What is it?
Parkinson’s disease (PD) is a progressive neurological condition of the central nervous system. Named after English doctor James Parkinson in 1817 and first described as the “shaking palsy”, Parkinson’s disease is characterised by shaking, rigid limbs, slow movement, and later thinking and behavioural problems alongside depression.
While scientists are still unsure why Parkinson’s disease develops, we know it is linked to the production and levels of the chemical dopamine, which is critical for motor control, caused by the death of brain cells. Parkinson’s disease is not life-threatening but symptoms get progressively worse over time.
Theories on what causes Parkinson’s disease include environmental toxins, oxidative stress, genetic factors or a combination of all three.
How common is it?
The Parkinson’s Disease Foundation estimates up to 10 million people are living with Parkinson’s disease worldwide, including 1 million in the United States, 127,000 in the UK and 66,000 in Australia. While Parkinson’s disease is linked with age, about 5% of people with PD are diagnosed before they turn 40. In the US, Parkinson’s disease is estimated to cost society $25 billion per year.
What is the limit of existing therapy?
There is currently no cure for Parkinson’s disease but a range of treatments do exist, although they can cost up to $1500 per year for medication and $100,000 for therapeutic surgery. Drug treatments work by restoring dopamine levels in the brain, but are known to have side effects including involuntary movements and compulsive behaviour.
What is Prana’s progress?
PBT434 is being advanced as a treatment for Parkinson’s disease, with research funded by the Michael J Fox Foundation. PBT434 has advanced through discovery, chemistry and screening and is currently in the final stage of preclinical testing.
PBT434 is the first molecule designed to inhibit the neurotoxic build-up of alpha synuclein in the brain to support the next generation of PD therapies.
In September 2013, Parkinson's UK awarded £150,000 (AU$260,000) to The University of Leeds to study the mechanism of action of PBT434. Principal Investigator Dr James Duce, from The University of Leeds UK, will collaborate with Professor David Finkelstein of The Florey Institute of Neuroscience and Mental Health (FINMH) in Australia and Associate Professor Robert Cherny of FINMH and Prana’s Head of Research on the Study.
The funds will be used over 18 months to study the mechanism of action of PBT434 in animal models of Parkinson’s disease and Parkinsonian syndromes. In particular, the ability of PBT434 to reduce the elevated iron levels in the Parkinsonian brain, attenuate oxidative stress and alter the function or abundance of iron regulated proteins.
Data presented at 2013 Annual Congress of Parkinson's Disease and Movement Disorders also showed PBT434 has significant disease-modifying capability in multiple animal models of Parkinson's Disease (PD), with potential utility in a range of movement disorders.
The findings were presented by Professor Colin Masters, Director of The Mental Health Research Institute at the Florey Institute of Neuroscience, in a plenary presentation, and Associate Professor David Finkelstein, Head of the Parkinson's Disease Laboratory at the Florey Institute.
A further 1000 novel MPACs are under screening and assessment for therapeutic applications in neurodegenerative diseases.