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Page 1: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents
Page 2: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Conference Underwriter

Page 3: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Stem Cell Innovation: The Next-Frontier Economy?

Page 4: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Polling Question:Which Would You Prefer the Federal Government to Do?

1. Place No Restrictions on Government Funding of Stem Cell Research

2. Ease the Current Restrictions to Allow More Stem Cell Research

3. Keep the Current Restrictions

4. Government Should not Fund Stem Cell Research at All

Page 5: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Polling Question:In General, Do You Think that it Is Morally Acceptable or Morally Wrong to Use Human Cloning Technology in Developing New Treatments for Disease?

1. Acceptable

2. Wrong

3. Don’t Know

Page 6: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Polling Question:How Important Do You Think it Is that California is a Global Leader in Medical and Scientific Research?

1. Very Important

2. Somewhat Important

3. Not Important

Page 7: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Polling Question:Where Will the Highest Rate of Return for California's Investment in Stem Cell Research Accrue?

1. Cures or Treatment of Disease for Residents

2. Jobs and Wages Created at Biopharmaceutical Firms in the State

3. State Government Guaranteed Rate of Return

Page 8: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Stem Cell Research

Page 9: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Stem Cell Research Centers

Page 10: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

World Stem Cell Map

Page 11: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

International RegulationsEmbryonic Stem Cell Research

Prohibition UtilisationUtilisation and

Extraction

Extraction and

Creation Austria Germany Australia SingaporeBrazil USA Canada UK

Costa Rica Greece IsraelDenmark Japan ChinaEquador SpainIreland Sweden

Peru The NetherlandsPoland France

Page 12: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Who is Leading the Way?Stem Cell Research

• SOUTH KOREAResearchers refined a process to clone stem cells in human embryos (nuclear transfer, therapeutic cloning).

• CHINAAs in South Korea, China’s scientists benefit from regulatory standards less strict than ours.

• SINGAPOREThe development of stem cell science is a national priority in Singapore.

• ISRAELIsraeli scientists have been leaders in the use of embryonic stem cells to study diabetes, heart disease and cancer.

• UKThe United Kingdom has long been a leader in embryo sciences, creating groundbreaking fertility procedures in the 1970s and cloning Dolly the sheep in 1996.

Page 13: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

TransplantationStem Cell Research

Page 14: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Stem CellsEmbryonic vs. Adult Stem Cells

• Embryonic Stem Cells are taken from the embryo that can form differentiated cells for all tissues in the body. Embryonic stem cells can self renew and be cultured as a cell line in vitro.

• Adult Stem Cells are undifferentiated cells found in a specialized tissue. Adult stem cells have the ability to make a limited range of specialized cell types. Adult stem cells are rare, difficult to isolate and have a limited capacity to divide.

Page 15: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Possible Sources of Stem Cells

Embryonic Stem Cells, Embryonic Germ Cells, Adult Cells

• Embryos created via IVF (for infertility treatment or for research purposes)

• Embryos or fetuses obtained through elective abortion

• Embryos created via SCNT (somatic cell nuclear transfer, or cloning)

• Adult tissues (bone marrow, umbilical core blood)

Page 16: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Five-Year Cancer Survival Rate

1950s-1990s

1990s1980s1970s1960s1950s

65

60

55

50

45

40

35

30

Percent

Page 17: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Economic Value of Eliminating Deaths

2000

U.S. Balance SheetAIDSStrokeCancerHeart Disease

50

40

30

20

10

0

US$ Trillions

Page 18: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Cost of Alzheimer’s Disease CA Community Residents with Alzh. Disease

2040 (est.)2020 (est.)2000

70

60

50

40

30

20

10

US$ Billions

Page 19: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Economic Costs of Major Illnesses

1995-1999

Illness Year Direct Costs Indirect Costs Total Costs

Ratio of indirect

to total costs

Injury 1995 89.0 248.0 337.0 74%Heart Diseases 1999 101.8 81.3 183.1 44%Disability 1986 82.1 87.3 169.4 52%Mental Disorders 1992 66.8 94.0 160.8 58%Cancer 1994 41.4 68.7 110.1 62%Alzheimer's Disease 1997 15.0 85.0 100.0 85%Diabetes 1997 44.1 54.1 98.2 55%Chronic Pain Conditions 1986 45.0 34.0 79.0 43%Arthritis 1992 15.2 49.6 64.8 77%Digestive Diseases 1985 41.5 14.7 56.2 26%Stroke 1998 28.3 15.0 43.3 35%Kidney and Urological Diseases 1985 26.2 14.1 40.3 35%Eye Diseases 1991 22.3 16.1 38.4 42%Pulmonary Diseases 1998 21.6 16.2 37.3 43%HIV / AIDS 1999 13.4 15.5 28.9 54%Other (10 further Illnesses) various 53.4 23.9 77.2 31%Total - 25 Illnesses 707.1 917.5 1624.0 56%

US$ Billions

Page 20: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Therapeutic Applications for Stem Cells

Regenerative Medicine

• Cardiomyocytes for Heart Disease

• Islet Cells for Diabetes

• Neural Cells for Parkinson’s

• Blood Cells for Cancer

• Chondrocytes for Burns

Page 21: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Stem Cells Could Potentially Cure

Stem Cell Research

Diseases Therapy

Cancer By rebuilding tissues destroyed by disease

Heart DiseaseBy replacing ischemic heart tissue with healthycardiac cells and by growing new blood vessels

Osteoporosis By repopulating bones with strong, new cells

Parkinson's Disease By replacing the brain's dopamine-producing cells

DiabetesBy infusing the pancreas with newinsulin-producing islet cells

BlindnessBy replacing the cells of the retina lost to macular degeneration, glaucoma or diabetic retinopathy

Spinal Cord Injury By replacing nerve cells in the spinal cord

Page 22: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Stem Cells Could Potentially Cure ─ Cont.

Stem Cell Research

Diseases Therapy

Kidney DiseaseBy replacing the cells, tissues, or even the kidney itself

Liver Disease By replacing liver cells or the liver itself

Amyotrophic Lateral SclerosisBy generating new nerve tissue throughout the spinal cord and body

Alzheimer's Disease By healing and replacing brain cells

Muscular DystrophyBy replacing muscle tissue and possiblydelivering gene-based cures

Osteoarthritis By helping the body re-grow cartilage

Autoimmune DiseaseBy repopulating the cells of the blood and theimmune system

Lung Disease By growing new lung tissue

Page 23: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Federal Policy on Embryonic Human Stem Cell Research

2001

• Federal funds can only be used on stem cell lines that have been derived before August 9, 2001.

• Lines have to be from unused blastocysts that were made for IVF.

• Originally 64 lines, now 21.

• All lines were made with mouse feeder cells. They have mouse antigens and many of the lines appear to have chromosomal rearrangements.

Page 24: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Funding for Stem Cell Research by NIH

FY 2003-2006 (est.)

2006 (est.)2005 (est.)20042003

700

600

500

400

300

200

100

0

US$ MillionsStem Cell ResearchHuman Embryonic Stem Cell Research

Page 25: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Top 15 Diseases, Conditions,Research Areas by Level of NIH Funding

FY 2003-2006 (est.)

FY 2003 FY 2004 FY 2005 (est.) FY 2006 (est.)Biotechnology 9,893 10,685 10,976 11,043Clinical Research 8,028 8,495 8,712 8,792Prevention 6,546 7,185 7,227 7,375Cancer 5,432 5,547 5,643 5,641Neurosciences 4,711 4,911 5,028 5,055Brain Disorders 4,740 4,821 4,931 4,961Genetics 4,236 4,535 4,620 4,637Women's Health 3,498 3,478 3,525 3,531Pediatric 3,066 3,132 3,195 3,204Infectious Diseases 2,441 3,055 3,102 3,104Behavioral and Social Sciences 2,684 2,932 2,992 2,998Clinical Trials 2,723 2,877 2,946 2,966HIV/AIDS 2,716 2,850 2,921 2,933Health Disparities 2,430 2,590 2,646 2,663Cardiovascular 2,286 2,360 2,409 2,420

NIH FundingUS$ Millions

Disease, Condition, Research Area

Page 26: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Funding for BiomedicalResearch by Source

2000-2004

Source 2000 2001 2002 2003 2004National Institutesof Health 17.1 19.8 22.3 26.4 26.9Other Federal 5.2 6.7 7.6 6.9 6.4State and LocalGovernment 3.5 3.7 4.0 4.3 N/AFoundations, Charities,and Other Private Funds 3.4 2.6 2.6 2.5 N/APharmaceutical Firms 21.4 23.5 25.7 27.0 30.6Biotechnology Firms 14.2 15.7 20.5 17.9 19.8Medical Device Firms 6.3 7.3 8.3 9.2 10.8Total 71.0 79.4 90.9 94.3 94.5Adjusted Total* 78.9 85.1 94.6 94.3

US$, Billions

*Adjuted by the Biomedical Research and Development Price Index

Page 27: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

NIH List of HumanEmbryonic Stem Cell Lines

Eligible for Use in Federal ResearchName

Eligible AvailableBresaGen, Inc., Athens, GA 4 3Cell & Gene Therapy Institute (Pochan CHA University), Seoul, Korea 2 N/ACellaritis AB, Goteborg, Sweden 3 2CyThera, Inc., San Diego, CA 9 0ES Cell International, Melbourne, Australia 6 6Geron Corporation, Menlo Park, CA 7 N/AGoteborg University, Goteborg, Sweden 16 N/AKarolinska Institute, Stockholm, Sweden 6 0Maria Biotech Co. Ltd. - Maria Infertility HospitalMedical Institute, Seoul, Korea 3 N/AMizMedi Hospital - Seoul National University, Seoul, Korea 1 1National Center for Biological Sciences/Tata Institute of Fundamental Research, Bangalore, India 3 N/AReliance Life Sciences, Mumbai, India 7 N/ATechnion University, Haifa, Israel 4 2University of California, San Francisco, CA 2 2Wisconsin Alumni Research Foundation, Madison, WI 5 5Total 78 21

Number of Stem Cell Lines

Page 28: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Investments in U.S. CompaniesCompanies Conducting Embryonic & Adult Stem Cell Research, 2003

Cythera, Inc.StemCells, Inc.GeronViaCell

50

40

30

20

10

0

US$ Millions

Page 29: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

FY 2006 President’s Budget Request

Total NIH Budget Authority: $28,740 Million

Other Research,

6%

Research Centers,

9%

Intramural Research,

11%

R&D Contracts,

11%

Research Project Grants,

56%

Res., Mgmt. & Support,

4%

Research Training,

3%

Page 30: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

History of Stem CellsResearch and Therapeutic Applications

Stem Cell Research

• 1998– First human embryonic stem cells derived and cultured

from a blastocyst– Potential for self renewal and differentiation

recognized

• 1997– Cloning of Dolly by SCNT signaled the potential to

produce embryonic stem cells with defined genetic make-up

Page 31: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Translation of New Technologies into Therapies Takes Time

Stem Cell Research

• Recombinant DNA (1960s;1970s)– Human Genome Sequence (2002)– Human Growth Hormone; Factor VIII (1980s)

• Monoclonal Antibodies (1970s;1980s)– New Cancer Therapies (Herceptin; Avastin) (2002-5)

• Stem Cells– May Aid Drug Discovery

– New Therapy (20??)

Page 32: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Stem Cell Research 2002-03 Bills and Laws

State

Prohibit allHuman Cloning

(incl. Therapeutic)

ProhibitReproductive

Cloning

AllowTherapeutic

CloningSpecifically

BanDestructiveEmbryonicResearch

Allow EmbryonicStem CellResearch

AL X XAR Law LawAZ X XCA X X Law X LawCO X XCT X XDE X XFL X XGAHIIA Law Law LawIDIL X X XIN X X XKS X X XKY X XLA X X Law The Laws that have already been enacted are indicated by the word "Law"The Bills are indicated by an "X"

Page 33: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Stem Cell Research 2002-03 Bills and Laws, Cont’d

State

Prohibit allHuman Cloning

(incl. Therapeutic)

ProhibitReproductive

Cloning

AllowTherapeutic

CloningSpecifically

BanDestructiveEmbryonicResearch

Allow EmbryonicStem CellResearch

MA X X XMD XMEMI Law Law XMNMO X XMSMTNCND Law LawNE X XNH X XNJ X X X XNMNVNY X X X XThe Laws that have already been enacted are indicated by the word "Law"The Bills are indicated by an "X"

Page 34: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Stem Cell Research 2002-03 Bills and Laws, Cont’d

State

Prohibit allHuman Cloning

(incl. Therapeutic)

ProhibitReproductive

Cloning

AllowTherapeutic

CloningSpecifically

BanDestructiveEmbryonicResearch

Allow EmbryonicStem CellResearch

OHOK X XOR X XPA XRI Law XSC X XSDTN X X XTX X X XUTVA Law XVT X XWA X X XWI X X XWV X XWYThe Laws that have already been enacted are indicated by the word "Law"The Bills are indicated by an "X"

Page 35: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Proposition 71 Stem Cell Research and Cures Initiative

• Establishes California Institute for Regenerative Medicine (CIRM) to regulate stem cell research and provide funding.

• Establishes constitutional right to conduct stem cell research; prohibits CIRM’s funding of human reproductive cloning research.

• Provides General Fund loan up to $3 million for CIRM’sinitial administration/implementation costs.

• Authorizes issuance of general obligation bonds to finance CIRM activities up to $3 billion subject to annual limit of $350 million.

Page 36: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Proposition 71 – Total Program Costs and Benefits

Case 1: Limited Therapeutic SuccessPhase 1 Phase 2 Phase 3Years 1-5 Years 6-14 Years 15-35 Total

Economic Costs to State Bugdet 56 1,289 4,010 5,355

Economic Benefits to State Budget1) Tax revenues from Proposition 71 direct spending 73 167 - 2402) Tax revenues from 2.5% increase in life sciences activity 54 355 1,796 2,2063) Cost savings from 1% reduction in state spending - 382 3,062 3,4444) Royalty revenues using 2% royalty rate - 10 527 537

Total 127 914 5,385 6,426% of Total Costs 227% 71% 134% 120%

Additional Benefits to Californians Not Incl. in State Budget

Health care cost savings from 1% cost reductions - 1,136 8,043 9,180% of Total Costs 0% 88% 201% 171%

Estimated Jobs Created (One Job for One Year = One Job Year)

Job years from Proposition 71 direct spending 14,272 33,209 - 47,480Job years from increase in life sciences activity 11,967 67,732 233,148 312,847

Total 26,239 100,940 233,148 360,328

US$ Millions

Page 37: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Proposition 71 – Total Program Costs and Benefits

Case 2: Increased Therapeutic SuccessPhase 1 Phase 2 Phase 3

Years 1-5 Years 6-14 Years 15-35 Total

Economic Costs to State Bugdet 56 1,289 4,010 5,355

Economic Benefits to State Budget1) Tax revenues from Proposition 71 direct spending 73 167 - 2402) Tax revenues from 5.0% increase in life sciences activity 108 711 3,592 4,4113) Cost savings from 2% reduction in state spending - 764 6,123 6,8874) Royalty revenues using 4% royalty rate - 19 1,054 1,073

Total 181 1,662 10,769 12,612% of Total Costs 324% 129% 269% 236%

Additional Benefits to Californians Not Incl. in State Budget

Health care cost savings from 2% cost reductions - 2,273 16,087 18,359% of Total Costs 0% 176% 401% 343%

Estimated Jobs Created (One Job for One Year = One Job Year)

Job years from Proposition 71 direct spending 14,272 33,209 47,480Job years from increase in life sciences activity 23,934 135,464 466,296 625,695

Total 38,206 168,672 466,296 673,175

US$ Millions

Page 38: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Proposition 71 – Total Program Costs and Benefits

Case 3: Expanded Therapeutic SuccessPhase 1 Phase 2 Phase 3Years 1-5 Years 6-14 Years 15-35 Total

Economic Costs to State Bugdet 56 1,289 4,010 5,355

Economic Benefits to State Budget1) Tax revenues from Proposition 71 direct spending 73 167 - 2402) Tax revenues from 5.0% increase in life sciences activity 108 711 3,592 4,4113) Cost savings from 10% reduction in state spending - 3,821 30,616 34,4374) Royalty revenues using 4% royalty rate - 19 1,054 1,047

Total 181 4,718 35,262 40,161% of Total Costs 324% 366% 879% 750%

Additional Benefits to Californians not incl. in State Budget

Health care cost savings from 10% cost reductions - 11,364 80,434 91,797% of Total Costs 0% 882% 2006% 1714%

Estimated Jobs Created (One job for one year = one job year)

Job years from Proposition 71 direct spending 14,272 33,209 47,480Job years from increase in life sciences activity 23,934 135,464 466,296 625,695

Total 38,206 168,672 466,296 673,175

US$ Millions

Page 39: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

CA ─ Stem Cell Research and Cures Initiative

Fiscal Fact Sheet

• Funds Life-Saving Research

• Generates New Revenue and Jobs for the State– California will benefit from Patents and Royalties– Construction of Research Facilities and new Jobs– Sales and Income Taxes generated from Bond Proceeds

are projected to reach $70 million in first 5 years

• Reduce Healthcare Costs by Billions– Californians spend more than $110 billion annually on

Healthcare Expenses

Page 40: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Korean Stem Cell Research

Fact Sheet

• Derived 11 new lines, including lines from males, females. Furthermore, the new lines include patients with spinal cord injury, inherited blood disease and diabetes.

• Egg donation from one woman sufficient to derive a line.

• Conclusive demonstration that the cell line was genetically identical to the nuclear donor.

Page 41: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

European Stem CellResearch

Organizations

• EuroStemCell (European Consortium for Stem Cell Research)

• Cambridge (UK) Stem Cell Institute• Institute for Stem Cell Research, University of

Edinburgh, Scotland• Lund Stem Cell Center (Sweden)• Karolinska Institute (Sweden)• Danish Stem Cell Research Center (Denmark)• National Center for Stem Cell Research (Norway)

Page 42: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Regulations in SelectedEU Member States

Human Embryonic Stem Cell (hES) Research

Country

Allowing procurement of hES cells from supernumary embryos by law

Prohibiting procurement of hES cellsfrom human embryos but allowing importation of hES cell lines

Prohibiting procurement of hES cells from human embryo

No specific legislation regarding human embryo research

Prohibiting creation of human embryo for research purpose and for procurement of hES cells by law

Austria X X Belgium X Germany X X Denmark X X Greece X X Spain X X Finland X X France X X Ireland X X Italy X X Netherlands X X Poland X Portugal X X Sweden X United Kingdom X

Page 43: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Somatic Cell Nuclear Transfer

Dolly the Sheep

Page 44: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Human Embryonic Stem CellsSelf-Renewing Source for the Scalable Manufacturing of Replacement Cells for Every Tissue in the Body

Neural Cells

Spinal CordInjury

Parkinson’sDisease

Cardiomyocytes

Heart Failure

Islets

Osteoblasts

OsteoporosisAnd BoneFractures

Blastocyst

Human Embryonic Stem Cells

HematopoieticCells

Bone MarrowTransplant

Chondrocytes

Hepatocytes

Drug Discovery

Arthritis

Diabetes

Page 45: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Take Home Message of CCST’s Interim IP Report

CA has taken a bold step CIRM-funded research will benefit state in significant ways

But, expectations of short-term revenue stream and quick availability of new treatments overstated

Near-term benefits:Magnet effect for talent to come to CAEnhanced business activity to support research

Long-term benefits:Creation of new high-tech jobsStimulation of the economy and tax revenues

Will result in new products and therapeutic treatments--but will take time

Overriding IP consideration for grantees should be to move technology from research to other entities as effectively as possible for the public

benefit

Page 46: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Expectations

Taxpayers’ return on investment is one issue that figured prominently in literature used to promote Prop 71.

We find that these proposals, and the statements and studies on which they were based, have unrealistic assumptionsabout the potential economic impact of CIRM’s research program.

All interested parties must be mindful of overestimating both the projected revenue stream from IP generated by CIRM-funded grants and the timeline to achieve it.

Page 47: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Examples of Unrealistic Assumptions

• Therapies will use many patents from many sourcesRoyalties from CIRM research alone small

• US universities receive about $1B from licensing for $40B investment

• Only 1 in 400 inventions generate more than $1M over lifetime

• CIRM’s $300M may generate $10M

• Companies pay for commercialization and will not partner if many restrictions

Page 48: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Expectations – Cont.

• While expectations of a short-term revenue stream and quick availability to the public of new treatments overstated, CIRM-funded research can be expected to benefit the state in significant ways, particularly in the long term.– Improved quality of life for those who suffer from

debilitating diseases– Attractiveness of CA as a location for biotech

companies– Development of research tools that will drive research

forward

Page 49: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Context of CA Stem Cell Funding

• CIRM funding– $300 M per year for 10 years

• NIH Biomedical research budget $30 B

• Federal funding of research in higher education, private research institutes, and national labs in California– $14 B per year

• Industry funded R&D in California– $45 B per year

Page 50: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Expenditures on Biological and Biomedical Research at Largest CA Universities, 2003

Institution Expenditures(millions)

UCSF $643

UCLA $607

Stanford $333

UCSD $330

UCD $248USC >$201

Total >$2.3 Billion

Page 51: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

State Funded R&D

Total state funded R&D approximately $300 M/year Major state programs:

PIER and Natural Gas Research $74.5 M/yearHIV-AIDS $9.2 M/yearBreast Cancer $14.9 M/yearTobacco-related Disease $14 M/yearSustainable Agriculture Research $7.5 M/yearHealth & Human Services $5.2 M/yearChild & Family Trust $17.9 M/yearGeothermal Resources Development $4.6 M/year

TOTAL $148 M/year

In addition, California Institutes for Science and Innovation $920M over five years

Page 52: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Current Stem Cell Research Centers in California

• The Burnham Institute• The Salk Institute• The Scripps Research Institute• Stanford University• UC Berkeley• UC Davis – Center for Regenerative Science and Therapies• UC Irvine – The Reeve-Irvine Research Center• UCLA Institute for Stem Cell Biology and Medicine• UC San Diego• UC San Francisco• UC Irvine – The Reeve-Irvine Research Center

Page 53: Conference Underwriter · Poland France. Who is Leading the Way? ... • Embryos created via IVF (for infertility treatment ... Cost of Alzheimer’s Disease CA Community Residents

Models for IP Policy

• Bayh-Dole Patent and Trademark Amendments Act of 1980 rationalized and simplified the process of moving technologies generated by federally funded research from university laboratories to the private sector

• Difficult to measure direct effect of Bayh-Dole on tech transfer, but is generally considered to have contributed positively to development of some technologies

• In many ways, situation in California today regarding state-funded research resembles federal situation prior to passage of Bayh-Dole

• Federal policy takes precedence over state policy

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Biomedical R&D Long and Costly

• Average time to development of a therapy from basic research: 10 years• Vast majority of inventions don’t lead to therapies

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War on CancerCase Study: Development of Rituxan by Biogen Idec

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Recommendations:CIRM IP Policy Objectives (1)

• Support open dissemination of research results and transfer of knowledge

• Ensure that discoveries & research tools are made broadly available

• To the extent possible, preserve ability for grantees to leverage non-CIRM funds in stem-cell related research

• Encourage practical application of CIRM-funded research results for broad public benefit

• Accelerate translation of discoveries from research to commercially available diagnostics and treatments

• To extent possible, balance existing investments with state investments so each receives appropriate return

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Recommendations:CIRM IP Policy Objectives (2)

• Promote collaboration between commercial entities and non-profit institutions

• Encourage private investors to invest in further research and development of new technologies resulting from CIRM-funded research

• Minimize costs of administering policies

• Be mindful of time delay and private investment needed before significant benefits accrue to the state

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Return on Investment (1)

• Ownership of intellectual property central issue to any IP policy• Under Bayh-Dole, ownership resides with grantee

• In considering IP ownership, important for CIRM to consider:• Relative importance of a policy consistent with Bayh-Dole statute

• Who is best able to manage resulting IP

• Existing financial models in place to make appropriate investments in protecting early-stage results

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Return on Investment (2)

• Defining return on investment complicated

• Revenues often modest and years in coming

• Various options for distribution of revenues

• State must balance desire for financial return on investment with effectiveness of CIRM-funded research; some federal agencies, such as the National Institutes of Health, have noted decreased effectiveness of programs when focus on financial returns to funder

• Whatever California chooses to do, must realize that conditions of CIRM grants need to be competitive

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General Principles for State IP Policies

• CIRM recommendations are in line with general principles we are likely to recommend for state-funded research in final report, which include:

• Consistency with the Bayh-Dole Act• Creating incentives for commerce in California from state-

funded research to the greatest extent possible• Encouraging timely publication of results• Diligent development of IP into products that benefit the public

• CIRM may want to adopt interim policies guided by this interim report and final policies after review of final report to state in December 2005

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Recommendations for CIRM IP Policies (1)

1. Permit grantees to own IP rights from CIRM-funded research.

2. Require that grantees (individuals, institutions, or both) provide a plan describing how IP will be managed for the advancement of science and California public benefit.

3. Grant basic research funds without requiring that grantees commit to providing a revenue stream to the state. If, however, a revenue stream develops over time, revenues will be reinvested in research and education.

4. Generally, make CIRM-developed research tools widely available to other researchers.

5. Require diligent efforts to develop CIRM-funded IP into therapeutics and diagnostics that can benefit the public.

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Recommendations for CIRM IP Policies (2)

6. Retain within CIRM Bayh-Dole-like rights to step in if the owner of IP is not undertaking appropriate steps to transfer technology to benefit the public.

7. Leave license particulars to the owner who is in the best position to judge how best to ensure that discoveries are made widely available through commercialization or otherwise.

8. Reserve the right to use IP by or on behalf of CIRM.

9. Establish and maintain a CIRM database to track all IP generatedthrough CIRM funding.

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CA’s Research Machine PowerfulUniversity of California Research Centers: Biotechnology (n=138)

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Industry Clusters AroundResearch Centers

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Wealth Created in Silicon Valley By Stanford Alums Is Staggering

• Of the 150 largest* publicly traded companies in the Valley, 37 Stanford alumni-founded companies (25% of total) represent

– $453 billion in market value (46% of total)**

– $138 billion in revenue (47% of total)

Read-Rite* Ranked by 2003 revenues

** As of March 31, 2004