New Parkinson’s Disease Discovery Could Bring Hope to Thousands

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Parkinson’s disease is known for affecting mostly older people, but the good news is that recent research suggests it can be preventable. Scientists have discovered that Parkinson’s disease is an illness that may actually be present in the brain right from the moment of birth or even earlier.

Scientists from Cedars-Sinai medical center have discovered than in the brains of Parkinson’s patient’s can be found malfunctioning neurons from the early stages of life, but it takes about 20 to 30 years for the symptoms to accumulate. The good news about this drug, already on the market and easily found, could help in preventing Parkinson’s disease from manifesting.

Parkinson affects the neurons in the brain that produce dopamine, causing muscle weakness and stiffness, balance problems and shaky hands. Even though Parkinson’s disease is an illness that is diagnosed most of the time in older people over the age of 60, around 20 percent of cases appear in people aged between 21 and 50.

Scientists began a new study to investigate if there are early warning signs of Parkinson in patient’s neurons, diagnosed before they turned 50. To do so, they have created induced pluripotent stem cells (IPSCs) from younger patients that suffer from Parkinson’s, which can be turned into any other cell in the body.

They used pluripotent stem cells to grow dopamine neurons in the lab. When watching the cells develop, the researchers noticed that cell structures (lysosomes) were malfunctioning.

Lysosomes are responsible for breaking down worn-out proteins. When unneeded proteins don’t work as well as they should, they begin to pile up. A type of protein that existed in higher amounts was alpha-synuclein, which can be found in many forms of Parkinson’s

“Our technique gave us a window back in time to see how well the dopamine neurons might have functioned from the very start of a patient’s life,” says Clive Svendsen, senior author of the study.

“What we are seeing using this new model are the very first signs of young-onset Parkinson’s. It appears that dopamine neurons in these individuals may continue to mishandle alpha-synuclein over a period of 20 or 30 years, causing Parkinson’s symptoms to emerge.”

The researchers also investigated if Parkinson’s could be prevented or treated. After many unsuccessful tests, they came across a drug that had potential. PEP005 is a drug that’s used against skin precancers and has already been approved by the FDA and it is used against skin precancers. The team also found that this drug works in lowering the levels of alpha-synuclein as well as another enzyme called protein kinase C, which has an unclear role in relation to Parkinson’s disease.

Even though this treatment may seem promising, for now, it is still in the early stages of lab work, only being tested on mice and other lab-grown cells, so it needs to be translated into human trials first. In addition, as they continue clinical testing, they also have to figure out how to make PEP005 adapt for use in the brain as currently, this drug is only available in gel form as it is exclusively used for treating skin cancer.

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