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NHLBI Working Group
Cell-Based Therapies for Regenerative & Reparative Medicine: Vision, Scope, and Directions

Executive Summary

The National Heart, Lung, and Blood Institute (NHLBI) convened a Working Group of investigators on May 1-2, 2002 to define the scientific state-of-the-art regarding cell-based therapies, discuss the implications of that knowledge for research and medicine, and identify opportunities and obstacles to successfully exploit cell-based therapies for repairing or replacing damaged, diseased, or defective tissue with new, functional tissue. The Working Group was organized by the Institute’s Cell-based Therapy Group to assist in the formulation of a strategic plan for new cell-based therapies.

The Working Group agenda was organized into three primary sessions. The first session on “Tools, Technologies and other Resources” included discussions on “Sources of Cells for Cell-based Therapies” and on “Models and Functional Assessment.” The second session on “Basic Science Knowledge” included discussions on “Lessons from Developmental Biology for Cell-based Therapy” and the “Immune Aspects of Cell-based Therapy.” The third session on “Clinical Applications” included discussions on “Disease Candidates for Cell-Based Therapies” and on “Clinical Applications.” In the final session, participants discussed recommendations for a “Master Plan: Vision, Scope and Direction” based on the summaries from the sessions on “Tools, Knowledge, Applications” as well as the group’s assessment of “Opportunities, Obstacles and Implementation Strategies.”

The recommendations of the Working Group have been organized into sixteen items in three broad categories depending upon whether implementation might best be initiated on a Divisional, or NHLBI-wide level, or whether multiple Institutes of the National Institutes of Health (NIH) should be involved. The complete list of these recommendations is included in section four of this report and the complete meeting agenda is included in the appendix.

The Working Group’s top recommendation is for a continued, strong basic research program in stem cell biology and cellular therapy. The key recommendations for the development of new programs are for: 1) definition of the stem cell niche both structurally and functionally; 2) identification of regeneration mechanisms at both the cellular and tissue level; 3) understanding the immunogenic response to cells intended for use as cell-based therapies; 4) the development of improved non-invasive imaging techniques to track cells in vivo; 5) an original effort promoting lung stem cell research; 6) and new research on the cardiomyognic potential of stem cells. In addition, the Working Group recommended continuation of programs supporting Bioengineering Research Partnerships and Tissue Engineering grants along with new and continued support of training programs for the isolation, culture and use of stem cells. Furthermore, the Group recommended the NHLBI continue to sponsor stem cell meetings for the purpose of building an interdisciplinary community of investigators to study stem cells and cell-based therapies.

The area of embryonic stem cells and their therapeutic potential was addressed. The Working Group supported this research area and proposed an embryonic stem cell data workshop to standardize data collection and facilitate data comparisons among laboratories. A greater availability of human embryonic stem cell lines to the scientific community was seen as essential. The Working Group saw the need for Institute-sponsored stem cell research centers, such as the Specialized Centers of Clinically Oriented Research (SCCORs) to encourage collaborative teams with multi-disciplinary basic and clinical investigators, including an assessment of stem cell delivery and safety.

The Working Group also recommended utilization of genomic and proteomic techniques to characterize the progression from a stem cell to adult cell types. A number of resource needs, including stem cell banks and animal models to establish treatment efficacy, were also identified.

PDF fileFull Report (PDF, 330 K)

See also: Working Group on Stem Cell Plasticity, March 2000

Last Updated June 2011

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