Regenerative medicine - Science and Technology Committee Contents


APPENDIX 5: VISIT TO CALIFORNIA INSTITUTE FOR REGENERATIVE MEDICINE (CIRM), UNITED STATES


Members visiting: Lord Krebs (Chairman), Lord Cunningham of Felling, Lord Patel, Baroness Perry of Southwark and Lord Willis of Knaresborough. In attendance: Mr Chris Atkinson (Clerk) and Professor Fiona Watt (Specialist Adviser).

Monday 3 December—Wednesday 5 December 2012, five members of the Committee, (accompanied by the Specialist Adviser and Clerk) visited the California Institute for Regenerative Medicine (CIRM). The aims of the visit were to learn from the work of CIRM, to see some of the groundbreaking translational work being undertaken in California, and to learn from the experience of those who have successfully commercialised regenerative treatments.

Day One

Introductions and welcome

Senator Art Torres, CIRM Board member; Dr Alan Trounson, CIRM President; and Ian Sweedler, CIRM Senior Counsel for International Programs, welcomed the Committee on behalf of the agency, Governor and Mayor. The "unique experiment" of CIRM was discussed including the proposition to create it (passed in 2004), the general obligation bonds which fund it, and the focus on getting treatments to patients.

Panel one

The Committee then met Dr Anne-Marie Duliege, Affymax and CIRM Board Member; Dr Edward Lanphier, Sangamo Biosciences Incorporated; Dr Thomas Okarma, BioTime; and Dr Edward Penhoet, Alta Partners and Member of the President's Council of Advisors on Science and Technology (by telephone), to discuss biotechnology venture funding and the biotechnology environment in California.

It was suggested that the Bay Area biopharmaceutical environment was extremely dynamic. This success was attributed in part to historical funding. Some were less optimistic currently because of the lower availability of capital, and because regulation was more significant and stringent. When specifically discussing stem cell research it was suggested that the path was less certain and consequently venture capitalists were not yet ready to support it widely so the Government should step in—as CIRM does. It was argued that it remains to be seen how costly it will be to bring stem cell to patients. It was noteworthy that the FDA had shown flexibility when it came to clear unmet medical need and orphan drugs, but on the whole it was perceived as becoming more conservative—wanting more certainty about efficacy and safety.

There was undoubtedly spectacular science in the field of regenerative medicine. To unlock patient benefit, research had to be encouraged, capital for translation provided, access to patients established and economic benefit demonstrated. It was argued that relying on federal government funding to adequately enable basic and early translational research was not sustainable and so private sector solutions and private sector incentives had to be sought. But as one moves away from drugs and monoclonal antibodies it was very hard to raise venture capital. Venture capitalists needed to see how they could make money and a near term return. Creating incentives for "big pharma" to invest would also be valuable. It was suggested that CIRM was a great alternative for capital, but not a long term solution to creating an economic model that drives incentives for early investment.

It was argued that there was less venture capital for autologous cell therapies, gene therapies and other regenerative medicines because there had been fewer successful business models when one compared regenerative medicine companies to other investments possibilities such as technology. Big pharmaceutical companies now have venture funds and are investing in this space. They can receive a tax free return on it from the investment tax credit. It was suggested that "the pull" through from basic to translational work was currently low because few products had got through successfully. One strategy to jumpstart the field and attract investment was investment in an array of opportunities so see quicker returns.

The decision of Geron to stop supporting regenerative medicine and to halt its spinal cord injury clinical trial was set out as a case study of how hard it was to do truly innovative work. Possible factors influencing that decision included the economic burden of developing human embryonic stem cell therapies, the long timeline for a return on investment and the significant risks involved. Relevant assets had been acquired by BioTime who would take the work forward but finding investment to do that had not been easy.

The importance of continued good relationships between the biotech industry and academic research was underlined. President Obama was very interested in maintaining the country's leadership in biotech and had commissioned his Council of Advisers on Science and Technology to undertake a study on the drug development process.

In further exploring the ecosystem of venture capital funding it was suggested that a quick return was always valued. Timeliness of return on investment in regenerative medicine was not consistent with investor expectations or wishes.

Finally, the difficulties associated with patenting regenerative medicine were compared with those in biotech. A comparison was drawn between the 20 years of research to optimise monoclonal antibodies before industry ("big pharma" and biotech) were convinced of the science and clinical application. It was suggested that because much of the invention in regenerative medicine was occurring in industry, this was riskier for investors.

Panel two

The Committee discussed manufacture, scale-up and GMP for cellular therapies, and clinical development of non-cellular therapies with Dr Gerhard Bauer, University of California (UC) Davis; Dr Patricia Olson, CIRM Executive Director of Scientific Activities; and Dr Phil Vanek, Lonza.

The Committee heard presentations about ongoing clinical work in UC Davis, including work to develop an HIV gene therapy treatment and collaborative work with Stanford University to manufacture induced pluripotent stem cells to treat epidermolysis bullosa. UC Davis does its GMP work in-house and also contracts out those facilities—around 40% of its contracts are private ones. Its GMP facilities are run on a quasi-commercial basis. It has six fully operational suites, which are running at capacity. CIRM had invested $12.5 million in this facility.

If a CIRM funded technology reaches a certain level of commercial success then a small portion of revenue from that goes back to the state general fund to repay taxpayers for investment in this research. The CIRM model was discussed further. Teams are encouraged to think early about how they will scale-up and manufacture any potential treatment. CIRM provides lots of tools and support for researchers such as webinars and access to consultants. The work of a disease team is milestone-driven and has specified outcomes. The CIRM model would be explored in greater detail later.

CIRM co-funds work with the UK MRC, China, Australia and other partners all over the world. They are very focussed on getting work into the clinic. Proposals submitted in response to requests for applications (RFAs) are evaluated by panels of reviewers who have expertise in various areas in addition to experts in the particular disease area. CIRM has a pool of reviewers (of approximately 150). They particularly encourage applications from multidisciplinary teams.

Lonza have been working on manufacturing challenges associated with cell therapy for around 12 years. It is seeking to answer the question: how can it help this industry materialise on a cost-effective practical basis? It considers key bottlenecks or challenges, and works to develop possible solutions. These challenges include keeping cells consistent, viable and recoverable in downstream processing.

Lonza starts with the end in mind: how can this treatment be mass produced for a patient population? Delivery at scale has many practical challenges such as dose and logistical issues. It was argued that manufacturing could not continue at current scale: Lonza wants to invent technologies that start with a lot size of 500-100 and to manufacture 5, 000-10, 000 doses per lot. These issues need consideration now before we run out of raw materials, such as serum. Automation and scale-up will be achieved through the next generation of technologies such as suspension bioreactors, and these new technologies could impact the development process.

CIRM provides some funding for considering these issues through its tools and technologies stream. The Committee then discussed delivery systems with the panel, including the specific example of how a macular degeneration treatment could be delivered to thousands of patients. Difficulties with achieving patents for processes were then discussed and it was suggested that patents were easily designed around. Transportation and shipping problems were discussed: there were specific needs for cryopreservation, validation and guarantees of time from manufacture to clinic. Down the line, a hospital-based cell pharmacy might be a necessity for allogeneic treatments. The question of whether one should bring the patient to the therapy or the therapy to the patient was raised. One model for addressing some of these issues was the Alpha clinic network which CIRM was exploring the feasibility of.

Panel three

The Committee met with Dr Larry Goldstein, UC San Diego and Sanford Consortium for Regenerative Medicine; Dr Michael Longaker, Stanford University; and Dr Thomas Rando, Stanford University and Palo Alto Veterans Affairs Medical Center, to discuss interdisciplinary centres and perspectives on the state of regenerative medicine science.

The Sanford Consortium for Regenerative Medicine is a partnership with independent charitable status, comprising universities and research institutes in the San Diego area. Through its layout and ethos it seeks to promote interdisciplinary working, in recognition of the need for collaboration between clinicians, scientists and engineers to deliver new treatments. It is striving to develop organisational systems to reward co-operation, and is bringing together groups to accelerate the movement of fundamental science into clinical applications.

The business model for regenerative medicine had not yet been proven. It was suggested that it was equally possible to develop commercially successful but medically less useful products as useful medical products which were not a commercial success.

Interdisciplinary research from collaboration between Stanford University and Veterans Affairs Medical Centres was discussed. They have a specific interest in disorders that often affect veterans and receive funding from the US Department of Defence. It was suggested that rehabilitation and regeneration go hand-in-hand—seeking to restore function and tissue. The Department of Defence also funds an Armed Forces Institute of Regenerative Medicine (AFIRM) which is a multi-institutional, interdisciplinary network working to develop advanced treatment options for our severely wounded servicemen and women.

Stanford University provides "accelerators" to progress basic science through to translation and commercialisation. It draws together legal expertise on IP and ethics, business skills to consider the business model, and the knowledge of the engineering school to drive entrepreneurship. Its medical centre has raised funding to build a therapeutics centre and it hopes to prevent the stem cell institute being an isolated "ivory towers". It will do clinical trials with bone marrow and stage four breast cancer in the first instance. One central office considers licensing and Stanford has a handful of excellent examples of patent return. Faculties often form companies and license use. If Stanford can't license it then they either drop prosecution of the patent or the investigator is free to start up a company to do so.

A question was raised about whether it was helpful to compare the model for IP and equity sharing during the technology boom with the situation now for stem cells and regenerative medicine. It was suggested that what was needed was to diversify risk by spreading it across a well-filled pipeline because regenerative medicine was perceived as high risk science and investment.

The CIRM disease team model was discussed further. It was thought that of the first round of teams at least seven of the 14 teams would get to clinical trial. The benefit of a four year deadline was a "flurry effect" of activity. Two projects are already in clinic. Academics had bought into the model relatively quickly. Where necessary, additional expertise and management could be brought in to help so that teams met their milestones. Typical investment in a disease team was around $20 million over four years. Each would consist of four or five investigators as well as six to ten people in labs.

It was suggested that biomedical science and engineering was "living off the fruits" of investment 10, 15, and 20 years ago. The private sector would not make investments in twenty year ROI propositions—so there was a role for the public sector to play. One of the major returns on investment in regenerative medicine would be a reduction in healthcare costs. In its more recent RFAs, CIRM had highly encouraged corporate partnerships which they argued was realistic as, because CIRM providing some of the capital investment, they were helping de-risk the proposal.

The Committee was encouraged to "be bold". Those who drafted proposition 71, which established CIRM, were now considered to be visionaries. The UK has an extraordinary scientific community. It needed to take risks in supporting this field. Disease has an enormous cost (for example, Alzheimer's Disease in the US has healthcare costs of $250-500 billion a year) not just from healthcare costs but in lost wages, the social bill and other indirect costs. A "can-do" approach like that of California was desirable. The UK needed, like CIRM, to build in front of its researchers: to think forward and prepare the space for where they are going. It was further suggested that money wasn't enough—incentives were needed and providing scientists with a way to do it. It was also important that universities recognised the value of translational and commercial work; assessment of the quality of science shouldn't rest solely on numbers of papers published. The importance of collaborative working was again stressed. Training grants were one lever to encourage medics to engage with research.

The UK Stem Cell Bank was described as "incompetent and intransigent". Dr Larry Goldstein had a very negative experience trying to secure the use of two cell lines in his research to the point that he gave up and used lines from elsewhere.

Panel four

The Committee then discussed models for translation through industry-academic relationships, including collaborations, spin-offs, and licensing with Dr Karen Aboody, City of Hope; Dr Dennis Clegg, UC Santa Barbara; Dr Peter Coffey, UC Santa Barbara; Dr Henry Klassen, UC Irvine; and Dr Clive Svendsen, Cedars-Sinai.

The Committee heard about the research and businesses of these researchers. For example, therabiologics was a spin-off company whereas jCyte Inc employed a virtual company model whereby it licensed the IP. It was suggested that, in the current economic climate, investors were very risk adverse and so researchers had to take development further than previously was the case before industry would step in. Industry was reluctant to pick up trials before they had phase II data. Academic-industry and philanthropic partnerships were possible solutions to this dual valley of death (as financing phase I trials was also problematic).

The California Project to Cure Blindness had some "big pharma" and VC interest already if it were taking its work to a phase III trial.

The London Project to Cure Blindness had been severely delayed by unclear interactions with GTAC. Professor Coffey was frustrated by delays and considered the UK regulatory pathway to be extremely complex. In contrast, he spoke highly of his interactions with the MHRA.

Cedars-Sinai hospital was a medical centre with a science and clinical side in the same hospital. Their focus is personalised medicine, and potentially getting stem cell therapies for a wide range of diseases. Medical centres were one important model for translation because they can do R&D without the commercial pressures. It was argued that private health insurers should be convinced of the savings afforded by regenerative medicine and also encouraged to invest.

CIRM host quarterly webinars with the FDA. It recognised that regenerative medicine is a learning process on both sides: for the FDA and people working in research. Through these webinars, meetings and papers CIRM seeks to help people understand what is required of them by regulators and to educate the regulators on the developing science. It was suggested that the FDA was getting much better at handling regenerative medicines.

Day two

Panel one

The Committee met with Mr Louis Breton, Calimmune; Dr Paul Laikind, Viacyte; and Mr Martin McGlynn, StemCellsInc, as witnesses from regenerative medicine companies in the translational through clinical stages.

Calimmune has the ambition to be the first company to provide a one-time cost effective HIV therapy. It was developing a combination therapy which was based on a natural mutation whereby people who lack CCR5 receptor have complete protection. It was about to embark on phase I/II trials in the US and Australia, and had investigator-initiated studies in the UK and France. Calimmune secured private investment because there was a well-developed and strong science base underpinning it. The company benefited from around 14 interactions with the FDA before submitting for IND (investigational new drug) approval.

Viacyte explained its VC-01 combination product which functions as a replacement pancreas delivering cells which differentiate to insulin and other cofactors and delivered using a propriety encapsulated delivery system. It was soon to begin phase I trials. This could be a cure for type one diabetes and an effective therapy for type two diabetes. CIRM's enthusiastic support for the project had been crucial.

StemCellsInc focuses on the central nervous system (CNS) and the liver. It started by developing an encapsulation technology and now sought to address unmet medical needs through the development of stem cells as therapeutic agents to treat damage to or degeneration of major organ systems. It was founded by four prominent academics. The company had benefited from the increasingly collaborative approach of the FDA and recommended that it become as much advisory as regulatory.

It was suggested that, in general, IP was not as valuable or useful in the reagents world as it was in that of therapeutics because prosecuting patents was very expensive and time consuming, and reagent life cycle can be very short.

The companies were already thinking about scale issues. A key challenge was demonstrating to regulators that stem cells could be reproduced at scale to the same, regulatory-required standard. Scalability was considered a critical requirement for attracting finance.

The attraction of the Australian R&D tax incentive was discussed. Views were mixed on whether "cash" or tax credits were more desirable. A further facet of CIRM's provision, namely its loans scheme, was discussed.

Panel two

Regulatory obstacles, pathways and engagement were discussed with Dr Lauren Black, Charles River Laboratories; Dr Joy Cavagnaro, Access BIO; Dr Ellen Feigal, CIRM Senior Vice President of Research and Development; and Dr Thomas Okarma, BioTime.

Geron's IND application was the first received by the FDA for an embryonic stem cell-derived therapy and the largest it had ever received (21, 000 pages). Geron had to invest substantially in animal modelling to demonstrate efficacy.

A lot was asked of FDA reviewers: to assess INDs at relative pace and to take a view on whether they were ready for humans and, if so, at what dose. The FDA was, however, viewed as a well-informed regulatory body. Regenerative medicines are much more complex than drugs and so there was a lot of uncertainty. To reduce some of the uncertainties, investment in animal modelling could greatly improve confidence. The majority of regulatory files submitted to the FDA Center for Biologics, Evaluation and Research's (CBER) Office of Cellular, Tissue and Gene Therapy were from research sponsors rather than commercial ones.

Insufficient harmonisation was identified as a problem—for example, Apligraf is regulated in different countries as a device, a biological or as a medicinal product. Unique and novel therapies can be daunting to regulators. The FDA was beginning to work internationally—such as its pilot programme of parallel scientific advice with the EMA. Dialogue was critical to its learning. Similarly, academia needed to understand more about assessing safety, efficacy and potency. CIRM has done a lot of work to educate investigators. It is uniquely placed to bring people together to increase knowledge on all sides. Webinars are one tool that CIRM use.

It was suggested that industry wants regulators to tell them what to do but they can't always because they don't have sufficient information on the various technologies to provide general guidance. One recent example of guidance the FDA had finally issued was Draft Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products- although it was suggested that this guidance document could become quickly dated as advancement in these fields were rapidly developing. Ways to improve the functionality of the FDA were discussed. There were mixed views about the efficacy of the FDA and the merits of the UK regulatory system.

Comparisons were drawn between the use of surrogate markers for HIV/AIDs and the need for similar initiatives to support orphan conditions, to increase the number of trial approvals. Any good regulatory framework for cell therapy needed to involve consultation with scientists, industry, the public and regulators. Patient advocate groups could be a powerful voice for change. It was suggested that the public needed better educated about risk-benefit.

CIRM bring in regulatory experts to support their disease teams. The FDA has also started approaching CIRM for assistance in gathering information or hosting events.

Panel three

Dr Alan Trounson, CIRM President; Dr Irv Weissman, Stanford University; and Mr Ian Sweedler, CIRM Senior Counsel for International Programs, discussed international collaborations with the Committee.

Professor Weissman described his scientific research and his experiences of commercialising this work. His CIRM funded leukaemia disease team was developing therapeutic antibodies directed against surface markers present in much larger amounts on LSC (leukaemia stem cells that are responsible for maintaining the disease) than on the surface of normal blood forming stem cells. This project is a collaboration with Dr Paresh Vyas at Oxford University, supported through CIRM-MRC collaborative funding.

He argued that the UK had better infrastructure for clinical trials than the USA because of its unified healthcare system and highlighted the potential for reimbursement this also provided. He observed that a permanent cure with one treatment required completely radical health economic models and pricing strategies. He continued: big companies will not invest until they are shown that it's a business for them.

Difficulties encountered trying to equip patients to make informed decision about unproven treatments were then discussed. The example of private cord blood banks making unproven claims about treating genetic diseases was given.

Alan Trounson recommended talking to academics about what they needed and founding a UK agency that delivered on that vision: assess where scientists are going and ask "what do they need to make this effective?" The UK should encourage collaboration and support scientists. He also introduced the concept of Alpha clinics which CIRM was exploring to deliver therapies.

Initial reactions from "big pharma" about the possibility of partnering with CIRM and gradually taking greater ownership (and providing more investment) as trials progressed from phases I-IV were positive. The sometimes conflicting desires of business executives and clinicians were discussed. The potential of investment from insurance companies was also considered. Investment by the Veterans Association was further explored.

It was also considered necessary to create a "revolving door" attitude in universities whereby it was normal and indeed recognised as valuable for academics to take leaves of absence to set-up companies.

Panel four

The Committee discussed regenerative medicine health care delivery barriers with Dr Graham Creasey, Stanford University; Dr Natalie DeWitt, CIRM Special Projects Officer; Dr Benton Giap, Santa Clara Valley Medical Center; Dr Steve McKenna, Santa Clara Valley Medical Center; Dr Bruce Quinn, Foley Hoag; and Dr Alan Trounson, CIRM President.

Some results of the (initially Geron run) stem cell based thoracic spinal cord injury treatment trial were discussed. The importance of looking, initially, for evidence of effect rather than cure was underlined. Issues surrounding patient identification and recruitment and multi-site trials were discussed. Research networks and logistical models needed further development. One of the possible solutions to difficulties with trial design was earlier interaction with regulators about outcome measures. The FDA was considered to be actively encouraging early interactions. Adaptive licensing was also discussed.

CIRM's alpha clinic network model to build clinical infrastructure to deliver cell therapeutics was considered further. These clinics would help identify what would work well for stem cell therapy trials, as well as helping define practical needs such as human resources. They could also work to help improve public perceptions, through education and counselling work.

The Canadian, German, US and UK healthcare systems were compared, including their reimbursement mechanisms. The benefits of the NHS as a single healthcare system were again highlighted.

Day three

Panel one

The Committee met with Ms Elona Baum, CIRM General Counsel and Vice President of Business Development; Dr Ellen Feigal, CIRM Senior Vice President of Research and Development; and Dr Alan Trounson, CIRM President, to discuss funding for research at various stages from translational through clinical—the "valley of death" and the CIRM model.

CIRM is seeking to build pathways to cures and accelerate relevant research. The cost of healthcare, as set out in analysis in a recent Ernst and Young report, is spiralling and regenerative medicine offers a hope for containing them. But, fundamentally, CIRM wanted to see patients made better. Their model is helping academics optimise their clinical development of research in such a way that it is investment ready.

CIRM has a strategic partnerships award to attract industry engagement and investment in CIRM funded stem cell research. The intent of the Initiative is to create incentives and processes that will: (i) enhance the likelihood that CIRM funded projects will obtain funding for phase III clinical trials (e.g. follow-on financing), (ii) provide a source of co-funding in the earlier stages of clinical development, and (iii) enable CIRM funded projects to access expertise within pharmaceutical and large biotechnology partners in the areas of discovery, preclinical, regulatory, clinical trial design and manufacturing process development.

This initiative requires applicants to show evidence of either having the financial capacity to move the project through development or of being able to attract the capital to do so. This may be evidenced by, for example, (i) significant investment by venture capital firms, large biotechnology or pharmaceutical companies and/or disease foundations; or (ii) a licensing and development agreement with a large biotechnology or pharmaceutical company or a commitment to enter into such an agreement executed prior to the disbursement of CIRM funding. CIRM strategic partnership awards are evaluated by scientists but they also have business and product development experts on the panel.

CIRM funding can be seen by other funders and industry as a validator—it lends credibility to research. This is true in terms of attracting "big pharma", small business innovation research and private interest. CIRM have spent a lot of time at the interface with angel, VC and pharma investors, showing them the potential in the field. To attract these groups in, CIRM are thinking creatively about how to interact with them—for example, offering them mentoring roles to projects and organising conferences.

Disease team management was discussed in greater detail. Success criteria and milestones are set and agreed in advance. Funding tranches are tied to these. A formal milestone review process is in place. Outcomes of these review meeting are the green light to go forward because they are on the right track, recommending a change of track or a change in milestones if that is realistic, or to terminate the project. CIRM can convert a disease team project back to translational research with reduced scope and budget if necessary. CIRM has withdrawn funding from underperforming projects. In between milestone review meetings, CIRM work with the teams to undertake: progress reports, annual reports, visits and regular phone calls. CIRM not only fund—they nurture, support and fund. CIRM is teaching external agencies about its milestone process and suggested that collaborative funders depend on them for this expertise. Finally, problems around shaping requests for applicants were discussed.

Panel two

The Committee then discussed financing models for regenerative medicine research and development with Dr Jonathan Thomas, CIRM Governing Board Chair; and Dr Alan Trounson, CIRM President.

The sale of general obligation bonds in California was discussed, including the CIRM bond as agreed by proposition 71. CIRM is funded by 30 year bonds. Ultimately, it is intended that this investment will be offset by reduced healthcare costs. The bonds are bought up quickly as they are seen as a good investment. CIRM has been exploring options for finance after the period covered by the bonds.

Bob Klein and political leaders including Governor Schwarzenegger had been instrumental in getting the proposition passed. Other countries have expressed interested in the finance model. Stem cell research in the US is being supported privately, including by philanthropists, and so other possible funding models include "venture philanthropy" as many philanthropists are interested in curing disease. Private health insurance might be a further source of investment. The establishment of public-private partnerships in the area would be helpful, perhaps even mega funds. A general principle observed was that investment attracts investment: when CIRM invested up to $20 million in Viacyte (who are developing a diabetes therapy), the juvenile diabetes foundation brought an additional $5 million to the project on the strength of CIRM's investment. The initial investment in CIRM was seen as a "pulse" that would start the ball rolling of investment in this field.

CIRM undertake a lot of outreach work. But they are careful not hype too high because that could destroy the integrity of its message. Finally, CIRM's governance structure was discussed.

Conclusions

The Committee then deliberated on key "take home" message from the visit and agreed the following:

Funding

·  Phase I and II clinical trials are unlikely to be funded by the private sector—the Government cannot expect this.

·  The importance of public-private partnership (private coming off the back of public). The necessity of incentives (Australian model). Is exploring a public bond a possibility?

·  There is a significant difference between cell therapies and drugs: they are so different that you can't generalise.

·  Different health economic models are required because potentially one could have one-time treatments with a higher up-front cost which offered long-term savings. The example was given of "curing" diabetes rather than managing it.

·  Does the UK have an incentive structure for academics setting up companies? Lessons should be learnt from the Stanford model, including the importance of a culture of being able to step from academia to industry and back.

Delivery and scale

·  For some treatments there will be a need for significant thought about how one delivers lots of cells to lots of patients around the world.

·  GMP facilities. Is there a possibility of a smaller number of facilities in the UK bringing more in? Could they have a more commercial model? They should draw in external users.

Regulation

·  There was conflicting evidence about the efficacy of the UK system but agreement on the need for greater engagement between regulators and stakeholders. There might be value in funding work on appropriate regulatory models.

·  It would be helpful if regulators were proactive in advising people rather than reactive to applications.

CIRM model

·  CIRM is transformative not just by providing money but through its leadership. We were impressed by the disease teams model—bringing people together to do things that mightn't do separately.

·  "Be bold", take risks, don't expect 100% success.

·  Four year target for getting to clinic; go-no go milestones; and support to achieve. CIRM truly did "lay down the gauntlet". It has impressive possible outcomes. Its interventionist style is markedly different from the UK's.

Other points of note

·  Critique of the UK Stem Cell Bank.

·  The unique advantage of the NHS for clinical trials.

·  The value of MD PHDs and the importance of opportunities for clinicians to work in labs.

·  A need for better public education.

·  Exploit the possibility of using the NHS to bring in international work. Do not fear contract working.

·  Good examples of hype and hope—such as private cord blood banks.

·  Positive examples: ARMD, HIV, artificial pancreas to treat diabetes.

·  The value of "can-do" collaboration. The importance of networks.


 
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