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Scientists may have created a drug that stops the progression of the disease
Last reviewed: 01.07.2025
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UCLA neurology professor Jeff Bronstein and his colleagues have created a new compound that can act as "molecular tweezers" by grabbing alpha-synuclein protein molecules at specific locations, preventing them from sticking together, MedicalXpress reports.
Alpha-synuclein is considered one of the factors that provoke Parkinson's disease: during the disease, its structure is disrupted, becomes amorphous and disordered, which leads to the formation of protein aggregates, as well as the death of neurons in the central nervous system.
The molecular tweezers created by Californian scientists not only prevent the formation of alpha-synuclein aggregates, but also suppress the toxicity of this protein and destroy existing aggregates. At the same time, it does not affect normal brain function.
Molecular tweezers are non-cyclic molecules that have two ends - two "arms" - that can grab other molecules through non-covalent bonds. The tweezers molecule for alpha-synuclein is called CLR01, is shaped like the letter "C" and has a chemical structure that allows it to "grasp" the protein chain at the sites where the amino acid lysine is found. This amino acid is found in most proteins.
The effects of CLR01 have been tested in both cell cultures and in vivo, using transgenic aquarium fish called zebrafish, which serve as a model for Parkinson's disease. Zebrafish are used as a laboratory model because they are easy to manipulate genetically and are transparent, which allows for the visualization of biological experiments.
Model aquarium fish carried alpha-synuclein labeled with green fluorescent protein, which allowed tracking the state of protein aggregates under the influence of molecular tweezers CLR01. In these experiments, as in cell cultures, the same effect was observed. CLR01 prevented the formation of alpha-synuclein aggregates, the death of neurons due to the toxic effect of protein aggregates, and also caused the destruction of existing aggregates.
These results have inspired the scientists to experiment with their molecular tweezers again: they are currently studying the effects of CLR01 in mouse models of Parkinson's disease, and hope that these studies will eventually lead to human trials.
Currently, there is only symptomatic treatment for people with Parkinson's disease; there are no drugs that stop the progression of the disease.
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