wps presentation - intro to rm (gab; nov 2010)
DESCRIPTION
An introduction to Regenerative Medicine.TRANSCRIPT
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Introduction to Stem Cells and Regenerative Medicine
Gregory A. BonfiglioProteus Venture Partners
Woodside Priory School (November 20, 2009)
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CONFIDENTIAL 2
Agenda
Introduction to RM - What is Regenerative Medicine? - The Promise of Regenerative Medicine
Stems Cells- What Are They?- What Can They Do?
Tissue Engineering - What Is Tissue Engineering? - What Can Tissue Engineered Products Do?
The Politics of Stem Cells - Controversy Around Human Embryonic Stem Cells - Therapeutic Cloning (SCNT) & Discarded IVF Embryos
Where to Find More Information About RM
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INTRODUCTION
1. Why Can’t We Be Like Salamanders?
2. What is Regenerative Medicine?
3. The Promise of Regenerative Medicine
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A Salamander Can Regrow Its Leg… …Why Can’t We?
CONFIDENTIAL 4
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What is Regenerative Medicine?
CONFIDENTIAL 5
Regenerative Medicine Is Set of Innovative Medical Technologies Based on Stem Cell Biology and Tissue Engineering That Harnesses the Body’s Own Healing Abilities.
RM Technologies Have The Capacity To:
1. Cure Diseases (Instead Of Treating Symptoms);
2. Repair or Regenerate Damaged Tissue; 3. Replace Entire Organs; and 4. Restore Function Lost to Disease or Injury.
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Promise of Regenerative Medicine:Repairing Nerve Damage
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Promise of Regenerative Medicine:Repairing Heart Tissue & Bladders
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CONFIDENTIAL 8
Regenerative Medicine: The Next Phase In Healthcare
1800 1900 2000
Prevention
Control &
Removal
Regenerate &
Regrow• Vaccines
• Bacteriology
• Control Germs
• Diagnostics
• Medical Devices
• Pharmacology
• Surgery
• Cell Therapies
• Tissue Engineering
• Regenerative Compounds
Era Of Regenerative Medicine
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Very Promising Technology
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STEM CELLS
1. What Are They?
− 3 Types of Stem Cells− Sources of Stem Cells
2. What Can They Do? − Therapeutic Applications
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What Is A Stem Cell?
Both Long-Term Self Renewal, and
Differentiate Into Specialized Cells
Stem Cells Have 2 Key Characteristics:
1. Repeated Self Renewal
2. Give Rise To Mature, Specialized Cells
Stem Cell
Mature Cell
Type I
Mature Cell
Type II
Progenitor
Differentiation
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Stem Cell TypeDifferentiation
AbilitySource
TOTIPOTENT(Germ Layer)
Can Form A Complete Organism
Very Early (1-3 days) Embryos
PLURIPOTENT(Embryonic)
Can Form Any Of The 200 Cell Types In The Body
Blastocyst Placental TissueAmniotic FluidiPS Technology
MULTIPOTENT(Adult)
Can Form Only A Limited Number Of Cell Types
Fetal TissueCord BloodBone Marrow & Fat
Stem Cells: 3 Broad Categories Based On Their Ability To Differentiate
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Embryonic Stem Cells (hESCs): Derived From the ICM of the Blastocyst
Remove ICM
Feeder Layer Supports The
Cells
hES Cell LineEstablished
Blastocyst
Inner Cell Mass
Cells Replicate
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Pluripotent (Embryonic) Stem Cells: Differentiate Into All 200 Human Cell Types
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hESCs Are Very Powerful, But They Can Form… Teratomas (Benign Tumor)
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CONFIDENTIAL 16
Mulitpotent (Adult) Stem Cells:Differentiate Into 3 Major Tissue Types
Nerve
Muscle
Blood
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CONFIDENTIAL 17
Adult Stem Cells:Derived From Adipose Tissue (Fat) + Bone Marrow
Liposuction
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CONFIDENTIAL 18
iPS Stem Cells: Pluripotent Cells Created From a Patient’s Skin Cells
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CONFIDENTIAL 19
What Can Stem Cells Do?Restoring Cardiac Function
Current SoC: Surgical Intervention, Medications. Only cure is a heart transplant.
RM Approach: Use of Cell Therapy to Regenerate or Restore Damaged Cardiac Tissue
Other Near Term Indications: Vision Disorders, Diabetes, Spinal Cord Injury
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What Can Stem Cells Do? Restoring Vision
CONFIDENTIAL 20
Fabricating anArtificial Cornea
Curing AMD with RPEsTransplant of Corneal Stem Cells
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CONFIDENTIAL 21
Cell Therapy Using ECMs To Regrow A Severed Finger
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Baseline 12 week
Cell “Therapy”Using ECMs to Regrow Hair
ECMs Produced 20-40% New Hair
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TISSUE ENGINEERING
1. What IS Tissue Engineering?
2. What Can Tissue Engineered Products Do?
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CONFIDENTIAL 24
Combining Human Cells With BiocompatibleScaffolds To Generate Functional Organs Or Tissues
Tissue Engineering: Cells + Scaffolds
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Engineered Bladder: Entire Organ Grown In Lab
Total Cystectomy Ureteral and Urethral Omental/Peritoneal Anastomosis Coverage
Surgically Implant The Neo-Bladder
Neo-Bladder Design• Autologous Bladder Cells
Seeded On Biodegradable Scaffold
• Grown in Lab for 8-10 Weeks
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Surgeon sends patient’s biopsy to Tengion.
Surgeon implants the neo-organ which regenerates and becomes functional.
Autologous Neo-organ DevelopmentAutologous Neo-organ Development
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Engineered Skin For Diabetic Ulcers
27
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Week 0
Week 5
Week 1
Week 8
Week 3
Week 10
5563i
Engineered Skin for Diabetic Foot Ulcers
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Clinical Results: Partial-Thickness Burns
Day 0 Day 6 6 Months After Burn
6138F
Using Engineered Skin: Burn Therapy
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30
New Heart Grown From Adult Stem Cells
University of Minnesota (2008)
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Growing A Windpipe From Stem Cells
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Tissue Engineered Articular Cartilage
Tissue-engineered Cartilage For Implantation
Bioreactor For Producing Cartilage
4523
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Articular Cartilage Surgical Implantation
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Potential Engineered Cartilage Products
Pre-formedCartilage
7128E
Cartilage / Anchor Cartilage /
Bone
Shape-to-FitCartilage
Cartilage
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Tissue Engineered Ear Reconstruction
7463 (Vacanti et. al., 1992)
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6559
TE Humor
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The Politics of Stem Cells
1. Controversy Around Human Embryonic Stem Cells
2. Therapeutic Cloning (SCNT) & Discarded IVF Embryos
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Research is Controversial
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Controversy Resulted In Limited US Funding….
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…. And Very Strong Feelings About The Issue
CONFIDENTIAL 40
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The Political Cycle of The Controversy
CONFIDENTIAL 41
1998 2001 2005 2008
Time
hESCs Created
Bush BanImposed
CIRM CreatedhESC Ban Lifted
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What Is The Controversy About?
1st: The Creation of hESCs Involves The Destruction of Embryos
CONFIDENTIAL 42
• Process Begins With A Human Egg From IVF Clinic or Donor
• Egg Matures To the Blastocyst Stage (8 Days)
• Inner Cell Mass Is Removed
• hESCs Created From The ICM
• Blastocyst Is Discarded
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CONFIDENTIAL 43
Blastocyst on Tip Of Needle
What Is The Controversy About?
There Are Over 400,000 Frozen Embryos In Storage…
…Most Will Be Destroyed
IVF Embryos
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What Is The Controversy About?
2nd: Nuclear Transfer Can Be Used to Clone Mammals
CONFIDENTIAL 44
Nuclear Transfer
• Process Begins With An Egg
• Nucleus (Genetic Material) Is Removed
• Nucleus From Donor is Inserted
• Egg Is Implanted and Grows To Maturity
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Nuclear Transfer: Removing The Nucleus
CONFIDENTIAL 45
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Nuclear Transfer: Inserting New Genetic Material
CONFIDENTIAL 46
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Result : Dolly The Sheep - 1st Cloned Mammal
Process Has Been Extended To Sheep, Cows, Goats, Pigs, Mice
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Could A Human Being Be Cloned?
CONFIDENTIAL 48
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How Does This Impact Regenerative Medicine?
CONFIDENTIAL 49
Nuclear Transfer Can Create Patient Specific Cell Lines
Would Eliminate The Need For Immune Suppression Drugs
And Cure Many Diseases
Therapeutic Cloning
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The Debate Continues…..
CONFIDENTIAL 50
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Cells Are Beautiful Things…
CONFIDENTIAL 51
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…Whatever Your Politics
CONFIDENTIAL 52
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Where to Find More Information About RM
1. National Institute of Health 2. Professional Organizations: ISSCR, ISCT, TERMIS3. Disease Advocacy Groups: JDRF, Michael J. Fox4. Websites
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Others:PenisTestisUterus
Ureter
Valve
Cartilage
Muscle
Trachea
Nerve
Bone
Kidney (epo prod’n and urine)
Bladder (multiple indications)
Vessels (AV shunt, bypass grafts)
Skin
Liver
Breast
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