According to a research that lays the way for a new medicine to cure the ailment, people who get Parkinson's disease before the age of 50 may have been born with disrupted brain cells that remained unnoticed for decades. Parkinson's disease develops when brain neurons that produce dopamine, a chemical that aids in muscle coordination, are damaged or destroyed. Slowness of movement, tight muscles, tremors, and loss of balance are symptoms that worsen with time.
According to study published in the journal Nature Medicine, 10% of patients are between the ages of 21 and 50, despite the fact that the majority are 60 or older when they are diagnosed. Because it affects individuals in the prime of life, "young-onset Parkinson's is exceptionally distressing," said Michele Tagliati, a professor at the Cedars-Sinai Medical Center in the US.
According to Tagliati, "this exciting new discovery gives hope that one day we may be able to diagnose and prevent this illness in at-risk people by taking early intervention." From the cells of individuals with young-onset Parkinson's disease, the study team created unique stem cells known as induced pluripotent stem cells (iPSCs).
This procedure includes returning adult blood cells to an early embryonic stage. The iPSCs are stem cells that have the potential to differentiate into a wide variety of cell types throughout development and early life.
The scientists created dopamine neurons from each patient's iPSCs, grew them in a dish, and examined the functioning of the neurons. Clive Svendsen, head of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, remarked, "Our technology gives us a window back in time to assess how well the dopamine neurons would have functioned from the very start of a patient's existence."
Two significant deviations were found in the dopamine neurons in the dish by the researchers. Alpha-synuclein, a protein that builds up in most cases of Parkinson's disease, was the first.
The second was malfunctioning lysosomes, which are cellular organelles that serve as the cell's "trash cans" for dissolving and discarding proteins. According to the researchers, alpha-synuclein might accumulate as a result of this defect.
The very first symptoms of young-onset Parkinson's are what are being seen using this new model, according to Svendsen. According to him, these people's dopamine neurons may continue to handle alpha-synuclein incorrectly for 20 or 30 years before developing Parkinson's symptoms.
Additionally, the researchers tested a range of medications that may be able to repair the defects they had seen using their iPSC model. They discovered that PEP005 lowered the high levels of alpha-synuclein in both the laboratory mice and the dopamine neurons in a dish.
The US Food and Drug Administration (FDA) has previously given PEP005 approval to treat skin precancers. Along with this aberration, the medicine also reduced increased levels of a protein kinase C variant whose significance in Parkinson's disease is unclear, the researchers discovered in the patients' dopamine neurons.
The goal of the study is to determine the best method for delivering PEP005, which is presently only accessible in gel form, to the brain in order to treat or prevent young-onset Parkinson's disease.