Stem Cell Technologies: World Market Outlook 2012-2022

yet feasible, due to the novelty of the cell type and the uncertainty about oncogenic potential, genomic stability, epigenetic abnormality and other features of the iPSCs. Modified stem cells as a delivery vehicle for the correction of genetic disorders are also a plausible future target.

In any given indication, stem cells must demonstrate safety before the question of efficacy can be broached. Autologous stem cell transplants have consistently met safety requirements (although they have not consistently shown sustained therapeutic effects). Allogeneic stem cell transplants generally incur higher safety risks and require immunosuppression to prevent the graft from being rejected. Mesenchymal stem cells have the benefit of being cleared rapidly from the body, which is a bonus from the perspective of safety considerations. This characteristic of MSCs does raise the question of how, if at all, they achieve therapeutic effects. It is believed that the MSCs, though only transiently present in the host body, initiate or modulate trophic effects (yet to be characterised) which confer therapeutic benefit.

Visiongains analysis concentrates on the companies involved in the field, which we have grouped under the major headings of oncology, auto-immune disease, diabetes, cardiological/ cardiovascular disease, neurological/cerebrovascular disease, ophthalmology and others. Table 5.1 and Figure 5.1 give an overview of the quantity of clinical stem cells work (academic and industry-sponsored) being undertaken in each of these therapeutic areas, according to the clinical trials register. Trials for adjunct products and other semi-relevant trials will generate false positives in this kind of analysis, which is therefore only used to suggest the current distribution of clinical attention. Cancer, the therapeutic area in which HSCT is an established practice, is the main therapeutic area for stem cells research, with cardiological/cardiovascular second.

Table 5.1 Quantity of Stem Cell Clinical Trials by Indication, 2012

Indication Oncology Autoimmune disease Diabetes Cardiological / Cardiovascular diseases Neurological disease Ophthalmology Phase I Phase II Phase III Phase IV Total

848 54 38 272 38 40

1577 58 42 519 44 37

390 5 7 124 7 7

59 1 8 35 1 2

2874 118 95 950 90 86

Source: clinical trial sources; visiongain 2012

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Figure 5.1 Quantity of Stem Cell Clinical Trials (All Phases) by Indication, 2012
Oncology 2874 Autoimmune,118

Diabetes 95

Cardiological/ Cardiovascular 950 Ophthalmological 86

Neurological 90

Source: visiongain 2012

Figure 5.2 Oncology Stem Cell Clinical Trials by Phase, 2012

1600 1400 1200 1000 800 600 400 200 0 I Source: visiongain 2012 II III IV

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Stem Cell Technologies: World Market Outlook 2012-2022

next in importance to stem cell therapies for the future development of the stem cell technologies market.

Figure 7.4 Pharmaceutical R&D Spending by Developmental Stage, 2010

Other 16.7% Other 25.0%

Pre-Clinical Stage 26.6%

Clinical Stage 56.7%

Cell-Based Assays 1.6%

Source: visiongain 2012

7.3.1 Axiogenesis In Vitro Disease Models

Axiogenesis is a stem cell services provider, and the self-described world-leader in murine embryonic stem cell derived products. Using proprietary platforms, the company creates in vitro differentiated cardiomyocytes, smooth muscle cells and endothelial cells.

Axiogenesis patented an in vitro disease model for the condition of cardiac hypertrophy, using its proprietary cardiomyocytes, which it describes as the gold standard for in vitro assays on cardiac toxicology and drug discovery. This represents part of Axiogenesis screening platform concept whereby stem cell-derived tissues are used to model pathological conditions, enabling target identification and patient stratification.

Axiogenesis is exploring iPS technology to expand its range of stem cell products.

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