stem cells a technology assessment june2009
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June 1st, 2009
STEM CELLS
Jie HeTony Jia
Ji Young LeeAndy Yen
HISTORICAL PERSPECTIVE AND SCIENTIFIC BACKGROUND
Timeline 1963 – Discovered by McCulloch and Till 1981 – Mouse Embryonic Stem Cells Isolated 1998 – First Human Embryonic Cell Line
Developed
Scientific Background Self-Renewing Pluripotent Two Main Types
Adult Embryonic
SCIENTIFIC BACKGROUND
Advantages/Disadvantages Embryonic Stem Cells will divide indefinitely Adult Stem Cells have a finite lifetime Not all tissues contain Adult Stem Cells Adult Stem Cells are not controversial Embryonic cell lines Media Reagents Safety
RESEARCH METHODOLOGY
Lots of Primary Research Industry and Academia
Some supporting Secondary Research Issues:
How Big? Future Potential Obstacles Driving Forces Most Promising Opportunities Intellectual Property Commercialization Business Model
LIST OF INTERVIEWS CONDUCTED Dr. David Baltimore
Robert Andrews Millikan Professor of Biology at Caltech, Nobel Laureate
Dr. Martin F. Pera Director of Eli and Edythe Broad Center for Regenerative Medicine and
Stem Cell Research at USC
Dr. Agnes Lukaszewicz Postdoctoral Scholar for Dr. David Anderson (Founder of StemCells, Inc.) at
Caltech
Dr. Owen Witte Founding Director of Eli and Edythe Broad Center of Regenerative
Medicine and Stem Cell Research at UCLA
Dr. Marie Csete Chief Scientific Officer of California Institute of Regenerative Medicine
Kenneth Aldrich Chairman and CEO of International Stem Cell Corporation
S CURVE ANALYSIS
DEVELOPMENT CYCLE
Three Phases
Biotech – long, complicated, expensive development cycle
Stem Cells Complexity of cellular biology Risks of new, unproven therapy
Research Testing/ApprovalCommercializatio
n
DEVELOPMENT - RESEARCH
Where is this research taking place and why?
DEVELOPMENT - RESEARCH
Most research is in academia CIRM grant data
Most Stem Cell companies NOT involved in research.
Research is still in the early stages. Current trend, private sector unwilling to
invest until Stage 2 or Stage 3 Too expensive and complicated Low success rate
DEVELOPMENT – TESTING AND APPROVAL
Human use – requires rigorous testing and approval
No precedent, very unique procedure A form of personalized medicine
How can FDA regulate biotech
they have no experience in?
GMP LabsResearchers must educate
the FDA
Physician Initiated Trials
COMMERCIALIZATION
Bone Marrow Transplantation Not a Commercial Product
Fixed Cost Similar to existing treatments
No potential for business applications
COMMERCIALIZATION
Month
Kidney Transplantation Immune response due to incompatibilities
Expensive Immunodepressant have to be taken throughout lifetime
Stem cell technologies avoids it Cost reduced dramatically
COMMERCIALIZATION
Windpipe Transplant
COMMERCIALIZATION
Patents WARF – Preparation of primate and human
embryonic stem cells Geron – Cell therapy and Drug screening
applications using cells derived from hESC Kyoto University– Derivation of iPS cells from a
somatic cell
Time Estimates vary, Hard to predict Depends on Therapy/Application
Macular degeneration: 3 - 5 years
Diseases in USA
0
5
10
15
20
25
Diabetes Parkinson'sDisease
Alzheimer'sDisease
Skin Cancer Other Cancer
Disease
# o
f C
ases
(M
illi
on
s)
POTENTIAL MARKETS
Stem Cell Therapy Most Obvious Most Future Potential
Peripherals Media and Reagents Supplies Cultures
Disease Study and Drug Development Unlimited Supply of Human Cells Use Stem Cells to test drugs Safer, more Efficient
Most VC+Startup is in the latter 2
DRIVERS
Mainly Intellectual Curiosity
Later: When more money is involved Demand for Cures “Money, Fame, Sex.” –David Baltimore
HOW BIG AND THE NEXT BIG THING
Lots of Funding at USC/UCLA CIRM Funding 180+ Startups Ken Aldrich – $100 billion market after
required FDA approvals.
Next Big Thing Induced Pluripotent Stem Cells (iPS) Macular Degeneration Advanced Melanoma
IPS CELLS
Essentially Pluripotent Stem Cells Derived from non-Pluripotent Adult Cells Uses Skin Cells
Ethical Considerations
Advantages DisadvantagesNot Limited by Existing Cell Lines
Low Efficiency
Can be Derived Universally High CostGenetic Compatability Very New
MACULAR DEGENERATION
Possible immediate applicability Leading cause of blindness and vision loss for
elderly people
Advantages Eye is a small organ Small number of cells required Wide Applicability Optical Tools Exist to Observe Results Eye is a noncritical organ
Age Percentage Afflicted66-74 10%75-85 30%
Advanced Melanoma
Already in Clinical trials Promising Results Stem Cells used as messengers to modify
patient’s immune system Uses Adult Stem cells Does not require detailed understanding of
how stem cells interact with human tissue. Easier to get approval for Stage 4 patients
ROADBLOCKS/RISKS
Cancer Teratoma
Dangers with iPS cells Oncogene retrovirus
These are not “killer” problems Biggest issue – sheer complexity of human
body High cost of production
Stanford GMP facility – $200M
BUSINESS MODEL
BUSINESS MODEL
CONCLUSIONS
Lots of therapies are possible Some are close: Macular Degeneration (3 years) Some are far off: Neural Diseases (5+ years) Impossible to Tell Peripherals Market
Limited by Regulation, Cost, and Science
Too Early for Private Investors Government Funding is Crucial for Basic
Research and Commercialization When it reaches the Market, initial cost will
be high but decrease over time
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