A collaborative study led by Dr. Nora Bengoa-Vergniory of the Oxford Parkinson's Disease Center showed that compounds called "molecular tweezers" may become promising disease-modifying treatments for Parkinson's.
A team of researchers has shown that tiny compounds called molecular "tweezers" may become a promising treatment for slowing Parkinson's disease. This new drug works by pulling apart toxic protein clumps that form in the brain during Parkinson's disease.
Previously, this therapy has shown the potential to target toxic protein clumps that form in neurodegenerative diseases such as Alzheimer's disease. Therefore, the research team investigated whether a specific molecular tweezer, CLR01, can reduce the formation of protein clumps in Parkinson's cells and mouse models.
Research shows that CLR01 can reduce the clusters formed by Parkinson's protein alpha-synuclein and prevent the death of human neurons made from stem cells.
Researchers tested CLR01 in a mouse model of Parkinson's disease, which stimulates the formation of protein clusters and mimics the motor symptoms experienced by people with this disease, including tremor and slow movement. As the mice age, CLR01 treatment reduces the appearance of movement problems and the formation of toxic protein clusters in the brain. Importantly, the research team showed that CLR01 is less effective in older animals with faster progression of Parkinson's disease.
This work shows that the use of protective therapy in the early stages of Parkinson's disease is essential for effective treatment. These combined results highlight that CLR01 represents a candidate for the treatment of Parkinson's disease and emphasize the need for further research in this area.
Lead researcher Dr. Nora Bengoa-Vergniory said: "Future investment in determining the appropriate therapeutic window for these therapeutic agents is critical to the success of these and other treatment strategies."