Prana Biotechnology has announced that it has begun recruiting healthy volunteers for its Phase I clinical trial of the experimental drug PBT434. The drug is thought to affect processes linked to Parkinson’s disease and atypical parkinsonism diseases, including multiple system atrophy and progressive supranuclear palsy. The full article can be found here, at Business Wire.
About Prana’s New Treatment for Multiple System Atrophy (MSA) and Progressive Supranuclear Palsy (PSP)
Prana’s initial targeted diseases for the drug PBT434 are multiple system atrophy (MSA) and progressive supranuclear palsy (PSP).
About Multiple System Atrophy (MSA)
MSA is a neurodegenerative condition caused by the deterioration of nerve cells in the brain. People affected by the disease may experience symptoms that include issues with their balance and movement, and problems with some automatic functions such as bladder control, breathing, blood pressure, and, in men, erectile dysfunction. The condition usually first appears between 50 and 60 years old, but some people may be affected as young as age thirty.
About Progressive Supranuclear Palsy (PSP)
PSP also affects the brain and is caused by damage to brain cells as a result of the protein tau building up. Tau is usually broken down, but in people affected by PSP, the protein is not adequately removed and instead forms clumps in the brain cells that damages them. The amount of tau and the areas of the brain it accumulates in varies between people who are affected resulting in different symptoms in different individuals. Some common effects of the condition include issues with movement, balance, speech, swallowing, and vision, as well as memory problems and behavioural changes. 4,000 people in the UK are estimated to have PSP, but since the condition is often misdiagnosed or undiagnosed, the actual numbers may be higher.
The Experimental Drug Explained
The experimental drug PBT434 has been proposed as a treatment for these two conditions, which both currently face a huge unmet medical need for effective treatments. PBT434 is a small molecule thought to inhibit the accumulation of specific proteins, including tau, which are linked to Parkinson’s disease and atypical parkinsonism conditions.
Previous research has found that PBT434 is effective at reducing accumulations of the targeted proteins. However, this research was carried out on animal models of disease, and the safety and tolerability of the drug need to be further evaluated in humans.
A Phase I clinical trial of the drug in healthy volunteers has recently received approval from an ethics committee and recruitment for the study has begun. It will study the safety and tolerability of the drug, and monitor how the drug is metabolised in humans. The trial is located in Melbourne, Australia and is being conducted by the Nucleus Network.
Further updates about the trial are expected to follow as it progresses.