Training Clinical Fellows in Pediatric Applications of Stem Cell Therapy

Funding Type: 
Research Training II
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Our clinical training institution proposes a CIRM type III program to train two Clinical Fellows yearly in stem cell research, with particular emphasis on translational research directed toward pediatric diseases. This program is designed to provide training opportunities for Clinical Fellows pursuing future careers in translational and clinical stem cell research. We are requesting funding to support two Clinical CIRM Fellows annually. The curriculum will consist of courses and practical, hands-on instruction in stem cell biology and laboratory research. The year-long course work, conducted at our nearby collaborating academic institution, will include these three new courses: Basic Biology of Stem Cells; Clinical Applications of Stem Cells; and Social, Legal and Ethical Implications of Stem Cell Research. In addition, CIRM Fellows will be expected to take the Human Embryonic Stem Cell Culture Training Course, developed by the Program Director, as PI, with the support of the NIH and successfully conducted over the past five years.

The CIRM clinical Scholars will enter a new track in our existing subspecialty fellowship training programs: after completing one year of clinical training in a subspecialty fellowship program, the trainee will spend one to two years in basic or translational stem-cell research. All CIRM Scholars will participate, during their first year of support, in a year-long sequence of three new courses: Basic Biology of Stem Cells; Clinical Applications of Stem Cells; and Social, Legal and Ethical Implications of Stem-Cell Research.

As a regional, tertiary-care, children’s hospital, our patients represent the entire range of pediatric diseases. Our scientists and clinicians currently direct a stem cell research and teaching enterprise working towards therapies for neurometabolic, immunologic, and hematologic disorders. Specifically, our therapy-directed stem cell research is focused on: lysosomal storage disorders such as Hurler’s disease, which we have already successfully treated with stem cell transplantation, and the childhood hematologic diseases such as leukemias and dysmyelopoeises including aplastic anemia and congenital marrow failure. We have been successfully treating these hematologic syndromes for many years with bone marrow, cord blood, and peripheral blood stem cells. Additionally, we have a nascent stem cell research program directed toward pediatric brain tumors. All of these diseases represent significant causes of mortality and morbidity in the pediatric population and all are potential targets for stem cell therapy.

Statement of Benefit to California: 

This CIRM Research Training Program will benefit the people and the state of California by producing clinician-scientists, well-trained in stem cell research, who will develop and apply future stem cell therapies.