Council of Councils > Concept

Project Concept

NIH Council of Councils
August 15, 2011
 

Program Title:

NIH Center for Regenerative Medicine

Program Goal:

To create a world-class center of excellence in iPS cell technology.
To use this facility to advance translational studies and ultimately cell-based therapies in the NIH Clinical Center

IC Director(s) or Senior IC Staff who provide general scientific leadership:

Steve Katz, Story Landis

Program Coordinator(s): IC staff who provide daily oversight and leadership for the program:

Mahendra Rao (newly recruited Center Director), David Owens (extramural program coordinator, NINDS)

Total # of Proposed Initiatives and their names:

• NIH Center for Regenerative Medicine Collaborative Projects

Overview: The NIH Center for Regenerative Medicine (NIH CRM) aims to be a foundation for excellence in stem cell technology and translational applications. This facility will be embedded within the NIH IRP and will capitalize on the substantive expertise and resources within the IRP. The Center will be led by Dr. Mahendra Rao, an international leader in stem cell biology, and will serve as a focal point for regenerative science at the NIH. Emphasis will be on potential clinical applications of stem cells, capitalizing upon the high throughput capabilities of the NIH Chemical Genomics Center and the unique clinical research opportunities provided by the NIH Clinical Center. The NIH CRM will serve as a ‘hub of excellence’ in induced pluripotent stem (iPS) cell technology, will develop experimental tools and cell lines, and will provide quality control and standards, thereby benefiting the broader research community and advancing personalized medicine. Finally, the Center goals include the development of translational projects with intramural or extramural collaborators.

These Collaborative Projects will not only result in the development of new therapies, they are expected to provide the scientific context to address cell therapy translational hurdles generally. Issues such as safety and cell tracking, manufacturing, and regulatory hurdles are expected to be addressed. In addition, standards for the use of iPS-based cell systems as platforms for small molecule screening are likely to be developed. Collaborative Project applications will be solicited and reviewed through the extramural grant process; therefore, this component of NIH CRM requires review and concept clearance by the Council of Councils.

Rationale for Collaborative Projects Initiative: The ability to reprogram somatic cells to iPS cells has the prospect for changing the face of medicine in the 21st century. There are enormous challenges to making this promise a clinical reality. The intent of NIH CRM is to address these challenges. Working on specific therapies through the Collaborative Projects initiative will allow the articulation of problem points in the cell therapy pipeline and will thus enable the Center to address and solve these problems.

The collaborative aspect of this initiative is critical. The Center is intended to provide expertise for the development of cell therapies, but the expertise in a variety of disease areas will require collaboration with other partners. Although the Common Fund will pay for these collaborative projects for the first five years, the expectation is that ICs will support future collaborations with the Center once it is established.

This initiative meets the Common Fund criteria as follows:

1. Is the program or initiative truly transforming – could it dramatically affect how biomedical and/or behavioral research is conducted within a predictable timeframe? This initiative is expected to transform the way in which cell therapies are developed by pinpointing problem steps and providing the context for overcoming the challenges. The therapies to be developed will move into clinical trials where they will hopefully provide a new avenue for therapy that can be widely used. In addition, the process improvements that are tested via these projects are likely to become the standard for development of additional cell therapies in the future.
2. Can the transformative outcome be accomplished or milestones reached within the period of funding from the Common Fund? Within the five year time frame of the proposed initiative, standards for the development of cell based therapies can be established. Individual projects to be conducted may be proposed at different stages of development. Those that are further along the translational pipeline can be expected to result in INDs by the end of the 5 year timeframe. Those that are at an earlier stage of development may not reach this point but will be well positioned for continuation at the end of CF support.
3.  Will the outcomes synergistically promote and advance the individual missions of NIH ICs to promote health? The Collaborative Projects Initiative Concept has been conceived with this goal in mind. The overarching goal of overcoming translational hurdles for cell therapies will enhance all IC-funded projects that aim to develop cell therapies. Moreover, the therapies to be developed through this initiative will enhance the mission of those ICs that are most interested in the diseases that are addressed.
4.  Is the program or initiative sufficiently complex that it requires coordination of the efforts of multiple ICs? Is the initiative not easily assignable to the mission of a single IC? The NIH CRM by its nature is cross-cutting in that stem cells can be useful for regenerative medicine approaches in many tissues. The management of the projects to be pursued through the Center will require coordination among all of the ICs with an interest in the work.
5. Is the proposed initiative something that no other entity is likely or able to do? Although the NIH CRM could potentially arrange individual collaborations, the Common Fund approach provides the opportunity for anyone, working on any disease, to apply for these resources. This approach would be unlikely to be funded by the ICs. Other institutions across the country and around the world are developing cell therapies, but other entities are not likely to be dedicated to community-wide process enhancements and working with appropriate regulatory groups to pave the way for future efforts. The intent of NIH CRM is not merely to develop therapeutic strategies but also to extrapolate lessons learned from these initiatives to benefit the community at large.