NIHR University College Hospitals Biomedical Research Centre

Progress on implementing our revised strategic plan NIHR University College London Hospitals Biomedical Research Centre

At the NIHR University College medicine activities and ensure we can make a London Hospitals BRC we have leading contribution to national rare disease been making brisk progress in our initiatives. UCL’s first rare diseases conference took relentless focus on world-class place in February 2013. activity of greatest therapeutic or Professor Bryan Williams’ appointment to the new diagnostic potential. As we enter our BRC Director post has strengthened our leadership. new phase we have expanded this Since August the four programmes have been led focus to drive forward strong and by substantive programme directors (PDs). The PD enduring partnerships with the role has required significant commitment (four life-sciences industry, both in the UK consultant PAs) from four outstanding international and internationally. leaders in experimental medicine: David Linch; Nick Wood; Bryan Williams; Deenan Pillay. All four are or As we push through home grown scientific have been NIHR investigators. discoveries into patient care, we are partnering Our new resource allocation models explicitly small medium enterprises (SMEs) and major target the translational potential of activities industry to share expertise, infrastructure and in nanotechnology, gene therapy, cell therapy/ resources. We are going to be flexible and creative regenerative medicine, bioengineering and in the ways we help create health and wealth. computer science. There are signs that the We are signing major new strategic partnerships increased focus on experimental medicine is with global pharmaceutical companies. A new impacting on the UCLH research portfolio. Between UCL partnership with Eisai Pharmaceuticals is 2011 and 2012 there was a 16 per cent increase now underway to develop novel therapeutics in the number of new early Phase clinical trials for neurological disorders. We are working with approved at UCLH. Over the same period we have GlaxoSmithKline to facilitate drug development for seen a 15 per cent increase in the number of fibrosis. We have also signalled our intent to occupy commercial contract clinical trials approved. space at the GSK supported Bioscience Catalyst We have seen an increase in investigator-led clinical in Stevenage to fast track home grown drug trials involving industry partnerships – reflecting development. Other new collaborations include changing attitudes to industry partnership and work with BioMarin Pharmaceuticals on gene UCL’s impressive track record in translational therapy for haemophilia. funding streams. Examples include a partnership with US-based BioMarin in gene therapy for Scientific focus haemophilia A and projects supported by the MRC- We have condensed activities AstraZeneca translational research partnership in from our previous theme mechanisms of disease. structure into four programmes Progress in our BRC Programmes over the last 12 – Cancer, Neuroscience, months includes: Cardiometabolic Science, and Infection, Immunity and • Drug discovery alliance with Eisai Inflammation. This structure is reflected in the Pharmaceuticals in which researchers from both UCL domain and UCLH clinical board structures, organisations will work together to investigate promoting easier science pull through and radical new ways of treating neurological maximising our leverage of the expertise, facilities diseases such as Alzheimer’s, Parkinson’s and and patient populations of our partner hospitals. other related disorders. The aim is to identify and validate novel drug targets, and develop We are, for example, leveraging the clinical, new therapeutics and evaluate them in proof- research and teaching expertise the partnership of-concept clinical trials. A joint Therapeutic offers in rare diseases. A new registry of rare Innovation Group of UCL and Eisai scientists diseases has been established to focus experimental will be formed to coordinate the discovery and

2 Progress on implementing our revised strategic plan

assessment of emerging therapeutic targets in Resource model neurological diseases. Research is expected to be carried out at UCL’s new Leonard Wolfson Review of existing BRC funding Experimental Neurology Centre, while Eisai commitments will provide drug discovery and development A review in 2012 of existing BRC funding resource and know-how, assay development commitments has enabled us to make significant capabilities and medicinal chemistry expertise. progress in using a new resource model that If successful, UCL will also receive milestone incentivises world-class activity and rewards and royalty payments on therapies brought to leverage: market. • Review of BRC funded staff – the activity • £500k investment in the MRC Neuromuscular of all BRC-funded staff (Consultant PAs and Centre at Queen Square, integrating our non-Consultant WTEs) has been reviewed and BRC activities with Great Ormond Street and research activity falling outside of the new Newcastle BRC activities. BRC strategy has been moved onto alternative • £900k investment in the Wolfson Drug Discovery appropriate funding streams – typically Research Unit with a focus on new interventions for Capability Funding (RCF). The newly refocused amyloid depletion budgets for Consultant level staff are maximising opportunities for engagement of NHS clinical • industry partnership with Cellectis Therapeutics researchers with UCL scientists. in gene and cell therapy, made possible by BRC investment in advanced therapies development • NHS Support Costs – We have simplified programme at UCL the methodology used to allocate funding for NHS Support Costs in line with the DH AcoRD • increased capacity in early phase clinical trials principles. in inflammatory arthritis with the launch of the BRC-supported Arthritis Research UK Centre for • Overheads – a detailed review of BRC Adolescent Rheumatology Research, including overheads has resulted in a clearer itemisation of a newly appointed Clinical Senior Lecturer in the BRC’s indirect costs. adolescent rheumatology The net effect of our financial review has been to • initiation of high throughput screening identify a core budget uplift from April 2013 of studies of novel agents including cell death in approximately £4.5m a year. osteosarcomas and head and neck cancer New strategic priorities for spend • further developments in the prognostic impact Our programmes have started to fund new of the mutational spectrum in acute leukaemia, initiatives in line with the key principle that BRC including new insights into the development of funds must, wherever possible, be used to leverage drug resistance in solid tumours ‘blue chip’ research or industrial income. We have • joint UCL-Yale MedTech conference in London to done this, for example, by: priming major grant leverage expertise and new product development applications (such as the MRC Neuromuscular across the two sites. Centre and the ARUK Centre for Adolescent Rheumatology); providing matched funding for initiatives and cross disciplinary collaborations (such as the country’s first PET/MRI facility); and allocating funds for the innovation pathway of high impact discoveries showing promise in therapeutics, diagnostics or MedTech.

3 NIHR University College London Hospitals Biomedical Research Centre

Devolved budgets to programmes Integrating NIHR resources As well as refocused staff budgets each programme We have continued to integrate NIHR infrastructure was allocated an initial £2.5m for 2012/13 to at UCLH and UCL. We have appointed a new deploy within their programmes. By reviewing Clinical Director Nursing to the CRF leadership funding commitments, we have been able to make team. The CRF is the home for our NIHR significant uplifts to the programme budgets from Experimental Cancer Medicine Centre activities. April 2013, according to their size and impact. All UCLH NIHR Clinical Core infrastructure budgets and high Research Network funding impact initiatives allocation is channelled through a new Research £2.5m has been allocated to the BRC Education Support Hub structure. NIHR and Training Programme to help attract and train Research Capability Funding the best of the next generation of clinical scientists (RCF) continues to be an in experimental medicine. extremely constructive funding We have allocated funding to support core stream for us. As well as supporting initiatives in experimental medicine infrastructure, including the later phase research such as health services research BRC leadership and operations teams, the Joint and clinical trials, RCF has been used to fund Research Office, the NIHR BioResource and the Consultant PAs not closely aligned with the BRC PET/MRI. We have carried out an internal review experimental medicine focus. The BRC uses RCF to of our NIHR Clinical Research Facility to ensure it is fund specialist regulatory project managers in gene optimally structured and supported. and cell therapy clinical trials – to help increase in the number of advanced therapy trials. A BRC strategic budget of approximately £10m has been established to support high impact, cross thematic, experimental medicine initiatives that will Governance deliver a step change in our experimental medicine The new BRC Executive Board came into effect capability. These initiatives will, by their very nature, from April 2012. Chaired by the BRC Director, the signal the intent of the new BRC to innovate at Executive Board membership consists of the BRC’s scale. A ‘High Impact Initiatives’ call went out in four programme directors and Education & Training February 2013. Several proposals have already Director, Director of R&D (UCLH), NIHR Dementia been identified for potential support, including BRU Director, BRC Chief Operating Officer and a partnership with UCL Enterprise, aligned with BRC Head of Finance. The Executive Board meets the recent Higher Education Funding Council for monthly to provide the key executive function for England Catalyst bid. the BRC, including assisting the Scientific Director in resource allocation decisions. Leveraging charitable support The BRC Strategic Board, chaired by the UCLH In the last year the BRC has consolidated its link Corporate Medical Director on behalf of the with the UCLH Charity. All new research legacies CEO, oversees delivery and development of the will be made available to the BRC for deployment BRC strategy. Its membership consists of BRC by the BRC Board. The BRC deploys charitable Scientific Director, UCL Vice Provost (Health), funds to support an experimental medicine Fast Managing Director UCL Partners, external industry Track research grant call. The most recent call representation, BRC Chief Operating Officer (November 2012) generated an unprecedented and BRC Head of Finance. The strategic board level of interest – a six-fold increase in applications, is also seeking to add two additional industry including many new groupings of NHS clinical representatives and includes a lay member. The researchers and UCL scientists. strategic board reports to the UCLH Board of Directors.

4 Progress on implementing our revised strategic plan

The four BRC programmes each have boards that resources from across UCL and UCLH, clarify the support the Programme Director and provide route for industry partners to engage with UCL/ leadership and direction for the experimental UCLH and bring the culture change needed to build medicine agenda. The programme boards include the industry portfolio. representation from the UCLH and UCL leadership, The BRC management team works closely with the as well as lay membership. UCL Translational Research Office (TRO) which was Each programme is supported by a Programme set up with pump priming support from the BRC. Operations Manager with responsibility for The TRO leads on projects aimed at promoting coordinating activities, Trust-UCL liaison, monthly industry partnership in experimental medicine metrics reports, resources management and through project managing new research projects industrial liaison. and negotiating new collaborations. The BRC has committed funding of £900k to the Enterprise, innovation and Wolfson Drug Discovery Unit which focuses on industrial partnership drug discovery in amyloidosis and other diseases Translating enterprise and innovation into creating depleting amyloid may have significant impact, health and wealth is a major and explicit strategic such as dementia. objective for the BRC. Our aim is to be national In 2012 we established a new Innovation Office for leaders in this area. We already have a growing UCLH which is providing support for staff with new portfolio of industry interactions and partnerships inventions. The Innovation Office is linked closely reflecting the impact of this new BRC strategy. with UCL Business. The BRC’s Proof of Concept UCL is committed to our partnership with (POC) fund continues to be administered by UCLB. GlaxoSmithKline in the Bioscience Catalyst in The POC fund, together with UCL Enterprise, is Stevenage. We are working closely with Cambridge supporting the novel pilot project Tissue Access for University on this and our recent bid to the Higher Patient Benefit, which is attracting considerable Education Funding Council for England Catalyst industry interest. Fund is a signal of our intent in this regard. We have identified three initial experimental medicine Education projects suitable for the new partnership. UCL has established an integrated Academic The new drug discovery alliance between UCL Careers Office (ACO), led by the BRC Director of and Eisai is an example of our focus on discovery Education and Training and supported by the BRC. and assessment of new therapeutic targets The ACO supports and helps develop academic and for neurological diseases, and we are currently clinical academic careers in the School of Life and considering options for leveraging experimental Medical Sciences (SLMS), the BRC and Dementia medicine opportunities from it. BRU. Five projects have been initiated by the ACO in the last year: The BRC Director is now a member of the UCL Enterprise Steering Committee which plays a key Clinical Research Training Fellowships role in developing the UCL enterprise agenda The BRC and the have and provides a link across large-scale enterprise created a framework to recruit interdisciplinary initiatives. The BRC Strategic Board includes Clinical Research Training Fellows (CRTFs). The representation from industry. We have drawn up BRC is jointly funding two CRTFs in 2013, and has plans for aligning the BRC’s secured funding support for a further two CRTFs industry-focused initiatives from the other two UCL BRCs. This initiative aims under a new BRC Enterprise to recruit the best trainee doctors to our three-year Partnerships concept so we PhD programme, with BRC funding used to support can better promote what the experimental medicine. If this pilot is successful, BRC has to offer industry, we will propose extending the framework to all leverage the expertise and UK BRCs.

5 NIHR University College London Hospitals Biomedical Research Centre

MiniMD two-week intensive course for Delivering through wider non-clinical scientists engagement The course offers basic scientists a direct insight into translational research and clinical medicine Engagement with national priority through out-patient clinics, ward rounds and initiatives operating theatre lists. The aim is to increase the We are actively engaged in the NIHR number of scientists pursuing experimental medical BioResource. questions and to encourage early-career biomedical UCL hosts all the National BioResource meetings. scientists in a translational direction. The first We have appointed a BRC BioResource Lead session of MiniMD will run in 2013. (Nick Wood) who is also the national lead for Experimental Medicine Conference Series Neuroscience, and we have appointed a senior (EMCS) BioResource Coordinator to establish essential The aim of these one-day conferences is to develop infrastructure and operations across the BRC. The interactions between the BRC programmes and local BioResource at UCL will act as the foundation between the programmes and external audiences. for studies of genotype to phenotype relationships. The EMCS will start in 2013 and will be delivered We see the development of these large phenotyped over the next five years. and genotyped populations as an essential capacity for experimental medicine across the UK. The UCL Gender equality BioResource will make a significant contribution The ACO is to the national endeavour by providing access coordinating the BRC’s to samples and data from 10,000 of the unique activities in relation population of volunteers and patients consenting to the Athena SWAN for recall to studies by genotype and phenotype charter, working through the UCL Partner Hospitals. This will closely with UCL. provide a powerful resource for studying disease Self-assessment teams mechanisms and the benefits and limitations of have been established emerging therapies. Samples will be collected, across SLMS and a processed and stored at UCL-RFH Biobank and UCL Women network data will be curated and stored at UCL. The BRC had its first meeting in has also explored other key opportunities for the January 2013. Athena BioResource, for example, mobilising the rare SWAN is referenced on disease cohorts at the GOSH NIHR BRC and in Royal the new BRC website and the BRC lead has been Free Immunology. appointed to UCL’s 50:50 gender equality group Data Integration and Informatics Programmes that develops policy on equality and diversity across all of UCL. We have been actively engaged in discussions around the CMO’s Grand Challenge since the The BRC has also recently committed to project’s inception. A project team has been supporting the highly successful UCL MBPhD established to ensure we have the flexibility and Studentship Programme the ability to act quickly as the national project The Programme has an exemplary 20-year track develops. The Centre for Health Academic record of developing academically-minded medical Partnership in Translational E-health Research students who have gone on to successfully (CHAPTER) is a consortium led from UCL bringing combine research with clinical medicine. together UCL, the London School of Hygiene Continued BRC support will secure re-orientation and Tropical Medicine, Queen Mary, University of this programme towards building capacity in of London, the Health Protection Agency, UCL experimental medicine. Partners, and MRC Clinical Trials Unit. CHAPTER, which is funded by a consortium of funding bodies including MRC and NIHR, will act as an

6 Progress on implementing our revised strategic plan

international centre of excellence in innovative The BRC Faculty has also been reinvigorated with health informatics research that will establish a clearer definition of the categories of Faculty translational research programmes, build capacity, membership – Senior Faculty, Faculty and Associate develop methodologies and promote engagement Faculty. The BRC Faculty includes NHS clinical between public, patients, clinicians and researchers. researchers and UCL scientists from the medical The BRC has recognised the importance of local sciences and health related faculties including harmonisation of ongoing activities around engineering and physics. bioresourcing, electronic patient data integration, bioinformatics and funding streams, in order to Engaging patients and public ensure effective project management and strategic The appointment of a Communications and PPI investment. Manager has enabled the BRC to offer more MRC/NIHR Phenome Centre support to researchers in preparing their research for the wider media. We have delivered a four-fold We have had one successful expression of interest increase in the number of research stories published in response to the Phenome Centre’s first call, on our BRC website over the last 12 months. A utilising our unique dataset in healthy humans greater focus on communications has also enabled exposed to environmental hypobaric hypoxia. the BRC to develop a culture of engagement with UCL Partners and harmonisation across London the NIHR Communications Unit. Further examples As part of our role in translating fundamental of recent progress made in our communications science into translational research we have and PPI activities include training sessions for significantly bolstered our work with the UCL researchers in PPI and a PPI facilitation service; Partners Academic Health Science Centre. We are introductory training for lay people carried out in represented on the UCLP Board, and our Scientific partnership with Macmillan Cancer and the NIHR Director contributed to the research strategy and BRCs at GOSH and Moorfields; and a BRC Summer bid prospectus for the UCLP Academic Health School funding lab placements for 20 gifted Science Network proposal and was part of the ‘A’ level students. We led a novel photograph UCLP interview panel for this bid. Likewise, the exhibition “Gathering Light” which highlighted UCLP Managing Director sits on the BRC Strategic the relationship between clinical researchers and Board. This is facilitating a London-wide focus patients. The exhibition is now touring other and role for the BRC. We are working with the centres, nationally. Our PPI initiatives have been NIHR BRCs at GOSH and Moorfields and the NIHR featured by the NIHR as case studies, notably our BRUs in dementia (UCL) and cardiovascular disease partnership working with Ataxia Research and our (Barts Health) on harmonising research efforts and PPI brochure. We have also secured £20k from the resources and fostering even greater collaboration Wellcome Trust for the development of patient and common approaches to BRC performance groups and a bursary funding pot. management. Our Joint Research Office has been a major contributor to the pilot project on harmonisation of clinical trials approvals which is delivering encouraging outcomes in terms of approvals timelines and industry engagement. Engaging the UCLH/UCL community A launch event for the new experimental medicine focussed BRC was held at the Wellcome Trust on 17 December 2012 and attended by over 130 staff. The event also marked the launch of our new BRC website (www.uclhospitals.brc.nihr.ac.uk) and the first edition of a new BRC e-Newsletter that is circulated to over 1,000 staff.

7 The National Institute for Health Research University College London Hospitals Biomedical Research Centre is a partnership between University College London Hospitals NHS Foundation Trust and UCL (University College London) and is part of the National Institute for Health Research.

www.uclhospitals.brc.nihr.ac.uk www.uclh.nhs.uk www.ucl.ac.uk 1st Floor, Maple House 149 London W1T 7NF Telephone: 020 7679 6639