Public Abstract:
A major goal of the Shared Research Laboratory (SRL) is to foster the development of new treatments for human diseases and disorders by serving as a leading regional center for human embryonic stem cell (hESC) research, clinical applications, and training. A critical component of this vision is a full service SRL. The SRL will provide space and equipment that is free of federal funding to allow pursuit of any study needed to discover the basic properties of hESCs, to understand disease processes, to accelerate drug development and to develop cell-based therapeutics. The research in the SRL includes a balance of studies into the basic biology of hESCs, disease mechanisms, and potential therapeutics. Results of these studies will increase our understanding of the causes and potential treatments of spinal cord injury, retinal disease, motoneuron diseases, Huntington’s disease, diabetes, multiple sclerosis, muscular dystrophy, heart disease, and Alzheimer’s disease. The SRL also hosts a hESC Techniques Course. This 5-day, intensive, hands-on course trains future stem cell researchers in techniques for cultivation, handling and differentiation of hESCs. We propose to develop new space for pre-clinical testing, to obtain key pieces of major equipment, and to support personnel in order to improve our ability to develop new FDA-compliant treatments for human diseases and disorders. The new space will allow us to expand our training effort to include procedures needed to conduct pre-clinical translational and transplantation projects. The expanded curriculum will include animal survival surgery, cell transplantation techniques, and methods for tracing transplanted cells in the animal. Currently few, if any, venues exist in which researchers can learn not only how to create potential hESC therapeutics, but also learn how to test potential treatments in animal models. Importantly, all treatment-oriented research will be done under strict FDA quality assurance guidelines, so researchers will not have to repeat experiments when they file with the FDA, streamlining processes and decreasing time to clinical trial. The research expertise and institutional support for hESCs puts us in a strong position to serve as a regional facility of excellence, bringing new researchers into the field, and leading the way toward realizing the potential of hESCs in treating human conditions.
Our institution is exceptionally strong in translating basic scientific discoveries to the clinic,and in particular, has FDA-compliant pre-clinical strength in translation of hESC discoveries. Indeed, preclinical studies undertaken through the SRL will be conducted under the guidance of existing Regulatory Quality Assurance Officers to ensure FDA-compliance. With the proposed additions to the SRL, our vision of serving as a regional resource for hESC research and training will bring us closer to hESC-based treatments.
Statement of Benefit to California:
Proposition 71’s primary goal is to translate basic research to clinical applications. Our program is exceptionally strong in moving basic scientific discoveries to the clinic and has FDA-compliant pre-clinical strength in translation of hESC discoveries.
The disability and loss of earning power and personal freedom resulting from a disease or disorder are devastating and create a financial burden for California. Therapies using human embryonic stem cells (hESCs) have the potential to change the lives of millions, and hESCs as models of diseases will help us understand the underlying causes of disease. For the potential of hESCs to be realized, California researchers need the equipment, lab space, and personnel to develop hESCs into viable treatments. Shared research laboratories (SRL) allow researchers to access critical, expensive equipment and concentrate expertise under one roof providing a favorable environment for collaboration. The federal constraints on hESCs create a critical need for SRL equipped and staffed with non-federal funds.
Our SRL is a regional resource currently used by scientists from 4 institutions, and hosts the quarterly hESC Techniques Course. Additional investment will result in a full service regional SRL where researchers can derive new hESC lines, develop cell-based treatments, and test potential therapeutics in animal models. Anticipated benefits of our SRL to the citizens of California include: 1) development of new cell-based treatments for a variety of human diseases and disorders; 2) improved methods for understanding normal development and the environmental risks to the early embryo; 3) development of intellectual property that could form the basis of new biotech startup companies; and 4) improved methods for drug development that could directly benefit citizens of the state. With the proposed additions, our vision of serving as a regional resource for hESC research and training will bring us closer to hESC-based treatments.
Progress Report:
Year 1
During the reporting period, the UC Irvine Regional Human Embryonic Stem Cell Shared Research Laboratory and Stem Cell Techniques Course continued to train scientists in the use and application of human pluripotent stem cells. We continued to train students, technicians, postdoctoral fellows and faculty in the use of Human Embryonic Stem Cells (hESCs). These individuals have come from all over the world. In the reporting period we trained 30 individuals in our training course. Most of the individuals were from UC Irvine, one from Kyoto University in Japan, one from West Coast University and two students from the newl-integrated Bridges to Stem Cell Research from Cal State Long Beach. These courses are tremendously popular and are oversubscribed. The training provided by the Stem Cell Techniques Course has enabled enormous scientific progress in a variety of areas related to the mission of the California Institute for Regenerative Medicine (CIRM). Scientists trained in the course have gone on to do groundbreaking research in many areas including Alzheimer’s disease, glioblastoma, multiple sclerosis, spinal cord injury and many others. In addition the Shared Research Laboratory has continued to serve as the training site for the Stem Cell Emphasis Track of the UC Irvine Masters Program in Biotechnology. This program was designed to train technicians for the biotechnology industry. Because of the paucity of technicians trained in the use of hESCs and other pluripotent stem cells (PSCs), this is a much sought after workforce. The facilities and equipment provided under this CIRM award have also been made available to many investigators across the university for carrying out stem cell-related research. In addition to the research mission of the program the Shared Research Laboratory has provided outreach to the community and education about stem cell research to members of the public. The staff of the Shared Research Laboratory has regularly provided tours to visitors from around the world as well as members of the local community. These include local high school teachers, local teaching organizations such as Vital Link and the Coastline Regional Occupational Program, members of the business community, philanthropic donors and our local Congressional representatives. The outreach activities of the Shared Research Laboratory serve to educate the public about our work and to address the needs of our local patient communities. They in turn provide important advocacy within the local community and with our representatives at the state and federal level. In short, the Human Embryonic Stem Cell Shared Research Laboratory and Stem Cell Techniques Course continues to serve a vital function within the stem cell research program at UC Irvine and within the larger research community in Orange County. Year 2
The UC Irvine Regional Human Embryonic Stem Cell Shared Research Laboratory and Stem Cell Techniques Course continues to be an invaluable element in supporting the mission of the Sue and Bill Gross Stem Cell Research Center to develop new treatments for human disease. The completion of Gross Hall: A CIRM Institute in May of 2010 allowed us to transition the activities of the lab and its associated teaching into a new world-class facility designed to be the hub of all stem cell research on the UC Irvine campus and in the region. Over the last year we have continued to train many students in stem cell biology at the didactic and practical levels and enabled their entry into the stem cell field. In addition, we have continued to support the research activities of many investigators on the Irvine campus who are already working in the field of regenerative medicine. During this period we have developed novel training methods and made all of our state-of-the-art equipment available to any researchers carrying out research relevant to the CIRM mission. Because of our new facility, we have been able to offer more hands-on training for the many specialized pieces of equipment in the building as well as providing didactic training courses for techniques and methods in the stem cell field. The facilities and courses continue to represent an essential element in the training of our fellows on the CIRM Training Grant II, other graduate students , postdocs, technicians and faculty on the Irvine campus and trainees on the CIRM Bridges programs. The Shared Research Laboratory also continues to host many outreach activities for members of the public. At these events, stem cell research is explained to a lay audience and members of the public are given an opportunity to see stem cells and learn about the incredible opportunities they provide for developing new treatments for human diseases and injuries. Many groups have visited the facility including high school kids, teachers, patients, patient advocates and caregivers as well as donors and potential donors. Each of these groups is inspirational in their own way and they are also inspired by the science and the potential of stem cell research. In short, the UC Irvine Regional Human Embryonic Stem Cell Shared Research Laboratory and Stem Cell Techniques Course continues to be an essential element in the success of the Sue and Bill Gross Stem Cell Research Center. Year 3
The UC Irvine Regional Human Embryonic Stem Cell Shared Research Laboratory and Stem Cell Techniques Course continues to be an invaluable element in supporting the mission of the Sue and Bill Gross Stem Cell Research Center to develop new treatments for human disease. Over the last year we have continued to train many students in stem cell biology at the didactic and practical levels and enabled their entry into the stem cell field. In addition, we have continued to support the research activities of many investigators on the Irvine campus who are already working in the field of regenerative medicine. During this period we have developed novel training methods and made all of our state-of-the-art equipment available to any researchers carrying out research relevant to the CIRM mission. In the past year we have also instigated a new training course designed to train researchers in methods of cell transplantation, a pre-requisite for translating stem cell research into clinical use. Because of our new facility, we have been able to offer more hands-on training for the many specialized pieces of equipment in the building as well as providing didactic training courses for techniques and methods in the stem cell field. The facilities and courses continue to represent an essential element in the training of our fellows on the CIRM Training Grant II, other graduate students , postdocs, technicians and faculty on the Irvine campus and trainees on the CIRM Bridges programs. The Shared Research Laboratory also continues to host many outreach activities for members of the public. At these events, stem cell research is explained to a lay audience and members of the public are given an opportunity to see stem cells and learn about the incredible opportunities they provide for developing new treatments for human diseases and injuries. Many groups have visited the facility including high school kids, teachers, patients, patient advocates and caregivers as well as donors and potential donors. Each of these groups is inspirational in their own way and they are also inspired by the science and the potential of stem cell research. On International Stem Cell Awareness Day alone over 200 schoolchildren from local high schools visited the center and learnt about stem cell research. In short, the UC Irvine Regional Human Embryonic Stem Cell Shared Research Laboratory and Stem Cell Techniques Course continues to be an essential element in the success of the Sue and Bill Gross Stem Cell Research Center. 