The Centers director, Dr. Mahendra Rao, continued early efforts involving co-sponsorship with the NIH Stem Cell Interest Group of both the Stem Cell Seminar Series and the annual Stem Cell Research Symposium, which showcased NIH CRM-funded research from its pilot grant program. The Research Symposium held 7 sessions covering topics ranging from rules and processes for using pluripotent stem cells where several representatives from OHSRP, OTT, and others provided valuable considerations for sending, receiving, and using pluripotent stem cells. Additional sessions covered new technologies for using iPSCs along with infrastructure enabled within the NIH IRP such as derivation of iPSC lines by the iPSC and Genome Engineering Core led by Dr. Guokai Chen. The final sessions at this years Stem Cell Research Symposium covered how to work with the NIH CC in developing an IND and then highlighting screening and therapy efforts made by NIH investigators. Through these continuing efforts and our pilot grant program, NIH CRM has been able to provide a forum for investigators to share their work and participate in fruitful discussion related to stem cell-based projects with tangible results along with a funding mechanism to advance the field. Moreover, NIH CRM in conjunction with FAES held a useful Stem Cell Industry Symposium showcasing innovations in four main areas: (1) cGMP manufacturing;(2) technologies to manipulate PSCs;(3) assays for characterizing PSCs;and (4) screening assays using primary cells. Twenty-eight leading companies in each of these areas presented their latest developments, ranging from reagents and tools to providing custom services that could benefit NIH investigators with their current research projects. This symposium also facilitated collaborations and partnerships for investigators with more advanced projects that were progressing toward the clinic (e.g., cGMP manufacturing of clinical-grade iPSC lines for cell-based therapies). Another set of efforts in conjunction with FAES was aimed at establishing relevant and timely stem cell research hands-on training courses. NIH CRM organized the following Bio-Trac courses: (1) TRAC CRM47: iPSC II: Human Induced Pluripotent Stem Cells (hiPSC);Differentiation to Neural Lineages (March 11-15, 2013);(2) TRAC CRM48: Using TALENs for genome engineering (July 29 - July 31, 2013);and (3) Trac CRM 49: Making iPSC from blood and pluripotent stem cell differentiation (August 12-16, 2013). These courses have collectively taught over 50 investigators (60% NIH, 40% extramural) valuable techniques that were logically paired with service provider discounts (10-30% off) in addition to reduced or free resources (e.g., NSCs, iPSCs, safe harbor TALEN and donor vectors) that will accelerate each investigators stem cell research activities. The center is also compiling an Intramural Pluripotent Stem Cell Protocol book that will be distributed to the intramural community this fall and will provide an invaluable resource to investigators who wish to have standardized and robust protocols for handling, generating, and manipulating stem cells. The center also co-authored a book chapter entitled Modeling CNS Disease Using iPS Cells that is currently in preparation for a book entitled Stem Cells: Regenerative Pharmacology, Drug Discovery and Development. This book will focus on the principal non-therapeutic applications of stem cell technology and will provide emphasis on the technology strengths, challenges, and future promises. In August, NIH CRM sponsored an important invitation-only two-day meeting along with NCATS and NIEHS that brought together the key government and industry players interested in investing and developing iPS cell-based assays for drug discovery and toxicology. The goals for this meeting were to present current efforts on developing iPSC banks, determine what roadblocks still exist before pharmaceutical companies will make major investments into using iPS cell-based screens in their drug development process, present a crowdsourcing developed business model for deposit and distribution of both research and clinical grade iPSC lines from repositories, and facilitate important public-private partnerships and collaborations. With more than 10 major pharmaceutical companies attending along with service providers for stem cell-based technologies and NIH officers and investigators from more than 15 institutes/centers, this symposium achieved many of its goals and was a benefit to the field by providing a useful forum for discussing current roadblocks and assembling the right partnerships to overcome challenges and advance the field. NIH CRM is currently preparing a position paper to summarize the meeting and disseminate the key points that were made so that the meeting can have broader impact for the field in advancing iPS cell-based assays for drug development and disease modeling. Another major milestone reached by NIH CRM in FY 13 will be the identification of major efforts, or big swings, that illustrate the highest potential for successful translation to stem cell therapy, for minimal investment. It is anticipated through the Therapeutic Challenge Program that 2-3 projects that also have support from their ICs will be funded and advance towards the clinic. The NIH CRM has completed negotiations with several companies to provide ready access to iPSC generation, differentiation, and genetic engineering services, and these are currently in place for impending use. These efforts supplement in critical ways the Centers efforts to provide services directly to the intramural community. In addition, NIH CRM has continued to work with representatives from the FDA, NIST, and other government groups involved in the regulatory process of cell therapies to provide researchers with the appropriate information and materials to move through the regulatory approval process more efficiently. Another meeting with the FDA is currently being organized for spring 2014 to provide further guidance for investigators navigating the regulatory process using cell therapies. In summary, the NIH CRM has established a clear path forward for screening and therapy efforts using PSCs and their derivatives. As part of its important focus on iPS cell-based assay development, optimization, widespread use, and commercialization, the NIH CRM has helped distribute over 125 iPSC lines to more than 30 investigators from 12 different federal institutes/agencies and 15 extramural institutes/organizations. NIH CRM identified companies permitting freedom to operate on various genomic engineering tools and advocated the need for a flexible approach to cell based assay development that the industry should consider. This along with the research symposia, training courses, seminar series, pilot grant program, cell distribution forms, and service contracts has proved the NIH CRM to be a valuable resource for the NIH IRP, having enabled the NIH to be a leader in the field of stem cell research. In the future, provided funding support continues, NIH CRM anticipates supporting NIH as a leader in the field by building on its numerous strategic efforts to facilitate the transition of key therapeutic and screening initiatives developed by the IRP to new therapy, screens, and commercialization.

Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
National Institute of Arthritis and Musculoskeletal and Skin Diseases
Zip Code
Rao, Mahendra; Ahrlund-Richter, Lars; Kaufman, Dan S (2012) Concise review: Cord blood banking, transplantation and induced pluripotent stem cell: success and opportunities. Stem Cells 30:55-60
Rao, Mahendra (2012) Stem cells and regenerative medicine. Stem Cell Res Ther 3:27
Mamidi, Murali Krishna; Pal, Rajarshi; Dey, Sovan et al. (2012) Cell therapy in critical limb ischemia: current developments and future progress. Cytotherapy 14:902-16
Rao, Mahendra (2012) The NIH and the regenerative medicine field. Regen Med 7:129-31
Lathia, Justin D; Venere, Monica; Rao, Mahendra S et al. (2011) Seeing is believing: are cancer stem cells the Loch Ness monster of tumor biology? Stem Cell Rev 7:227-37
Rao, Mahendra (2011) Keeping things simple. Nat Methods 8:389-90
Chiu, Arlene Y; Rao, Mahendra S (2011) Cell-based therapy for neural disorders--anticipating challenges. Neurotherapeutics 8:744-52
Vemuri, Mohan C; Chase, Lucas G; Rao, Mahendra S (2011) Mesenchymal stem cell assays and applications. Methods Mol Biol 698:3-8
Rao, Mahendra (2011) Straight talk with... Mahendra Rao by Dolgin Elie. Nat Med 17:1163
Rao, Mahendra S (2011) Funding translational work in cell-based therapy. Cell Stem Cell 9:7-10