The Proposed CIRM Major Facility The proposed facility will support the establishment of the Neurogenesis Institute of Stem Cell Technology on our campus. The scientists from relevant disciplines will use the facility to conduct research in drug discovery for Alzheimer’s disease using stem cells. We also plan to use the facility for advanced training of working professionals in stem cell technology and stem cell research lab management. The major facility will house labs for culturing and storing stem cells, producing drug-like compounds from natural plants, analyzing these compounds’ effect on stem cells and in the brain tissue. The research lab space will have an open layout with research bay areas shared by scientists and their students. This design will facilitate on-going interactions and exchange of results and ideas. The conference room will be used both as a meeting room and a small classroom for training purposes. The Stem Cell Research Program Our goal is to find drug-like compounds in treating Alzheimer’s disease (AD) and other related diseases. AD is the most common neurodegenerative disease with a debilitating and fatal outcome that afflicts 15 million people worldwide. The geriatric demographic dictates an increase in this patient population to 45 million by 2050, with 16 million in the United States. In the absence of an effective treatment, the cost to society and the extent of human suffering are incalculable. The drugs for AD patients lose their effectiveness 2-3 years following diagnosis and do not treat the root cause of the disease. Stem cell therapies require direct delivery to the brain via surgery, which would be highly impractical and risk-ridden for this large elderly patient population. The ideal treatment for AD would be to orally administer a drug that can penetrate into the brain to stimulate the local stem cells to develop and proliferate into new and functional neurons. This approach would avoid brain surgery and offer hope for a preventative treatment of cognitive decline across the general population. We will use a proven chemical conditioning process on natural plants to produce novel and drug-like compounds. These compounds will be screened using neural stem cells. Their effects on stem cells will be studied and the drug-target interactions analyzed. Finally, the drug-like compounds’ effect in the brain will be examined in preclinical studies. This program covers a broad scope of stem cell research as well as an in-depth pursuit of drug-like compounds in treating AD and related diseases. The Benefits of the Major Facility Currently, we do not have a designated facility for stem cell research. Such a facility will allow scientists to work effectively to achieve the common goals of finding practical therapeutic cures for AD and other related diseases. The advanced training in evenings and weekends will produce master’s level stem cell technologists that are much needed to support stem cell scientists in CA.
Statement of Benefit to California:
The Proposed Facility and the Stem Cell Program The facility will house a set of labs with open layout to facilitate on-going interactions and exchange of results and ideas. The facility will be used for dual purposes. 1) For research, we will use a proven chemical conditioning process to produce drug-like compounds from plant extracts. The compounds will be screened and their effects on cellular development and aging will be studies using neural and embryonic stem cells. Further drug-target interactions and the drug effects will be carried out in preclinical studies. This program covers a broad scope of stem cell research with a specific focus and an in-depth pursuit of drug-like compounds in treating Alzheimer’s (AD) and related diseases. 2) For training, we plan to add to our existing successful professional science master’s degree in biotechnology/MBA with specially tailored courses in advanced stem cell techniques and stem cell lab management. These classes will be offered in the facility evenings and weekends to train working professionals at the master level so that they could provide much needed support to the senior scientists in stem cell research field. The Benefits of the Major Facility and the Stem Cell Program to California AD is the most common neurodegenerative disease with a debilitating and fatal outcome that afflicts 15 million people worldwide. Over 500,000 Californians are affected by this illness, with the most conservative estimation predicting the population to reach 1.6 million in California by 2050. The cost to the State and local governments, the extent of human suffering, and the loss of productivity due to family members providing long-term care to their afflicted loved ones are immeasurable. Current drugs for AD become ineffective after 2-3 years and they do not provide a cure. Stem cell therapy requires brain surgery to deliver stem cells into a large patient population, which is impractical, costly and risk-ridden. Our research is to find drugs that can be formulated for oral intake, a therapeutic approach that is innovative, practical and economical. We have received over $2 million in donations from the private sector to support our endeavors and we have sufficient matching fund required by this grant. The proposed facility will allow scientists to work effectively to achieve the common goal of finding cures for AD and other related diseases. Our research results will lead to discovery of effective drugs in treating AD patients for which thousands of Californians will benefit. The State of California does not have a program to provide advanced training in stem cell technology and stem cell lab management. However, the need in specially trained personnel is staggering in support of the $3 billion Stem Cell Initiative in the State. Our training program will produce much needed professionals at the master’s level to join the scientific workforce in stem cell research in California.