Scientists may have created a drug that stops the development of the disease
Last reviewed: 16.10.2021
All iLive content is medically reviewed or fact checked to ensure as much factual accuracy as possible.
We have strict sourcing guidelines and only link to reputable media sites, academic research institutions and, whenever possible, medically peer reviewed studies. Note that the numbers in parentheses ([1], [2], etc.) are clickable links to these studies.
If you feel that any of our content is inaccurate, out-of-date, or otherwise questionable, please select it and press Ctrl + Enter.
A professor of neurology at the University of California, Los Angeles, Jeff Bronstein and colleagues created a new compound that can serve as a "molecular forceps": it captures the molecules of the alpha-sinuclein protein in certain places, preventing them from clumping together, reports MedicalXpress.
Alpha-synuclein is considered one of the factors that provoke Parkinson's disease : the disease breaks down its structure, becomes amorphous and disordered, which leads to the formation of protein aggregates, as well as to the death of neurons of the central nervous system.
Molecular tweezers, created by Californian scientists, not only inhibit the formation of aggregates of alpha-synuclein, but also suppresses the toxicity of this protein and destroys the already existing aggregates. However, it does not affect the normal functions of the brain.
Molecular tweezers are non-cyclic molecules that have two ends - two "hands", capable of capturing other molecules through non-covalent bonds. The tweezer molecule for alpha-synuclein is called CLR01, it has the shape of the letter "C" and the chemical structure, through which it "wraps" the protein chain in the places where the amino acid lysine is located. This amino acid is part of most proteins.
The action of CLR01 was tested both in cell cultures and in the living organism, on transgenic zebrafish aquarium fish, which served as a model for Parkinson's disease. Danio is used as a laboratory facility because it is easy to carry out genetic engineering manipulations with them, and in addition they are transparent and this allows you to visualize biological experiments.
Model aquarium fish carried alpha-synuclein, labeled with a green fluorescent protein, which made it possible to track the state of protein aggregates under the influence of the molecular tweezers CLR01. In these experiments, just as in cell cultures, the same effect was observed. CLR01 prevented the formation of aggregates of alpha-synuclein, the death of neurons due to the toxic effect of protein aggregates, and also caused the destruction of existing units.
These results inspired scientists to new experiments with molecular tweezers: they are currently studying the action of CLR01 in mice - models of Parkinson's disease and hope that these studies will ultimately lead to trials in humans.
Currently, for people with Parkinson's disease there is only symptomatic treatment, there are no drugs that stop the development of the disease.
[