In a new study, researchers found a small molecule that may slow down or stop the progression of Parkinson’s disease.
The research was conducted by a team at Rutgers University and Scripps Research
Parkinson’s, which affects 1 million people in the United States and over 10 million worldwide according to the Parkinson’s Foundation, is a neurodegenerative disorder with no cure.
Symptoms develop slowly over time and can be debilitating to patients, who most recognizably develop tremor, slow movements, and a shuffling gait.
A key feature of Parkinson’s disease is a protein named α-synuclein, which accumulates in an abnormal form in brain cells causing them to degenerate and die.
However, it has been difficult to target α-synuclein because it does not have a fixed structure and keeps changing its shape, making it very difficult for drugs to target.
Because higher levels of the protein in the brain speed the degeneration of brain cells, scientists have been looking for ways to decrease protein production as a form of treatment.
The team explored a novel idea for treating Parkinson’s disease using new technology.
The method matches RNA structure with small molecules or drug-like compounds.
The team believed this innovative technology could be used to find a drug that targets the messenger RNA that codes for α-synuclein, which causes the disease, in order to reduce the production of the protein in the brains of Parkinson’s patients.
In the study, by targeting messenger RNA, the team found a compound that prevents the harmful Parkinson’s protein from being made.
This new compound, named Synucleozid, reduces specifically α-synuclein levels and protects cells against the toxicity of the misfolded form of the protein, suggesting that it has the potential of preventing disease progression.
This new compound has the potential to do that and could change the course of life for people with this devastating disease.
The team says this discovery is ‘highly promising’ and is eager for the next steps in optimizing and testing the compound.
Additionally, this can benefit another devastating disease that also has α-synuclein clumps, known as Dementia with Lewy Bodies.
Further, this new concept of targeting RNA to reduce protein production may be applied to other challenging diseases because of their similar undruggable proteins including Alzheimer’s disease.
The study is published in PNAS.
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