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CIRM MAJOR FACILITIES GRANT APPLICATION #FA1-00619-1

Recommendation: Recommended for further consideration as a CIRM Institute
Element X Score: 76
Element Y Score: 78
Element Z Score: 72
Use & Contribution Score: 80

Public Abstract (provided by applicant)

The CIRM Facility, at the School of Medicine of {REDACTED}, will be the home of {REDACTED}, a comprehensive and integrated program of basic, pre-clinical and clinical stem cell research arising from scientific collaboration between six institutions. The scientists of {REDACTED} bring together a diverse set of skills and expertise in stem cell biology, chemistry, engineering, and medicine, and the CIRM Facility will be a focal point that catalyzes interdisciplinary work to accelerate their discoveries towards cures. {REDACTED} is committed to the development of a stem cell program, eventually comprising eighteen research teams, that will be housed in the new Facility and will form the core of the research activity. The Facility will also devote a significant amount of space for scientists from other departments at {REDACTED} institutions to train in stem cell research and carry out pilot research projects that will explore promising new ideas. By sharing facilities, reagents, and discoveries, {REDACTED} scientists will make efficient use of state stem cell resources and accelerate the progress of research to the clinic. The stem cell program of the host Institution has already attracted eight new scientists to California from leading laboratories around the world; construction of the new Facility is essential to retaining these new investigators and to attracting more. Research on fundamental stem cell biology is one important area scientists in the new facility will pursue, for there is much still to learn about how embryonic stem cells multiply and specialize to form tissues like the eye and heart, and much to discover about how to tap the potential for adult tissue stem cells to regenerate and repair damaged organs. This fundamental research is then integrated into technological advances that will lead to industrial scale production of cells, to development of new medicines that will help drive tissue regeneration after injury, and to the production of important new research reagents that will be used in laboratories around the world. These technologies will then be applied to the treatment of disease. Scientists in {REDACTED} will be concentrating on four organ systems and diseases affecting them: eye and ear, heart and blood vessels, liver and pancreas, and blood cells and cancer. These focus areas are ones in which {REDACTED} has particular strengths in clinical research, and they also encompass disease states within the reach of regenerative medicine in the mid to long term.

Statement of Benefit to California (provided by applicant)

The construction of the new CIRM Facility will bring great scientific, educational, economic, and health benefits to Californians. The Facility will form a hub for collaborative work in stem cell research and regenerative medicine between six institutions, which together form the {REDACTED}, with unique and complementary scientific expertise. {REDACTED} will thus catalyze synergistic interactions that will accelerate the progress of research towards achieving cures for disease. By sharing facilities, reagents, and expertise, the institutions in {REDACTED} will achieve highly efficient use of state stem cell funding. The host Institution, whose medical school campus will be home to the Facility, is building a new stem cell research focus, and is committed to the recruitment of a group of 18 scientists who will conduct basic and applied stem cell research. In the first year of its existence, this new center has already attracted eight scientists to California from leading laboratories around the world and that effort continues. These scientists will work with colleagues from six institutions in the region to study fundamental biology of stem cells and to apply this knowledge to developing the new technologies that are essential to the production of stem cell based therapies. While ultimately the goal is to develop cellular therapies for disease, there are many spinoffs from stem cell research undertaken in the new facility that will have an impact on human health and the regional, state and national economy. These include development of new processes for expansion of cells, new research reagents, new drugs to stimulate and control tissue regeneration and growth, new monoclonal antibodies for diagnosis and treatment of disease, and new platforms for virology research and vaccine development. This work will integrate with clinical programs in four areas: sensory systems, cardiovascular medicine, liver disease and diabetes, and hematology and oncology. This clinical research will lead to treatments for retinal disorders that lead to blindness, heart disease, liver disease, diabetes and cancer. The new Facility will provide a training center for scientists, technicians, and the academic physicians who will be essential to the future progress in this sector of medicine. Moreover, the location of the new Facility at the Medical School of {REDACTED} is of strategic significance, because the hospitals associated with this institution serve the needs of a large patient population with a wide range of socioeconomic, ethnic and racial diversity. This context places {REDACTED} in a special position amongst California institutions to develop and deliver regenerative medicine for underserved segments of the population. Plans to develop a biomedical technology park adjacent to the CIRM Facility will ensure that {REDACTED} becomes a focal point for the growth of stem cell biotechnology in the region.

Review Report

Executive Summary

The proposed CIRM Institute at the lead institution will be home of a regional Stem Cell Scientific Collaboration (SC3), which will facilitate collaborations among six institutions resulting in a comprehensive and integrated program of basic, pre-clinical and clinical stem cell research. A well thought out and detailed administrative structure for the collaborating institutions has been put in place. Over half of the facility will be devoted to laboratories for a critical mass of eighteen stem cell investigators, all from the lead institution. Space will be dedicated to four core facilities available to these scientists and to SC3 investigators from other locations; to a small vivarium, to administrative space and to space for collaborative training and a venue for seminars. The lead institution has a long track record of accomplishments in hematopoietic stem cell biology, transplantation and gene therapy, which is combined with excellence in epigenetics, developmental biology, and translational and clinical research. An internationally renowned stem cell scientist has been recently recruited as the new stem cell program director, filling a critical leadership position and enabling the recruitment of an energetic group of seven new faculty members studying human embryonic stem cells and tissue regeneration and repair. The lead institution has made a substantial investment in this arena and is committed to hiring ten additional faculty bringing the total to 18 research teams to be housed in the facility. The facility would specifically house studies on hESCs that are outside Federal guidelines. The basic research program (Element X) will focus on stem cell renewal and stability, stem cell differentiation in regeneration, stem cell niche and organ morphogenesis . Y element programs that bridge scientific advances and clinical applications build on strengths in applied technology in areas of cell culture technology, chemical genomics and drug discovery, monoclonal antibodies, cell phenotype and function, disease models and virology research. Finally the clinical focus of this collaborative program would build on strengths in sensory (neural) systems, cardiovascular disease, liver disease and diabetes, and in hematology/oncology. The six institutions serve a large population base. This places them in a special position among California universities to develop and deliver regenerative medicine for underserved segments of the population. The new facility coincides with plans to construct a biomedtech building adjacent to it.

Element X comprises four programs of fundamental research in stem cell biology. The program is anchored by the recent addition of an outstanding stem cell scientist and his/her success at subsequent recruitment of seven additional very strong stem cell scientists. These eight scientists form the core of the stem cell program, are predominantly focused in Element X research and will be housed in the proposed facility. In addition to the eight core scientists from the lead institution, the four research programs include over 20 collaborating scientists from the lead institution and from the collaborating institutions. The reviewers commented that the quality of the programs and participating investigators appeared high. The reviewers found it difficult to “get their arms around” the collaborative programs as well as to understand how interaction and collaboration within the lead institution and among the five other collaborating institutions would occur. Reviewers questioned the actual degree of collaboration, although discussants noted a previous history of collaboration with some of the institutions, in addition to the recent formal memorandum of understanding among all six collaborating institutions. The reviewers agreed that putting the core investigators together in a facility, especially given such a prominent director, would serve as a magnet for internal as well as external collaboration. At least two of the core services planned for the facility (hESC core laboratory and chemical genomics core) were considered as important resources for investigators at collaborating institutions.

Element Y focuses on six research areas including cell culture technology, drug discovery, monoclonal antibodies, cell phenotype and function, disease models, and virology research. Some parts of the program are directed towards the clinical focus areas of Element Z, while others are directed at development of generic technologies that will accelerate application of stem cells in drug discovery research and treatment. These programs bring together researchers from all the partner institutions working in a variety of disciplines from cell biology, chemical biology, protein engineering, nanotechnology, genomics, epigenomics, and virology. Each of the projects is led by a senior and accomplished scientist/investigator from the lead institution, and each project could stand alone as presented. The reviewers noted that the proposed chemical genomics core in the new facility will enable investigators at collaborating institutions to conduct studies that otherwise would have been difficult for them to perform. One reviewer noted that the proposal suffered from a lack of definition of interactions among the projects with little evidence for synergies or interdisciplinary strategies.

The collaborating institutions will focus on four areas of clinical research in Element Z, including sensory systems, cardiovascular disease, liver disease and diabetes, and hematology/oncology. The reviewers commented that a project focused on alloimmunity was part of one of these programs and that this was a key consideration for stem cell therapies. The actual clinical work will be carried out at the lead institution and two of the collaborating institutions, which are located in proximity to each other and serve a large and diverse patient population. The reviewers noted that collaboration among the institutions was most obvious in this element, although to one reviewer it was not clear how the CIRM facility would facilitate collaborations or move the ongoing research forward at a faster pace than it is currently progressing.

Among the collaborating institutions there is expertise and experience in liver cell and islet transplantation and in hematopoietic stem cell transplantation including transplantation of genetically modified HSC. Through the multi-institutional collaboration, researchers have access to a state-of-the-art production facility designed to purify and characterize DNA, proteins, viral vectors, and modified or expanded cell populations, as well as to process development capabilities and expertise in cell therapy. The reviewers considered this to be the weakest element among the three elements discussed primarily because there were no current stem cell clinical trials in areas of clinical focus (with the exception of hematology/oncology) and because the specific research programs described in the focus areas fell more into Element Y rather than Z. The lack of current stem cell clinical trials was a criticism that potentially could be leveled at most programs.

The proposed CIRM Institute will provide space for researchers at the lead institution who are currently located at sites on the campus on loan to the stem cell program. The Institute will act as a focus for a multi-institutional stem cell program spanning basic through clinical research through providing a home for a core group of stem cell researchers and core resources. The current cadre of stem cell scientists is outstanding, is led by an internationally recognized stem cell scientist, and is primarily focused on basic and early translational research. Additional recruits are predominately planned in the translation and clinical research areas. The programs in research elements X, and Y were considered stronger than programs in element Z. Implementation of collaborations and interaction among programs was again raised as a concern. The lead institution clearly has a strong commitment to the program as it is providing start-up and ongoing support for each core stem cell program PI, including the cost of core programs for this developing and collaborative stem cell program.

Detailed Summary

Element X

Score: 76

SCIENTIFIC PROGRAM: The program focuses on four core areas. These include stem cell renewal and stability, stem cell differentiation in regeneration, stem cell niche, and organ morphogenesis. Investigators from all the collaborating institutions participate in this discovery research, although those associated with the lead institution predominate. The work in stem cell renewal, stability and early lineage choice is led by an internationally renowned stem cell scientist who was recently recruited and is one of the pioneers in this field. The reviewers believe that this work and that of collaborating scientists at the lead institution will provide important novel insights into maintenance of pure populations of stem cells in defined conditions with retention of genetic integrity. This program will be housed in the proposed facility. The work is complemented by that of others, especially by studies on epigenetics led by a collaborating Principal Investigator at the host institution. Studies on pathways of stem cell differentiation in regeneration and repair will be conducted at all collaborating sites. Research by seven investigators from three institutions was highlighted. The laboratories of these investigators are using human and other ES cells to investigate factors that drive differentiation down pathways towards products that can be used for stem cell therapy. Two PIs from the lead institution who are planned to be housed in the proposed facility were highlighted for their studies on the stem cell niche. These studies focus on one of the best characterized tissue stem cell populations and on the hematopoietic stem cell niche in the marrow. The organ morphogenesis and regenerative medicine aspects will be investigated by four PIs, two from the lead institution who will be housed in the facility and two from an SC3 collaborating institution.

These are interesting experimental programs and the scientific quality of both the programs housed within and outside the facility to be high. The availability of the facility is likely to foster interdisciplinary synergy and collaboration. One reviewer was concerned that there was no clear strategy for collaborations and interactions among the researchers from collaborating institutions.

FORMAL INSTITUTIONAL COLLABORATIONS: The six institutions represented in the SC3 have agreed to a Memorandum of Understanding which establishes the collaboration and its governing body, the Scientific Advisory Committee. A key component of the memorandum is commitment of space within the CIRM facility for training under the cooperative CIRM Training Grant and Shared Facilities grant, and for pilot projects and collaborative research. A number of the scientists have already established a track record of collaboration. However, no details are provided about the nature of the collaborations and interactions.

CORE SERVICES: The CIRM facility will contain four cores that will be made available to members of the SC3. This includes a Human Embryonic Stem Cell Core Laboratory, a FACS Core, an Imaging Core, and a Chemical Genomics Core. The cores will be managed by core managers. Use of the cores will be determined by the Scientific Advisory Committee. One reviewer felt that the hESC and the chemical genomics cores would be especially useful to members of the SC3 outside of the lead institution. The FACS and Imaging Cores will likely be more important to investigators who are on site. Nevertheless, all four cores are likely to be important elements for the basic and discovery research by members of SC3.

PLANS FOR GROWTH: The lead institution has made a major commitment to ES cell biology in the eighteen months of the stem cell program’s existence. It has grown to eight research groups and over fifty scientists. The predominant research focus of these groups is Element X. The lead institution intends to recruit ten more scientists for a total of eighteen core principal investigators by the time the facility opens. Most of these recruits will be in elements Y and Z. Establishing the CIRM facility will enhance not only the ability of the lead institution, but also of the collaborating institutions to recruit outstanding stem cell researchers.

DISCUSSION: This application is from an institution with a good track record that is collaborating with five other institutions. The reviewers found the involvement of the other institutions difficult to grasp, and were divided on the actual degree of collaboration among institutions. One reviewer noted that s/he had trouble “getting my arms around this one”. The scientific program read like a like a phone book of investigator names. Each investigator cited is prominent, but one reviewer did not see evidence that they were going to interact at all. Another reviewer noted that faculty of a collaborating institution were also cited in another proposal so the degree of involvement is not clear. On the more supportive side of the collaborations, another reviewer noted that an attempt was made to put people in different programmatic groupings and state what they would accomplish. Yet another reviewer noted, based on his/her personal experience, that it will be a great advantage for investigators who are currently spread over the campus of the lead institution, to be co-located in a new facility. One reviewer was very impressed by the collaborations with one SC3 member institution. A discussant pointed out that there were very strong ties between the lead institution and at least one of the collaborating institutions including joint appointments.

All reviewers highlighted the huge success of the lead institution in the recruitment of the new director of the stem cell program and the quality of his/her program. The reviewers noted that this PI would be a focus for others to collaborate and build around his/her expertise. The reviewers commented on the quality of the local investigators at the lead institution and at collaborating institution(s). They noted that the core investigators at the lead institution who will be moving into the facility is “a group of really good people”. The reviewers cited the planned cores in the facility as good and as a useful resource for all collaborating member institutions. A discussant commented that a good conceptual model of this proposal is the lead institution as an ‘anchor’ that would be the focus for collaborative research among the institutions. The reviewers agreed and noted that in this regard the proposal was very strong.

Element Y

Score: 78

SCIENTIFIC PROGRAM: This element will focus on six research areas. These include cell culture technology, chemical genomics/drug discovery, monoclonal antibodies, cell phenotype and function, disease models, and virology research. Some parts of the program are directed towards the clinical focus areas identified in Element Z, while others are directed at development of generic technologies that will accelerate application of stem cells in drug discovery research and treatment.

The cell culture technology area will be headed by the director of the stem cell program at the lead institution and involves other investigators from the lead institution as well as from a collaborating institution. S/he will be housed in the CIRM facility. Many barriers remain before stem cells can be grown in large numbers in sufficient purity for clinical application, and it is unclear whether many of the cell culture systems described in the literature are robust for expansion and clinical application. This is one focus of the proposed program and will remain a pivotal area of further development.

The chemical genomics/drug discovery program will be led by an investigator at the lead institution who will be housed in the CIRM facility. This investigator is known for the development of small molecule templates, which have proven activity against a wide range of protein targets including G-protein coupled receptors and peptidases. The application states that one of these compounds is about to enter phase I clinical trials. The focus of this program is to develop small molecule compounds, to maintain human ES pluripotency or to direct lineage-specific differentiation.

The program to develop monoclonal antibodies is led by an investigator at the lead institution who has extensive expertise in monoclonal antibody generation and development for therapeutics and diagnostics. This investigator will not be housed in the proposed facility but will collaborate with “core” investigator housed in the facility to develop monoclonal antibodies against human ES cells and progenitors for reagents and potentially for use as antibody-based therapies.

The program in cell phenotype and function is focused on genomics, epigenomics and proteomics and is led by two investigators, both at the lead institution, who are experts in cancer epigenetics. These investigators are planned to be housed in a technology core in the cancer center which is outside the proposed facility. This technology center will provide support for the stem cell program in the form of high throughput analysis of gene expression in single cells, microfluidics for cell sorting, and cellular physiology. Collaborations are already in place between these investigators and core PI(s) who will be housed in the CIRM facility.

The preclinical disease model program is led by an investigator at the lead institution who will not be housed in the proposed facility but working in collaboration with lead institution investigators, both those internal and external to the proposed facility. The aim of this program is to produce disease models in a species that is more amenable to physiological and neurological investigations than is the model most commonly used. Diseases to be modeled overlap with the focus for clinical investigation including disease of the liver and pancreas, the heart, sensory systems, and modulation of inflammatory responses in fibrosis. The eventual aim of this program is to use the preclinical disease models for exploration of stem cell therapies.

The virology research program is led by a noted recent recruit at the lead institution. This PI is planned to be housed outside the proposed facility. This program is directed towards using ES cells as a platform for the study of viral pathogenesis and the potential for vaccine production by providing clean, well characterized human cells capable of large-scale expansion and differentiation into cell types of appropriate infectivity.

Overall, element Y brings together researchers from all SC3 partners working in a variety of disciplines from cell biology, chemical biology, protein engineering, nanotechnology, genomics, epigenomics, and virology. Each project is led by a senior and accomplished scientist/investigator and each could stand alone as presented. At lease one reviewer noted that the proposal suffered from a lack of definition of interactions among the projects with little evidence for synergies or interdisciplinary strategies. The CIRM facility will provide a focal point for the coalition of this expertise. The preclinical Disease Models platform will enable testing of the potential therapeutics developed in the other research areas of this element. Eventually, this will be translated into the clinical setting.

FORMAL INSTITUTIONAL COLLABORATIONS: Collaborations between the lead institution and another collaborating institution in technology relevant to cell culture and high throughput methodology for gene expression analysis have been ongoing. Most reviewers believed that the CIRM facility will accelerate these collaborations although one reviewer was not convinced that the formation of the new CIRM sponsored institute will further enhance the shared use of these facilities.

CORE SERVICES: The chemical genomics/drug discovery and ES cell cores planned to be housed in the CIRM facility will support the Element Y work of groups within the SC3. The availability of a drug discovery core will enable investigators at collaborating institutions to conduct studies that otherwise would have been difficult for them to perform, including sophisticated application of cell-based assays in screening that requires close interactions between biologists and chemists. Further, the establishment of the SC3 will make available to the collaborators core facilities at a collaborator institution which includes a state-of-the-art production facility designed to purify and characterize DNA, proteins, viral vectors, and modified or expanded cell populations. Further, collaborating institutions would have access to this institution’s dedicated process development laboratory for cell therapy candidates. This institution has substantial experience at working with and providing cells for clinical research.

PLANS FOR GROWTH: The CIRM facility will set the stage for development of scale-up systems for ES and progenitor cell populations as well stem cell banks. The lead institution intends make several of the 10 anticipated hires who will be housed in the CIRM facility in this research Element Y. In addition, the lead institution plans to develop a new biomedical research park on land adjacent to the campus. This will provide excellent opportunities for start-up companies by faculty.

DISCUSSION: The reviewers were overall very positive with respect to this research element. The scientific programs in cell culture, disease models and drug discovery were regarded as particularly strong; the former two were also highlighted as very important technologies for the field with cell culture technology, in particular as being critical for the ESC field. One reviewer, as with Element X, again did not see strong evidence for interaction and collaboration.

Element Z

Score: 72

SCIENTIFIC PROGRAM: Collaborators at the SC3 institutions will focus on four areas of clinical research, including sensory systems, cardiovascular disease, liver disease and diabetes, and Hematology/Oncology. The actual clinical work will be carried out at the lead institution and two of the collaborating institutions, which are located in proximity to each other and serve a large and diverse patient population. These three institutions have collaborated to form one of the largest NIH-funded general clinical research centers in the country.

The program in sensory systems is focused on ocular disorders, including macular degeneration and retinitis pigmentosa, both of which are caused by death or dysfunction of the retinal pigment epithelium. The program is led by an investigator from the lead institution and has collaborating PIs from the lead institution as well as from another collaborating institution. The lead institution investigators will not be located in the CIRM facility. There is experience in preclinical studies of retinal and retinal progenitor transplants as described in Element Y. Research is focused on generation of the target cell for therapy from human ESC, on alternative stem-cell based strategies for visual repair and on technologies for assessment of visual therapies. There are, as yet, no clinical trials.

A number of investigators have formed a nucleus of the cardiovascular program that at this stage is more focused on early translational issues. This program is led by a PI from the lead institution and involves investigators from the lead and a collaborator institution. These investigators collaborate with cardiothoracic and vascular surgeons, as well as immunologists who focus on alloimmunity, which is critical to success of ES therapies. There is also a vascular biology program focusing on the ability to deliver molecules capable of modifying the responses of host vessels to injury. Again, there are, as yet, no clinical trials.

The lead institution is well positioned to conduct clinical research in liver disease based on an excellent liver transplantation service. This capability will facilitate the basic and preclinical discoveries that are applicable to this research focus. Collaboration in early preclinical studies are underway between stem cell investigator(s) and the director of the institution’s liver disease program. As for pancreas and diabetes, early preclinical studies of islet precursors derived from human ES cells in vitro are underway at the lead institution. Active recruitment is ongoing in this program area. Clinical programs in adult and pediatric diabetes as well as an active islet transplant program at the host institution will provide clinical expertise for moving therapies forward.

The clinical program in hematology/oncology is located at a collaborating institution under the leadership of a pioneer in cell and gene therapy. The clinical program includes an active pediatric hematopoietic stem cell transplantation program and a program in genetically modified hematopoietic stem cells in patients with genetic diseases and HIV.

There are active phase I and II trials of anti-cancer agents. Work is ongoing in collaboration with a stem cell investigator to initiate clinical trials in colorectal cancer investigating a signaling antagonist thought to act specifically on stem cells.

FORMAL INSTITUTIONAL COLLABORATIONS: There is a history of collaborations between the lead institution and a collaborating institution; all of the collaborating institution’s faculty have appointments at the lead institution. A collaborating institution, as already indicated, has an active gene therapy and stem cell therapy program. Similarly, there is close collaboration with another collaborating institution, which has very active clinical programs in the areas of cancer including hematopoietic cell transplantation, cardiology, molecular diagnostics, and others.

CORE SERVICES: A number of clinical support resources currently exist at the lead institution. The lead institution has a clinical research core service which guides investigators through the complex processes required to identify a potentially translatable finding and move it through the development of preclinical studies and regulatory filings into clinical applications. The lead institution also has an associated organization that provides services for the conduct of clinical trials. A well developed core resource at the lead institution in imaging provides state of the art imaging instrumentation for small animals, large animals and humans and conducts research and develops tools in the area of imaging informatics. Finally, several biostatistic resources at the lead institution can provide statistical support of clinical trials. ,No additional clinical support cores are planned as a result of the establishment of the CIRM facility although in at least one instance an expansion is proposed to address cell therapies. Access to collaborating institution’s facilities and expertise in good manufacturing practice (GMP) production and cell manufacturing will add greatly to the capacity of the proposed facility. Finally, existing clinical protocols at a collaborator institution provide precedent for transfer of cells from these facilities to patient treatment areas.

PLANS FOR GROWTH: The majority of the 10 recruits planned to be housed in the CIRM facility are in the translational and clinical areas.

DISCUSSION: The reviewers noted that collaboration among the institutions was most obvious in Element Z, although to one reviewer it was not clear how the CIRM facility would facilitate collaborations or move the ongoing research forward at a faster pace than it is currently progressing. The reviewers considered this to be the weakest element among the three elements discussed primarily because there were no current clinical trials in areas of clinical focus and the description of specific programs often included investigators who were involved in research that was Element Y stage. One reviewer pointed out that the criticism of no current stem cell clinical trials can be applied to almost everyone. The reviewers noted that overall, stem cell therapy clinical trials among the collaborating institutions were primarily limited to the use of adult hematopoietic stem cells, which have been a standard in the treatment for hematological disorders for many years. A reviewer did note however, that a collaborator institution has been conducting clinical trials of genetically modified hematopoietic stem cells. A reviewer also noted that this proposal highlighted a very good to excellent clinical research program on alloimmunity which is a very important consideration for stem cell based therapies. Experience in islet transplantation was also highlighted.

Use & Contribution

Score: 80

The proposed CIRM Institute will provide space for researchers at the lead institution who are currently located at sites on the campus that are on loan to the stem cell program. The use and design of the new facility is well planned and described in detail, and forms the basis for this whole application. It appears that most of the growth projections are centered in this facility. By the time the facility is opened in 2010, the lead istitution will have recruited additional PIs; and, at capacity (within a year of opening) plans to have 18 PIs and their research groups located within the facility. The lab space in the facility is predominantly allocated to Element X, then Y, then Z. The building will also contain cores for drug discovery, human ES cells, flow cytometry and imaging. Space is provided for conferences and will accommodate meetings of the therapeutic focus groups. Scientists in the facility will concentrate on four organ systems and diseases affecting those systems: eye and ear, heart and blood vessels, liver and pancreas, and blood cells and cancer. These focus areas are those in which SC3 has particular strengths both in laboratory and clinical research. They also encompass diseases within the reach of regenerative medicine in the mid to long term. The lead institution clearly has a made a strong commitment to this developing stem cell program given the accomplishments in program development over the last few years.

Programmatic Discussion

 

A motion was made to recommend this application for consideration as a CIRM Institute (3 elements): Some discussants felt that that scoring for this application was too low. Note was made that the lead institution has shown great commitment to stem cell research by successfully recruiting a major stem cell researcher, then recruiting an additional seven strong stem cell scientists to develop a program comprising over fifty investigators. In addition the lead institution and five other institutions have formed a collaborative program for stem cell research. A panelist commented that the application is “a powerhouse” for Element X and Y programs. Panelists noted that the Element Z was the weakest program and the score was fair, but also commented that this institution with its collaborating institutions will, in the future, be among the first that attempt stem cell clinical studies. A panelist considered the clinical program to be a programmatic strength given the clinical experience at the lead institution as well as that at two of the collaborating institutions. This positions the lead institution with it’s collaborators to be clearly “in line” for applying the basic and translational to the clinic. A panelist noted that other institution(s) are currently conducting clinical studies with stem cells (other than hematopoietic stem cells) rather than having plans on paper. The motion to recommend this application for consideration as a CIRM Institute passed.

The following Working Group members had a conflict of interest with this application and were therefore recused from participating in review of, discussion of, and voting on the application:

• Feit, Marcy
• Sheehy, Jeff