Stem Cell-Based Therapeutic Delivery
Challenges & Opportunities
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| “Stem cell-derived therapeutics are on the brink of a new era, with a handful of therapies expected on the market within the next 2 or 3 years. Big pharma and biotech companies such as Pfizer and Genyzme have recently invested large sums in this exciting and fast-moving area, further demonstrating its growing maturity. The results of numerous Phase 1/2 clinical trials that are underway now will provide a clearer picture of the potential of stem cell-derived therapies for the future.” Dr Sara Sleigh The pace of stem cell research is astonishing. It is less than 30 years since the discovery of the embryonic stem cell, and less than a decade since scientists determined the right conditions under which human embryonic stem cells (hESCs)could be directed to differentiate into a variety of cell types. Within the last few years, companies have built up sufficient data to support applications to start human clinical trials based on hESCs that have differentiated into oligodendrocytes for the treatment of spinal cord injury. Embryonic stem cells are just part of the stem cell story. Adult stem cells, and those from other sources such as umbilical cord blood, have also been the subject of intense research over the past few years. While there has been a precedent for the use of bone marrow stem cells in the treatment of hematological malignancies, the development of new therapies based on cultured, expanded populations of specific cell types opens up a vista of new possibilities. Regenerative medicine has a wide definition covering therapies that aim to repair, replace, restore and regenerate damaged or diseased cells, tissues and organs. Innovative therapies derived from stem cells fall within this definition and will, in the coming years, provide options for the treatment of a variety of diseases including: autoimmune diseases such as diabetes, Crohn’s disease and osteoarthritis, CNS disorders and neurodegenerative diseases, heart diseases, as well as eye diseases. Stem cell therapies are already offered at various locations across the globe, including China and Thailand, despite a lack of robust, randomized clinical trials demonstrating their safety and efficacy. The fact that stem cell therapies are available in these locations and that patients’ travel across the globe to receive them illustrates the high unmet medical need associated with some of the targets being investigated. Clinical evidence from high quality clinical trials is now imperative in order to protect these vulnerable patient populations. The challenges facing companies developing stem cell-derived therapies are high and range from ethical discussions surrounding the use of hESCs to difficulties associated with manufacture of cell-based products at scale and proving that products lack the potential to cause an immune response or cancer in a recipient. Nevertheless a growing pool of companies is investing heavily to bring this rapidly moving science to the clinic. |
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| Keywords: allogeneic, adipose tissue, ALS, autologous, cardiovascular disease, cancer, CNS, COPD, Crohn's disease, diabetes, embryonic stem cell, GvHD, hESC, hematological malignancies, iPS cells, Parkinson's | |
 
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By Dr Sara Sleigh / Publication Date: 15th December 2008
Contents:
Key Findings 10
Stem Cells & Cell-Based Delivery 10
The Market for Stem Cell-Based Therapies10
Stem Cell-Based Therapies for Cardiovascular Disease 11
Stem Cell-Based Therapies for CNS Disorders 11
Stem Cell Therapies for Diabetes 12
Stem Cell Therapies for Cancer 13
Stem Cell-Based Therapeutics: Challenges & Opportunities 13
Future of Stem Cell-Based Therapeutics & Market Trends 14
Chapter 1 Stem Cells & Cell-Based Delivery 16
Key Points 16
Introduction 17
An introduction to stem cell science 19
Embryonic stem cells 19
Adult stem cells 20
Creating pluripotent cells from adult tissue 21
Nuclear transfer/therapeutic cloning 22
Combining embryonic stem cell therapy with gene therapy 24
Stem cell therapy – a discovery timeline 25
Major scientific challenges for the development of stem cell therapies 27
Report structure 30
Chapter 2 Stem Cell-Based Therapies: Pipeline Analysis 33
Key Points 33
Key therapy areas covered by stem cell-based therapies 34
Product pipelines 34
Stem cells for drug discovery 39
Stem cell culture, expansion and manufacturing 40
Stem cell companies analysed in this report 40
Chapter 3 Stem Cell-Based Therapies for Cardiovascular Disease 43
Key Points 43
Introduction 44
Autologous cell platforms 49
Case Study: MyoCell (Bioheart Inc) 50
Case Study: Myoblast (Advanced Cell Technology) 54
Case study: CardioCure (Gamida Cell Ltd) 56
Case Study: C-Cure (Cardio3 BioSciences) 57
Case Study: Celution System (Cytori Therapeutics) 59
Allogeneic cell platforms 61
Mesenchymal stem cell therapies 62
Case Study: Prochymal (Osiris Therapeutics) 62
Case study: Revascor? (Angioblast Systems Inc) 65
Case study: PLX-PAD (Pluristem Therapeutics Inc) 67
Human embryonic stem cell therapies 69
Case Study: ACT Cellerate (Advanced Cell Technology) 69
Cell delivery technologies 72
Our opinion on stem cell-derived therapies for CV diseases 75
Where the technology is now, its evolution, achievements and pitfalls 75
Competition 75
Potential future applications 76
Activity in the market 76
Major players 77
Winners 77
Chapter 4 Stem Cell-Based Therapies for CNS Diseases 80
Points 80
Introduction 81
Autologous cell platforms 85
Case Study: NurOwn (BrainStorm Cell Therapeutics Inc) 86
Case study: NeuroGeneration 87
Allogeneic cell platforms 88
Stem cells from donated tissue 89
Case Study: HuCNS-SC® (StemCells Inc) 89
Case Study: ReNeuron 91
Case Study: Neuralstem 94
Case study: Q-cells (Q Therapeutics Inc) 95
Case study: StemedicaMCT (Stemedica USA and Stemedica International) 96
Stem cells from umbilical cord blood 97
Case study: Beike Biotechnology 97
Human embryonic stem cells 98
Case Study: GRNOPC1 (Geron) 98
Case study: hESCs from California Stem Cell Inc 101
Cell delivery technologies 102
Our opinion on stem cell-based therapies for CNS diseases 104
Where the technology is now, its evolution, achievements and pitfalls 104
Competition 104
Potential future applications 104
Activity in the market 105
Chapter 5 Stem Cell-Based Therapies for Autoimmune, Inflammatory & Other conditions 107
Key Points 107
Introduction 108
Crohn’s disease 112
Case study: Prochymal for Crohn’s disease (Osiris) 112
Case Study: Ontaril (Cellerix) 113
Diabetes 115
Autologous stem cell transplants for diabetes 116
Allogeneic stem cell transplants for diabetes 117
Osteoarthritis 121
Lung diseases 122
Chronic obstructive pulmonary disease 122
Pulmonary arterial hypertension 122
Our opinion on stem cell-based therapies for autoimmune and other diseases 124
Where the technology is now, its evolution, achievements and pitfalls 124
Competition 124
Potential future applications 124
Major players and winners 125
Chapter 6 Stem Cell-Based Therapies for Graft versus Host Disease, Hematological Malignancies and Cancer 127
Key Points 127
Introduction 128
Cancer stem cells 128
Stem cells as a delivery vehicle 129
Graft versus host disease 132
Expanded umbilical cord blood transplants for malignant diseases 135
Case Study: StemEx (Gamida Cell) 137
Stem cells as delivery systems 139
Lung cancer 140
Metastatic cancer and intracranial tumors 140
Hematological malignancy 142
Multi-lineage progenitor cells (BioE) 142
Conclusion 143
Our opinion on stem cell-derived therapies for GvHD, hematological malignancies and cancer 144
Where the technology is now, its evolution, achievements and pitfalls 144
Competition 144
Potential future applications 145
Activity in the market, major players and winners 145
Chapter 7 Challenges & Opportunities for Stem Cell-Based Therapies 147
Key Points 147
Introduction 148
Ethical issues for stem cell research 149
Funding stem cell research 150
Intellectual property 155
Manufacturing stem cell-based products 156
Regulation 157
Reimbursement 159
Delivering stem cell-based therapies and tissue engineering 159
Challenges 159
Opportunities 160
Chapter 8 Market size, Evolution & Prospects 162
Key Points 162
Key stem cell therapy companies 163
Recent deals and alliances 168
The global stem cell market 2008-2014 170
Analysis parameters 170
Market forecast 2008-2014 175
Global stem cell-based therapy market in 2020 176
Drivers and market trends 176
Summary & Conclusions 178
Acknowledgements 180
Bibliography & Endnotes 181
List of Tables:
Table 1: Pros and cons of different stem cell types 29
Table 2: Diseases targeted by stem cell-based therapeutics 37
Table 3: Stem cell therapy companies discussed in this report 41
Table 4: Leading companies evaluating stem cell-based CV therapies 46
Table 5: Leading academic institutions researching the use of stem cells to treat CV diseases 48
Table 6: Leading companies evaluating stem cell-based therapies for CNS diseases 83
Table 7: Leading institutes evaluating stem cell-based therapies for CNS diseases 85
Table 8: Geron’s stem cell-based product pipeline 99
Table 9: Leading companies evaluating stem cell therapies for autoimmune and other diseases 109
Table10: Leading academic institutes evaluating stem cell therapies for autoimmune and other diseases 111
Table 11: Leading companies evaluating stem cell therapies for GvHD and hematological malignancies 130
Table 12: Academic institutions with an interest in GvHD, hematological malignancies and cancer 131
Table 13: Other clinical trials of mesenchymal stem cells for the prevention or treatment of GVHD 133
Table 14: Results of Phase 1/2 trial for StemEx 137
Table 15: Global positions on research on embryonic stem cells derived from IVF procedures 150
Table 16: Current funds available from individual US states for embryonic and adult stem cell research 153
Table 17: Summary of leading stem cell-based therapeutics companies 163
Table 18: Recent licensing deals and alliances with big pharma/biotech 168
Table 19: Stem cell-based therapies included in the market forecast 173
Table 20: Forecast of pipeline stem cell therapy products 2008-2014 (US$m) 175
Table 21: Forecast of pipeline stem cell therapy products 2015-2020 (US$m) 176