We propose to create a world-class human Pluripotent Stem Cell (hPSC) Repository in California with an initial task of making the 3,000 induced pluripotent stem cell (iPSC) lines generated as part of this CIRM initiative and an additional 1,500 hPSC lines generated within California widely available. iPSCs are extremely valuable as they are able to self-renew and can be converted into virtually all human tissues. In addition, since they are made from adult cells obtained from a specific donor, they can be used to model the biological profile of that donor, which is especially relevant if the donor has a particular diseases. Therefore, iPSCs can be used to create cells or tissues that mimic disease and can be used in drug discovery efforts. Based on our experience, a critical component of operating the hPSC Repository will be to ensure users of the cell lines have the technical skills to take advantage of this great resource. To that end, we have provided numerous training programs through our existing facility and have established collaborations in California to bring the leaders of the field for similar programs on how to differentiated PSCs into relevant adult cell types and to utilize them in the development of drug discovery assays. This Repository has the opportunity to transform the landscape of stem cell research and we hope to be the group helping to make sure this becomes a reality
Statement of Benefit to California:
As a result of CIRM’s creation, California has established itself as a key player in all areas of stem cell research. If awarded this grant, our group will be able to integrate our unique high-throughput human pluripotent stem cell (hPSC) characterization, expansion, and distribution platform into this rich environment. Based on our previous experience, our efforts to build innovative programs to change the landscape of stem cell research have enabled us to accelerate research and coordinate the stem cell communities at the institutions near our home location, which we hope to replicate in California. The use of iPSCs as a technology for basic biological research, drug discovery, and therapeutic applications has enormous potential, but requires major advances before translation. As an organization that has significant research experience and a proven track record of operating a hPSC repository, we are confident we have both the technical expertise and foresight regarding the future use of these cell lines to ensure the Repository becomes a vital component of the California research enterprise and economy. These activities should allow California and its citizens to be among the first communities to see the innovations resulting from this research in the form of medical breakthroughs and commercial opportunities.
This application was provisionally recommended by the Grants Working Group. The applicant proposes to establish a pluripotent stem cell repository in California that will maintain and distribute human induced pluripotent stem cell (hiPSC) lines from 3000 donors as well as 1500 additional human pluripotent cell lines from California investigators. The applicant also submitted a proposal under RFA 12-03 hiPSC Derivation Awards (ID1-06617) that is intended to complement this repository proposal. This banking and distribution effort will utilize automated processes for cell line expansion and testing. It will feature a comprehensive data tracking system to ensure appropriate documentation and quality control in all aspects of cell line deposit, expansion, cryopreservation, QC testing, inventory, and ordering/shipping. The proposed repository also will provide appropriate training and support for users. Quality of Proposed Repository -Many features of the proposed repository including the experience of the applicant organization, the quality control plans and the data management system for tracking and documentation are particularly strong. -Reviewers were enthusiastic about plans for automated and high throughput procedures. -Plans for cell line distribution and facility management were judged as excellent. -Reviewers appreciated planned storage of original tissue samples. Feasibility -Overall plans for establishment and running of the repository were considered highly feasible. -It was unclear whether the proposed banking procedures would be entirely compatible with derivation handled by another organization. -Reviewers expressed some concerns about the adequacy of the space available for the facility -Absence of a clear sustainability plan was viewed as a weakness of the proposal. -Reviewers appreciated the applicant’s interest in developing multiple collaborations with California researchers. Program Director (PD) and Team -The PD has a strong track record and management experience that is both substantial and appropriate. -The management team was judged to have good experience in cell banking and related activities. -Reviewers expressed some concern about the need to train a significant number of new personnel to operate the facility. Appropriateness of the Budget -Overall, reviewers viewed the budget as reasonable. -There was some concern as to whether costs were adequately budgeted if the applicant received only the Repository award.