Stem Cell-Based Therapeutic Delivery: Challenges & Opportunities

Stem Cell-Based Therapeutic Delivery: Challenges & Opportunities published by PharmaVision & Espicom in December, 2008. This report consists of 180+ PAGES and the price starts from US $ 1655.

Introduction

Abstract

This cutting edge and insightful report can be used to interpret and assess the real potential of stem cell-based medicines

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.

In March 2009, President Obama relaxed federal restrictions on ESC research in the US and this is likely to fuel further uptake of this technology in the developed world. Meanwhile, stem cell therapies are already offered at various locations across the globe, including China and Thailand, despite a lack of robust, randomised 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 and investment is growing

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 the 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.

"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."

A FAST GROWING SECTOR WITH GREAT PROMISE

Stem cells have grabbed the attention of scientists across the globe and the pace of research has been astonishing. It is just 10 years since the first human embryonic stem cells (hESCs) were isolated and cultured; new therapies based on this research are now reaching the clinical phase of testing.

KEY FINDINGS IN THE REPORT SHOW

• Our research has revealed a total of 122 stem cell-derived therapeutic projects being undertaken in companies across the globe. The majority of these projects are in the earliest stages (28%) or in preclinical studies (43%). Of the projects that have reached clinical trials, only a handful have passed initial Phase 1/2 testing, with a total of eight (7%) being investigated in Phase 3 studies.
• The market for stem cell therapeutics is forecast to grow from 2011, as the first products enter the marketplace, and is expected to generate global sales in excess of US$1.2 billion by 2015.
• Autologous products, derived from a patient' s own tissue, will be the first to reach the market, but ' off-the-shelf' allogeneic products may offer greater commercial potential. The first allogeneic product to achieve approval is likely to be Osiris Therapeutics' Prochymal for graft versus host disease.
• Delivery of stem cell-based therapeutics can be challenging. Osiris' Prochymal is unique among those reviewed, as it is delivered intravenously, offering obvious commercial advantages. Other therapies are being delivered directly to the site of injury, involving state-of-the-art catheters and imaging systems. The delivery of stem cells for CNS disorders remains a challenging area.

THIS REPORT PROVIDES REAL INSIGHTS...

For executives working in industry and commerce, the need to understand the issues, players and products in this sector of high growth potential is essential. That is why Espicom is making this report available, published by UK pharma consultancy PharmaVision.

In over 180-pages, Stem Cell-Based Therapeutic Delivery: Challenges & Opportunities (published December 2008) summarises some of the latest developments and analyses some of the most promising solutions which companies are providing to address the use of stem cells in key areas of unmet clinical need. Packed with facts, statistics and case studies, the report is the most comprehensive study on this sector of great clinical and commercial potential.

This report answers key business questions

• Which companies & academic institutes are actively involved in stem cell therapy research?
• What are the key areas of therapeutic focus for stem cell therapeutics in the near & long-term?
• What are the key obstacles companies & delivery technologists need to overcome to commercialise stem cell-derived therapies?
• How will the delivery technology drive the development of innovative stem cell-derived therapies in the future?
• Which stem cell technologies & agents are likely to win in the near-term & long-term, & why?
• Where are the market opportunities now & in the future?
• When are stem cell-derived medicines likely to reach the market?
• What do we predict will be the value of the stem-cell derived therapeutics by 2015 & 2020?

Table of Contents

KEY FINDINGS

• Stem Cells & Cell-Based Delivery
• Stem Cell-Based Therapies: Pipeline Analysis
• Stem Cell-Based Therapies for Cardiovascular Disease
• Stem Cell-Based Therapies for CNS Diseases
• Stem Cell-Based Therapies for Autoimmune, Inflammatory & Other Conditions
• Stem Cell-Based Therapies for Cancer, Graft versus Host Disease & Hematological Malignancies
• Challenges & Opportunities Challenges & Opportunities for Stem Cell-Based Therapies
• Future of Stem Cell-Based Therapeutics & Market Trends

CHAPTER 1 STEM CELLS & CELL-BASED DELIVERY

• Key Points
• Introduction
• An Introduction to Stem Cell Science
  • Embryonic stem cells
  • Adult stem cells
  • Creating pluripotent cells from adult tissue
  • Nuclear transfer/therapeutic cloning
  • Combining embryonic stem cell therapy with gene therapy
• Stem Cell Therapy - A Discovery Timeline
• Major Scientific Challenges for the Development of Stem Cell Therapies
• Report Structure

CHAPTER 2 STEM CELL-BASED THERAPIES: PIPELINE ANALYSIS

• Key Points
• Key Therapy Areas Covered by Stem Cell-Based Therapies
  • Product pipelines
• Stem Cells for Drug Discovery
• Stem Cell Culture, Expansion & Manufacturing
• Stem Cell Companies Analyzed in This Report

CHAPTER 3 STEM CELL-BASED THERAPIES FOR CARDIOVASCULAR DISEASE 41

• Key Points
• Introduction
• Autologous Cell Platforms
  • Case study: MyoCell (Bioheart Inc)
  • Case study: Myoblast (Advanced Cell Technology)
  • Case study: CardioCure (Gamida Cell Ltd)
  • Case study: C-Cure (Cardio3 BioSciences)
  • Case study: Celution System (Cytori Therapeutics)
• Allogeneic Cell Platforms
  • Mesenchymal stem cell therapies
  • Case study: Prochymal (Osiris Therapeutics)
  • Case study: Revascor (Angioblast Systems Inc)
  • Case study: PLX-PAD (Pluristem Therapeutics Inc)
  • Human embryonic stem cell therapies
  • Case study: ACT Cellerate (Advanced Cell Technology)
• Cell Delivery Technologies
• Our Opinion on Stem Cell-Derived Therapies for CV Diseases
  • Where the technology is now, its evolution, achievements and pitfalls
  • Competition
  • Potential future applications
  • Activity in the market
  • Major players
  • Winners

CHAPTER 4 STEM CELL-BASED THERAPIES FOR CNS DISEASES

• Key Points
• Introduction
• Autologous Cell Platforms
  • Case study: NurOwn (BrainStorm Cell Therapeutics Inc)
  • Case study: NeuroGeneration
• Allogeneic Cell Platforms
  • Stem cells from donated tissue
  • Case study: HuCNS-SC (StemCells Inc)
  • Case study: ReNeuron
  • Case study: Neuralstem
  • Case study: Q-cells (Q Therapeutics Inc)
  • Case study: StemedicaMCT (Stemedica USA and Stemedica International)
  • Stem cells from umbilical cord blood
  • Case study: Beike Biotechnology
  • Human embryonic stem cells
  • Case study: GRNOPC1 (Geron)
  • Case study: hESCs from California Stem Cell Inc
• Cell delivery technologies
• Our Opinion on Stem Cell-Based Therapies for CNS Diseases
  • Where the technology is now, its evolution, achievements and pitfalls
  • Competition
  • Potential future applications
  • Activity in the market

CHAPTER 5 STEM CELL-BASED THERAPIES FOR AUTOIMMUNE, INFLAMMATORY & OTHER CONDITIONS

• Key Points
• Introduction
• Crohn' s Disease
• Case study: Prochymal for Crohn' s disease (Osiris)
• Case study: Ontaril (Cellerix)
• Diabetes
  • Autologous stem cell transplants for diabetes
  • Allogeneic stem cell transplants for diabetes
• Osteoarthritis
• Lung Diseases
  • Chronic obstructive pulmonary disease
  • Pulmonary arterial hypertension
• Our Opinion on Stem Cell-Based Therapies for Autoimmune and Other Diseases
  • Where the technology is now, its evolution, achievements and pitfalls
  • Competition
  • Potential future applications
  • Major players and winners

CHAPTER 6 STEM CELL-BASED THERAPIES FOR CANCER, GRAFT VERSUS HOST DISEASE & HEMATOLOGICAL MALIGNANCIES

• Key Points
• Introduction
  • Cancer stem cells
  • Stem cells as a delivery vehicle
• Graft versus host disease
• Expanded Umbilical Cord Blood Transplants for Malignant Diseases
  • Case study: StemEx (Gamida Cell)
• Stem Cells as Delivery Systems
  • Lung cancer
  • Metastatic cancer and intracranial tumors
  • Hematological malignancy
  • Multi-lineage progenitor cells (BioE)
• Conclusion
• Our Opinion on Stem Cell-Derived Therapies for Cancer, GvHD and Hematological Malignancies
  • Where the technology is now, its evolution, achievements and pitfalls
  • Competition
  • Potential future applications
  • Activity in the market, major players and winners

CHAPTER 7 CHALLENGES & OPPORTUNITIES FOR STEM CELLBASED THERAPIES

• Key Points
• Introduction
• Ethical Issues for Stem Cell Research
• Funding Stem Cell Research
• Intellectual Property
• Manufacturing Stem Cell-Based Products
• Regulation
• Reimbursement
• Delivering Stem Cell-Based Therapies and Tissue Engineering
  • Challenges
  • Opportunities

CHAPTER 8 FUTURE OF STEM CELL-BASED THERAPEUTICS & MARKET TRENDS

• Key Points
• Key Stem Cell Therapy Companies
• Recent Deals & Alliances
  • The Global Stem Cell Market 2008-2014
  • Analysis parameters
  • Market forecast 2008-2014
  • Global stem cell-based therapy market in 2020
  • Drivers and market trends
• Summary & Conclusions
• Acknowledgements
• Bibliography & Endnotes

List of Figures

• Figure 1: Source of embryonic stem cells: the blastocyst
• Figure 2: Differentiation of embryonic stem cells
• Figure 3: Somatic cell nuclear transfer
• Figure 4: Combining gene therapy and cell therapy for sickle cell anemia in a mouse model
• Figure 5: Stem cell research: a timeline
• Figure 6: Stem cell therapeutics: defining therapeutic areas
• Figure 7: Number of projects in the different stages of pharmaceutical development
• Figure 8: Number of projects in each therapeutic area, by stage of development
• Figure 9: Proportions of autologous and allogeneic technology platforms
• Figure 10: Stem cell sources (embryonic, adult or cord blood/placenta)
• Figure 11: MyoCell: Heart failure treatment process
• Figure 12: Advanced Cell Technologies Myoblast Phase 1 programme; results
• Figure 13: The Celution system from Cytori Therapeutics
• Figure 14: Preclinical data demonstrating the effects of Prochymal therapy on the heart
• Figure 15: PLX cells growing in Pluristem' s PluriX bioreactor
• Figure 16: ACTCellerate technology
• Figure 17: Schematic of cell-based therapy administration to the myocardium
• Figure 18: Cells affected by different CNS diseases/disorders
• Figure 19: NeuroGeneration' s Parkinson' s disease therapy
• Figure 20: StemCells Inc technology platform
• Figure 21: ReNeuron' s process for generating therapeutic cells
• Figure 22: Stemedica' s established cell lines
• Figure 23: Results of animal studies with Geron' s oligodendroglial progenitor cells derived from human ESCs
• Figure 24: Stem cell sources under investigation to treat type 1 diabetes
• Figure 25: Stem cells applications in cancer and other malignant diseases
• Figure 26: Strategies for potentiating stem cells for tumor treatment
• Figure 27: Challenges and opportunities for stem cell science
• Figure 28: Ethical issues with stem cells from different sources
• Figure 29: Federal funding of embryonic stem cell research in the US: a timeline
• Figure 30: US patent ownership in the stem cell field
• Figure 31: Stem cell-based therapeutics: market drivers

List of Tables

• Table 1: Pros and cons of different stem cell types
• Table 2: Diseases targeted by stem cell-based therapeutics
• Table 3: Stem cell therapy companies discussed in this report
• Table 4: Leading companies evaluating stem cell-based CV therapies
• Table 5: Leading academic institutions researching the use of stem cells to treat CV diseases
• Table 6: Leading companies evaluating stem cell-based therapies for CNS diseases
• Table 7: Leading institutes evaluating stem cell-based therapies for CNS diseases
• Table 8: Geron' s stem cell-based product pipeline
• Table 9: Leading companies evaluating stem cell therapies for autoimmune and other diseases
• Table 10: Leading academic institutes evaluating stem cell therapies for autoimmune and other diseases
• Table 11: Leading companies evaluating stem cell therapies for GvHD and hematological malignancies
• Table 12: Academic institutions with an interest in cancer, GvHD and hematological malignancies
• Table 13: Other clinical trials of mesenchymal stem cells for the prevention or treatment of GvHD
• Table 14: Results of Phase 1/2 trial for StemEx
• Table 15: Global positions on research on embryonic stem cells derived from IVF procedures
• Table 16: Current funds available from individual US states for embryonic and adult stem cell research
• Table 17: Summary of leading stem cell-based therapeutics companies
• Table 18: Recent licensing deals and alliances with big pharma/biotech
• Table 19: Stem cell-based therapies included in the market forecast
• Table 20: Forecast of pipeline stem cell therapy products 2008-2014 (US$m)
• Table 21: Forecast of pipeline stem cell therapy products 2015-2020 (US$m)

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