A key problem in using stem cells to better understand human disease and to develop new therapies is the need to generate large quantities of highly purified, rigorously characterized, functionally robust and viable cells of various types. Here we propose to develop the tools and flow cytometry technology needed to solve this problem in the specific case of neurons in the human brain that are damaged in Parkinson's Disease and in Alzheimer's Disease. Our goal is to develop tools to generate and purify these special neurons from human ES and iPS cells.
The lessons we learn in the solution of these cases will then be extended to the generation of a general technological approach to solving in vitro differentiation problems in any cellular system using flow cytometry and surface markers.
We seek to define systems that enable and permit safe and effective utilization of the many approaches in regenerative medicine that are being developed from hESC or iPS cell sources, to be safely used in Phase I and subsequent trials to establish their utility in clinical therapy.
Such purified cells will also be crucial reagents for finding and testing new drugs for the treatment of AD, PD, and other diseases.
Statement of Benefit to California:
Developing better understanding and treatments for a variety of diseases using human stem cell technology requires not only inducing differentiation of the required cell types, but also requires purifying them sufficiently that they would be useful for research or safe for transplantation. Our proposal is to develop methods to generate and purify two defined types of useful neurons for AD and PD, which has the long-term benefit on contributing to therapy development for very costly and devastating diseases.
In addition, the methods and tools we develop may be commercializable and stimulate new business and beneficial economic activity in California.
It is our expectation that this technological approach to greatly reducing one of the key identified risks in the potential therapeutic use of hESC derived differentiated cells, may thus more rapidly enable the initiation of Phase I and further cllnical trials of various cell types being developed in California under the CIRM program.