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T2-00005: Stem Cell Training Grant

Application Number:  T2-00005

Proposal Abstract as Submitted by Applicant

This Level II Training Grant will support seven PhD Post-Doctoral and three MD Clinical Fellows at Keck/USC and CHLA for training in stem cell biology, and the clinical and ethical implications of stem cell research. CHLA’s research has been consistently ranked in the top six of the nation’s pediatric stand alone institutions by the NIH. Over the past 20 years, CHLA has built an internationally renowned research program in stem cell biology and its clinical applications. The program was founded on the fields of human hematopoietic stem cell biology, transplantation and gene therapy. In the past decade, the program has been expanded to include somatic stem cells from lung, pancreas, liver, gut, bladder and mesenchyme. In the past three years, investigators at CHLA have developed expertise in human embryonic stem cells (hESC) and established a hESC training and tissue culture core for CHLA investigators. A unique focus of the Training Program at CHLA will be the application to pediatric disorders such as diabetes, monogenic inherited disorders (cystic fibrosis, muscular dystrophy, sickle cell disease, etc), and congenital birth defects.  It is our central hypothesis that childhood disorders will be especially responsive to therapies produced by the use of stem cells; advances in the use of stem cells to treat childhood illnesses will then lead the way to treatments for the many disorders that occur later in life. The training program includes: a didactic course on “Stem Cell Biology, Research Methods and Stem Cell Therapies”, a course titled “The Ethics of Stem Cell Research and Therapies”, participation in multiple existing training activities at CHLA, training in laboratory methods in hESC and joint participation in a didactic course taught by scientists from CHLA, USC Keck School of Medicine and the California Institute of Technology. A Steering Committee with stem cell researchers, clinical physician/scientists and medical educators will oversee selection and supervision of trainees, the mentoring process, and other activities of the training program. The biomedical environment and strength of stem cell research at CHLA combine to provide a rich milieu for training the next generation of physicians and scientists who will use stem cells as the basis for research and therapy.

Benefit of this Program to California

This program will benefit the people and the state of California by providing high-quality training in the scientific, clinical, social, and ethical aspects of stem cell research to the scientists and clinicians who will develop and apply future therapies in this rapidly emerging field.