NIH Center for Regenerative Medicine

National Institute Of Arthritis And Musculoskeletal And Skin Diseases

Investigators

Linked publications & trials

Abstract

The NIH Center for Regenerative Medicine (NIH CRM) represents an initiative within the intramural program supported by the Common Fund. NIH CRM aims to be a foundation for excellence in stem cell research, with a focus on induced pluripotent stem cells (iPSC) technology and clinical translation. This facility is embedded within the NIH IRP and capitalizes on the substantive expertise relevant to the development and clinical applications of this powerful technology. There is an emphasis on potential clinical applications, capitalizing on the high throughput capabilities of the NCATS and the unique clinical research opportunities provided by the NIH Clinical Center. During the 2012 fiscal year the NIH CRM made further progress in establishing itself as a resource for the clinical translation of stem cells, extending the efforts led by Dr. OShea. The Centers new director, Mahendra Rao, continued early efforts involving co-sponsorship with the NIH Stem Cell Interest Group both the Stem Cell Seminar Series and the annual Stem Cell Research Symposium showcasing NIH CRM-funded research. Through these continuing efforts, NIH CRM has been able to provide a forum for the investigators to share their work and participate in fruitful discussion related to stem cell-based projects with tangible results. In addition to the annual meeting co-sponsored with SCIG, NIH CRM sponsored two new meetings. In May, NIH CRM in conjunction with FAES held a successful industry symposium showcasing newly available resources. In August, NIH CRM sponsored an important invitation-only meeting that brought together the key government and industry players involved in achieving stem cell therapy. Several all-hands working sessions yielded specific next steps as well as interest in continuing the discussion. The Center has continued to implement the Directors vision of how best to leverage most rapidly its resources and relationships to make stem cell therapy a clinical reality. Having reached wide consensus on consent and distribution issues and developed standard forms and agreements, these materials have been made widely available to intramural researchers, stem cell consortiums nationwide, as well as research groups globally. This has been possible due to outreach to outside groups, to establish infrastructure for widely disseminating the standardized tools and processes while simultaneously facilitating research collaborations. New agreements reached have been with a second group at Kyoto (IFMS), and with the LV Prasad Eye Institute (LVPEI) in India to accelerate RPE research. At the governmental level, the post-LOI US-India Joint Statement in development is now in final review. In addition to finalizing several agreements currently in the works, the next focus for outreach will be the UK, with a trip planned for November. Significant developments have also resulted from NIH CRMs first agreement, with CIRM. A large PD workshop with CIRM will take place in the next few months, and a collaboration has been forged between NIHs Restifo and CIRM researcher Ribas, on a study recently awarded $20 million by CIRM that will launch an immune therapy clinical trial. Also critical to the Centers success in making cell lines available, with minimal reach-through, has been its successful negotiation with one major repository, Rutgers, where cell lines have already been deposited according to the newly established terms. Additional repository agreements are in the pipeline. Another major milestone reached by NIH CRM in FY12 was the identification of major efforts, or big swings, that showed the highest likelihood for successful translation to stem cell therapy, for minimal investment. These have now been identified, encompassing both therapy and screening. For therapy, the big swings are RPE, PD, T cell therapy, and CGD. Screening is aligned with therapy, with swings identified in RPE and PD. In other significant developments, NIH CRM has completed negotiations with several companies to provide ready access to iPSC generation, differentiation, and genetic engineering services, and these should be in place by the end of the fiscal year. 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. With the administrative and oversight infrastructure established for the Center, Dr. Rao completed key recruitments for the Center. Following Scott Lipnicks departure in May 2012, a new AAAS fellow (Becker) and Research Fellow (Hunsberger) joined at the end of FY12. In addition, NIH CRM completed the upgrade to the Centers main conference room in the building to facilitate international interactions, and launched the new NIH CRM website. In the future NIH CRM will continue to build on its various strategic efforts to facilitate the transition of key therapeutic initiatives developed by the IRP to the clinic.