The National Institute of Diabetes & Digestive & Kidney Diseases

Trans-NIDDK Strategic Planning: Stem Cells and Developmental Biology Agenda

September 19, 2000

Bethesda Hyatt Regency Hotel

Introductions

Opening Remarks
Allen M. Spiegel, MD, Director, NIDDK
Jeffrey L. Gordon, MD, Discussion Leader

Brief Discussion of Background Material

1. Review of current relevant grant listings
2. Description of recent initiatives
3. Related NIDDK planned initiatives (e.g. trans-NIDDK RFA on stem cells and HL/DK RFA on hematopoietic stem cell plasticity)
4. Previous recommendations (stem cell, pancreas, zebrafish, kidney/urology, functional genomic initiatives and resources)

1. Identification & isolation of stem cells in selected tissues (state of understanding, including available morphologic, molecular, and clonigenic assays)
   • Bone marrow
   • Stomach/intestine
   • Liver
   • Kidney
   • Prostate
   • Bladder
   • Pancreas
   • Neuromuscular system
   • Bone
   • Other
2. Stem cell origin
3. Genes controlling stem cell division (asymmetric/symmetric) and commitment of progeny to a particular fate
4. Properties of immediate committed daughters
5. Functional genomics: gene expression profiling; surface markers
6. Issues related to niche: interactions with microenvironment that help define 'stemness'
7. Normal functions of stem cells in organ of interest: role in morphogenesis; maintaining homeostasis in self-renewing cell populations
8. Role in disease pathogenesis
9. Stem cells as therapeutic targets (see 4)

1. Plasticity
2. Transplantation
3. Targets for gene therapy; modulation of activity for therapeutic benefit (includes chemo- and radiotherapy)

1. Lack of molecular markers and clonigenic assays for retrieval and characterization of stem cells and committed lineage progenitors
2. Cultural barriers: organizing scientific community across model systems and organ systems
3. Other technological or resource needs

Discussion of Committee's work: Presentation of report to NIDDK

1. Define state of knowledge and critical scientific needs in area of stem cell research: Should there be a subdivision into working groups that focus on each organ system?
2. Provide examples of diseases whose pathogenic mechanisms are believed to involve disruption of normal stem cell functions
3. Provide examples of therapeutic interventions or opportunities that do, or could, center on stem cells and/or their immediate committed daughters
4. Describe a strategy for supporting new and innovative research on stem cell biology; define scope of resource needs and format for NIDDK/NIH- sponsored research initiative.
5. Coordinate report with efforts of the NIDDK committee working on functional genomic and bioinformatics initiatives (Consideration of cross-committee membership and reporting).

Other issues as time permits: Research infrastructure

1. Technology development. How to stimulate innovation and risk-taking in the scientific community? Clear NIDDK/NIH definition of intent with this initiative for stem cell research. Definition of scope of research; scope of allocations; available expertise for peer review process
2. Bio-informatics: engineering access to deposited searchable databases generated from microarray studies (and ? proteomics); sharing of reagents and model systems (includes providing funds for reagent distribution)
3. Interactions with biotechnology and large Pharma.
4. Training and manpower

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