Funding opportunities

Stem Cell Training Grant

Funding Type: 
Research Training II
Grant Number: 
TG2-01168
Principle Investigator: 
Funds requested: 
$2 518 227
Funding Recommendations: 
Recommended
Grant approved: 
Yes
Public Abstract: 
This Level II Training Grant will support seven PhD Post-Doctoral and three MD Clinical Fellows for training in stem cell biology, and the clinical and ethical implications of stem cell research. The program is based in one of the top six of the nation’s pediatric stand alone Institutions. Over the past 25 years, we have 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 two decades, integration of research in developmental biology and tissue regeneration have expanded the reach of the program into other types of somatic stem cells including lung, pancreas, liver, gut, bladder and mesenchymal tissue. In the past 6 years, we have developed expertise in human embryonic stem cell (hESC) culture and differentiation and have established an hESC tissue culture core. A unique focus of Stem Cell Training will be on applications 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" that will review the ethical, legal and social implications of research with human stem cells, participation in multiple existing training activities, an introductory training in laboratory methods in hESC and a didactic training course taught in concert with the [REDACTED]. 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 complex biomedical environment and the strength of stem cell research 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.
Statement of Benefit to California: 
This Level II Training Grant will continue to support seven PhD Post-Doctoral and three MD Clinical Fellows for training in stem cell biology, and the clinical and ethical implications of stem cell research. The program is based in one of the top six of the nation's pediatric stand alone Institutions, located in California. Over the past 25 years, we have 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 two decades, integration of research in developmental biology and tissue regeneration have expanded the reach of the program into other types of somatic stem cells including lung, pancreas, liver, gut, bladder and mesenchymal tissue. In the past 6 years, we have developed expertise in human embryonic stem cell (hESC) culture and differentiation and have established an hESC tissue culture core. A unique focus of Stem Cell Training will be on applications 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" that will review the ethical, legal and social implications of research with human stem cells, participation in multiple existing training activities, an introductory training in laboratory methods in hESC and a didactic training course taught in concert with [REDACTED]. 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 complex biomedical environment and the strength of stem cell research 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. This will provide unique benefits to the State of California and its citizens by providing a superbly trained group of stem cell experts whose work is focused on relieving childhood diseases in California. The trainees in this program will also strengthen and accelerate our research efforts in this field.
Review Summary: 
This is a Type II application to support seven postdoctoral and three clinical trainees. The program is associated with a children’s hospital, and is focused on pediatric disorders. Didactic courses will be required in stem cell methods and ethics, and a training course on human embryonic stem cells will be taught at a neighboring institution. Reviewers were very supportive of this application and its focus on pediatric disorders. The program is well structured and articulated; it clearly builds upon a strong training environment. There is a nice balance of didactic and hands on activities with careful attention to mentorship and evaluation. Due to its focus, the research program is not as strong or comprehensive as some of the other institutions discussed in this review session. However, reviewers agreed with the applicant’s statement that "the young, developing organism is a biologically ideal host for stem cell transplantation and 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 Program Director (PD) is well qualified as a scientific leader and has substantial experience as a mentor and director of training programs. Reviewers commented that the scientific and administrative oversight is appropriate. The institution has a Mentor Training Program (an NIH K12 award), so the faculty already has mentors experienced in supervising PhDs and MDs. The mentor pool has good depth and breadth with very substantial track records of mentorship. The PD is an experienced mentor, although it is unclear to what extent his/her role as vice-chair of the Department of Pediatric Surgery may limit the time available to devote to this training program. The faculty at this institution has a strong focus on transplantation, particularly in the area of hematopoietic stem cells. The depth and strength of basic science in the stem cell area is not as strong as at the major institutions, but reviewers felt that this institution provides a venue for trainees to work on the bridge between basic and applied efforts in the field. The current other training programs are strong, though perhaps not at the level of the top California institutions. The institution received funding in the first round of CIRM Training grants. The reviewers did not notice particular efforts at reaching out to minorities in the recruitment of trainees for the program. The institution has a strong commitment to translating science to the pediatric setting and a strong commitment to the stem cell area, as exemplified by its long-standing support of bone marrow transplantation and organ transplantation. The strength of the research is not at the very highest levels seen in some other applications, but it is certainly of sufficient quality to provide a good place for training of fellows, particularly in areas relevant to pediatrics.
Conflicts: 

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