For over two decades, researchers and pharmaceutical companies have been trying to develop a drug that will slow or stop the progression of abnormal protein aggregation that can damage the brain or other organs. There are currently over 30 disorders including Parkinson’s and Alzheimer’s disease that involve protein buildup and none of them have a cure.
New research conducted at UCLA is bringing new hope. They have developed a compound that slows and reverses toxic protein clumps in the brains of animals genetically modified to develop Parkinson’s disease. With further study and successful human trials, this drug may help millions of people with Parkinson’s and other disorders involving protein aggregation.
How the Drug Works
A protein called α-synuclein, which is normally present throughout the brain is thought to be a pathway to Parkinson’s disease when it binds together in clumps and damages neurons.
Their compound, called CLR01 is shaped like a “C” and acts like a tweezer, grabbing chains of lysine, an amino acid that makes up most proteins. But, it only targets abnormal aggregates, nothing else.
In their study, CLR01 not only blocked the formation of α-synuclein aggregates it also reversed those that had already formed. And, the compound did not interfere with normal function of α-synuclein protein in the brain or show any other signs of toxicity.
This study used zebrafish, a tropical freshwater fish commonly found in aquariums. The zebrafish is a popular animal for research because it is easily manipulated genetically, develops rapidly and is transparent, making the measurement of biological processes easier.
Hope for a Cure
Being able to prevent protein aggregates, prevent toxicity and break up existing aggregates is a very encouraging result, but the research is still in its early stages.
Nonetheless, said the researchers, CLR01 holds great promise as a new drug that can slow or stop the progression of Parkinson's and related disorders. “This takes us one step closer to a cure," said study author, UCLA professor of neurology Jeff Bronstein.
The researchers are already studying CLR01 in a mouse model of Parkinson's and say they hope this will lead to human clinical trials.