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Stem Cell Research at the NIH


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Title: Stem Cell Research at the NIH

Stem Cell Research at the NIH
What are the unique properties that make human
embryonic stem cells (hESCs) special?
  • hESCs have an unlimited capacity for self renewal
    in culture
  • hESCs have the capability to differentiate into
    any adult cell type (under the right conditions)

Non-Embryonic Stem Cells
  • Include Fetal, Umbilical Cord Blood, and Adult
    Stem Cells
  • Found in many organs such as bone marrow, gut,
    skin, nervous system, and liver
  • Relatively rare (1/1000 to 1/10,000)
  • Limited capacity for self renewal in the
  • Limited capacity for differentiationusually
    limited to cell types in organ of origin

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Have hESCs or Adult Stem Cells Treated Any Human
  • Although hESC are thought to offer potential
    cures and therapies for many devastating
    diseases, research using them is still in its
    early stages since there discovery in 1998 and
    initiation of federal funding in August 9, 2001.
  • Adult stem cells, such as blood-forming stem
    cells in bone marrow, are currently the only type
    of stem cell commonly used to treat human
    diseases such as leukemia, lymphoma and other
    inherited blood disorders.

Image of hESCs in Culture
hESC Line Pictured HSF-6
Intro To Human Development
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August 9, 2001 801 P.M. CDT
THE PRESIDENT  Good evening.  I appreciate you
giving me a few minutes of your time tonight so I
can discuss with you a complex and difficult
issue, an issue that is one of the most profound
of our time. The issue of research involving
stem cells derived from human embryos is
increasingly the subject of a national debate and
dinner table discussions
Presidents Policy
As a result of private research, more than 60
genetically diverse stem cell lines already
exist. They were created from embryos that have
already been destroyed, and they have the ability
to regenerate themselves indefinitely, creating
ongoing opportunities for research.   I have
concluded that we should allow federal funds to
be used for research on these existing stem cell
lines, where the life and death decision has
already been made.
Eligibility Criteria for Federal Funding
NIH Stem Cell Registry
Worldwide Survey of Stem Cell Lines
  • According to the journal Stem Cells there are
    over 400 hESC lines available
  • More than 300 are ineligible for federally funded
  • More lines are being created overseas than in the
    United States

Recent Legislation
  • H.R. 810 - Research Enhancement Act of 2005
  • Passed House/Senate Vetoed by President
  • S. 2754 - Alternative Pluripotent Stem Cell
    Therapies Enhancement Act
  • Passed Senate Failed in the House
  • S. 3504 - Fetus Farming Prohibition Act of 2006
  • Passed House/Senate Signed into law

NIH Supports Research on Many Types of Stem Cells
  • Early phase of researchbreakthroughs with
    clinical relevance might emerge from research on
    many types of stem cells
  • FY2005 investment in human adult stem cell
    research Approximately 200 million
  • FY2005 investment in human embryonic stem cell
    research Approximately 40 million
  • Promote growth in both areas in the future

The Immediate Challenge for NIH and hESC Research
  1. Generate and characterize distribution quality
    human ES cell lines from the NIH Registry
  2. Stimulate more research on basic biology
  3. Train investigators to culture and use stem cells

Infrastructure Awards
  • Awards to organizations with entries on NIH Stem
    Cell Registry available for Federal funds to
    develop into distribution-quality cell lines
  • Two year period of support
  • Nine awards for total of 6,953,815
  • Led to development of 21 hESC lines ready for

National Stem Cell Bank (WiCell Research
  • Ready source of hESCs in one location
  • Compare and expand hESCs available to
    NIH-supported scientists
  • Ensure consistent quality control
  • Reduce cost to obtain cells listed on the NIH
    Stem Cell Registry
  • 8 hESC lines available for distribution
  • Negotiations underway for additional lines from
    the providers.

NIH Intramural Research
  • Several 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
  • http//

NIH Stem Cell Unit
NIGMS Exploratory Centers for hESC Research
  • Awards were made to multi-investigator teams to
    conduct research using hESCs at
  • Albert Einstein College of Medicine
  • The Burnham Institute
  • Mount Sinai School of Medicine
  • University of Michigan Medical Center
  • University of Wash/Fred Hutchinson Cancer
    Research Center
  • WiCell Research Institute
  • The Exploratory Center P20 Grant can lead to a
    Specialized Center P50 application

Centers of Excellence for Translational Stem Cell
  • Multidisciplinary teams of stem cell experts,
    clinical researchers, and transplant surgeons
  • Speed translation of basic knowledge to clinical
    therapies for human disease
  • Test adult and embryonic stem cell therapies for
    many diseases including blood cancers, kidney
    disease, and neurological disorders
  • Awards made to UC-Davis and Northwestern

Stem Cell Training
  • Short-term Courses in hESC Cell Culture
    Techniques held at
  • Burnham Institute
  • Childrens Hospital of Orange County
  • Jackson Laboratory
  • Technion-Israel Institute of Technology
  • University of Georgia
  • University of Minnesota
  • University of Pittsburgh/Magee-Womens Research

Recent Scientific Advances Using Stem Cells
  • Further Evidence that Human Embryonic Stem
    Cells May Help Treat Vision Loss
  • (Laboratory of R. Lanza, Cloning and Stem Cells
    8 189-199)
  • Signaling Molecules May Help Adult Brain Repair
    Itself Following a Stroke
  • (Laboratory of R. McKay, Nature advance online
  • Neurons Grown from Embryonic Stem Cells
  • Restore Function in Paralyzed Rats (Laboratory
    of D. Kerr, Annals of Neurology 60 3244)

Recent Scientific Advances Using Stem Cells
  • Blood Stem Cells Are Already Used to Treat
  • (National Marrow Donor Program,
  • ESCs Cure Mouse Model of Hemophilia
    (Laboratories of O. Smithies and J. Frelinger,
    Proc Natl Acad Sci 102 2958-2963)
  • Human Stem Cells Help Rats with Spinal Cord
    Injury (Laboratory of A.J. Anderson, Proc Natl
    Acad Sci 102 1406914074)

Recent Scientific Advances Using Stem Cells
  • Protein Responsible for Balancing Stem Cell
    Growth, Aging, and Cancer
  • (Laboratory of S. Morrison, D. Scadden, and N.
    Sharpless, Nature Advanced Online Publication)
  • Transplanted Cells Improve Symptoms in a Monkey
    Model of Parkinson's Disease (Laboratory of N.
    Hashimoto, J Clin Invest 115 102109)
  • Scientists Generate Motor Neurons from hESC
    (Laboratory of S-C Zhang, Nature Biotechnology
    23 215221)

NIH Stem Cell Internet Sites
  • Stem Cell Information
  • http//
  • NIH Stem Cell Registry
  • http//
  • Email

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