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 Institutes
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 groups 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 Groups 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.
Full Report (PDF, 330
K)
See also: Working Group on Stem
Cell Plasticity, March 2000
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