Regen: the industry responsible for cell-based therapies

Abstract

Regenerative MedicineVol. 4, No. 6 ForewordFree AccessRegen: the industry responsible for cell-based therapiesChris Mason and Elisa ManzottiChris Mason† Author for correspondenceAdvanced Centre for Biochemical Engineering, University College London, Roberts Building, Torrington Place, London, WC1E 7JE, UK. Search for more papers by this authorEmail the corresponding author at chris.mason@ucl.ac.uk and Elisa ManzottiFuture Medicine Ltd, Unitec House, 2 Albert Place, Finchley Central, London, N3 1QB, UK. ; www.futuremedicine.com Search for more papers by this authorEmail the corresponding author at e.manzotti@futuremedicine.comPublished Online:10 Nov 2009https://doi.org/10.2217/rme.09.72AboutSectionsPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInRedditEmail Today, we have a global healthcare industry dependant upon three main pillars: pharmaceuticals, biopharmaceuticals and medical devices. To this list will soon be added a fourth and equally important new pillar, the cell therapy industry. Whilst presently small in overall size and poorly defined in composition, it is none the less universally recognized as an emerging new healthcare sector capable of delivering substantial health benefits as well as sustainable wealth creation.Cell therapy industryIn simple terms, each of the three current pillars represent a different platform technology with unique core competencies, namely, small molecules, biologically produced molecules and devices upon which each of the particular major healthcare sectors is built – pharma, biotech and the medical device industry, respectively. The different underpinning technologies, chemistry (pharma), molecular biology (biotech) and engineering (medical devices), define the expertise and products of the specific industrial sector. Each has their own strengths, weaknesses, opportunities and threats. But all contribute to healthcare and the capturing of the commercial value of their respective core technology. Likewise, the emerging cell therapy industry with its living cell-based products has its own unique set of challenges. For example, cell therapies are undoubtedly a disruptive technology requiring not only novel discovery, development, manufacture and marketing of living products, but also an entire service infrastructure to support their deployment in the market, including appropriate regulation, reimbursement and clinical expertise. In addition, new business models need devising in order to capture the value of cell- and tissue-engineered products. Unfortunately, current pharma and biotech models do not seem to be adaptable to cellular therapies. In part this is due to the final living product and its manufacturing process being totally inseparable, coupled to the spectrum of opportunity ranging from patient-specific therapies that are service orientated, through to universal commercial products that are more capable of scalable manufacture. A more complete list of specific barriers to the cell therapy industry has been published by Plagnol et al. in the July 2009 issue of Regenerative Medicine[1]. Just as for biotech, the new industry is not just about technology, but also about financial, social and ethical, political, regulatory and market forces that shape today’s world [2]. The cell therapy industry, like biotech before it, must also demonstrate that stem cells are not just an esoteric science with little promise for substantial and sustainable financial returns [3]. The regen flagship company, Organogenesis (Canton, MA, USA) is already well on the way to being a $100 million p.a. turnover company, but this is just a start.ScopeBig pharma, biotech and the medical device industry all cover a huge diversity of medical specialities and applications, so too the cell therapy industry. These include or are likely to include: ▪ Permanent cell-replacement therapies▪ Bioaesthetic cell-based treatments▪ Cell cancer vaccines▪ Immunomodulation cell therapies▪ Transient cell therapies that disrupt/reduce natural diseases progression▪ Gene therapy using cells as delivery vehicles▪ Tissue engineering▪ Xenografts▪ Organoids/primordia▪ Conventional cell, tissue and organ transplantationThe list above is for humans but could equally be applied to veterinary applications. Conventional cell, tissue and organ transplantation is included since there are increasing overlaps with the new and emerging cell therapy industry, including the need for significantly better preservation of donor organs [4] and common immunology challenges [5]. Which are regenerative medicine and which are not depends upon one’s definition of regenerative medicine. Using the brief definition, “regenerative medicine replaces or regenerates human cells, tissue or organs, to restore or establish normal function” [6,7], we would suggest that all the above could be included under the regenerative medicine umbrella. All certainly have the vision to deliver therapies that avoid pure mechanical solutions and/or lifelong pharmacotherapy. Regenerative Medicine actively supports all these various different cell therapy approaches. Indeed, this November issue is typical, in that the majority of the different living cell-based methodologies are included amongst its diverse content.Regenerative medicine products are not only cell basedHowever, to quote Shakespeare, “All that glistens is not gold”, regenerative medicine products are not only cell based. Small molecule drugs, recombinant proteins and even medical devices (e.g., scaffolds for tissue engineering) all have an important role to play. Whilst the term ‘regenerative medicine’ is well understood, the term ‘regenerative medicine industry’ is unfortunately confusing. Many believe it to be the cell- and tissue-engineering community only. However, the clinical delivery of regenerative medicine requires the products from all four pillars of healthcare either singularly or in combination. A few years ago, Dunnill and Mason proposed the term ‘regen’ as “the industry that develops and sells regenerative medicine products” [7,8]. In its subsequent use, the definition has been narrowed to, “the industry responsible for cell-based therapies”. This term has been increasingly adopted by a number of leading individuals, organizations and events, for example the Genetic Policy Institute and their 2009 World Stem Cell Summit. Whilst we are not precious about the exact term used, we do firmly believe that the industry that is based on ‘cells as therapies’ is both distinct and requires its own identity if it is to flourish and reach its full potential. Whilst regenerative medicine is broad and draws upon pharma for small molecules, biotech for products of recombinant DNA technology and the medical device industry for scaffolds, it fundamentally relies upon the regen industry for its cell-based products and services.Today, the regen industry is undoubtedly in its infancy. It is recognized by patients, the public and governments to have huge potential but at present is only just starting to make an impact. The analogies to other industries based upon paradigm shifts is clear. For example, the personal computer was clearly a disruptive technology to the mainframe computer industry. At the start micro-computers, later called personal computers, were not as capable as the established mainframes. However, over time, personal computer technology evolved, new business models were developed and their value in the workplace and home became established [9]. This gradual emergence of efficacy is exactly the same route that cell-based therapies are now undergoing [10]. The regen industry with its disruptive cell-based therapies and yet to be defined business models is already starting to challenge the status quo and may eventually produce and deliver advanced living therapies and cures for many of the most difficult chronic and degenerative disorders that effect the global population. Disruptive healthcare technologies are the fundamental drivers of real changes in the prevention, treatment and cure of human diseases and disorders. The addition of the fourth pillar of products to the clinician’s armoury is highly welcome; however, to deliver living cells as therapies, a clearly defined industry is needed – one that focuses on the core technology of living cells just as the fledgling biotech companies embraced recombinant DNA technology in the 1970s. This fourth pillar needs its own voice and lobby groups to specifically champion cells as therapies [11,12].Pharma, biotech and medical devices are all outstanding at producing therapies based on their core competencies. Recombinant eryrthropoetin and bone morphogenic protein are both phenomenally successful billion dollar products [13]. Both act in a regenerative manner but both are products of the biotech industry. The frequent claims that the regenerative medicine industry is already a multibillion dollar sector are therefore highly misleading when the vast majority of this figure is regenerative successes from the established biotech industry. Far better to acknowledge that the cells as therapy industry is at present a few hundred million dollars per year but growing rapidly to become a fully competitive and sustainable forth pillar in its own right. For example, this would present an unambiguous baseline to governments, states and other funding agencies wanting to support the emerging cell therapy sector but are confused by the present multibillion dollar claims.The reason why we need to define the industry is that cells as therapies are a paradigm shift in healthcare, their successful delivery depends upon an industry that has totally different needs to the present and very well established three pillars of healthcare. The light bulb did not evolve out of the candle industry, likewise cell therapies did not evolve out of the present healthcare industry. The lesson of history is that step-change technologies are driven by new companies totally focused on their particular disruptive innovation. The first products are rudimentary, the technology evolves and the industry finally flourishes. To quote a leading biotech pioneer, Roberto Crea, writing in 1993 about the “biotech class of 1982–83” and his advice to would be biotech entrepreneurs, “Be prepared for total involvement and struggle” [14]. Total involvement means focus. The focus of the fourth pillar of the global healthcare industry is very simple: it is cells as therapies. Regen is the industry responsible for cell-based therapies.Financial & competing interests disclosureThe authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.No writing assistance was utilized in the production of this manuscript.Bibliography1 Plagnol AC, Rowley E, Martin P, Livesey F: Industry perceptions of barriers to commercialization of regenerative medicine products in the UK. Regen. Med.4(4),549–559 (2009).Link, Google Scholar2 McCormick DK: So, we have survived again. Biotechnology11(3),243 (1993).Crossref, Google Scholar3 Drews J: Into the 21st century. Biotechnology and the pharmaceutical industry in the next ten years. 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This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.No writing assistance was utilized in the production of this manuscript.PDF download