The Immediate Challenge for NIH
and Human ES Cell Research
Generate and characterize distribution
quality human ES cell lines from the
NIH Registry
Stimulate more research on basic
biology
Train investigators to culture and use
stem cells
NIH hESC Awards
FY2002-FY2003
9 Infrastructure Awards
27 Investigator-initiated Awards
88 Administrative Supplements
2 Pilot & Feasibility Projects (Beta Cell Consortium)
3 Postdoctoral Fellowships
6 Training Grants
5 Short Term Cell Culture Training Courses
3 Exploratory Center Research Grants
Infrastructure Awards
Awards to organizations with entries on NIH
hESC Registry available for Federal funds to
develop into distribution-quality cell lines
Two year period of support
Nine awards for total of over $6M
Led to development of twelve hESC lines ready
for shipment with more to come in near future
Program Announcement to be reissued
Program Announcements (PAs)
Short-term Courses in Human Embryonic Stem
Cell Culture Techniques
– T15 Mechanism (Continuing Education
Training)
– Supported by 11 NIH Institutes
– $1M direct costs committed (FY2002)
– Three-year support ($150,000/year direct
maximum)
– Five awards have been made
Program Announcements
Career Enhancement Award for Stem Cell
Research (K18) and Career Development Award
(K08, K23)
NIDDK, NIAAA, NINR, NIAID, NHLBI
Mid-career investigators, 6-24 month salary and
$50,000 direct/year
Training to use stem cells in research
– grants.nih.gov/grants/guide/pa-files/PAR-02-069.html
Requests for Applications (RFAs)
Exploratory Center Grants for hESC Research (NIGMS)
(Letter of intent 2/13/03; receipt date 3/13/03)
Multi-investigator teams to conduct multidisciplinary
research using hESCs
Infrastructure, growth and maintenance, biochemical/
molecular markers for hESCs, unique properties of hESCs,
pilot studies leading to additional investigator-initiated
research grant applications
P20 Exploratory Center Grant, leading to P50 application
$2M in 2003, funding 2-3 P20 grant applications for up to 3
years at $500,000 direct/year
Additional PAs/RFAs
Innovative Concepts and Approaches to Developing
Functional Tissues and Organs for Heart, Vascular,
Lung, and Blood Applications (R21) (NHLBI)
Plasticity of Human Stem Cells in the Nervous System
(R01) (NINDS, NIA, NIMH, NHLBI)
Basic and Applied Stem Cell Research for Arthritis and
Musculoskeletal Diseases (RO1) (NIAMS)
Stem Cells in Development/Repair of Orofacial
Structures (NIDCR)
Additional PAs/RFAs
Basic Research on Mesenchymal Cell Biology (NIA,
NHLBI)
Comprehensive Programs in Beta Cell Biology (NIDDK)
Hematopoietic Cell Lineage Genome Anatomy (NIDDK)
Cellular Repair Studies of the Auditory and Vestibular
System (NIDCD)
Research on Stem Cell Biology and Cell-based
Therapies for Heart, Lung, Blood, and Sleep Disorders
(NHLBI)
Stem Cell Research for Alcohol-related Disorders
(NIAAA)
NIH Intramural Research
Nine laboratories at NIH are currently
using hESC in their research
Expanding interest as cell line availability
becomes more straightforward
Creation of a stem cell characterization
unit within Intramural Research Program
at NIH
Human ES Cell Research
Symposium
Showcased research conducted and
supported by NIH on June 13, 2003
Podium presentations and posters
Workshops on topics of interest (cell
culture, policy, intellectual property)
Immediately after the GM Stem Cell
Symposium on June 10-11, 2003
Highlights of Human ES Cell
Research Progress
Neural differentiation—protocol described
to generate dopamine secreting neurons
similar the midbrain neurons lost in
Parkinson’s disease
Pluripotency/Self Renewal—discovery of
Nanog, a transcription factor that serves
as a master regulator of pluripotent and
self renewing state of human ES cells
Highlights of Human Es Cell
Research (cont.)
Hematopoietic differentiation—culture
conditions being defined that promote
specialization of human ES cells into blood
forming cells
Cardiac myocyte differentiation—
functional characterization of atrial,
ventricular, and nodal cardiac myocytes
differentiated from human ES cells in
culture
Research Advances using Other
Types of Stem Cells
Neuronal stem cells—discovery and
characterization of a source of selfrenewing neural progenitor cells in adult
brain white matter
Dental pulp stem cells—discovery of a
pluripotent population of stem cells in
dental pulp of deciduous teeth which can
be cultured and divides
Research Advances using Other
Types of Stem Cells (cont.)
Hematopoietic stem cells—discovery that Wnt
pathway can promote self renewal of
hematopoietic stem cells in culture
Multipotent Adult Progenitor Cells (MAPC)—
bone marrow mesenchymal stem cell with
capacity to self renew and become many
different specialized cells
Umbilical cord stem cells—research to define
culture conditions that promotes expansion of
cells obtained from a single unit in culture
Research advances (cont.)
Human EG cells—in a rat model for ALS,
scientists restored movement by
transplanting EG cells, which secrete
factors that promote survival of
endogenous neurons
Muscle stem cells (satellite cells)—
scientists determined that the Wnt
signaling pathway activates muscle
formation
NIH Supports Research on Many
Types of Stem Cells
Early phase of research—breakthroughs
with clinical relevance might emerge from
research on many types of stem cells
FY2002 investment in human adult stem
cell research--$170,745,000
FY2002 investment in human embryonic
stem cell research--$10,689,000
Promote growth in both areas in the future
On the Research Horizon
Definition of standardized human ES cell
culture conditions that obviate the need for
either mouse or human feeder cells
Enabling tools and technologies to further
characterize stem cells as they become
specialized cells (specific antibodies, etc.)
Define the molecular pathways that
specify differentiation into different
specialized cells
Horizon (cont.)
Determine the factors/conditions critical for
long-term survival and function of
transplanted cells in a host
Understand control of cell division—
essential to expand cells before
specialization, but must be tightly
regulated after transplantation for therapy
NIH Stem Cell Web Sites
Stem Cell Information
– stemcells.nih.gov
Human Embryonic Stem Cell Registry
– escr.nih.go