Research Training II
This proposed Comprehensive Training Program will include 16 CIRM Scholars, three clinical fellows, eight postdoctoral fellows, and five pre-doctoral fellows. Our multidisciplinary program will provide training opportunities in stem cell biology, engineering, and medicine, with a focus on applying stem cell research toward the goal of treating human disease. Coursework offered as a part of this program will cover basic stem cell biology and current and potential uses of stem cells in regenerative medicine, as well as ethical, legal and social issues in stem cell research. CIRM Scholars' research projects may investigate fundamental biomedical problems such as the molecular basis of stem cell pluripotency, stem cell differentiation into mature tissues such as blood, nerve and muscle, or the reduction of stem cell regenerative capacity with ageing. CIRM Scholars whose expertise is in engineering may work to develop controlled environments for expansion and tissue-specific differentiation of human embryonic stem cells, or devices to enable or evaluate the results of stem cell transplantation. Clinical Fellows will integrate basic and translational research, with particular emphasis on cord blood transplantation for genetic or malignant blood diseases. This program will train leading young physicians, scientists and engineers who will contribute to the understanding of stem cell biology and to the development of stem cell-based therapies, as well as advancing the medical, intellectual, technological and economic strength of the State of California.
Statement of Benefit to California:
This proposed training program will develop highly skilled scientists, engineers, and clinical researchers to help ensure California’s continued leadership in biomedical research and engineering. The basic biological research component of this program seeks to identify molecules or biochemical pathways that could function as targets for enhancing or blocking stem cell differentiation. The resulting information could thus serve as a catalyst for drug development. Translational research projects that will be part of the proposed program include development of synthetic materials to support stem cell culture and tissue engineered blood vessel or nerve grafts. The program also includes engineering of novel equipment for imaging and isolation of stem cells. A primary goal of this work is to build new products to be developed and marketed by California biotechnology companies for research and clinical use. The clinical research component of our proposed program will attempt to expand the circumstances under which umbilical cord blood transplantation can be used to cure genetic blood diseases such as sickle cell anemia and thalassemia. These conditions afflict a significant number of individuals in California, particularly in underserved populations, and require expensive and tissue-damaging treatment throughout patients’ lives. Success of this program will benefit the State of California by providing extended and enhanced quality of life for patients with genetic blood diseases, as well as a reduction in the anticipated amount of medical treatment cured patients will require over their lifetime.
This is a request for renewal of a Type I program to fund 16 scholars (5 predoctoral, 8 postdoctoral, 3 clinician scientists). The basic science fellows have access to a variety of well-outlined topics, while the clinical fellows will be trained on the use of human cord blood cells. The CIRM Scholar selection process has already been worked out. The training environment and resources at the applicant university are superb and are supplemented by a collaborating clinical institution. Reviewers considered the integration between stem cell biology, bioengineering and medicine a strength of the program. Although not described in detail, imaging training is included in the program, adding another dimension emphasizing clinical translation. In addition, the availability of electives in law, ethics and humanities related to stem cell issues enhances the program as a whole. To this effect, two pre-JD fellows are included. The usual seminars, yearly retreat and symposia round out the training of the scholars. In spite of the overall educational strengths of the program, the required Stem Cell Biology and Regenerative Medicine course was considered a weakness. No description is given of the topics to be covered, rather the content will change to reflect the current literature and the fellows’ interests. The course apparently will meet only once per month, and overall appears to be more like a journal club than a well thought out course. A unique strength of the program is the placing of trainees within a clinical pediatrics environment, although one reviewer expressed concern whether the clinical fellows will be on a trajectory to emerge as leading clinician-scientists in stem cell research, since it was not clear whether they would engage in basic science at the applicant institution. The current Program Director (PD) will continue in this position, he/she is well qualified and the overall team has nice breadth of expertise and perspectives. The PD is aided by an Associate PD from the collaborating clinical institution, who is responsible for all aspects of the clinical fellows program. The steering committee consists of strong faculty at the various institutions. These team members have been working together well during the previous funding period as evidenced by the success of the program. The degree of assessment of trainees’ progress by the program leadership could be stronger. All scholars will meet individually with the PD at the midpoint of their annual appointment period. Scholars will present their work once yearly at the annual retreat, but no written reports are required for the scholars program. Faculty mentors will be monitoring individual trainees within the context of their laboratories. Approximately 40 mentors are involved in this training program. They all appear to be well-versed in mentoring given the number of past trainees. The pool of clinician-scientists for mentoring the Clinical Fellows is very solid but not as large or robust as in some other programs. There is a strong base of training programs and training experience at the applicant institution. The 3-year CIRM comprehensive training program already in place has successfully produced several trainees, giving reviewers confidence that the proposed program is likely to succeed. Competition to get into the program is increasing, and thus the demand is present to support the increased number of slots requested. The existing scholars in this program have published in top tier journals. The applicant institution runs outreach programs for summer research opportunities for undergraduates across the US. The institution has a very good diversity record, and of the 22 CIRM scholars appointed to date, 4 are from underrepresented groups in science. The research and intellectual environment at the applicant university is outstanding. In addition, it has significantly invested in stem cell research on campus. There already is substantial cohesion among different stem cell scientists, as evidenced by the previous CIRM comprehensive training grant round. Many investigators participating the training program are leaders in their field.