干细胞相关策略成就细胞治疗的商业化

Abstract

Traditionally, treatment of human diseases is carried out by small molecular weight pharmaceuticals or large biotherapeutic molecules that modulate the biology of specific cell types. In many cases, such as with cancer, the aim of the treatment is to kill targeted cells, leading to the physiological benefit for the patient. In other circumstances, the targeted cells are modulated by a drug to perform a specific function that they normally do not. A variety of molecules have been utilized as therapeutic agents. These include:

• Small molecules
• Peptides
• Proteins
• Antibodies
• Anti-sense RNAs
• Ribozymes

In the case of cell therapy, as the name implies, treatment is carried out using cells instead of therapeutic molecules. In cell therapy, cells are given to the patient to achieve a therapeutic benefit.

Table of Contents

Chapter 1: Executive Summary 1--1

Chapter 2: Introduction and Background to Cell Therapy 2--1

• Introduction 2--1
• Cells for Therapeutic Applications 2--2
• Epithelial Tissue 2--3
• Connective Tissue 2--3
• Muscle Tissue 2--4
• Nervous Tissue 2--5
• Basic and Advanced Cell-Based Therapy Approaches 2--5
• Single Cell Therapy 2--5
• Multiple Cell Therapy Strategies 2--5
• Cell Isolation Techniques 2--6
• Primary Cell Isolation Protocol 2--6
• Mononuclear Cell Isolation 2--6
• Immunologic Procedures for Cell Isolation 2--7
• Developments in Cell Selection 2--7
• Ex vivo cell expansion 2--8
• Peripheral Blood Stem Cell Harvesting 2--9
• Dendritic Cells 2--9
• Background 2--9
• Dendritic Cell-Based Cancer Therapy 2--10
• Dendritic Cells in Cancer Therapeutics 2--11
• Stem Cells 2--11
• Background 2--11
• Stem Cells: Classification and Characterization 2--12
• Embryonic Stem Cells: Classification and Characterization 2--14
• Adult Stem Cells: Classification and Characterization 2--14
• Stem Cell Therapy: Basic and Advanced Principles 2--18
• Embryonic Stem Cells for Therapeutic Transplants 2--19
• Advantages and Disadvantages of Embryonic Stem Cells 2--19
• Potential Uses of Human Embryonic Stem Cells 2--20
• Genetic Manipulation of Human Embryonic Stem Cells 2--21
• Transplantation Research--Restoring Vital Body Functions 2--21
• Basic Research Applications of ESCs 2--22
• Therapeutic Delivery Systems for SCs 2--23
• Other Applications of Stem Cells 2--23
• Critical Evidence and Questions about Stem Cell Research 2--23
• Known facts About Human Pluripotent Stem Cells? 2--24
• Comparisons of Adult Stem Cells and Embryonic Stem Cells 2--25
• Conclusions 2--25

Chapter 3: Market Analysis of Cell Therapy Space 3--1

• Overall Forecast for the Cell Therapy Field 3--6

Chapter 4: Cell Therapy in The News 4--1

• Stem Cell Injection Restores Ability to Walk 4--1
• Stem Cells Can Help Fight Against HIV 4--1
• Iowan Makes Strides After Spine Surgery 4--2
• Stem Cell Trigger Identified 4--2
• Successful Treatment of Stroke Victim Who Received Stem Cell Transplantation Therapy 4--2
• 'Long Road' for Stem Cell Research 4--3
• S.F. Clinic Recruiting Egg Donors for Stem Cell Research 4--3
• FDA Approves Brain Stem Cell Transplant 4--3
• Successful Trial of Gene Therapy in France Uses "Adult" Stem Cells 4--4
• Adult Stem Cells Reverse Diabetes in Mice 4--4
• Bone Marrow Stem Cell Transplant Used to Treat Children with Cartilage Defect 4--4
• Neural Stem Cells Able to Repair Damage Throughout Brain 4--4
• Corneal Stem Cells Help Restore Sight 4--4
• Human Bone Marrow Stem Cells Can Form Liver 4--5
• Adult Bone Marrow Stem Cells Can Generate Nerve Cells 4--5
• Large Numbers of Adult Bone Marrow Stem Cells Can Be Generated in Culture 4--5
• Adult Bone Marrow Cells Can Form Multiple Tissue Types 4--5
• Adult Stem Cells Reverse Ddiabetes in Mice 4--6
• Scientists Identify Human Adult Pancreatic Stem Cells 4--6
• Comprehensive Review Finds Adult Stem Cells Present Fewer Biological Problems for Producing Insulin Secreting Pancreatic Beta Cells 4--6
• Clinical Successes in Cancer Treatment Again Show Versatility, Pluripotent Abilities of Adult Stem Cells 4--6
• Adult Stem Cells Again Proved Capable of Numbers to Meet Clinical Demand 4--7
• Treating Heart Disease with adult Stem Cells 4--7
• Adult Stem Cells Advance Toward Treating Degenerative Neural Disorders 4--7
• Sonic Hedgehog" Paves Way for Potentially Unlimited Supplies of Adult Stem Cells; Improved Treatment for Cancer Patients 4--8
• Stem Cells from Skin Develop into Brain Cells and Other Tissues 4--8
• Stem Cell-Rich Cord Blood Successfully Treats Often Fatal Blood Disorder 4--8
• Scientists Purify Pluripotent Adult Neural Stem Cells from Brain 4--8
• Adult Stem Cells Show Success in Treating Autoimmune Disease 4--8
• Adult Stem Cells Treat Potentially Fatal Skin Disorder 4--9
• Adult Stem Cells Repair Heart Damage 4--9
• Adult Stem Cells Can "Be Cultured and Expand Indefinitely" 4--9
• Adult Stem Cells Offer Hope without Hype 4--9
• Successful Repair of Heart Damage Using Adult Stem Cells 4--9
• Japanese Scientists use Neural Stem Cells to Decrease Parkinson's Symptoms 4--10
• Human Adult Bone Marrow Stem Cells Can Form Kidney Tissue 4--10
• Treatment of Multiple Sclerosis Using the Patient's Own Cells 4--10
• Umbilical Cord Blood Effective At Treating Adult Blood Disorders 4--10
• Sickle Cell Anemia Treated with Umbilical Cord Blood Stem Cells 4--10
• Stroke Treatment Follow-up Shows Patient Improvement 4--11
• Tissue-Engineered Arterial Grafts from Adult Blood Cells 4--11
• Functional Brain Cells Generated From Adult Bone Marrow Stem Cells 4--11
• Spinal Cord Regeneration Using Adult Cells 4--11
• New Corneas Grown from Patients' Own Adult Stem Cells Treat Corneal Scarring 4--11
• Treating Parkinson's with Adult Stems Cell and Other Alternatives 4--12
• Retinal Cell Implants Improve Parkinson's 4--12
• Stimulating Adult Brain Stem Cells Decreases Parkinson's Symptoms 4--12
• Progenitor Cells Reverse Severe Parkinson's Symptoms in Rats 4--12
• Adult Liver Stem Cells Make Pancreatic Cells, Reverse Hyperglycemia in Diabetic Mice 4--12
• Adult Bone Marrow Stem Cells Transformed Into Functional Liver Cells 4--12
• Adult Muscle Stem Cells Form Multiple Tissues; Regenerate Dystrophic Muscle 4--13
• Chapter 5: Analysis of End-user Survey and Expert Opinions 5--1

• Interview with Dr. Nevenka Kregar Velikonja, Managing Director, Educell d.o.o. 5--1
• Interview with Stephen Bartelmez Ph.D., President and CEO, Ixion Biotechnology 5--5
• Survey Analysis 5--7
• Chapter 6: Company Profiles 6--1
• Aastrom Biosciences, Inc. 6--1
• Advanced Cell Technology, Inc. 6--4
• AllCells, LLC. 6--9
• Amcyte Inc. 6--10
• Argos Therapeutics 6--11
• Athersys Inc. 6--12
• BioE 6--13
• Bioheart, Inc. 6--14
• BioMark International 6--16
• BioTissue Technologies GmbH. 6--17
• Cambrex 6--18
• Cell Transplant International 6--21
• Cellartis AB 6--26
• CellECT Bio, Inc. 6--28
• Cellerant Therapeutics, Inc. 6--29
• Cellerix 6--30
• CepTor Corporation 6--31
• Chromos 6--32
• Cryosite Limited 6--34
• Cytomatrix LLC 6--35
• Cytori Therapeutics, Inc. 6--36
• Dendreon Corporation 6--37
• Educell, d.o.o. 6--39
• ES Cell International Pte Ltd 6--40
• Gamida Cell Ltd. 6--41
• GenOway 6--43
• Genzyme 6--44
• Geron 6--45
• Histostem, Ltd. 6--47
• Ixion Biotech Inc. 6--49
• Medra, Inc. 6--50
• Nephros Inc. 6--51
• Neuronyx Inc. 6--53
• Neurotech USA 6--54
• Northwest Biotherapeutics Inc. 6--55
• Orion Biosolutions 6--57
• Osiris Therapeutics, Inc. 6--58
• Progenitor Cell Therapy, LLC 6--61
• ReNeuron 6--62
• Stem Cell Technologies 6--64
• StemCells Inc. 6--67
• Theravitae Inc. 6--69

Chapter 7: Cell Therapy: Patent Analysis 7--1

Appendix: Basic Properties of an Embryonic Stem Cell Appx--1

• What are Some of the Questions that Need to be Answered about
• Stem Cells? Appx--1
• Timeline of Human Embryonic Stem Cell Research Appx--3
• Stem Cells Appx--5
• Web Resources Appx--5
• 1. Stem Cell Information and Background Appx--5
• 2. Stem Cell-related Organizations (US) Appx--5
• 3. International Stem Cell-related Organizations Appx--5

TABLE OF EXHIBITS

• Exhibit 1.1 Basic Principle of the Cell-based Therapy 1--1
• Exhibit 1.2 Basic Principle Behind Cell Therapy Strategy 1--2
• Exhibit 1.3 Number of Publications in the Cell Therapy area in the Past Decade 1--3
• Exhibit 1.4 Technologies Driving the Development of Stem Cell Therapy 1--4
• Exhibit 1.5 Lead Companies in the Cell Therapy Space 1--5
• Exhibit 2.1 Treatment of Human Diseases Using Dendritic Cells 2--10
• Exhibit 2.2 Stem Cell Hierarchy 2--12
• Exhibit 2.3 Classifying Embryonic and Adult Stem Cell Types 2--13
• Exhibit 2.4 Stem Cell Types and Their Characteristics 2--13
• Exhibit 2.5 Generating Autologous Ttissue Grafts Using the Therapeutic Cloning Strategy 2--18
• Exhibit 3.1 Market Forecast for Cell Therapy Treatments 3--2
• Exhibit 3.2 Distribution of Companies in the Cell Therapy Area 3--3
• Exhibit 3.3 Distribution of Companies Based on the Targeted Organ 3--3
• Exhibit 3.4 Distribution of Companies Based on Specific Therapeutic Areas 3--5
• Exhibit 3.5 Distribution of Companies based on Specific Disease Indication 3--6
• Exhibit 3.6 Expected Growth in the Number of Clinical Treatment Studies in Four Major Therapeutic Indications 3--7
• Exhibit 6.1 Production Cycle for Three Cellerix Products 6--30
• Exhibit 6.2 Applying Stem Cells in Developing Human Therapeutic Applications 6--46
• Exhibit 6.3 Stem cell discovery cycle 6--68