|
Memorandum submitted by the East of England Stem Cell Network (Bio 23)
INTRODUCTION
1. This evidence is submitted to the Parliamentary Committee on Science and Technology (the (Committee") on behalf of the East of England Stem Cell Network ("EESCN", the "Network"; www.eescn.org.uk) by its Managing Director, Julian Hitchcock, in response to the Committee's invitation of 4 November 2009. 2. EESCN was established in 2004 with the objective of ensuring that the East of England prospers in terms of both economic output and patient benefit from stem cell biology and regenerative medicine. The Network supports the development of regenerative medicine innovation and enterprise, facilitating collaborations and attracting inward investment in an open innovation framework. Its aim is to secure Cambridge's position as an international centre of excellence in regenerative medicine. 3. EESCN's method is: • to foster an integrated "ABC" culture, in which Academics, Businesses and representatives of the Clinical sector (including patients) are in frequent contact with one another, for example by means of its regular "ABC" meetings; • to build upon the region's preeminence in stem cell science, physics, computing, material science, manufacturing and standard setting, for example by advancing inter-disciplinary projects and introducing disruptive thinking; • to identify bottlenecks, opportunities and value in the emerging regenerative medicine economy, so as to direct skills and effect introductions to meet them; • to facilitate public engagement and education in regenerative medicine and stem cell science; • to advocate the interests of members in public policy, law making and the regulatory arena; and • to act as an international gateway to, and ambassador for, the region's regenerative medicine capability. 4. EESCN presently funds one of its Directors, Julian HItchcock, to run the Network on a one-day-per-week basis. In reality, Mr Hitchcock and his fellow Directors spend considerably longer supporting the enterprise, taking significant time out of their senior day jobs to do so. 5. EESCN is part funded by the East of England Development Agency ("EEDA"); part by a small number of sponsors. At the time of writing, EESCN anticipates that further funding may become available on a match-funding basis from EEDA, although neither the match-funding nor EEDA funds are confirmed.
What is the UK's research capacity?
6. The fundamental and translational research capacity of the EESCN region is world class and substantial. Over 30 groups, at the University of Cambridge and at surrounding research institutes such as the Babraham Institute, are conducting stem cell/regenerative medicine projects such as: • basic research, including developmental genetics, cell biology, genomic imprinting, cell signaling and tissue engineering; • interdisciplinary research, including stem cell physics, biomaterials and manufacturing; • translational research, including for example the clinical application of stem cells for neuronal repair and pancreatic islet transplantation. 7. The EESCN region also enjoys the supportive clinical environment of the Cambridge University Hospitals NHS Foundation Trust.
How easy is it to translate and commercialise research?
8. The EESCN region is also notable for the intensity of its scientific entrepreneurship and for its culture cluster. It includes institutions such as the Judge School of Business and Centre for Entrepreneurial Learning actively actively involved in regional life. Cambridge Enterprise, the technology transfer arm of the University of Cambridge, is a very professional company which successfully exploits technologies arising from the University's research. 9. Nevertheless, it is extremely hard to translate and commercialise regenerative medicine research, especially for consortia. Case Study 1 10. The following case report from Dr Su Metcalfe sets out her personal experience of attempting to progress a novel medical device with "huge implications in healthcare" and how she has been impeded by discontinuous support within the UK. The events are from 2009. Background 11. "Through my research at the University of Cambridge I have identified novel biological activities linked to the stem cell growth factor "LIF". 12. "Technical innovation created a novel medical device for targeted delivery of LIF and other growth factors in vivo. The device incorporates LIF within a biodegradable nanoparticle coated with targeting antibody. The materials are FDA approved. 13. "Indications include (i) neurodegenerative diseases; (ii) immune dysfunction; and (iii) stem cell therapy. For example, in the UK alone, over 500,000 people suffer from incurable neurodegenerative disorders of the brain, costing several billions to the NHS. 14. "The University of Cambridge chose not to patent the device. 15. "Ownership of the IP was assigned to myself from UofC and I self-funded patent of the device. NIHR i4i Initiative " Invention for Innovation" (phased funding scheme (FPD1 - 4)) 16. "The NIHR i4i strategic initiative provided a well structured route for translation and commercialisation of the nano-LIF. FPD1: Proof of Concept Study i4i (1y) 17. "Support from the i4i FPD1 funding scheme led to successful PoC of nano-LIF as a novel medical device for immune modulation in both mouse and Rhesus monkey models." FPD2: Translation and Commercialisation i4i (3y) 18. " 'The main objective of an FPD2 project is to build on the results of a completed assessment of the feasibility to produce a medical device or product through technological improvements or developments and that provides further evidence of its capacity to deliver improved healthcare outcomes and commercial opportunities' Translation 19. "Based on the successful PoC under FPD1, a bid for continued support under the FPD2 scheme was made, entitled 'NANO-THERAPEUTIC DELIVERY OF LIF FOR BRAIN REPAIR'. 20. "This bid included myself and was led by Dr Roger Barker, a clinical neurologist (Brain Repair Unit University of Cambridge) who is a world specialist in both Parkinson's Disease and Huntington's Disease, and member of the MRC Stem Cell Liaison Committee. As co-applicants we directly linked "bench to bedside", driving forward innovation in an entirely novel treatment for neurodegenerative disease." Commercialisation 21. "A start-up biotech company was established to progress commercial development of nano-LIF and related technologies. Directors included international expertise in biotech start-ups with focus on stem-cell-related initiatives. 22. "The FPD2 bid was rejected at the preliminary assessment stage. Current Status General 23. "Given the lack of UK funding support, new collaborations within the USA are advancing our work, including three groups at Harvard University and one group at Yale. 24. "The start-up company has been dissolved." Intellectual Property 25. "Preliminary filing disclosure by Yale University on the LIF-nano device for treatment of neurodegenerative diseases is in place. Other filings via Yale are planned. 26. PCT and National Patents on linked technologies remain owned by myself." 27. Dr Metcalfe adds a personal comment: 28. "The UK funding structure for translation of research and its commercialisation is strongly biased towards consortia. This funding structure should not be at the expense of small groups with proven track record in innovation and strong networking skills. (NB: relatively low cost). 29. "I funded from my own pocket the IP protection of the inventions rejected by the UofC. 30. "I funded from my own pocket significant sums toward the Biotech Company that was established for commercial progression of this IP." 31. Dr Metcalfe refers to the NIHR Invention for Innovation Programme's i4i Future Product Development Funding Opportunities (FPD2): Criterion 1: There needs to be strong evidence of clinical need and high relevance to the NHS" 32. Dr Metcalfe replies in two words: "Neurodegenerative diseases" Case Study 2 33. A second case study reached EESCN directly after a Board meeting of the otherwise promising spin out company, Odontis. Professor Paul Sharpe writes: 34. "I am founder and scientific director of a university stem cell spin-out company Odontis, and I have just returned from Odontis had been funded by a 3 year Wellcome Trust strategic Translation award that had been running for 2 years. However because it was clear that we would not have a "product" ready for translation by the end of the third year, Wellcome decided not to continue funding for the final year." 35. "The basic problems in the UK in this field are: (I) "Milestone based funding is not appropriate for cell-based research. (II) "The timescales for taking research from Discovery" to Translation are unrealistic. (III) "The is no provision in the system for flexibility. A project like ours is making progress but we reached a stage where we needed to spend some time going back to basics in order to progress further. This is not acceptable in translational funding."
How do UK and international regulations affect research and translation?
4. In addition to my role as Manager and a Director of EESCN, I am an intellectual property and life science lawyer specialising in the law and regulation of cells and tissues and, especially, regenerative medicine (based at Field Fisher Waterhouse LLP, London). I have been the intellectual property representative to the UK National Stem Cell Network's Working Committee on commercialisation and have recently co-written the chapter on European intellectual property law landscape for a forthcoming textbook, edited by Professor Dame Julia Polak and Dr Cathy Prescott, on commercialising regenerative medicine. I also sit on behalf of EESCN as an interested party at the Committee for Advanced Therapies of the European Medicines Agency ("EMEA") 5. I am very familiar with the regulation affecting this area. As a lawyer, I have for example, acted for NHSBT in connection with a range of stem cell trials and on the Wellcome Trust funded collaboration for the production of embryonically derived human red blood cell concentrates and for Axordia in connection with the regulatory aspects pertaining to the use of the first stem cell line banked at the UK Stem Cell Bank. 6. I do not propose in this written evidence to provide a detailed review of the regulation. However, I can report my overall feelings. 7. In my judgment, the level of regulation is unavoidable and essential. In effect, European regulation, and UK regulation in particular, may exact considerable international advantage to the UK insofar as it may provide an assurance of quality. The same demands drive the value of new enabling technologies, thereby providing new strategic opportunities. 8. However, there is no doubt that well-meaning efforts of the relevant authorities (HFEA, HTA and MHRA) to set out a road map of regulation, have had the opposite effect. The "Monopoly Board" diagram serves to confirm suspicions that this is at least a complex area. Faced with such hurdles and such a long path to eventual success, few private investors will opt for regenerative medicine. In fact, the regulatory position,while certainly demanding, is less complex than it at first appears. 9. I do not think the solution to this complexity is to unify the relevant agencies (an idea already rejected during passage of the HFE Bill in connection with the HFEA and HTA). Nor do I think a unifying piece of legislation is required. What is needed is some activity and experience for the rules to work upon. With government support for clinical trials, we may gain somewhat. 10. A matter of some concern are the commercialisation rules of the UK Stem Cell Bank in relation to cell progeny. Essentially, the concern is that a right of access to cells in effect deprives owners of the exclusivity bought by their investment in the research. However, I feel that there may be some basic confusion going on. I should be happy to discuss if called to give oral evidence. 11. The Committee's questions are directed towards regulation as opposed to law. However, I do have some concerns about the way in which induced pluripotent stem cells may be treated in law, notably in the light of the recent WARF decision by Enlarged Board of Appeal of the European Patent Office and the revised definition of "embryo" under the Human Fertilization and Embryology Act 2008 (which amends the 1990 Act of the same name). 12. In my personal judgment, the bases set out in the Biotechnology Directive should also be subject to further review as technology and attitudes develop. My concern is that valuable research may be hampered by imaginary or theoretical ethical problems.
How CAN THE UK MAINTAIN AND GROW ITS INTERNATIONALLY COMPETITIVE POSITION? 13. I have conducted research (with King's College London) into the stem cell patenting policies of China and India and am familiar with the positions in the United States and European territories. 14. Undoubtedly, regulation plays a key role in success in this area. 15. I will be able to provide more detail under oral examination, but these brief comments may set the scene. 16. First, I very much doubt that governments have fully grasped the economic significance of regenerative medicine. In principle, a technology with reduces the rocketing cost of degenerative disease and makes societies more economically competent ought to be worth a vast amount to governments, insurers and individuals. By implication, those territories with the most key patents will tend to be yet better off: in effect, by exacting royalties from the others. Further, the economic activity associated with the endeavour will have benefits in terms of employment. 17. This suggests that government should address regenerative medicine in the most strategic way. 18. A related aspect of this concerns the use of intellectual property generated in the UK. 19. In September 2009, the Vine Street meeting (which I organised) brought senior stakeholders and policy makers together for the first time to discuss the potential of patent pooling in regenerative medicine. This had been mooted four years' previously by the Pattison Report, but never done. The meeting concluded that the technology was developing so fast as to make it very hard to identify essential platform technologies, but that greater coordination among technology transfer officers, acting under the guidance of regenerative medicine expert technology finders, could help to bring together relevant UK and foreign intellectual property to create valuable new intellectual property. (A copy of the Vine Street Meeting report appears at www.ffw.com/pdf/Patent-Pooling-Report.pdf ). 20. However, the Vine Street participants were clear that greater funding is required: that UK companies could not afford technology which was then acquired (cheap) by US companies. East of England Stem Cell Network |