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European Biopharmaceutical Review

Finding the Right Place

The growth of the cell therapeutics industry brought with it an expectation of a paradigm shift in the way that today’s untreatable or poorly treated diseases are dealt with, and this promise is now beginning to bear fruit..

The cell therapy industry (CTI) is coming of age with its most exciting commercial year yet and it has been predicted that the 2011 turnover will break the $1 billion mark (1). This figure is expected to rise and hit $5.1 billion by 2014. It has certainly been attracting its fair share of media attention, with hardly a day passing without an article featuring news on some form of cell therapy. Some of the most recent CTI highlights include:
  • Reneuron’s pilot investigation of stem cells in stroke (PISCES) study in the UK is the pioneering first-inman, neural stem cell (nonembryonic) therapeutic for disabled stroke patients (2)
  • Dendreon receiving FDA approval for Provenge, the autologous cancer vaccine for the treatment of advanced prostate cancer.This product is set to be the first cell therapy industry blockbuster with predicted sales in the region of $1.75 billion per annum by 2014 (3).This approval has also shown that autologous therapies can work commercially
  • Ground-breaking steps have also been made in human embryonic stem cell therapy where Geron received the landmark FDA approval to commence clinical trials of their allogeneic oligodendrocyte product for acute spinal cord injury. The company have just treated their second patient (4)
  • Two clinical trial approvals for Advanced Cell Technology: allogeneic stem cell-derived retinal pigment epithelium product for Stargardts disease and age-related macular degeneration (5,6)
Past and Current Perspectives

So, all in all, the last 12 months have firmly positioned the CTI as a credible sector of the healthcare industry, which has driven enhanced interest from the investment community. Even traditional pharma companies are beginning to make concerted moves into the field.

Large pharmaceutical companies have traditionally been slow to enter emerging technology markets but their interest in the CTI seems to be growing, perhaps driven by the opportunities afforded by cell therapies for a future of truly restorative or disease modifying therapies, rather than the standard focus of palliative treatment we have today (7). Within the last six months there have been some large deals between pharma and stem cell companies. For example, Cephalon took a 20 per cent stake in Mesoblast Ltd for the rights to market their adult stem cell therapies for heart and nervous system conditions in a deal potentially worth more than $2 billion (8). Shire is the latest to get a piece of the action with its purchase of Advanced BioHealing for $750m in cash, in order to gain access to its Dermagraft product – a skin substitute that assists in restoring damaged tissue (9). It is believed that these moves are just the start as other large pharma and biotechnology companies will most likely follow suit. Their participation is of immense value to the industry, as with their support clinical trials can be elevated to well-designed, large scale Phase 3 trials right through to the industrialisation of cell therapy production. This will help to bring therapies to market faster and to elevate cell therapy as the standard of care.

Aside from the positive news-flow from the CTI, their productivity has also been impressive. Up until the end of 2010 there had been over 675,000 therapeutic regenerative medicine cell therapies manufactured and 323,000 patients treated (10). Organogenesis, a company based in Canton, Massachusetts, constitutes over twothirds of all these cell-based products with Apligraf, which was one of the first FDA-approved living, human cell-based products, indicated for the treatment of venous leg and diabetic foot ulcers. It is now one of the leading regenerative medicine brands in the world where to date, according to their website, they have treated over 250,000 patients with Apligraf. If we take a global view of the number of companies working in cell therapeutics with products either on the market or in some stage of late pre-clinical or clinical development, then there are approximately 275 companies showing the breadth and depth of development activity within the sector (11). This is similar to the early activity observed within the biologics market, which is now a multi-billion dollar industry.

The factors which are driving this level of interest from the life sciences community are in part due to the capacity of the CTI to transform the medical industry. Cell therapy can be described simply as the process of introducing live cells into a tissue in order to repair it or restore lost or defective functions, regardless of the cell type or therapeutic indication. This transformation will be realised through the impact of the application of cell therapeutics to a wide range of disease markets that will benefit, aligned with the potential substantial impact on economic costs.Mason and Dunhill summarised this by looking at those diseases that could be impacted relatively soon and the associated effect on economic costs (12). Even though the numbers are relatively small, the realisable benefits are of major significance to indications of heart failure, insulin-dependent diabetes, stroke, Parkinson’s disease, spinal cord, Alzheimer’s and renal disease (see Table 1).



Future Predictions

The promise of cell therapies could therefore have a major impact on restoring normal function within these diseases, driving enhanced patient outcome while lessening the healthcare economic burden and increasing productivity, potentially saving up to $250 billion a year. Studies also show that there are potential wins from early introduction of cures, for example a cell therapy treatment for juvenile diabetes coming five years earlier – in 2030 instead of 2035 – is estimated to generate nearly $12.5 billion in additional revenue (13). These are strong drivers for the CTI and the healthcare sector as a whole, especially when combined with increasing life expectancy rates, which are set to cause a major burden in relation to chronic and degenerative diseases. This is an area where the CTI could offer useful therapeutic solutions – for example in the US alone the percentage of people over 65 in 2010 was 12 per cent and this is estimated rise to 19 per cent by 2030 (14).

It is clear that the CTI will have an important role to play in our future healthcare armoury and as such it will need regulatory, legislative and technology support to realise its full potential. Translating the process of generating cell therapies in the laboratory to one which has the capacity to reproducibly manufacture enough product and incorporate an efficient distribution system to scale, is presenting some interesting challenges and, consequently, demanding innovative thinking from those in the industry. Some of the challenges and questions that face cell therapeutic companies in the process of commercialising a new product include the choice of cell type, autologous versus allogeneic, cryopreserved product, cell only versus a combination cell/device product, single versus multiple treatments, centralised processing hub or multiple hubs, reimbursement strategy and the overall business model, to name a few.

One of the rudimentary elements and challenges of producing a cell therapy product is down to characterisation – a challenge which is shared by all in the industry. This was also seen in the results of a recent survey carried out by the International Society for Cellular Therapy where its membership expressed a desire for cell characterisation standards, cell processing and manufacturing standards, and technology assessment (15). Characterisation is fundamental as it spans from early R&D through the production process from scale-up or scale-out, right through to full-scale manufacture so that the cell product can be produced consistently and reproducibly,which in essence will ensure successful cell therapy product production.

Characterisation needs to be evaluated at both the product development stages, as well as for comparability testing of the final product throughout manufacture, as it is vital to understand the characterisation of the starting materials as well as the final cell therapy product. In this way we have a clear understanding, or global view, of the cell throughout the manufacturing process and an understanding of quality, safety and efficacy which is linked to the final product. The recommended characterisation of cell products from the regulatory bodies includes sterility, purity, potency, ID markers, stability, safety and efficacy. These are in place to ensure more efficient characterisation of cells at all stages of development, ultimately resulting in increased product performance and less variability. At the end of the day, it all boils down to product quality and consistency. However, characterisation using today’s tools is challenging, as well as labour- and time-intensive, such that it is thought that characterisation will become the bottle-neck in progressing these therapies through manufacture to the market (see Table 2 for a summary of characterisation techniques for cell therapy products) (16). These tools are not entirely fit for purpose and it will be important that as an industry we focus on producing and supporting innovative ways to characterise.



Conclusion

More sophisticated tools for molecular characterisation and the ability to link this back to the biology will be instrumental to delivering full characterisation of the cell, the process and, consequently, the product. There are several new technologies that have been developed in this vein. For example, the use of gene expression and micoRNA (miRNA) profiling which allows enhanced characterisation and provides a link back to the biology. It will be important for these technologies, as they develop, to be standardised for use in the industrial setting, and this will be done as part of consortia efforts and collaborative investigations between the developers and the end users. There has been some recent news-flow on collaboration between industries to focus on characterisation standardisation and, as more and more of these cell therapies enter the market, the requirement for new tools and technologies that understand these systems will be essential. The need for standardisation within this young industry was also backed up by a survey published by the Genetics Policy Institute. This report indicated that 45 per cent of researchers working with animal stem cells and 30 per cent of researchers working with embryonic stem cells cited a lack of optimal, standardised experimental protocols. The industry therefore has an important role in facilitating the development of stem cell biology research (17).

The CTI is rapidly gaining pace and hopes to truly deliver both patient and economic benefits in an overarching healthcare sector which has not been delivering of late. In order for the full potential of this industry to be realised, there will need to be a concerted effort to establish the regulatory, legislative and technology support that is necessary to aid the development of the CTI. One can only wait with great expectation to see the products of this wave of opportunity and answer the question of fact or fiction.

References
  1. Mason C, Brindley DA, Culme-Seymour SJ and Davie NL, Cell therapy industry: billion dollar global business with unlimited potential, Regen Med 6(3): 265-272, 2011
  2. ReNeuron cell therapy programmes, www.reneuron.com/company_info/ cell_therapy_programmes
  3. Larkin C, Dendreon cancer drug faces US review tied to its sole product, Bloomberg Businessweek, www.bloomberg.com/news/2010-11- 16/dendreon-cancer-drug-faces-u-sreview- tied-to-its-sole-product.html
  4. Krassowska A, Geron to proceed with first human clinical trial of embryonic stem cell-based therapy, www.geron.com/media/pressview.aspx?i d=1229
  5. Douglas B, Advanced cell technology’s RPE cells granted orphan drug status from FDA for treatment of Stargardt’s macular dystrophy, www.advancedcell.com
  6. Schustack D, Advanced cell technology receives FDA clearance for clinical trials using embryonic stem cells to treat age-related macular degeneration, www.advancedcell.com
  7. McKernan R, McNeish J and Smith D, Pharma’s developing interest in stem cells, Cell Stem Cell 6: pp517-520, 2010
  8. Waters R and Gale J, Cephalon buys Mesoblast stake: drug rights in potential $2 billion deal, Bloomberg, www.bloomberg.com/news/2010-12- 08/cephalon-buys-20-stake-inmesoblast- rights-to-adult-stem-celltherapies. html
  9. Wechsler P, Shire’s purchase of advanced biohealing for $750 Million Hits ‘Sweet Spot’, Bloomberg, www.bloomberg.com/news/2011-05- 17/shire-s-purchase-of-advancedbiohealing- for-750-million-hits-sweetspot-. html
  10. Mason C and Manzotti E, Regenerative medicine cell therapies: numbers of units manufactured and patients treated between 1988-2010, Regnen Med 5(3): pp307-313, 2010
  11. Buckler RL, Opportunities in regenerative medicine: the global industry and market trends, BioProcess International 9(1): pp14-19, 2011
  12. Mason C and Dunhill P, The strong financial case for regenerative medicine and regen industry, Regen Med 3(3), pp351-363, 2008
  13. Longaker MT, Baker LC and Greely HT, Proposition 71 and CIRM-assessing the return on investment, Nature Biotechnology 25(5), 2007
  14. US Census Bureau, www.census.gov
  15. Deans R et al, A changing time: the International Society for Cellular Therapy embraces its industry members, Cytotherapy 12: pp853-856, 2010
  16. Characterisation of cell therapy products, BSI, Draft PAS 93, 2011
  17. Glover C and Chiu T, Filling the commercialisation gap in stem cell technologies: case studies of successful partnerships between academia and industry (The Genetics Policy Institute), The World Stem Cell Report: 150-153, 2008
  18. Recreated from Mason and Dunnill, The strong financial case for regenerative medicine and the regen industry, Regen Med 3(3): pp351-363, 2008


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Verna McErlane is Director of Commercial Operations at Sistemic and is actively engaged in the cell therapy markets. Verna gained her PhD in cancer drug development and spent time at the Gray Cancer Institute, London, and SPEAR Therapeutics Ltd prior to moving to the University of Dundee, Scotland. Subsequently, she was chosen to take part in the distinguished Saltire Foundation Fellowship programme, an experiential MBA where she spent time working on commercial cell therapy projects with Genzyme and market entry strategies for Axis-Shield. This has allowed Verna to further her passion for commercialising science into realisable patient benefits. Email: verna.mcerlane@sistemic.co.uk
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