Innovative Stem Cell Tools for Parkinson's Assay Development

Funding Type: 
Tools and Technologies I
Grant Number: 
RT1-01038
Investigator: 
ICOC Funds Committed: 
$0
oldStatus: 
Closed
Public Abstract: 
Parkinson's disease is a neurodegenerative disease resulting in the loss of a specialized class of cells in the brain that control movement and coordination. Current pharmaceutical treatments for Parkinson’s disease typically supplement the neurotransmitter produced by the cells, yet unfortunately the disease is very progressive and even more of these cells are lost. A very promising treatment, therefore, would focus on a strategy to protect these cells and prevent the disease from progressing. One way to protect these cells is to use pharmaceutical medicines. But in order to identify candidate drugs, most strategies require growing large number of these cells in a dish and testing compounds that can protect them from the effects of Parkinson’s-causing chemicals or genetic predisposition. To date, insufficient sources of cells have existed, making large-scale screening impossible. Embryonic stem (ES) cells have the marvelous capacity to divide indefinitely, making them an ideal resource to derive large numbers of specialized cells. Yet the differentiation process, by which they are made into specialized cells, varies for each type of cell desired. Previously, a lengthy process was required in order to derive the specialized cells involved in Parkinson’s disease, but recent research has advanced this goal such that a simple differentiation technique may be soon realized. Our strategy takes advantage of previous results to produce two genetically modified human embryonic stem cell lines for use in a drug screening assay to protect Parkinson’s cells from similar toxins that cause the disease. One key advance of our strategy will be the inclusion of recombination sequences in key genes, allowing other researchers to ‘hot swap’ genes and other markers of interest for a variety of purposes. The assay described in this proposal will be the first of its kind in California, and will pave the way towards the development of large-scale pharmaceutical screens. The proposed research provides long-term benefits to California State citizens through design of a novel in vitro neuroprotective assay that could lead to novel and safer drugs for Parkinson's patients, significant academic research tools to aid in the understanding of the biology Parkinson's disease, intellectual property gains fostering commercial development, increase in the biotechnology strength in the state, generate jobs, generate sales of reagents and supplies from California companies, and bring in revenues from companies outside the state via corporate partnering.
Statement of Benefit to California: 
By approving Proposition 71, voters in California agreed to fund ground breaking stem cell research in regenerative medicine to improve the lives of California citizens, and to benefit the State economically. Our proposal is designed to combat Parkinson's disease, which affects an atypically large percentage of California State citizens compared to most states. Parkinson's disease is a neurodegenerative disease resulting in the loss of a specialized class of dopamine-producing neurons. Although there are genetic origins of Parkinson's disease, the large majority of cases are idiopathic. According to many reports, pesticides such as paraquat and dieldrin used by California's significant agricultural community may be linked to many of these cases. The successful completion of the goals proposed here will result in two genetically modified stem cell lines for use in a drug screening assay to protect these neurons from toxic insults. Further insights into Parkinson’s disease may also alter pesticide application and will translate into better protection for California State citizens. As the first of its kind in California, this assay will provide long-term benefits leading to new preclinical pharmaceutical development for environmental and genetic Parkinson's disease, improving immeasurably the lives of thousands of California State citizens and reducing associated health care expenditures. As well, it is anticipated that the cell lines generated from this project will serve as significant research tools for improving the differentiation of dopaminergic neurons for clinical purposes, the novel in vitro neuroprotective assay developed with this funding could lead to novel and safer drugs for Parkinson's patients, significant academic research tools to aid in the understanding of the biology Parkinson's disease, intellectual property gains fostering commercial development, increase in the biotechnology strength in the state, generate jobs, generate sales of reagents and supplies from California companies, and bring in revenues from companies outside the state via corporate partnering.

© 2013 California Institute for Regenerative Medicine