Stem Cell Technologies World Market Outlook 2015 2025

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Contents

1. Report Overview

1.1 Global Stem Cell Technologies and Applications Market Overview

1.2 Global Stem Cell Technologies and Applications Market Segmentation

1.3 Why You Should Read This Report

1.4 How This Report Delivers

1.5 Key Questions Answered by This Analytical Report

1.6 Who is This Report For?

1.7 Methodology

1.8 Frequently Asked Questions (FAQ)

1.9 Associated Visiongain Reports

1.10 About Visiongain

2. Introduction to Stem Cell Technologies and Applications

2.1 What are Stem Cells?

2.2 Timeline of Stem Cell Research

2.2.1 Embryonic Stem Cells (ESCs) Enter the Picture

2.3 Classifying Stem Cells by Potency

2.3.1 Stem Cell Categories: ESCs, SSCs, IPSCs

2.3.2 Parthenogenetic Stem Cells: Ethical ESC-Like Cells?

2.3.3 Autologous versus Allogeneic Stem Cells: Towards Universal Stem Cell Products?

2.3.4 Advantages and Disadvantages of Different Cell Types

2.4 Regulation of Stem Cell Research

2.4.1 Regulation in the US

1. Report Overview

2. Introduction to Stem Cell Technologies and Applications

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Contents

2.4.1.1 Understanding ‘Homologous Use’

2.4.1.2 Embryonic Stem Cell Research Remains Controversial

2.4.1.3 FDA v Regenerative Sciences: Regulating MSC Therapy

2.4.2 Regulation in Europe

2.4.2.1 The Brüstle Ruling: Dead and Buried?

2.4.2.2 The UK Leads in European Stem Cell R&D

2.4.3 Regulation in Japan

2.4.4 Regulation in Other Regions

2.4.4.1 South Korea Paves a Path for MSC Therapies

2.4.4.2 Liberal Chinese Laws for Stem Cell Research

2.4.4.3 Israel: A Strong Presence in Stem Cell Research

2.4.4.4 India: An Emerging Stem Cell Industry

2.5 Phases of Clinical Trials

2.6 Scope of this Report

2.7 Currency Exchange Rates in This Report

3. Stem Cell Technologies and Applications: World Market 2015-2025

3.1 The World Stem Cell Technologies and Applications Market in 2014

3.2 World Stem Cell Technologies and Applications Market: Regional Breakdown, 2014

3.3 Stem Cell Technologies and Applications Market Segmentation, 2014

3.4 Stem Cell Technologies and Applications Market: Sales Forecast 2015-2025

3.5 Stem Cell Technologies and Applications Market: Sales Forecast by Segment, 2015-2025

3.6 How Will Segmental Market Shares Change to 2025?

3. Stem Cell Technologies and Applications: World Market 2015-

2025

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Contents

3.7 Non-Therapeutic Applications Lead the Stem Cell Technologies and Applications Market

3.8 Stem Cell Technologies and Applications Market: Drivers and Restraints 2015-2025

3.9 Recent Developments

3.9.1 STAP Cell Scandal in Japan

3.9.2 Tracheal Transplant Patient Dies

3.9.3 Stem Cell Rush in Japan after Regulatory Changes?

4. Stem Cell Cancer Therapeutics Segment 2015-2025

4.1 Stem Cell Cancer Therapeutics: Overview

4.2 The Stem Cell Cancer Therapeutics Market in 2014

4.3 Stem Cell Cancer Therapeutics: Market Forecast 2015-2025

4.4 Haematopoietic Stem Cell Transplantation (HSCT)

4.4.1 An Established Treatment for Haematological Cancers

4.4.2 Autologous HSCT Continues to Dominate

4.4.3 Allogeneic HSCT: More Rejections, Fewer Relapses

4.4.4 GvHD: The Major Issue for HSCT

4.4.5 Peripheral Blood: More Anti-Tumour Effect, More GvHD Risk

4.4.6 The Rise of Umbilical Cord Blood

4.4.7 80,000 HSCT Operations Performed Worldwide in 2014

4.4.8 HSCT Costly, Medicare Coverage Selective

4.4.9 Haematological Malignancies are the Major Indication for HSCT

4.4.10 HSCT Addresses Cancers Representing a $20bn Market

4.5 From Procedures to Products: Cord Blood Stem Cell Approvals

4.5.1 Hemacord (New York Blood Center)

4. Stem Cell Cancer Therapeutics Segment 2015-2025

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Contents

4.5.2 HPC, Cord Blood (ClinImmune/University of Colorado Cord Blood Bank)

4.5.3 Ducord (Duke University School of Medicine)

4.5.4 Allocord (SSM Cardinal Glennon Children’s Medical Center)

4.5.5 HPC, Cord Blood BLA 125432 (LifeSouth Community Blood Centers)

4.6 Stem Cell Cancer Therapeutics: Pipeline Review 2015-2025

4.6.1 Apceth: Genetically Modified MSCs for Gastrointestinal Cancer

4.6.2 Athersys: MultiStem in Haematological Malignancies

4.6.3 Cellerant Therapeutics: CLT-008 and CLT-009

4.6.4 Cynata Therapeutics: Mass Producing MSCs for GvHD?

4.6.5 Fate Therapeutics: Modified HSCs for Haematological Malignancy

4.6.6 Gamida Cell: Shifting Focus from StemEx to NiCord

4.6.6.1 Survival at 100 Days Improved by StemEx in Latest Trial

4.6.6.2 NiCord Heads Up a Pipeline of Novel Cell Therapies

4.6.6.3 Novartis in Talks to Acquire Gamida Cell

4.6.7 ImmunoCellular Therapeutics: Stem Cells for Cancer Immunotherapy

4.6.8 Mesoblast: Using Mesenchymal Precursors to Enrich Cord Blood

4.6.8.1 Prochymal (remestemcel-L/MSC-100-IV, Mesoblast): Limited Market Access

4.6.8.2 The Development of Prochymal

4.6.8.3 Success in Subsets of GvHD Patients

4.6.8.4 An Important Role to Play in the Future of HSCT

4.6.8.5 Prochymal: Sales Forecast 2015-2025

4.6.8.6 Phase 3 Trial in Haematological Malignancies

4.6.8.7 A Competitor for Gamida Cell?

4.6.9 Novartis Developing Cord Blood Transplant Candidates

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Contents

4.6.9.1 HSC835 Expanding Umbilical Cord Blood

4.6.9.2 Novartis Partners with Regenerex for Cell-Based Therapies

4.7 Other Stem Cell Oncology Prospects

4.7.1 Ancillary Products for the HSCT Setting

4.7.2 Targeting Cancer Stem Cells (CSCs)

4.7.3 Immunovative Therapies: Building on HSCT for a Cellular Anti-Cancer Therapy

4.8 Stem Cell Cancer Therapeutics: Drivers and Restraints 2015-2025

5. Stem Cell Cardiovascular Therapeutics Segment 2015-2025

5.1 Stem Cell Cardiovascular Therapeutics: Overview

5.2 The Stem Cell Cardiovascular Therapeutics Market in 2014

5.3 Stem Cell Cardiovascular Therapeutics: Market Forecast 2015-2025

5.4 Cardiovascular Diseases Dominate the Global Pharmaceutical Market

5.4.1 Cardiovascular and Cerebrovascular Conditions Are the World’s Most Fatal Diseases

5.4.2 AMI, CLI and Stroke: Major Ischaemic Disease Targets

5.5 The State of Stem Cell Research for Cardiovascular Diseases

5.5.1 Can Stem Cells Heal the Heart and Vasculature?

5.5.2 Stem Cell Treatments for Stroke

5.5.3 Defining Cardiovascular Stem Cells

5.6 Safety and Efficacy of MSCs in Clinical Trials

5.6.1 The Mysterious Efficacy of Adult Stem Cells in the Heart

5.6.2 Therapeutic Effects Despite Rapid Clearance

5.6.3 Doubts over the Efficacy of MSCs in the Heart

5.6.4 Looking Beyond MSCs

5. Stem Cell Cardiovascular Therapeutics Segment 2015-2025

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Contents

5.7 Stem Cell Cardiovascular Therapeutics in the Market

5.7.1 Pharmicell: Hearticellgram-AMI, One of the First Approved Cardiovascular Stem Cell

Treatments

5.7.1.1 Hearticellgram-AMI Sales 2012-2013

5.7.1.2 Difficulty Accessing Markets Outside South Korea

5.7.1.3 Hearticellgram-AMI: Sales Forecast 2015-2025

5.7.1.4 Pharmicell: Pipeline Products

5.7.2 Reliance Life Sciences: CardioRel

5.8 Stem Cell Cardiovascular Therapeutics: Pipeline Review 2015-2025

5.8.1 Cytomedix Rebrands as Nuo Therapeutics, Exits Stem Cell R&D

5.8.2 Aastrom Biosciences (Vericel Corporation): Ixmyelocel-T

5.8.2.1 New Focus on Dilated Cardiomyopathy

5.8.3 Apceth: MSCs for Advanced Peripheral Vascular Disease

5.8.4 Arteriocyte: Nanex Technology to Treat CLI

5.8.5 Athersys: MultiStem in Stroke and Acute Myocardial Infarction

5.8.6 Baxter: Phase 3 in Refractory Angina

5.8.7 BioCardia: MSC Therapies for Heart Failure and AMI

5.8.8 Biogenea-Cellgenea: Still Developing Cardiogenea?

5.8.9 Bioheart: Phase 3 in Heart Failure, But Needs Extra Funding

5.8.9.1 Seeking Expanded Access for Compassionate Use

5.8.10 Capricor: Cardiosphere-Derived Cells for Heart Repair

5.8.11 Cardio3 BioSciences: Cardiopoiesis for Heart Regeneration

5.8.11.1 Phase 3 Trial Begins Amid Controversy

5.8.12 CellProthera: Myocardial Tissue Regeneration

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Contents

5.8.13 Cytori Therapeutics: Stem Cells for Acute Myocardial Infarction

5.8.14 Human Stem Cells Institute: Developing Gemacell and Cryocell

5.8.15 Mesoblast: Mesenchymal Precursor Cells for Heart Failure and Myocardial Infarction

5.8.15.1 CEP-41750 Moving to Phase 3 in Congestive Heart Failure

5.8.15.2 Phase 2 in Acute Myocardial Infarction

5.8.15.3 Promising Preclinical Findings in Ischaemic Stroke

5.8.15.4 Teva Collaborating on Cardiovascular Stem Cell Therapy R&D

5.8.16 NeoStem: Diversified Outfit with One of the Leading Candidates

5.8.16.1 NBS10 to Treat Acute Myocardial Infarction

5.8.17 Stemedica Cell Technologies: Ischaemic-Tolerant Stem Cell Platform

5.8.17.1 Phase 2 in Acute Myocardial Infarction, Ischaemic Stroke, Heart Failure and

Cutaneous Photoaging

5.8.18 Stempeutics: Emerging Indian Company Targets CLI

5.8.19 Other Stem Cell Cardiovascular Prospects

5.9 Can Stem Cells Change The Treatment Paradigm for Cardiovascular Disease?

6. Stem Cell Central Nervous System Therapeutics Segment 2015-2025

6.1 Stem Cell Central Nervous System Therapeutics: Overview

6.2 The Stem Cell Central Nervous System Therapeutics Market in 2014

6.3 Stem Cell Central Nervous System Therapeutics: Market Forecast 2015-2025

6.4 The State of Stem Cell Research in CNS Diseases

6.4.1 Stem Cells the Best Hope for Many Serious CNS Conditions

6.4.2 Awaiting a First Breakthrough Approval

6. Stem Cell Central Nervous System Therapeutics Segment 2015-

2025

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Contents

6.4.3 Human NSCs Successfully Isolated

6.4.4 NSCs Reach the Clinic

6.4.5 CNS Disorders are the Major Focus for ESC Research

6.4.6 MSCs: Can Glial Cell and Astrocyte Formation Help Neurological Conditions?

6.5 Progress in Specific CNS Conditions

6.5.1 HSCT in MS: Could ‘Resetting’ the Immune System Treat MS?

6.5.2 ALS: Rare Disease Could be the First Neurodegenerative Condition for Stem Cell

Treatment

6.5.3 Parkinson’s Disease: Moratorium on Stem Cell Research Ends

6.5.4 Dry Age-Related Macular Degeneration: A Major Unmet Need

6.5.5 Several Multi-Billion Dollar Potential Markets in the Sights of CNS Stem Cell Developers

6.6 Stem Cell Central Nervous System Therapeutics: Pipeline Review 2015-2025

6.6.1 Advanced Cell Technology: Stem Cells for Eye Diseases

6.6.1.1 Stargardt’s Macular Degeneration: Moving to Phase 2

6.6.1.2 Early Signals of Efficacy, or Confounded Results?

6.6.1.3 Advancing to Phase 2 in Dry AMD

6.6.2 Athersys: Multistem for Ischaemic Stroke

6.6.3 BrainStorm Cell Therapeutics: Neurotrophic Factor-Releasing Stem Cells for ALS

6.6.3.1 Phase 2 Trial in 48 ALS Patients Underway

6.6.4 Cellonis Biotechnologies: Switching From Diabetes to Stroke

6.6.5 Corestem: MSCs for ALS

6.6.6 International Stem Cell Corporation: Preclinical CNS Treatments

6.6.7 InVivo Therapeutics: Neuro-Spinal Scaffold for Spinal Cord Injury

6.6.8 Mesoblast: Phase 2 in Wet AMD, Preclinical in Neurology

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Contents

6.6.9Neuralstem: Allogeneic Neural Stem Cells for Synaptic Repair and Neuroprotection

6.6.9.1 Long-Term Follow-Up Data is Promising in ALS

6.6.9.2 Chronic and Acute Spinal Cord Injury Treatment to Follow

6.6.9.3 Ischaemic Stroke: Phase 1/2 Trials Underway in China

6.6.10 Preclinical Work in Other Neurological Disorders

6.6.11 NeuroGeneration: Investigating Human NSCs

6.6.12 Pfizer: Collaborating on the London Project to Cure Blindness

6.6.13 Q Therapeutics: Glial-Based Stem Cells as an ALS Treatment

6.6.14 ReNeuron: ReN001 is Demonstrating Potential in Stroke

6.6.14.1 Foetally-Sourced Cells Have Wide Potential Application

6.6.15 RhinoCyte: Stem Cells from the Nasal Passage to the Spinal Cord

6.6.16 SanBio: SB623 for Ischaemic Stroke

6.6.16.1 Future SanBio Programmes at Preclinical Stage in 2014

6.6.17 StemCells: a Leader in Neural Stem Cells

6.6.17.1 HuCNS-SC Product Proving Itself in Rare Indications

6.6.17.2 Moving on to Spinal Cord Injury and AMD

6.6.17.3 Alzheimer’s Disease and Stroke to Follow?

6.7 Other Companies with Technologies of Interest

6.7.1 iPierian – Bringing IPSCs to the CNS Market?

6.8 Stem Cell CNS Therapeutics: Drivers and Restraints 2015-2025

7. Stem Cell Therapeutics in Other Disease Areas 2015-2025

7.1 Stem Cell Therapeutics in Other Disease Areas: Overview

7.2 Stem Cell Therapeutics in Other Disease Areas: The Market in 2014

7. Stem Cell Therapeutics in Other Disease Areas 2015-2025

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Contents

7.3 Stem Cell Therapeutics in Other Disease Areas: Market Forecast 2015-2025

7.4 Stem Cell Therapeutics for Other Diseases in the Market

7.4.1 Osteocel Plus (NuVasive): The Leading Stem-Cell Orthobiologic

7.4.1.1 Osteocel to Osteocel Plus: A Chequered History

7.4.1.2 Better than Autograft?

7.4.1.3 Osteocel Plus: Sales Forecast 2015-2025

7.4.2 Trinity Elite and Evolution (Orthofix): Second-Generation Stem Cell Orthobiologics

7.4.2.1 Trinity Elite and Evolution: Sales Forecast 2015-2025

7.4.3 Cartistem (Medipost): ‘World’s First Allogeneic Stem Cell Drug’

7.4.3.1 Cartistem: Sales Forecast 2015-2025

7.4.4 LiquidGen (Skye Orthobiologics): Liquid Amniotic Allograft

7.4.5 AlloStem (Allosource)

7.4.6 Map3 (RTI Surgical)

7.5 Stem Cell Therapeutics for Other Diseases: Research Areas

7.5.1 HSCT for Orphan Diseases

7.5.2 Genetically Modified Stem Cells for HIV and Other Diseases

7.5.3 Stem Cells Are Osteogenic

7.5.4 Perianal Fistula Treatment Indicates Stem Cells’ Potential in Tissue Repair

7.5.5 Autoimmune Disorders Offer Real Market Potential

7.5.6 A Potential Cure for Diabetes?

7.5.7 Stem Cells in Active Liver Repair

7.5.8 Long-Range Possibilities

7.6 Stem Cell Therapeutics in Other Disease Areas: Pipeline Review 2015-2025

7.6.1 Alliancells Bioscience Corporation: Possible RA Treatment

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Contents

7.6.2 AlloCure: AC607 Phase 2 Study Terminated

7.6.3 American CryoStem: ATCELL Preclinical Programmes

7.6.4 Anterogen: South Korean Stem Cell Company

7.6.5 Athersys: MultiStem Fails Phase 2 in Ulcerative Colitis

7.6.6 BioRestorative Therapies: Exploring Disc Disease and Diabetes

7.6.7 BioTime: Multiple Subsidiaries, Multiple Therapies

7.6.7.1 Asterias Biotherapeutics: Neurology and Oncology

7.6.7.2 Cell Cure Neurosciences: Phase 1/2a in Dry AMD

7.6.7.3 OncoCyte: Cancer Diagnostics

7.6.7.4 OrthoCyte: Orthopaedic Stem Cell Treatments

7.6.7.5 ReCyte: ESCs and IPSCs for Cardiovascular Diseases

7.6.7.6 Other BioTime Subsidiaries

7.6.8 Calimmune: Dual Anti-HIV Gene Therapy via Stem Cells

7.6.9 Celgene Cellular Therapeutics: Targeting Inflammatory Conditions

7.6.10 Cellular Biomedicine Group: MSCs for Knee Osteoarthritis

7.6.11 IntelliCell BioSciences: Adipose-Derived Stem Cells

7.6.12 Life Stem Genetics: Adipose-Derived Stem Cells

7.6.13 Medipost: Four Programmes in Development

7.6.14 Medistem: Acquired by Intrexon

7.6.15 Medistem Panama: Investigating MSCs

7.6.16 Mesoblast: MPCs for Crohn’s Disease, Diabetes and Rheumatoid Arthritis

7.6.16.1 Prochymal in Crohn’s Disease: Phase 3

7.6.16.2 Allogeneic MPCs for Diabetes in Phase 2

7.6.16.3 Allogeneic MPCs for Rheumatoid Arthritis in Phase 2

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Contents

7.6.16.4 Allogeneic MPCs in Intervertebral Disc Repair and Spinal Fusion: Phase 2

7.6.17 Pluristem Therapeutics: Placental Stem Cells for Cardiovascular Diseases

7.6.18 Regeneus: Taking Adipose-Derived MSCs into Clinical Trials

7.6.19 S-Evans Biosciences: Menstrual Stem Cells for Hepatic Disease

7.6.20 TiGenix: Expanded Adipose-Derived Stem Cell Therapies

7.6.20.1 Cx601: Phase 3 Data Expected In July 2015

7.6.20.2 Cx611: Phase 2 in Rheumatoid Arthritis

7.6.20.3 Cx621: Intralymphatic Stem Cells for Autoimmune Diseases

7.6.20.4 ChondroCelect Already on the Market

7.6.21 ViaCyte: Revolutionising Diabetes Treatment with VC-01?

7.6.22 ViroMed: Genetically Modified Stem Cells for Chronic Granulomatous Disease

7.7 Genetically Modified Stem Cell Therapy Pipeline

7.7.1 Bluebird Bio: Gene-Modified HSCs for Orphan Diseases

7.7.2 GSK: Ex-Vivo Stem Cell Gene Therapies for Rare Diseases

7.7.3 Sangamo BioSciences: Genetically Modified Stem Cells for HIV

8. Stem Cell Non-Therapeutic Applications 2015-2025

8.1 Stem Cell Non-Therapeutic Applications: Overview

8.2 Stem Cell Non-Therapeutic Applications Market in 2014

8.3 Stem Cell Non-Therapeutic Applications: Market Forecast 2015-2025

8.4 Stem Cell Banking: Growing Demand Worldwide

8.4.1 Dental Stem Cell Banking: An Alternative to Cord Blood Banks?

8.5 Stem Cell Supply and Processing: IPSCs the New Driver

8.6 Stem Cell-Based Assays: Major Potential for Preclinical Screens

8. Stem Cell Non-Therapeutic Applications 2015-2025

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Contents

8.7 Research, Reagents, and Other Non-Therapeutic Stem Cell Activities

8.8 The Outlook for Non-Therapeutic Stem Cell Activities

9. Qualitative Analysis of the Stem Cell Technologies and Applications

Market 2015-2025

9.1 SWOT Analysis of the Stem Cell Technologies and Applications Market

9.2 Strengths

9.2.1 HSCT is an Established Procedure

9.2.2 Recent Approvals of Stem Cell Therapies

9.2.3 Relaxation of Regulatory Barriers

9.3 Weaknesses

9.3.1 Uncertain Mechanism of Action in Stem Cell Therapies

9.3.2 Few Late-Stage Pipeline Products

9.3.3 Regulatory and Reimbursement Concerns

9.4 Opportunities

9.4.1 Genetic Modification of Stem Cells

9.4.2 Unmet Clinical Needs

9.4.3 Non-Therapeutic Uses: Cord Banking and Cell-Based Assays

9.4.4 Interactions with Related Technologies

9.5 Threats

9.5.1 Financial Risks

9.5.2 Pipeline Failure

9.5.3 Long-Term Safety Concerns

9.6 STEP Analysis of the Stem Cell Technologies and Applications Market

9. Qualitative Analysis of the Stem Cell Technologies and

Applications Market 2015-2025

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Contents

9.7 Social Factors

9.7.1 Increasing Burden of Disease

9.7.2 Biological Insurance through Stem Cell Banking

9.7.3 Stem Cell Tourism: the Hype and the Hope

9.8 Technological Factors

9.8.1 Increasing Research Output

9.8.2 IPSC Breakthroughs

9.8.3 Greater Understanding of Stem Cell Differentiation

9.8.4 Biomaterials, Bioprinting and Other Developments

9.8.5 CRISPRs: A Breakthrough in Genome Editing

9.9 Economic Factors

9.9.1 Grey Market for Stem Cell Therapies

9.9.2 Broad Changes in Pharma/Healthcare Markets

9.9.3 The Business Model Needs Further Development

9.10 Political Factors

9.10.1 Ethical Objections to Embryonic Stem Cell Research

9.10.2 Strong Support from National Governments

9.10.3 Pressure to Deregulate the Stem Cell Therapies Market

10. Research Interviews

10.1 Interview with Mr Brock Reeve, Executive Director, Harvard Stem Cell Institute,

Massachusetts, US

10.1.1 Recent Developments in Stem Cells

10.1.2 Stem Cell Techniques and Therapies in 2015-2025

10. Research Interviews

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Contents

10.1.3 Potential Applications of Genome Editing

10.1.4 Therapeutic Areas for Stem Cell Therapies

10.1.5 The Need for New Business Models?

10.1.6 Developing the Stem Cell Therapy Market

10.1.7 Other Trends and Developments

10.2 Interview with Dr Mark Berman, Founder, Cell Surgical Network, US

10.2.1 Cell Surgical Network: Stem Cell Therapies

10.2.2 Cell Therapy as Investigative Treatment

10.2.3 Promising Therapeutic Applications

10.2.4 Future Developments for CSN and Stem Cell Therapies


10.2.6 Regulatory Issues in Japan and Elsewhere

10.2.7 The FDA and Cell Surgical Network



10.3 Interview with Dr Leonard Zon, Director of the Stem Cell Program at Children's Hospital

Boston, Massachusetts, US

10.3.1 Recent Developments in Stem Cells

10.3.2 Research at Zon Laboratory

10.3.3 Stem Cell Techniques and Therapies in 2015-2025




10.4 Interview with Dr Artur Isaev, General Director, Human Stem Cells Institute, Moscow,

Russia

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Contents

10.4.1 Future Prospects for Cord Blood Stem Cell Banking

10.4.2 Increasing Competition in Stem Cell Banking

10.4.3 Progress with Gemacell and Cryocell

10.4.4 Opportunities in Russia and Other Markets

10.4.5 Challenges for Stem Cell Companies Over 2015-2025



10.5 Interview with Dr Alan Trounson, Emeritus Professor, Monash University, Melbourne,

Australia

10.5.1 Improved Funding in the Stem Cells Field

10.5.2 MSCs: Major Potential or Merely a Placeholder?

10.5.3 CIRM Investment Strategies

10.5.4 The Changing Regulatory Environment Worldwide

10.5.5 Key Trends for the Next Five to Ten Years

10.6 Interview with Dr Andras Nagy, Senior Scientist, Samuel Lunenfeld Research Institute,

Mount Sinai Hospital

10.6.1 Overview of the Current State of the IPSC Field

10.6.2 Genomic Stability and Other Potential Issues for IPSCs

10.6.3 Potential for Treating and Modelling Diseases with IPSCs

10.6.4 Reprogramming Without Transgenes

10.6.5 Possibilities for Directly-Converted Cells

11. Conclusions

11.1 Stem Cell Technologies and Applications: An Emerging Market

11. Conclusions

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Contents

11.2 The Stem Cell Technologies and Applications Market in 2014

11.2.1 Current Leading Stem Cell Technologies and Applications Segments

11.2.2 Leading Regional Markets

11.3 World Stem Cell Technologies and Applications Market Forecast 2015-2025

11.4 Stem Cell Technologies and Applications Late-Stage Pipeline

11.5 The Future of the Stem Cell Technologies and Applications Market

11.5.1 Challenges for the Stem Cells Market

11.5.2 Concluding Remarks

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Stem Cell Technologies and Applications: World Industry and Market Outlook 2015-2025

Aside from cardiomyocytes, the other major cell type in the heart is the stromal fibroblast cells,

which have a structural and supportive role but do not carry out the functional activity associated

with cardiomyocytes. Fibroblasts are similar to MSCs: MSCs are stromal cells first isolated from

the bone marrow, but now identified in many other bodily tissues, including adipose tissue. The

close resemblance between fibroblasts and MSCs suggests that introduction of MSCs into the

heart may help promote the growth of stromal tissue which may have some beneficial cardiac

function. However, there is little evidence that MSCs or other stromal cells can contribute to the

functional cell lineages. Without genetic manipulation to alter their cellular phenotype, MSCs do not

differentiate into cardiomyocytes, though under some circumstances they may display gene

expression associated with these cell types.

5.6 Safety and Efficacy of MSCs in Clinical Trials

Although the existing adult stem cells as they currently stand may not have the potential to fully

treat cardiovascular conditions, some relatively strong signs of efficacy have been noted. Left

ventricular function and cardiac ejection fraction have been improved by intracoronary injections of

stem cells, though sustained cardiac response or myocardial regeneration have yet to be

demonstrated. Regulators focus on safety and efficacy, with mechanism of action an optional

extra.

• MSCs have strong evidence of safety: some studies have indicated that treatment of acute

myocardial infarction with MSCs and G-CSF may increase in-stent re-stenosis rate, but

there is little statistically meaningful evidence for this.

• MSCs have some evidence of efficacy, but not a completely convincing case as yet. This

may be because large-scale trials to date have not reached the effective dose threshold

needed to translate the faint efficacy signals detected into a robust and demonstrable

clinical benefit. If so, the current trials will help to resolve this question. The efficacy of

MSCs is further discussed below.

5.6.1 The Mysterious Efficacy of Adult Stem Cells in the Heart

Early clinical studies so far suggest that both stromal (MSC) and blood (HSC) stem cells may, if

implanted into the heart, improve cardiac function by a mechanism of action not yet clearly

delineated. There are several possibilities, all or none of which may apply in each different case:

• Implanted adult stem cells improve cardiac function by secreting trophic factors or

cytokines at the site of cardiac tissue damage

• Implanted adult stem cells stimulate the action of endogenous adult cardiac stem cells



Table 5.5 Pharmicell: Hearticellgram-AMI Revenue ($m), AGR (%), CAGR (%), 2014-2025

2014 2015 2016 2017 2018 2019 Hearticellgram-AMI ($m) 7 8 9 9 10 10 Annual Growth Rate (%) 20.0 10.0 5.0 5.5 3.2 CAGR (%, 2014-2019) 8.6

2020 2021 2022 2023 2024 2025

Hearticellgram-AMI ($m) 11 11 11 11 11 11 Annual Growth Rate (%) 3.2 2.0 1.0 1.0 1.0 1.0 CAGR (%, 2019-2025) 1.5 CAGR (%, 2014-2025) 4.7

Figure 5.5 Pharmicell: Hearticellgram-AMI Revenue ($m), AGR (%), 2014-2025

5.7.1.4 Pharmicell: Pipeline Products

Pharmicell’s clinical trials indicate that the company is developing stem cell therapies for spinal

cord injury and liver failure caused by alcoholic liver cirrhosis. Livercellgram, a phase 2 stem cell

therapy, involves autologous bone marrow-derived mesenchymal stem cells injected into the

hepatic artery via a catheter. Due to the less stringent regulatory framework in South Korea,

Livercellgram could potentially reach the market soon after results from the current phase 2 trials

0.0

5.0

10.0

15.0

20.0

25.0

0

2

4

6

8

10

12

2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025

AGR

(%)

Sale

s ($

m)

Year Sales ($m) AGR (%)

Source: visiongain 2014




extrapolate findings in phase 2 trials into late-stage pivotal trials due to the lack of repeatable

success in the field.

Table 6.3 Stem Cell CNS Therapeutics: Phase 1 and 1/2 Pipeline, 2014

Company Name Description Indication Phase BioTime (Cell Cure Neurosciences)

OpRegen hESC-derived RPE cells Dry AMD 1/2a

Stemedica Cell Technologies Stemedyne-MSC ischaemic-tolerant

allogeneic MSCs Ischaemic stroke 1/2a

Advanced Cell Technology RPE cell therapy RPE cells derived from

hESCs Myopic macular degeneration 1/2

Cellonis Biotechnologies

BMSCs and EPCs

autologous bone marrow stromal cells and endothelial progenitor cells

Ischaemic stroke 1/2

Cytori Therapeutics

ADRCs via Celution system

autologous MSCs from adipose tissue Glaucoma 1/2

Medipost Neurostem allogeneic cord blood-derived MSCs

Alzheimer's disease 1/2

Neuralstem NSI-566 human spinal cord-derived NSCs Spinal cord injury 1/2

Neuralstem NSI-566 human spinal cord-derived NSCs Ischaemic stroke 1/2

SanBio SB623 modified allogeneic MSCs Ischaemic stroke 1/2

SanBio SB623 modified allogeneic MSCs

Traumatic brain injury 1/2

StemCells HuCNS-SC allogeneic NSCs Dry AMD 1/2 Stempeutics stempeucel allogeneic MSCs Stroke 1/2

BioTime (Asterias Biotherapeutics) AST-OPC1

hESC-derived oligodendrocyte progenitor cells

Spinal cord injury 1

CoreStem HYNR-CS-Allo injection

allogeneic bone marrow-derived MSCs ALS 1

StemCells HuCNS-SC allogeneic NSCs Pelizaeus-Merzbacher disease

1


Stem Cell Technologies World Market Outlook 2015 2025

Business

stem cell rush

stem cell categories

world stem cell technologies

global stem cell technologies

embryonic stem cell

timeline of stem cell

emerging stem cell industry

european stem cell rd