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Safety of Cell Therapies Derived from Human Embryonic Stem Cells

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Safety of Cell Therapies
Derived from Human
Embryonic Stem Cells
CTGTAC #45
April 10, 2008
1
Public Scientific Discussions
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Advisory Committee Meetings
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Human Stem Cells as Cellular Replacement Therapies for
Neurological Disorders. July 13-14, 2000.
Unrelated allogeneic hematopoietic stem/progenitor cells from
placental/umbilical cord blood for hematopoietic reconstitution
February 27, 2002.
Allogeneic Pancreatic Islets for Type 1 Diabetes October 9-10,
2003
Cellular Products for the Treatment of Cardiac Disease March 1819, 2004
Cellular Products for Joint Surface Repair March 3-4, 2005
Draft Guidance for Industry for minimally manipulated, unrelated
allogeneic cord blood, and the regulatory approach to minimally
manipulated unrelated allogeneic peripheral blood
stem/progenitor cells. March 30, 2007
Workshops
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FDA/NIST Sponsored Workshop In Vitro Analyses of Cell/Scaffold
Products December 6-7, 2007
2
Stem Cell-based Products:
Scientific Considerations and
Stem
Cell
Safety Evaluation
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Properties of embryonic stem cells
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Safety Evaluation
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Immature
Cells
Inappropriate differentiation
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Tumorigenicity
Ectopic tissue formation
Mature
Cells
2
tumorigenicity
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Self renewal
Pluripotency
Differentiation in culture
Safety Assessment
21 CFR, part 312.23(a)(8):
Pharmacologic & Toxicologic Studies
“…adequate information about the
pharmacological & toxicological studies…on
the basis of which the sponsor has concluded
that it is reasonably safe to conduct the
proposed clinical investigations. The kind,
duration, & scope of animal and other tests
required varies with the duration & nature of
the proposed clinical investigations.”
4
Focus
пЃ® Safety
issues regarding the use of
cellular products derived from
human embryonic stem cells
пЃ® Discussion from CTGTAC and
additional expert panelists
5
Discussion Issues..
Inappropriate
Differentiation/Tumorigenicity
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Preclinical safety assessment
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Animal species/model selection criteria
Clinically relevant study designs
Site of cell implantation
пЃ® Fate of cells after administration
пЃ® Cell dose extrapolation from animal to human
пЃ® Study duration
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6
Discussion Issues..
Product Characterization
пЃ® Characteristics
related to safety
пЃ® Ability
to predict inappropriate
differentiation/tumorigenicity
пЃ® Sensitivity/specificity of assays to
distinguish degree(s) of cell
differentiation
7
Discussion Issues..
Clinical Trial Design
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What tools exist for monitoring the fate of
infused cells during clinical trials?
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Where do the cells go in the body?
Can we monitor inappropriate cellular
differentiation in the body?
Cell dose levels:
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Balancing possibility of therapeutic effect with
need to ensure maximum safety
8
Overview
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Guest Presentations
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Dr.
Dr.
Dr.
Dr.
Dr.
Carpenter, Novocell
Dinsmore, ACT
Lebkowski, Geron
Isacson, Harvard
Bulte, Johns Hopkins
Open Public Hearing
Panel Discussion of FDA Questions
9
CTGTAC #45
Thanks to all
participants!
10
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