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Workshop Report

Summary of Workshop Recommendations for the Establishment of a Common Fund NIH Center for Regenerative Medicine (NCRM)

The NIH Director invited a panel of distinguished experts to give counsel on the establishment of a new NIH Center for Regenerative Medicine (NCRM) on the NIH Campus. The meeting took place at the NIH on January 15, 2010. The list of invited participants and their affiliations are provided below. The participants presented state of the art research findings from their own research programs and conveyed current trends in the field of regenerative medicine. A panel discussion at the end of the meeting centered on ways in which the NIH’s own intramural program was positioned to advance regenerative medicine based therapies to correct or replace diseased/damaged tissues through the establishment of NCRM.

The research talks and discussions highlighted: (1) the absence of robust, agreed-upon protocols for the derivation of so-called “induced pluripotent stem cells” (iPS cells) that are a type of adult stem cell that can be derived by reprogramming a variety of cell types in the human body in to an embryonic stem cell-like state; (2) the need for a better understanding of how cells are reprogrammed in to iPS cells as well as the precise steps in developmental pathways by which iPS cells can subsequently differentiate in to any cell type needed for a cellular therapy; (3) the incomplete understanding of the differences between iPS cells and human embryonic stem cells, which are the current “gold standard” for regenerative medicine therapies, and (4) the need to satisfactorily address safety concerns stemming from the ability of stem cells to produce tumors.

There were several specific recommendations including:

  • Finding new strategies for making iPS cells that do not involve introducing foreign DNA in to adult cell types to reprogram them in to iPS cells (foreign DNA can cause tumors to form), for example, by working with the NIH Chemical Genomics Center to conduct high throughput screens to identify small molecules that can reprogram adult cell types in to iPS cells
  • Using these same screening strategies to identify small molecules that can subsequently be used to differentiate iPS cells in to any cell type needed for a particular cellular therapy
  • Optimizing iPS cell derivation and differentiation protocols such that one can generate sufficiently large, uniform batches of iPS cells, and from these, specific cell types needed for a cellular therapy
  • Ensuring correct differentiation of iPS cells in to a desired cell type by tracking specific molecules that can serve as indicators of different stages of cellular differentiation
  • Developing technologies to track iPS cell-derivatives in vivo to see if they specifically integrate in to the targeted organ/site as well as methods to kill any cells that go awry, for example, those that form tumors
  • Interacting with the NIH gene therapy group to correct gene defects in iPS cells before they are used to make cell types that will be reintroduced in to a patient to correct a genetic disease
  • Using iPS cells to establish in vitro models of disease
  • Making mouse models with “humanized” tissues for pre-clinical studies of iPS cells
  • Addressing problems related to immune system rejection of foreign cell types, that is, “graft vs. host” disease
  • Establishing cell banks that can house ethnically diverse panels of normal and patient-derived iPS cells with complete genomic, epigenomic, gene expression, and phenotypic analyses, for research purposes, as well as clinical grade cells for cellular therapies. NCRM has access to patients with rare diseases through the NIH Clinical Center and cells from these patients should be included in the cell banks
  • Making all information related to the iPS cells, for example, the protocol by which they were derived, openly accessible. Ideally, information on how the cells perform after they are distributed should also be made available
  • Extending work to beyond iPS cells to include other types of regenerative medicine therapies for example trans-differentiation of skin cells in to neurons for targeted neuronal therapies

A final general recommendation was to synergize NCRM’s efforts with other national and international activities to maximize investments and reduce unnecessary duplication.

Invited Participants:

  • Dr. Kristin Baldwin (Scripps Research Institute)
  • Dr. Helen Blau (Stanford University)
  • Dr. Linzhao Cheng (Johns Hopkins University)
  • Dr. James Ellis (University of Toronto)
  • Dr. Peggy Goodell (Baylor University)
  • Dr. Ron McKay (National Institutes of Health)
  • Dr. Rene Reijo Pera (Stanford University)
  • Dr. Raj Puri (Federal Drug Administration)
  • Dr. David Scadden (Harvard University)
  • Dr. Lorenz Studer (Memorial Sloan-Kettering Cancer Center)
  • Dr. Shinya Yamanaka (University of Kyoto)
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