California's Stem Cell Agency

In year 3, the training grant funded the stipends of 6 postdoctoral trainees, enabling them to receive training in human stem cell biology relevant to regenerative medicine. Projects included skeletal muscle stem cell renewal, use of stem cells to understand the developmental basis of Alzheimer's Disease, development of drugs for blood vessel regeneration, and pancreatic beta cell regeneration for Diabetes. Overall, the program supports the training of a new generation of stem cell researchers, some with clinical training, and also expanded the ability of our research laboratories to engage in regenerative medicine and create the researchers of the future. Administrative accomplishments by the Program included: - A multi-institutional course in stem cell biology, research ethics, and legal/business issues relevant to stem cell biology and regenerative medicine that was taught in conjunction with similar CIRM programs at the Salk Institute, the Scripps Research Institute, and the University of California, San Diego.This interdisciplinary lecture/discussion course was developed specifically for this program to cover human embryonic and other stem cells and their uses in basic science, translation, and clinical research and application. The course has units on basic embryology and on specific clinical applications. - The Training Program conducted a search for new trainees. 6 new trainees were selected following a competitive application process. - A one-day retreat was organized at which trainees presented their research to colleagues, and discussed emerging technologies and advances in regenerative medicine. - A stem cell seminar series and a trainee seminar series - The program funded trainees to attend up to one nationally recognized scientific meeting annually. Scientific accomplishments by our trainees included: - Discovery of a gene that enhances dopaminergic neuron production by human stem cells in a Parkinson’s Disease model, Shing Fai Chan (PLoS One, 2011). - Discovery of a method to produce skeletal muscle cells directly from human pluripotent stem cells, Sonia Albini (Cell Reports, 2012) - Identification of drugs that activate pancreatic beta cell differentiation, Ron Piran (ACS Chemical Biology, 2013). - Discovery of the biological mechanism that senses stretch in heart muscle and hence contributes to heart failure, Cecilia Hurtado and Ke Wei (both trainees) (Nature, 2012). - Synthesis and characterization of a new drug-like molecule that induces cardiomyocytes from human pluripotent stem cells, Erik Willems (Cell Stem Cell, 2012) The Director administered the program with the support of an Executive Committee consisting of Fred Levine, MD, PhD, Alessandra Sacco, PhD, Guy Salvesen, PhD, and Evan Snyder, MD, PhD. The Executive Committee advised the Director on the 1) selection of trainees for appointment, 2) select faculty for appointment and evaluate participation, 3) long-range direction of the program, 4) review the curricula and course requirements and, importantly, 5) trainee progress.

This annual progress report documents the activities of the CIRM-funded type 3 grant to train postdoctoral scientists at the Sanford-Burnham Medical Research Institute (SBMRI). SBMRI faculty direct a large stem cell research and teaching enterprise that comprises over 100 biologists, chemists, engineers and clinicians with extensive expertise in stem cell biology and in allied disciplines dedicated to stem cell-based therapies for cardiovascular, neurodegenerative, hematopoietic and metabolic disorders. During the past fiscal year, the SBMRI CIRM training program trained 6 postdoctoral fellows who were selected from an applicant pool by a committee of faculty who are experts in stem cell biology and experienced mentors of young scientists. The trainees conducted research on stem cell engineering, directed differentiation and drug discovery. Most of the Trainees’ projects focused on the use of stem cells to treat degenerative diseases, although a few use patient specific stem cells to develop therapies for other diseases. In addition, the program organized lecture, journal club and laboratory courses jointly with similar CIRM programs at the Salk Institute, Scripps Research Institute and the University of California, San Diego. Our curriculum included intensive lecture courses on Stem Cell Biology (>48 hours lectures), and Ethics, Intellectual Property and Regulatory Affairs (>20 hours lectures) and a 3-week hands-on, intensive laboratory courses. These core courses were required of all CIRM trainees at the four institutions. The program also funded instructors to provide specialized workshops in hESC techniques and we co-hosted a retreat of all CIRM program trainees in Salk Institute, Scripps Research Institute and the University of California, San Diego. Our trainees also attended local La Jolla and CIRM statewide trainee meetings to augment interchange and education. In addition, the training program provided a research stipend to defray the costs of the trainees’ research. The program is designed to offer comprehensive training in stem cell research and allied disciplines required to translate basic discoveries to clinic, biotechnology and pharmaceutical settings. The program created an enduring benefit to stem cell research by introducing new technology into established laboratories, and training a new generation of scientists.