The transplantation field is moving fast, as new products and technologies emerge. Scrip's report assesses the world transplantation market, discussing organ donation, xenotransplantation and artificial organs, with consideration of the important ethical and social issues. Special attention is given to current and emerging transplantation drugs in this expanding market, and the latest clinical developments are reviewed. Also included are detailed profiles of the key companies involved in the transplantation area.

Companies profiled include: American Home Products; Fujisawa; Genzyme; Novartis; Pharmacia & Upjohn; Roche.

PUBLICATION: FEBRUARY 1998
REF: BS913E
PAGES: 150+
PRICE: £370/$780/¥89,000

ABBREVIATIONS

CHAPTER 1 INTRODUCTION
1.1 Background
1.2 Objectives of the report
1.3 Methodology
1.4 Report structure
1.5 Boundaries of the report

CHAPTER 2 TRANSPLANTATION
2.1 Introduction
2.2 Types of transplantation
2.2.1 Autografts
2.2.2 Allografts
2.2.3 Isografts
2.2.4 Xenografts
2.3 Immune responses
2.3.1 An overview
2.3.2 Specific features
2.3.2.1 The role of the major histocompatibility complex
2.3.2.2 B cells, antigen-specific antibodies, and complement
2.3.2.3 T cells and T cell receptors
2.3.2.4 Cytokines
2.3.2.5 Adhesion molecules
2.4 Rejection description and classification
2.4.1 Hyperacute rejection
2.4.2 Acute rejection
2.4.3 Chronic rejection
2.4.4 Graft-versus-host disease
2.4.5 Immunological accommodation or tolerance
2.5 Indications
2.5.1 Kidney, pancreas, and kidney + pancreas
2.5.2 Liver
2.5.3 Heart, lung, and heart + lung
2.5.4 Gastrointestinal tract
2.5.5 Haematopoietic/lymphopoietic stem cells
2.5.6 Skin
2.6 Organ demand
2.7 Organ availability
2.8 Ethics
2.8.1 Living donors
2.8.2 Cadavers as donors
2.8.3 Use of foetal material
2.9 Transplantation regulations in major markets

CHAPTER 3 XENOTRANSPLANTATION
3.1 Review of the latest research
3.1.1 Xenotransplantation of vascularised 'solid' organs
3.1.2 Xenotransplantation of cell 'suspensions'
3.1.3 Other ways to enhance xenograft acceptance
3.2 Ethics

CHAPTER 4 CELL THERAPIES
4.1 Review of products
4.1.1 ADA gene therapy
4.1.2 Gene therapies for cardiovascular disease
4.1.2.1 Ad-GAX1
4.1.2.2 a-glucosidase gene therapy
4.1.2.3 BST-3001
4.1.2.4 iNOS gene
4.1.2.5 LDL receptor gene therapy
4.1.2.6 PKG gene therapy
4.1.2.7 VEGF-B gene
4.1.2.8 VEGF-2 gene
4.1.2.9 Others
4.1.3 Gene therapies for chronic granulomatous disease
4.1.4 Gene therapies for CNS diseases
4.1.4.1 Gene therapies for cognitive disorders
4.1.4.2 Gene therapies for Huntington's disease
4.1.4.3 Gene therapies for focal epilepsy
4.1.4.4 Parkinson's disease therapy
4.1.4.5 Gene therapies for stroke
4.1.4.6 Others
4.1.5 Gene therapies for cystic fibrosis
4.1.5.1 GR-213487
4.1.5.2 Others
4.1.6 Gene therapies for diabetes
4.1.7 Gene therapies for Gaucher's disease
4.1.8 Gene therapies for haematological/immunological diseases
4.1.8.1 Aviheme
4.1.8.2 CD5/8 gene therapy
4.1.8.3 Erythropoietin gene therapy
4.1.8.4 Gene therapies for haemophilia A (Factor VIII)
4.1.8.5 Gene therapies for haemophilia B
4.1.8.6 GM-CSF gene therapy
4.1.8.7 MDL-104396
4.1.8.8 Gene therapy for b-thalassaemia
4.1.8.9 Others
4.1.9 Gene therapies for hormone deficiencies
4.1.10 Gene therapy for Hurler's syndrome
4.1.11 Gene therapies for acute liver failure
4.1.12 Gene therapies for muscular dystrophy
4.1.13 IL-1ra gene therapy for rheumatoid arthritis
4.1.14 Retinal gene therapy
4.1.15 Technologies
4.1.15.1 AAV vectors
4.1.15.2 bcl-xs gene therapy
4.1.15.3 BRCA2 gene
4.1.15.4 d-like protein
4.1.15.5 ELVS
4.1.15.6 Gene gun
4.1.15.7 HSV vectors
4.1.15.8 MDR gene therapy
4.1.15.9 Non-viral gene therapies
4.1.15.10 Regulated gene therapy
4.1.15.11 Retroviral vector
4.1.15.12 Stem cell gene therapy
4.1.15.13 Others
4.2 Review of markets
4.2.1 Potential markets in cardiovascular medicine
4.2.2 Potential market for CNS disorders
4.2.2.1 Alzheimer's disease
4.2.2.2 Amyotrophic lateral sclerosis
4.2.2.3 Cognitive disorders
4.2.2.4 Focal epilepsy
4.2.2.5 Huntington's disease
4.2.2.6 Parkinson's disease
4.2.2.7 Stroke
4.2.3 Potential market for cystic fibrosis
4.2.4 Potential market for diabetes
4.2.5 Potential market for Gaucher's disease
4.2.6 Diseases for which stem cell transplants might find application
4.2.6.1 Stem cells for malignant diseases
4.2.6.2 Stem cells for non-malignant diseases
4.2.7 Potential market for growth hormone replacement
4.2.8 Potential market for acute liver failure
4.2.9 Potential market for muscular dystrophy
4.2.10 Potential market for rheumatoid arthritis

CHAPTER 5 CURRENT AND EMERGING DRUG THERAPIES
5.1 Current market trends
5.2 Descriptions of the current and emerging medicines
5.2.1 Primary immunosuppressants
5.2.1.1 Cyclosporin
5.2.1.2 Tacrolimus
5.2.2 Adjunct immunosuppressants
5.2.2.1 Azathioprine
5.2.2.2 Mycophenolate mofetil
5.2.2.3 Corticosteroids
5.2.3 Anti-rejection antilymphocyte (antithymocyte) globulins
5.2.3.1 Atgam
5.2.3.2 OKT3
5.2.3.3 Thymoglobulin
5.2.4 Others
5.2.4.1 Anti-CD3 monoclonal antibody
5.2.4.2 Gusperimus
5.2.4.3 Ior-t3
5.2.4.4 Mizoribine
5.2.4.5 Pentostatin
5.2.4.6 Protein G
5.3 Analysis of older generation versus new generation immunosuppressants
5.4 Market implications

CHAPTER 6 MEDICINES IN DEVELOPMENT
6.1 Research trends
6.2 A review of products in R&D
6.2.1 Cytotoxic immunosuppressants
6.2.1.1 AI-502
6.2.1.2 Apoptosis modulators
6.2.1.3 Atiprimod dihydrochloride
6.2.1.4 Brequinar sodium
6.2.1.5 Busulfan
6.2.1.6 Castanospermine
6.2.1.7 CG-1088
6.2.1.8 FR-901459
6.2.1.9 FTY720
6.2.1.10 Ginkgolide B
6.2.1.11 ISIS-2302 and related compounds
6.2.1.12 KE-298
6.2.1.13 Leflunomide
6.2.1.14 MC-1288
6.2.1.15 NE-0501
6.2.1.16 Peldesine and other PNP inhibitors
6.2.1.17 PG-27
6.2.1.18 PIC-060
6.2.1.19 Rapamycin
6.2.1.20 SDZ RAD
6.2.1.21 SIDR-1026
6.2.1.22 SDZ 281-240
6.2.1.23 SP100030
6.2.1.24 TAQ-588
6.2.1.25 TBC-1269
6.2.1.26 Tresperimus triflutate
6.2.1.27 VX-497
6.2.2 Biological immunosuppressants
6.2.2.1 ABX-CBL
6.2.2.2 Allotrap-2702
6.2.2.3 Anti-gp39 monoclonal antibody
6.2.2.4 Anti-Tac(Fv)-PE40
6.2.2.5 Basiliximab
6.2.2.6 BMS-188667
6.2.2.7 BTI-322
6.2.2.8 CAB-2
6.2.2.9 Campath-1H
6.2.2.10 CD2
6.2.2.11 CD4 inhibitors
6.2.2.12 CD45RB
6.2.2.13 CD95 modulators
6.2.2.14 CDP-571
6.2.2.15 CDP-865
6.2.2.16 CMI-392
6.2.2.17 Cytokine agonists
6.2.2.18 Cytokine traps
6.2.2.19 Daclizumab
6.2.2.20 Enlimomab
6.2.2.21 Growth and differentiation factors
6.2.2.22 Hu23F2G
6.2.2.23 hu1124
6.2.2.24 IL-2:diphtheria toxin monoclonal antibody
6.2.2.25 Immunophilins
6.2.2.26 Interleukin-4 fusion toxin
6.2.2.27 Interleukin-4 receptor
6.2.2.28 Interleukin-10
6.2.2.29 Ior-t1
6.2.2.30 LDP-01
6.2.2.31 LFA3TIP
6.2.2.32 mAb-24
6.2.2.33 MEDI-507
6.2.2.34 Monoclonal-porphyrins
6.2.2.35 Odulimomab
6.2.2.36 PRIMATIZED antibodies
6.2.2.37 Protein A
6.2.2.38 Recombinant PAF-AH
6.2.2.39 SCA proteins
6.2.2.40 sCR1
6.2.2.41 Superoxide dismutase
6.2.2.42 T10B9
6.2.2.43 Toleragens
6.2.2.44 5G1.1-SC
6.2.3 Others
6.3 Comments on the potential of these products
6.4 Predictions for the direction of future research

CHAPTER 7 MARKET DATA
7.1 Market trends
7.2 Market size
7.3 Current sales of major drugs
7.4 Predicted sales of major medicines
7.5 Pricing information of major medicines
7.6 Market size by major geographical region
7.7 Marketing strategies
7.8 Pharmacoeconomics data

CHAPTER 8 COMPANY PROFILES
8.1 Company profiles
8.1.1 American Home Products
8.1.2 Baxter International
8.1.3 BioTransplant Inc
8.1.4 Celltech
8.1.5 Diacrin
8.1.6 Fujisawa
8.1.7 Genzyme Corporation
8.1.8 ICOS Corporation
8.1.9 Nippon Kayaku
8.1.10 Novartis
8.1.11 Roche
8.1.12 Signal Pharmaceuticals
8.1.13 T Cell Sciences
8.1.14 Titan Pharmaceuticals (Theracell)
8.2 'Biotech bubble under pressure'

CHAPTER 9 DIRECTORY

CHAPTER 10 CONCLUSIONS

REFERENCES

LIST OF TABLES
Table 2.1 Some characteristics of the major histocompatibility complex
Table 2.2 Some of the important properties of the various classes of immunoglobulins
Table 2.3 Helper T cell populations distinguished by their anti- (Th1) or pro- (Th2) inflammatory profile of secreted cytokines
Table 2.4 Adhesion molecules on endothelial cells and leukocytes and involved in leukocyte migration into organ grafts
Table 2.5 Grading of graft-versus-host disease severity in the three main target organs
Table 2.6 Construction of an overall grade for graft-versus-host disease severity from disease scores in the three major target organs
Table 2.7 The number of patients awaiting organ transplants in the US at the end of each year, 1988-1996
Table 2.8 The number of deaths amongst patients awaiting organ transplants in the US, 1988-1996
Table 2.9 The number of patients awaiting organ transplants in the UK in December 1996
Table 2.10 The number of transplant operations performed in the US, 1988-1996
Table 2.11 The number of bone marrow transplants in North America in 1994
Table 2.12 The number of organ transplants recorded in countries of Europe in 1996 according to the European Transplant Coordinators Organization (ETCO) - I. Austria to Germany
Table 2.13 The number of organ transplants recorded in countries of Europe in 1996 according to the European Transplant Coordinators Organization (ETCO) - II. Greece to Spain
Table 2.14 The number of organ transplants recorded in countries of Europe in 1996 according to the European Transplant Coordinators Organization (ETCO) - III. Sweden to UK & Ireland and region totals in comparison with the US
Table 4.1 The advantages and disadvantages of some of the vectors being evaluated for gene therapies
Table 4.2 Percentage of patients with dementia in various age groups - the Framingham study
Table 4.3 Estimated new cancer cases and cancer deaths (x103) in the US in 1994 for which autologous or allogeneic stem cell grafts have been attempted in a significant number of patients
Table 5.1 Comparative nephrotoxicities of the two Novartis formulations (Sandimmun and Neoral) of cyclosporin in living donor kidney recipients
Table 5.2 Comparative pharmacokinetics of Neoral and Sandimmun and the frequency of rejection episodes
Table 5.3 Pharmacokinetics of tacrolimus
Table 5.4 Comparison of tacrolimus and cyclosporin (Sandimmun) in the European Multicentre Study: data at 1-year post transplantation
Table 5.5 Comparison of tacrolimus and cyclosporin (Sandimmun) in the US Multicenter Study: data at 1- and 2-years post transplantation
Table 5.6 Representative list of widely-available oral anti-inflammatory corticosteroids
Table 6.1 The effect of combining FTY720 with cyclosporin on survival of dogs given a renal allograft
Table 6.2 Summary of the results of a multicentre Phase III trials of daclizumab (with cyclosporin, azathioprine, and corticosteroids) in renal allograft recipients: 6-month data
Table 7.1 Total US market 1991-2001 for organ transplantation products and artificial organs (including skin)
Table 7.2 Total US market 1992-2002 for monoclonal antibodies
Table 7.3 September 1997 sales of some important immunosuppressive medicines
Table 7.4 Estimated peak sales (for organ transplantation) of some major immunosuppressant medicines. I - Current and emerging drugs
Table 7.5 Estimated peak sales (for organ transplantation) of some major immunosuppressant medicines. II - Drugs in development
Table 7.6 UK pricing information of the major immunosuppressant medicines. I - Primary immunosuppressants
Table 7.7 UK pricing information of the major immunosuppressant medicines. II - Adjunctive immunosuppressants
Table 7.8 A comparison of direct medical (in-hospital) costs for patients given Neoral or Sandimmun over the 4 months post-transplantation
Table 7.9 Comparative data on the cost-effectiveness of tacrolimus and OKT3 in liver transplant recipients
Table 7.10 A comparison of the cost-effectiveness of mycophenolate mofetil and azathioprine (in combination with corticosteroids) for the treatment of acute rejection in renal allograft recipients
Table 7.11 A comparison of the cost-effectiveness of mycophenolate mofetil and azathioprine (in combination with cyclosporin and prednisone) for the treatment of acute rejection in cadaveric renal allograft recipients
Table 8.1 American Home Products' financial figures ($ million) 1990-1996
Table 8.2 American Home Products' sales by geographical region and business segment ($ million) 1996
Table 8.3 Baxter's financial figures ($ million) 1990-1996
Table 8.4 Baxter's sales by geographical region and business division ($ million) 1995
Table 8.5 Celltech's financial figures (� 000) 1992-1996
Table 8.6 Diacrin's financial figures ($ 000) 1992-1997
Table 8.7 Fujisawa's financial figures (¥ million) 1992-1996
Table 8.8 Fujisawa's sales by geographical region and by business segment (� million) 1996
Table 8.9 Genzyme's financial figures ($ 000) 1991-1996
Table 8.10 ICOS's financial figures ($ 000) 1992-1996
Table 8.11 Nippon Kayaku's financial figures (¥ million) 1991-1996
Table 8.12 Novartis' financial figures (SwFr billion) 1995-1997
Table 8.13 Novartis' sales by geographical region (in SwFr billion in 1995) and company division (in SwFr billion in 1996)
Table 8.14 Roche Group's financial figures (SwFr million) 1990-1996
Table 8.15 Roche Group's sales by geographical region (in SwFr million in 1995) and business segment (in SwFr million in 1996)
Table 8.16 T Cell Sciences' financial figures ($ 000) 1992-1996
Table 8.17 Titan's financial figures ($ 000) 1995-1996
Table 8.18 Major UK biotech companies: their share prices (as of early November 1997) and market capitalisation
Table 8.19 Cell adhesion programmes in some biotech pharmaceutical companies
Table 9.1 Pharmaceutical companies with their major development-stage and launched transplantation medicines

LIST OF FIGURES
Figure 2.1 Schematic representation of the immune response
Figure 2.2 The major histocompatibility gene complex
Figure 2.3 Generic structure of an antibody molecule
Figure 2.4 Schematic representation of the complement system
Figure 2.5 Important steps in the development of T cell anergy as a potential mechanism for the development of tolerance to organ transplants
Figure 3.1 An overview of some genetically-engineered, vascular-based changes which would be expected to promote xenograft survival

• Order this report.
• Request a catalogue.

© PJB Publications Ltd. 2001 All rights reserved.