Parkinson’s Disease Research at NYSCF

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About Parkinson’s Disease

Parkinson’s Disease Definition

Parkinson’s disease is a chronic, progressive, neurodegenerative disorder that affects over one million Americans and ten million people worldwide, including over 1% of people over the age of 60. The disease stems from dysfunction and deterioration of brain cells, mainly dopamine-producing neurons. 

Parkinson’s Disease Symptoms

The main motor symptoms of Parkinson’s are tremors, rigidity, slowness of movement, and impaired balance or coordination. A plethora of other debilitating symptoms are often observed, including depression, anxiety, irritability, insomnia, constipation, impulse control disorders, pain, fatigue, and vision problems.

Parkinson’s Disease Causes

While decades of research has begun to reveal genetic and biological factors that contribute to Parkinson’s disease, the precise mechanisms are not known yet, and there are no current treatments to change the disease course, only symptom management approaches.

Parkinson’s Disease Diagnosis and Prognosis

A Parkinson’s disease diagnosis cannot be determined by a single test but is rather assigned based on a patient’s medical history, symptoms, and a series of neurological and physical exams. Each patient’s Parkinson’s disease prognosis is different, as the disease is unique to each person. While all patients will experience some degree of motor dysfunction, the severity and course that the disease takes may differ from person to person.

Parkinson’s Disease Research at NYSCF

At NYSCF, we are studying the actual human brain cells implicated in the disease to develop novel Parkinson’s treatments. We are doing this in several steps:

1. We are using our unique, powerful robotic system for creating patient stem cells. Our NYSCF Global Stem Cell ArrayTM can rapidly, cleanly, and reproducibly create stem cells from skin or blood, and then reprogram them to become the brain cells implicated in Parkinson’s.

2. These patient-specific brain cells allow us to investigate the molecular and cellular causes of Parkinson’s as it develops so that we may see the actual decline and failure of diseased cells.

3. We can also use these cells to develop and test drugs, essentially running a “clinical trial in a dish” to identify the safest and most promising compounds.

Parkinson’s Disease Stem Cell News

Publications

Below are select publications on recent advancements in Parkinson’s research by NYSCF Research Institute scientists.

Directed Differentiation of Human Pluripotent Stem Cells to Microglia.
Douvaras P, Sun B, Wang M, Kruglikov I, Lallos G, Zimmer M, Terrenoire C, Zhang B, Gandy S, Schadt E, Freytes DO, Noggle S, Fossati V.
Stem Cell Reports. 2017. doi: 10.1016/j.stemcr.2017.04.023.

In this study, NYSCF researchers developed a protocol for turning stem cells into microglia (the immune cells of the brain). Microglia have recently been implicated in PD, and having an effective method to generate them will allow scientists to elucidate their precise role in the disease.

iPSC-Derived Dopamine Neurons Reveal Differences between Monozygotic Twins Discordant for Parkinson’s Disease.
Woodard CM, Campos BA, Kuo SH, Nirenberg MJ, Nestor MW, Zimmer M, Mosharov EV, Sulzer D, Zhou H, Paull D, Clark L, Schadt EE, Sardi SP, Rubin L, Eggan K, Brock M, Lipnick S, Rao M, Chang S, Li A, Noggle SA.
Cell Reports. 2014. doi: 10.1016/j.celrep.2014.10.023.

In this study, NYSCF scientists derived stem cells and then dopamine neurons from a genetically identical set of twins (one with Parkinson’s disease and one without). This revealed unexpected molecular differences between the twins’ cell lines that indicate which genetic and environmental factors may trigger Parkinson’s onset.

FAQs 

Is Parkinson’s genetic?

About 10% of Parkinson’s cases have been linked to genetics. Most cases are non-familial (are not accompanied by a genetic mutation). There are several genetic mutations known to be associated with increased risk for developing Parkinson’s, but not all people with these mutations will get the disease.

What is the most common Parkinson’s disease medication?

The most common medication for Parkinson’s disease is levodopa (L-dopa). L-dopa is a precursor to dopamine, the neurotransmitter that is depleted in Parkinson’s. This medication can only help with symptoms and does not stop or cure the disease. Current medications are also limited by serious side effects such as hallucinations, cognitive deficits, dizziness, sleepiness, and compulsive behaviors. At NYSCF, we are committed to finding safe and effective therapies that will do more than mask Parkinson’s disease symptoms, instead targeting the root of the disease.