Funding opportunities

Training Clinical Fellows in Pediatric Applications of Stem Cell Therapy

Funding Type: 
Research Training II
Grant Number: 
Funds requested: 
$763 686
Funding Recommendations: 
Not recommended
Grant approved: 
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.
Review Summary: 
This application proposes a Type III training program that intends to train two clinical fellows per year. The program of training is focused on stem cell research with an emphasis on translational research directed toward pediatric diseases. After one year of standard clinical training the fellows will pursue 1-2 years in basic or translational stem cell research as a CIRM Scholar. During the first year of CIRM training, fellows will participate in year-long courses at a nearby university. The course curriculum includes three new courses in basic biology of stem cells, clinical application of stem cells and social/ legal/ethical implications of stem cell research administered and organized by the partnering organization. In addition, an intensive human ES cell culture training course developed by the program director will be required. The reviewers appreciated the clinical emphasis of this proposal but were discouraged by the poorly described training plan. The proposal provides a description of ongoing research of each faculty mentor but does not adequately focus on the more relevant details of the courses and training that it will provide. Reviewers were concerned that the training program is superimposed on the current clinical fellowship program. From the proposal it was not clear if the host institution has a pool of applicants that have strong research orientation or background likely to benefit from this clinician-scientist training program. A reviewer commented that there is a universal challenge of training clinician-scientists who become nationally competitive and remain in academia as investigators rather than pursue private practice. This challenge is magnified when there are very few clinician-scientist role models who serve as living testimonies to the feasibility of this choice for young trainees. The faculty at the applicant institution appears to lack depth in this regard according to the reviewer. One reviewer expressed concern that the partnership between the applicant institution and the partnering university – although promising – did not appear fully developed at this time. Letters of support did not convince reviewers of a strong and robust collaboration between the two institutions. Moreover, viability of the application is further compromised by the failure to fully leverage the resources and faculty at the partnering institution. The Program Director was considered well qualified and experienced to oversee the program. Reviewers felt that the Program Director was a clear champion of this training effort but appears to lack adequate support from the applicant institution and would benefit from greater collaborative outreach to other institutions. Reviewers were concerned by the insufficient depth and breadth of opportunities for the trainees as evident from only five mentors listed for this program. The relatively few clinician scientists engaged as mentors for the clinical fellows was considered problematic. The applicant institution has demonstrated experience in the training of clinical fellows in subspecialty pediatric disciplines but reviewers noted that the applicant institution has no NIH or CIRM training grants at present. The human ES cell culture course was highly regarded and viewed as the “shining light” of the program. Institutional commitment was evident only through the support of laboratory space and relevant core facilities but otherwise appeared low. Reviewers were also not convinced that the applicant institution was fully committed to diversity, as the applicant’s statement was seen as vague and not compelling. Overall, reviewers found the training proposal to offer very limited opportunities to trainees. The program of training was not adequately described and reviewers were not convinced that the program would foster the development of competitive clinician-scientists engaged in stem cell research.

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