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ISSN 1746-0751Regen. Med. (2012) 7(5), 645–648 64510.2217/RME.12.52 © 2012 Future Medicine Ltd

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Conference SceneTrends in the stem cell and regenerative medicine industry

Dusko IlicHuman Embryonic Stem Cell Laboratories, Guy’s Assisted Conception Unit, Division of Women’s Health, King’s College London School of Medicine, London, UK Tel.: +44 20 7188 0547 dusko.ilic@kcl.ac.uk

World Stem Cell Regenerative Medicine CongressLondon, UK, 21–23 May 2012

The World Stem Cell Regenerative Medicine Congress series, now in its 7th year, is organized annually in the USA, Europe and Asia by Terrapinn, a business media company with its head office in London, and has grown over the last several years into the largest and probably the most important strategic stem cell conference where regulators, investors, big pharma, and small and medium enterprises gather to share and create synergy in developing and commercializing stem cell applications. The conference, held in London on 21–23 May 2012, only confirmed that this series is the meeting to attend if you want to get a clear understanding of trends in the stem cell and regenerative medicine industry.

Chris Mason, Professor at University College London (UK), opened the con-ference and moderated the first morn-ing panel discussion on accelerating and translating stem cell research to the mar-ket. The panel pointed out that stem cell therapy actually requires the pharmaceuti-cal industry to change the approach from ‘one product for one target’ to ‘one product for many targets’. Big pharma are reluctant to enter the arena of regenerative medi-cine and stem cell-based therapy with full force and need to be convinced that there is value in this area. Success stories in the field are still not up to big pharma stand-ards in spite of over 325,000 patients that have been treated with US FDA-approved cell therapy products so far. The panel reminded us that a successful product is not only innovative and effective but also reimbursable [1].

David Willetts, UK Minister for Universities and Science, gave an over-view of the national take on innovation and emergence of the new field of regen-erative medicine driven by unmet clinical needs. The well-balanced, rigorous but permissive regulatory regime in the UK apparently provides a fertile environment for clinical trials. A third of clinical trials on stem cells in Europe are conducted in the UK. Keith Thompson, Head of Cell Therapy Catapult, an independent not-for-profit organization with core funding of GB£10 million (US$15.5 million) a year over the next 5 years from the Technology Strategy Board outlined the modus oper-andi of this translational center. The Catapult will work with businesses across the cell therapy field, coordinating activity,

expertise and information as they seek to innovate. One goal is to add a further GB£20 million (US$31 million) a year from these activities without affecting intellectual property rights of companies using the Catapult. Frank-Roman Lauter introduced a global consortium of six regenerative medicine translational centers, The Regenerative Medicine Coalition.

Gil Van Bokkelen, Chairman of the Alliance for Regenerative Medicine and Chief Executive Officer (CEO) of Athersys, spelled out and offered solutions for the main issues that hamper preclinical develop-ment: a clear need for creating better regula-tory transparency; establishment of a more streamlined and cost-effective paradigm to demonstrate consistency of cell therapy-based product or process in safety and efficacy of clinical treatment; lack of trans-parency for the efficient reimbursement process; and getting investors excited about the sector. Partnership with Pfizer, ongoing discussion with the FDA on Accelerated Approval Pathway and a Phase II clinical study investigating the treatment of inflam-matory bowel disease with their stem cell-based product MultiStem® suggest that some of the proposed solutions certainly worked, at least for Athersys.

The second panel of the morning revis-ited a need for translational centers; big pharma has no experience in obtaining reimbursement for stem cell-based therapy and small and medium enterprises cannot expect help or guidelines here. Moreover, in the field of regenerative medicine, big pharma seems to be more inclined to buy ready-to-sell products than invest in the building of a new one.

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Christopher Bravery, Director of Consulting on Advanced Biologicals, a specialist regulatory consultancy prima-rily serving the regenerative medicine industry, chaired a plenary session of the second day. Maria Cristina Galli and Paula Salmikangas, members of the EMA Committee for Advanced Therapy, gave a regulatory perspective on inspecting and guaranteeing the quality of the manu-facturing process of cell-based medicinal products (CBMPs) versus other biologi-cals [101]. The number of clinical trials in advanced therapy medicinal products (ATMPs), which are medicines for human use that are based on gene therapy, somatic cell therapy or tissue engineering, is rapidly growing in the EU, from only 22 clini-cal trials in 2005 to nearly 500 in 2011. There are over 300 companies worldwide developing CBMPs. Approximately 20–30 CBMPs approved on national markets in the EU are expected to submit a centralized marketing authorization application by the end of 2012. Both speakers re-emphasized that a dialog between authorities and devel-opers is essential for a successful marketing authorization application [2].

The panel discussion following their presentation further tackled the feasibility of the canonical Phase I, II and III clini-cal trials in bringing ATMPs, especially CBMPs, to the market. The point made was that I, II and III are just numbers, and that one has to know both product and disease in order to design an appropriate clinical trial.

Different approaches in obtaining a reimbursement for cell therapy were the topics of the next plenary session. Cytori, Advanced BioHealing, Intercytex and NeuralStem, all reasonably successful in translation of cell-based therapy from bench to bedside, presented their busi-ness development strategies and views on making profitable ATMPs in the shortest time possible. In a captivating presenta-tion, Dean Tozer, Senior Vice President of Corporate Development from Advanced BioHealing, spoke about the importance of ‘owning disease’, creating and owning the entire infrastructure through invest-ing heartily in enabling customer coding, coverage and reimbursement. Diabetes wounds, for example, can be found throughout the healthcare system, from

primary care and home health to dialysis centers and assisted living facilities, and it is practically impossible to have a tar-geted campaign that would reach out to all branches caring for these patients. The company is, therefore, dispatching business development specialists who will work with the system, being advocates for patients in order to get the referral system function-ing. The take-home message was that the key point in winning reimbursement is to take costs out of the system; improv-ing quality of life will not get the product reimbursed. Paul Kemp, CEO and Chief Scientific Officer of Intercytex, enthralled the audience with multiple examples of how a proper navigation through defini-tions of unmet medical needs and use of risk-sharing agreements could shape busi-ness plans and bring profit. Disgruntled with the FDA, Richard Garr, CEO of NeuralStem, changed gears and ended the session by advocating for medical tourism.

Using surveys of all parties involved, Edward Tenthoff, a senior research analyst from Piper Jaffray, attempted to define and address barriers to technology development and commercialization of tissue engineering and regenerative medicine products. From an investors’ point of view, three primary barriers to investment were clinical transla-tion, commercialization/reimbursement and lack of regulatory pathway clarity, with the latter being the main one. Ram Mandalam, President and Chief Executive Officer of Cellerant Therapeutics, explained how the company has shown on its own exam-ple how to advance the stem cell pipeline through mutually beneficial partnership. Cellerant is developing their universal cell-based drug, myeloid progenitor cells CLT-008, in partnership with US Department of Health and Human Services’ Biomedical Advanced Research and Development Authority (BARDA). BARDA is support-ing the development of products to treat bone marrow, gastrointestinal, lung and skin injury caused by radiation.

The panel discussion on how to get the second round of funding did not provide any new insight. We learned that Teva is looking for a innovative and blockbuster niche, but so are we all. The final two presentations, followed by a panel discus-sion, compared and discussed concepts of values and recommendations for cell

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therapies from the viewpoints of the UK’s NICE and American Center for Medicare and Medicaid Services [3].

Two streams – Cell Therapy and Regenerative Medicine and Stem Cells for Drug Discovery – were running in parallel for all 3 days. Although Cell Therapy and Regenerative Medicine drew bigger crowd, Stem Cells for Drug Discovery also had a few very interesting presentations. Here is a brief overview of three of the most inter-esting presentations from each of the two streams.

Gary Rabin, new Chairman and CEO of Advanced Cell Technology, briefed us about the company’s retinal pig-ment epithelium clinical program and mesenchymal stem cell program. No doubt, everybody was impressed with the

retinal pigment epithelium success story [4]. Donna Skerrett, Chief Medical Officer of Mesoblast, presented another success story. Pleiotropic properties of Mesoblast’s ‘off-the-shelf ’ mesenchymal precursor cells allowed diverse disease application and development of a multiproduct pipe-line that strictly follows a regulatory and commercialization pathway. A strong cash position (US$232 million) and relationship with Teva and Lonza make Mesoblast one of the strongest contenders in the field of stem cell therapy and regenerative medicine today. Eduardo Bravo, CEO of TiGenix, a company known for the first and only approved cell therapy product in Europe for cartilage regeneration in the knee, ChondroCelect®, reviewed the data from clinical trials in treatment of perianal and

Box 1. Japanese technology is entering the stem cell arena.

� Professor Takashi Asada from the Institute for Integrated Cell–Material Sciences (iCeMS), Kyoto University, gave an overview of the New Energy and Industrial Technology Development Organization (NEDO) project. The aim of this NEDO project is the “development of core technologies for industrial applications of human stem cells.” In recent years, great progress has been made worldwide in developing drug discovery screening and cell therapy applications for human pluripotent stem cells, in terms of both human embryonic stem cells and induced pluripotent stem cells. However, large-scale pluripotent stem cell culture methods vary widely across the globe and are in need of standardization. Initiated last year in Japan, this 6-year national project supported by the Ministry of Economy, Trade and Industry (METI) aims to develop not only large-scale production methods, but also the technologies necessary for stem cell evaluation. Eight companies and five academic research groups are collaborating in this effort.

� Interestingly, the majority of the main players were not coming from big pharma. Sumitomo Bakelite developed Oligosacharride Purification Kit BlotGlyco®, which is used as a cell quality evaluation reagent. BlotGlyco beads are chemically synthesized polymer particles containing high-density hydrazide groups, which form stable covalent binding with aldehyde groups at the reducing end of glycans and, as a result, can selectively capture glycans from a variety of biological samples. Shimadzu, established in 1875 and known for its high-quality analytical and measuring instruments, is developing evaluation instruments for stem cells, whereas Hamamatsu Photonics, known for its imaging and analyzing systems, opto-semiconductors, photomultiplier tubes and light sources, are now involved in stem cell imaging. Nissan Chemical Industries, founded in 1887 as Japan’s first chemical fertilizer manufacturer, is venturing into development of stem cell culture substrates and materials. Nipro (Japan), known for advancing the frontiers of minimally invasive technology, developed automated gas-permeable bag culture systems for large-scale and low-risk stem cell production. Takara Bio, with a forte in gene therapy and genetic engineering and a side business in health food products, is now developing evaluation reagents for stem cell research. In collaboration with Diagenode SA, Belgium, Genetein has developed the Automated Epigenetics System, which uses magnetic particles and a movable magnet (Magtration® technology [102]) to provide accurate and reliable information and characterization of stem cells; chromatine immunoprecipitation, methylated DNA immunoprecipitation and hydroxymethylated DNA immunoprecipitation, which discriminate between hydroxymethyl cytosine, methyl cytosine and unmodified cytosine, are fully automated.

� ReproCELL (www.reprocell.com), founded in 2003, is working on commercial development of stem cell technologies originated in the laboratories of Professor Norio Nakatsuji at the Kyoto University and Professor Hiromitsu Nakauchi at the Tokyo University.

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rectovaginal fistulas with allogeneic adi-pose stem cell platforms and outlined the strategy of progress into Phase III with an orphan indication.

Nancy Stagliano, CEO of iPierian, pre-sented impeccable and incontestable logis-tics of how to use disease-specific induced pluripotent stem cells for drug discovery, whereas Chris Parker, Vice President and Chief Commerical Officer of Cellular Dynamics International, showed how, with a good business sense, one can take an opportunity and build from scratch a company that has become, in a very short period of time, the number one provider of induced pluripotent stem cells worldwide. With examples of spinal muscular atrophy and Huntington’s disease, Arnaud Lacoste from Novartis reminded us that although big pharma might be reluctant to use stem cells for therapy, it is certainly not reluctant to use them as a tool in drug discovery.

The next Stem Cells and Regenerative Medicine Congress is focused on the USA and it will be held on 20–21 September 2012 in Boston (MA, USA). However, given the huge market (Box 1) and lack of legislation in most Asian countries, the 4th Annual World Stem Cells and Regenerative Medicine Congress Asia, which will be held on 18–21 March 2013, in Singapore, is definitively worth paying attention to.

Financial & competing interests disclosureThe author has 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 manu-script. This includes employment, consultancies, honoraria, stock ownership or options, expert testi-mony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

References1 Ilic D, Salaway T. Logistics of stem cell

isolation, preparation and delivery for heart repair: concerns of clinicians, manufacturers, investors and public health. Regen. Med. 3, 83–91 (2008).

2 Maciulaitis R, D’Apote L, Buchanan A, Pioppo L, Schneider CK. Clinical development of advanced therapy medicinal products in Europe: evidence that regulators must be proactive. Mol. Ther. 20, 479–482 (2012).

3 Jensen TS, Jacques LB. Medicare coverage: engaging on evidence. Regen. Med. 6(6 Suppl.), 99–101 (2011).

4 Schwartz SD, Hubschman JP, Heilwell G et al. Embryonic stem cell trials for macular degeneration: a preliminary report. Lancet 379, 713–720 (2012).

� Websites101 Committee for Medicinal Product for

Human Use (CHMP). Guideline on human cell-based medicinal products. www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003894.pdf

102 Magtration® Technology.

www.youtube.com/watch?v=88S-lnNol00