Parkinson’s disease is a progressive neurological disorder where the brain does not produce enough dopamine – a neurotransmitter that relays chemical messages between cells in the brain.

Dopamine is used by the brain in many ways, but is particularly important in controlling movement. This lack of dopamine is what causes the most well-known symptoms of Parkinson’s – body tremors, muscle rigidity and difficulty moving, which gradually progress and worsen over time. However, people with Parkinson’s can also be affected by a broad range of non-motor symptoms including cognitive impairment and mood disorders.

The cause of Parkinson’s is unknown, with available treatments only masking the symptoms of the disease. There is no way for clinicians to detect and diagnose the disease early, meaning that a person diagnosed with Parkinson’s will already have irreversible symptoms of the illness, with current treatments unable to stop, or even slow its progression.

An international effort

Without a medical breakthrough, the number of Australians diagnosed with this devastating illness is set to double every fifteen years. To address the critical need for more effective treatments, the Garvan Institute of Medical Research in collaboration with the Shake It Up Australia Foundation, Parkinson’s Australia, The Cure Parkinson’s Trust (UK) and The Michael J. Fox Foundation for Parkinson’s Research (USA) have launched the Australian Parkinson’s Mission (APM), a world-first clinical trial design.

The APM, an Australian-led initiative, will take a new approach to researching Parkinson’s treatments by integrating clinical trials which will test four repurposed drugs, with whole genome sequencing and biomarker analysis. Each patient enrolled in the clinical trial will have their genome – their entire DNA – sequenced with the aim to reveal the different disease subtypes of Parkinson’s and uncover which drug will work for each subtype.

Analysis of patient biomarkers – naturally occurring measurable indicators of a disease – will further enable researchers to identify and measure the efficacy of the repurposed drugs.

Trial to patient, fast-tracked

“One of the aims of the APM is to be able to deliver effective treatments to people with Parkinson’s as quickly as possible,” says Associate Professor Antony Cooper, Head of the Neurodegeneration and Neurogenomics Program at Garvan who is spearheading the program.

An ongoing mission

The APM will begin enrolling people with Parkinson’s onto the program in mid-2019 when clinical trials are due to begin, but the breadth of this program will see it evolve over time.

“We’ve identified complementary expertise from across the globe to create what can be a truly game-changing clinical program for people with Parkinson’s”, says Associate Professor Cooper.