Tantalising results: A summary of the World Stem Cells and Regenerative Medicine Congress 2013

stem cells congress day one

The conference hall at Victoria Park Plaza, London, UK was humming with the buzz you might expect as thought-leaders in the stem cell and regenerative medicine field congregated for the 8th annual World Stem Cells and Regenerative Medicine Congress Europe 2013. As several speakers were to note over the coming three days, it is an extremely exciting time for regenerative medicine. In the past year, researchers have gathered data from human trials and the results have been tantalising. No more are these promising results just coming from trials with laboratory rats, but from real trials in real humans – and looking at the speaker list for the conference, we were sure to hear about some very exciting results indeed.

But what was most striking to me was how the conference tracks also focused on financing and commercialisation of stem cell-based therapies, really highlighting just how close this field is to achieving market penetration and delivering innovative breakthrough therapies that will transform global healthcare systems. Stem cell therapy was once so alien, a dream, just a concept – but when we start to hear discussions on the stem cell reimbursement landscape, it hits home how close regenerative medicine is to becoming a commercial reality.

It was fitting, therefore, that the morning session of day one covered exactly that – focussing on a ‘Roadmap for Commercialising Stem Cell-Based Therapies'. The session was chaired by Professor Chris Mason, of the Regenerative Medicine Bioprocessing Unit at the Advanced Centre for Biochemical Engineering, University College London. He introduced the first speaker of the morning, Lord John Krebs, Chairman of the House of Lords Science and Technology Committee, who delivered a keynote address on "Mapping a route for the commercialization of cell therapies". Regenerative medicine, highlighted Lord Krebs, was one of the government's eight priority targets. He discussed the value of regenerative medicine, and spoke about the Committee's latest inquiry into regenerative medicine. [Read more: Commercial Value, Capacity and Intellectual Property]

Following on from Lord Kreb's fascinating and insightful talk, Keith Thompson, Cell Therapy Catapult CEO, gave us some insights on the purpose and achievements of the Cell Therapy Catapult its future projections. The Cell Therapy Catapult was established in 2012 as a centre of excellence for cell therapy and regenerative medicine to stimulate innovation in this area. Mr Thompson discussed funding the so-called "Valley of Death", and he addressed the barriers leading to this translational gap. The regenerative medicine industry will be bigger than monoclonal antibodies if we do it right, said Mr Thompson. [Read more: Roadmap for Commercialising Stem-Cell Based Therapies].

To continue with the commercialisation theme, the second session of the morning was on ‘Alternative Methods of Financing.' Claudia Jiménez, Senior Director Corporate Development, TiGenix, discussed the financing lifecycle of a biotech company, and why biotech is a financing challenge. We learned from TiGenix's experience how to raise venture capital funds and what factors were key to gaining that funding. Following on from Claudia Jiménez's talk, Navid Malik, Head of Life Science Research, Cenkos Securities, presented "Alternative Financing Models: Floating On The Stock Markets To Gain Investment." He discussed the risks and costs involved in floating a company, and mirroring Mr Thompson's words early, monoclonal antibodies provide a roadmap for regenerative medicine. Grants such as those from the Wellcome Trust, says Dr Malik, are highly regarded by financers as they bestow both accreditation for the research and some level of validation to help investors breathe easy before signing new regenerative medicine deals. [Read more: The Biotech Bull and its impact on the future of Regenerative Medicine].

Following on from Dr Malik's talk, Ed Field, Chief Operating Officer, Cytomedix, presented on "Commercialisation Through Collaboration: What Does Partnering In This Industry Actually Look Like?" Apparently, like other technology areas but without the volume of deals. Pharma may currently be reluctant to get involved in partnering, but government agencies are already playing an active role with patient advocacy groups becoming more active. To conclude the session on alternative methods of financing, the audience were treated to a fascinating and in depth panel discussion which dug deep into the key issues surrounding "Non-traditional sources of finance – debating the best strategies for gaining investment". On the panel was Ram Mandalam, President and CEO of Cellerant Therapeutics, Greg Bonfiglio, Managing Partner of Proteus Venture Capital; and Navid Malik, Head of Life Science Research, Cenkos Securities. [Read more: Regenerative Medicine: Alternative Methods of Financing].

The afternoon session was split in to two sections – one on ‘Cell therapy and regenerative medicine' and ‘iPS cells for therapy'. In the former stream, late stage clinical development was the flavour of the afternoon. Dolores Baksh, Director of R&D at Organogenesis spoke on "Improving product development efficiency using scientific, risk-based approaches", which covered Quality by Design (QbD) principles in product development. Antonio Lee, Associate Director, Business Development, MEDIPOST, gave a presentation on "Cord blood cells for Alzheimer's disease and bronchopulmonary disease (BPD)". He outlined the use of umbilical cord blood for mesenchymal stem cells, and talked us through three exciting MEDIPOST products –  CARTISTEM for degenerative osteoarthritis, PNEUMOSTEM for BPD, and NEUROSTEM for Alzheimer's disease. Yael Margolin, President and CEO of Gamida Cell, gave a presentation wonderfully titled "The Gamida Cell chronicles: diary of a clinical adventure", taking us on a journey through Gameda Cell's TEPA (copper chelator) and NAM (nicotinamide) technology and their respective uses in StemEx and NiCord products. Roland Gordon-Beresford, RA and IP Senior Director, Cardio3 BioSciences, gave a presentation on "Balancing financial contrains and regulatory requirements within a clinical development plan for cell therapies". He gave a brief case study of C3BS and how they develop regenerative therapies for the treatment of cardiac diseases, before delving into the C-CURE multicentre Phase II trial and the development of C-CATHez for the Phase III trial [Read more: Trends in Regenerative Medicine Late Stage Clinical Development].

Meanwhile, next door, the ‘iPS cells for therapy' stream was running concurrently. Aidan Courtney, Chief Executive Officer of Roslin Cells was chairman for the session. Thomas Fellner, director of Innovation in pluripotent Stem Cell technologies at the contract manufacturing giant, Lonza, spoke about "Establishing a cGMP-compliant process for the generation of Induced Pluripotent Stem Cells". Dr Fellner described how Lonza are addressing the key technical and economical challenges surrounding the scale up and commercialisation of iPSC technology [Read more: IPSC manufacturing in 2013]. Marc Peschanski, Director, Institute for Stem cell Therapy and Exploration on Monogenic diseases (i-STEM), then spoke about the "European ScreenTox project: using cells derived from embryonic stem cells for predictive toxicology". He addressed the congress attendees on the recent challenges and achievements of the ‘Stem Cells for a relevant, Efficient, Extended and normalized Toxicology (SCR & Tox) programme, an EU FP-7 funded program which aims to eliminate the requirement for animal testing. SCT & Tox is focused on harnessing creating cell based toxicity assays to detect early cellular changes in toxicity pathways of a variety of cell types, and early results show stem cell derived tissue models are not only capable of filling the gap left by the animal testing ban but can also be an improvement [Read more: EU Ban on Animal Testing in Cosmetics Spurs Stem Cell Derived Toxicity Assay Development].

Back in the ‘Cell therapy and regenerative medicine' stream, the focus had turned to ‘Challenging traditional clinical development'. Christopher J Calhoun, Chief Executive Officer, Cytori Therapeutics, spoke about "Evolving clinical research into multi-centre studies". We learned about the use of adipose derived regenerative cells (ADRCs) the Celution system, an experimental thermal burn treatment funded by the Biomedical Advanced Research and Development Authority (BARDA). Francois Binette, VP Research and Business Development, Orthocyte Corporation, gave a presentation on "Tailoring effective stem cell therapies for the aging spine". Back pain presents a huge economic burden and effects nearly everybody in their productive life. Binette highlighted the large therapeutic gap between the initial inflammatory response and later structural collapse. The vertebral disc is apparently the largest avascular tissue in the body, and so is a great target for regenerative medicine as a pharmaceutical approach won't work. There is therefore a real opportunity and a real market. Binette discussed the use of PureStem and HyStem cell delivery system in orthopaedic regenerative medicine, and how the product pipeline addresses the entire care continuum for disc degeneration. To conclude the session, Paul Kemp, CEO and CSO, Intercytex Ltd, discussed "Accelerated approval for breakthrough therapies: streamlining clinical development" where he spoke about the Cell2Therapy translation service to take cell therapies rapidly into the clinic.

Day Two was equally exciting. The morning plenary sessions saw Dr Thomas Okarma, President and CEO of Asterias Biotherapeutics Incorporated, introducing Asterias as a new consolidator in regenerative medicine. Asterias is focusing on the commercialisation of proof of concept (POC) stage cell therapies having acquired and combined the vast expertise and patent portfolio of two regenerative medicine giants, Biotime and Geron Stem Cells. Whilst applauding the enormous research and development efforts of its parent companies, Dr Okarma was keen to emphasise Asterias's resolve to learn from their mistakes [Read more: Biotime and Geron combine to form Asterias Biotherapeutics]. Gil Van Bokkelen, President and CEO, Athersys, teamed up with Micheal Werner, Alliance for Regenerative Medicine Co-founder and Executive Director, to discuss "Early stage dialogue: operating reimbursement in parallel with accelerated regulatory approval". They discussed recent advances on the regulatory front, such as FDA accelerated approval and breakthrough therapies, while also discussing the reimbursement of regenerative medicine products and the tying of payment to accelerated approval. Before presenting the reimbursement plan to potential investors, a CEO should tick all the boxes of the "reimbursement checklist" [Read more: Cell therapy approval and reimbursement must pace in synch within the modern scenarios]. Professor Richard Linford, Clinical Epidemiology, Public Health and Biostatistics, University of Birmingham, then gave a very entertaining presentation on "Health economics: how to value your cell therapy product". In a speech filled with enthusiasm and humour, Prof. Linford described health economics at the supply side and the use of cost-effectiveness analysis to calculate headroom. He highlighted the importance of using decision gates to help make decisions when experts give different estimated QALYs.

As the afternoon sessions split into two streams, attendees could enjoy either ‘Cell Therapy & Regenerative Medicine' or ‘Cell Therapy Manufacture & Scale Up'. In the former, Matthew Vincent, Director of Business Development, Advanced Cell Technology, gave a presentation on "David versus goliath: how to tackle a giant indication". Paul Anderson, Managing Director, OrthoCell, discussed "Innovative approaches to the regeneration of tendon, cartilage and soft tissue injuries". OrthoCell have established a variety of products and Paul Anderson, CEO at Orthocell, illustrated features and applications of one of these, which is the first product to be commercialized for autologous tenocyte implantation: Ortho-ATIâ„¢ [Read more: Autologous Cell Therapy can treat Tendinopathy]. Following on from this, a panel discussion focussed on "The evolving role of big pharma: what are they looking for?" On the panel were Tim Allsopp, Head of Improve Outreach Programme, Pfizer Regenerative Medicine; Matthias Steger, Global Head of Research and Technology Partnering, Hoffman-La Roche; and Sven Kili, Director, Global Medical Affairs – Cell Therapy and Regenerative Medicine, Sanofi.

Meanwhile, in the ‘Cell Therapy Manufacture & Scale Up' stream, the focus was on ‘Thinking ahead: The need for manufacturing foresight'. Robert Shaw, Commercial director of the Stem Cell Initiative at EMD Millipore discussed the state of manufacturing processes in the regenerative medicine industry today, focussing on "A time for innovation: is the next phase of growth to be accelerated by the bio-manufacturing community?".  In spite of the rapid progress in clinical trials, he began, the industry is moving slowly in terms of developments in manufacturing. He attributed this in part to a lack of standardization and tools for large scale processing. Professor David Williams, Head of Group, Centre for Biological Engineering, University of Loughborough, followed with a clear message about product characterisation and comparability in his presentation "New technologies: incremental improvements and disruptive innovations". Without comparability, argued Professor Williams stated, there can be no process changes, no multiple sites, no scalability, no roll out following commercial success and, accordingly, no effective manufacturing strategy [Read more: Unite and Standardize; the Need for Manufacturing Foresight in Regenerative Medicine].

After a networking lunch, back in ‘Cell Therapy & Regenerative Medicine', speakers were focussed on the evolving regulatory landscape. Christian Schneider, Chair, CAT, European Medicines Agency, presented on "Regulatory perspective of advanced therapies and recent scientific development". He discussed the advanced therapy medicinal product (ATMP) landscape in Europe, and the involved for the Committee for Advanced Therapies (CAT). ATMPs are special, said Schneider, and rather than adapting the product to the agency's thinking, the agency needs to adapt their thinking to the product. He pointed out that the EMA has recommended some advanced therapies, such as a gene therapy, so advanced therapies are coming! Michael Werner, Executive Director, Alliance for Regenerative Medicine (ARM), presented on "What does the process of interacting with the regulatory authorities look like?" There are 141 current members of ARM, said Werner – a list that includes companies, research institutions, investors, affiliates, patient advocates and foundations. The ARM regulatory agenda is to create a predictable and efficient review and approval process that enables approval of safe and effective products as quickly as possible. Werner also discussed improving communication between the FDA and sponsors, with ARM having published a paper that provides information to sponsors regarding effective communication with the agency. Rob Janssen, Secretary-General, Alliances for Advanced Therapies (AAT), gave a talk on "Regulatory harmonization and predictability in Europe: the hospital exemption as a case". He spoke about the work of AAT in promoting the interests of the advanced therapies sector in Europe. Janssen described the need for regulatory harmonization to improve clarity and predictability, and how such harmonization was necessary for stimulating investment and for lowering costs of therapy development. He also described the Hospital Exemption (HE) and the criteria an ATMP needs to fulfil in order to qualify for HE. It became clear that there is a need for European harmonization, including a proper implementation of the HE.

As the focus turned back to the convergence of cell and gene therapy, the session ended with a talk from Rahul Aras, CEO of Juventas Therapeutics, who discussed "Commercial challenges in the next generation market: Gene therapy activating cell therapy pathways". Aras discussed Juventus' lead product, JVS-100, which is in Phase II clinical trials in heart failure and critical limb ischaemia. JSV-100 encodes SDF-1, which is a "natural repair system" that recruits endogenous stem cells to damaged organs, promoting new blood vessel formation. The product is in Phase II clinical trials in heart failure and critical limb ischaemia. Aras also spoke about the development of JVS-200 which encodes monocyte-specific cytokine 3 (MCP3) as an adjunct to enhance autologous and allogeneic cell therapy strategies.

Meanwhile, in ‘Cell Therapy Manufacture & Scale Up', Progenitor Cell Therapy's (PCT) President and chief scientific officer Dr. Robert Pretti spoke about "Creating an efficient, robust and cost-effective cell therapy development and commercialization program". The emphasis was on the strengths of utilizing contract research and manufacturing organizations at the early stages of therapy development and commercialization [Read more: Contract Development and Manufacturing Organisations- Why Would You?]. Michael May, President and CEO, Centre for Commercialization of Regenerative Medicine (CCRM), discussed "Potency assays for thorough product characterisation and efficient commercialisation" [Read more: Predictive surrogate assays for potency could drive efficient clinical translation and commercialization].

Alongside conference roundtables on translational research, financing models, regulatory approval, iPS cells & disease modeling, strategic partnering, cord blood banking and reimbursement & market access, the morning of Day Three focused on ‘Designing an economic process’. Jose Castillo, Director of Cell Culture Technologies, ATMI LifeSciences, spoke about “How to drive overall cost of goods down with the right process in place”, focusing on ATMI’s Integrity Xpansion Multiplate Bioreactor. One Xpansion biorector is the equivalent to 20 open systems. Eric Halioua, CEO, Promethera Biosciences, also discussed cell therapy and scale-up. Promethera is a cell therapy company dedicated to the treatment of liver diseases, and the audience learned about the use of Promethera’s processes in treating inborn errors of liver metabolism – such as Crigler-Najjar disease, urea cycle diseases, phenylketonuria and glycogen storage disease type 1. Suzanne Farid, Associate Professor, Department of Biochemical Engineering, University College London, discussed “How can the cell therapy sector achieve cost-effective manufacture at the commercial scale?”. Suzanne Farid highlighted the need for more advanced tools to determine the most cost-effective strategies early on in development. She discussed the scope of UCL decisional tools, which focus on process synthesis & facility design, and portfolio management & capacity planning.

The morning also included talks from Alexander Vos, CEO, PharmaCell, Klaus Kuehlcke, Managing Director, EUFETS GmbH, and Robert Sexauer, EVP Clinical Development, IntelliCell BioSciences. Aby Mathew, Senior Vice President and Chief Technology Officer at

BioLife Solutions, spoke about "Ensuring the appropriate cryopreservation of your stem cell-based therapy" and gave us some heads up about the importance of ensuring biopreservation at all levels (upstream and downstream) of the bioprocess for the successful commercialization of cell therapy products [Read more: Critical stability and Bioprocess consensus for manufacturing, storage and clinical delivery of cell and tissue products].

The World Stem Cell and Regenerative Medicine Congress 2013 closed with two talks on ‘Adapting to the Current Environment for Future Success’. Richard Garr, Director & CEO, Neuralstem, discussed “Understanding the practicalities of using innovative therapies on patients”. He discussed Neuralstem technology in amyotrophic lateral sclerosis (ALS), spinal cord injury (SCI), ischaemic stroke and glioblastoma. In the case of ALS, the cells synaptically integrate and therefore this is not a transient therapy. He discusses the success of the NSI-566/ALS phase I trial, which showed the therapy to be safe and also showed signs of efficacy. 5 out of 6 ambulatory patients showed improvement or very slow progression of the disease post-surgery. Richard Garr also discussed the plans for the future with ALS trials, and also NSI-566/SCI and NSI-566/Ischaemic stroke. The conference closed with a very entertaining talk from Fiona Fox, Chief Executive, Science Media Centre, who discussed the importance of interacting with the media about scientific discoveries for accurate reporting.

The list of presentations above is by no means exhaustive, as many other fascinating talks were given over the next few days. However, I'm sure you'll appreciate that it would not be possible to cover them all in one write-up!

Were you at the 8th annual World Stem Cells and Regenerative Medicine Congress Europe 2013. What were your event highlights? You can join our discussion on LinkedIn, or leave a comment below. Want more from Total BioPharma? Sign up to our newsletter – it doesn't cost anything and only takes a minute.

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