A new therapeutic strategy that can slow the progression of Parkinson's disease by inhibiting the production of a specific lipid component in brain cells has been developed in South Korea.
A research team led by Professor Oh Chang-myung of the Department of Biomedical Science and Engineering at Gwangju Institute of Science and Technology (GIST) confirmed that the accumulation of ceramide is a key cause of protein aggregation and neuronal damage, and published findings showing that inhibiting ceramide can mitigate the pathological progression of Parkinson's disease, the scientific community reported Tuesday.
Ceramide is a metabolite that functions like a lipid within brain cells, regulating cell structure and signal transduction. It is known to accumulate abnormally during aging and in neurodegenerative diseases. In Parkinson's disease specifically, ceramide is known to promote the aggregation of alpha-synuclein, a protein that damages nerve cells.
The researchers tested the effects of administering myriocin, a substance that inhibits ceramide production, using Parkinson's disease animal models and patient-derived cells.
In the experiments, myriocin was administered for five to seven months to laboratory mice engineered to develop abnormal alpha-synuclein protein clumping. The results showed reduced protein aggregation, improved motor function and memory, and significantly less damage to dopaminergic neurons.
Myriocin also lowered the expression of inflammation-related genes and restored signal transduction in dopaminergic neurons — which regulate movement, memory and concentration — to near-normal levels.
The researchers further confirmed that mitophagy, the process of removing damaged mitochondria within cells, was activated, while neuroinflammation and cell death decreased. This suggests that the activation of mitophagy serves as a key mechanism in improving Parkinson's disease pathology.
