The NYSCF scientific team is leveraging recent advances in stem cell research to study Parkinson's through the creation of disease-specific stem cell lines.
Chris Fasano, PhD,
2008 NYSCF-Druckenmiller Fellow
Parkinson’s disease affects nearly one million people in the United Stated and ten million people worldwide. Parkinson’s is a chronic and movement disorder that results from the death of neurons in the brain. This debilitating disease may be marked by tremors, impaired coordination, and slow movements among many other symptoms.
Made possible by the NYSCF – Golub Stem Cell Research Initiative for Parkinson's Disease and in collaboration with scientists from around the country, we are developing stem cell disease models and stem cell arrays for Parkinson's disease. The derivation of these stem cell lines will allow us to create models for the sophisticated investigation of the molecular and cellular causes of the disease. These in turn will lead to the discovery of cell-based treatments and new drugs for Parkinson's patients.
In the first phase of this research, being led by Dr. Scott Noggle, we will study the progression of the disease using these arrays, which will enable us to hone in on the individual triggers that cause Parkinson's. Second, in the drug discovery phase, we will search for drug-like compounds that block the progression of the disease. The goal of the drug discovery phase is to generate lead compounds that pharmaceutical partners can develop into clinically viable drugs tailored to individual patients.
These stem cell lines will enable NYSCF to overcome the fundamental challenges that have kept scientists and clinicians from fully understanding this devastating neurological condition.
NYSCF Lab Director Dr. Scott Noggle
Why Stem Cells?
Stem cell research holds the promise for scientists to overcome one of the most challenging problems in studying human disease: the fact that by the time the disease is diagnosed, many of the events that led to it have already occurred, preventing scientists from understanding its precise origin. It also gives us an unprecedented opportunity to create the cells needed for cell replacement therapy and for screening drugs on the actual cells that are getting sick in diseases.
The problem encountered by scientists attempting to study disease or injury is analogous to that faced by investigative teams trying to understand the causes of a plane crash before the advent of the "black box," when understanding the often subtle events that led to the incident was nearly impossible. The cell-based models of disease created in the NYSCF laboratory will serve as "data recorders" for the study of disease. Just as flight recorders allow the Federal Aviation Administration to replay air accidents over and over again to understand the underlying causes of a crash, these cell models will enable us to repeatedly replay the development of disease until it is fully understood.
Scientific research has been further hampered by the inability to obtain adequate numbers of the actual type of cells needed for research in specific diseases or other medical conditions. As a result, scientists have traditionally turned to the study of rodent models, which often poorly recapitulate human disease. NYSCF scientists have shown that human pluripotent stem cells carrying the genes causing a specific disease can replicate many of the degenerative processes that occur in humans with that disease. The ability of these cells to grow indefinitely in culture, while retaining the capacity to differentiate into all the cells of the body, will allow us to produce a limitless quantity of these degenerating cells for use in disease studies and drug identification.
Recent Parkinson's Disease Research:
“Direct generation of functional dopaminergic neurons from mouse and human fibroblasts”
Scientists under Dr. Vania Broccoli have discovered three factors that were able to elicit conversion of mouse and human fibroblasts to dopaminergic neurons. Direct generation of dopaminergic neurons from somatic cells may have significant implications for understanding critical processes for neuronal development and cell replacement therapies.
“Protein-based human iPS cells efficiently generate functional dopamine neurons and can treat a rat model of Parkinson disease”
Stem cell have shown promise for the treatment of Parkinson’s disease, but the safety and efficacy of transplanted cells has been variable. Now, scientists under Sang-Hu Lee at Hanyang University have compared the safety of several types of human iPSCs and found that those derived via protein-based techniques were able to generate healthy dopamine neurons and rescue motor deficits in a rat model of Parkinson’s.