Open Session MEH Board

Joint Research (MEH and UCL IoO)

Produced by:

Professor Sir Peng Khaw & Professor Phil Luthert

Board of Directors Meeting

20 November 2014

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Item 1

Open Session MEH Board

MEH Trust Board Report November 2014 – Joint Research (MEH and UCL IoO)

1. Introduction This paper provides an update on research funding and output performance across

Moorfields Eye Hospital (MEH) and UCL Institute of Ophthalmology (IoO) to indicate the

progress being made against implementation of the Joint Research Strategy (JRS).

2. Context Summary

This is the first full year of the JRS Implementation. The timing for the joint strategy is ideal in

terms of positioning the organisations for the key future milestones including:

1. National Institute for Health Research Biomedical Research Centre (NIHR BRC) &

Clinical Research Facility (CRF) award renewals in 2016-17 worth potentially >£33M.

2. Sir Jules Thorn Clinical Investigation Centre: New facility build for 2020 worth >£5M.

3. Academic Health Sciences Centre (AHSC) of UCL Partners (UCLP) Eyes and Vision

Programme.

4. Working with the Crick Institute from 2015.

5. Punching above our weight in translational medicine for the UK’s > £1.72Bn public

sector research fund.

6. Delivering on the UK life-science economy and Health to Wealth agenda.

The unified organisational vision and demonstrable championing of research excellence is

pivotal to ensure that our global leadership position is maintained in life-changing research

for patients.

Global competition is fierce and the scarcest resource is research talent. Keeping,

developing and acquiring talent in areas of excellence or potential areas of excellence is the

priority. Providing the right environment for such people to flourish is imperative.

Supporting Prof Sir Peng Khaw and Prof Phil Luthert in their leadership roles is the

Joint Strategy Board (JSB) Chaired by Sir Roger Jackling and membership includes John

Pelly, Declan Flanagan and Prof Alan Thompson (UCL). This Board oversees the

prioritisation, resource deployment, progress and delivery of the joint research strategy.

The support mechanism for the JSB and the Researchers is the Joint Management

Team comprised of the Moorfields Eye Hospital (MEH) and UCL Institute of Ophthalmology

(IoO) managers, who operationalise, facilitate and deliver outputs and results.

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Open Session MEH Board

3. Background

a. Research Funding

2007/08 2008/09 2009/10 2010/11 2011/12 2012/13 2013/14£0

£2,000,000

£4,000,000

£6,000,000

£8,000,000

£10,000,000

£12,000,000

£14,000,000

Moorfields Eye Hospital Research Funding

Other

Commercial (Supported & Sponsered)

Research Council

Special Trustees

Charity

NHS/NIHR grants

NIHR FSF/RCF

CLRN Funding

DoH Culyer funding

NIHR CRF

NIHR BRC

Figure 1: Research Funding to MEH 2007 – 2014.

• The funding gap created by the phasing out of Department of Health (DoH) Culyer has

been replaced by National Institute for Health Research (NIHR) funding (BRC, Research

Capability Funding (RCF), Research Network (CLRN/LCRN)

• Growth in NIHR infrastructure / experimental medicine research funding has occurred

year-on-year from 2011/12, driven by increased BRC funding (2012-17; including NIHR

Rare Disease Translational Research Collaboration) and NIHR Clinical Research

Facility funding from 2012.

• Greater than 75% growth in research funding to MEH since 2009/10. Greater than 50%

growth in research funding to MEH since 2011/12.

• Funding to MEH from commercial partnerships has increased 6-fold since 2011/12

reflected by a 6-fold increase in commercial study number since 2010-11 (Figure 5).

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Open Session MEH Board

2009-10 2010-11 2011-12 2012-13 2013-14£0

£5,000,000

£10,000,000

£15,000,000

£20,000,000

£25,000,000

UCL Institute of Ophthalmology Research Funding

HEFCE Research Councils NHS/NIHRCommercial Charity Special TrusteesMEC EU Government Other

Figure 2: Research Funding to IoO 2009 – 2014.

• IoO funding by sector is variable year-on-year, except for commercial funding which has

been stable (at around £3m per year).

• Total funding mean is £19m per year (2009 – 2014).

2009-10 2010-11 2011-12 2012-13 2013-14£0

£5,000,000

£10,000,000

£15,000,000

£20,000,000

£25,000,000

£30,000,000

£35,000,000

Combined MEH / IoO Funding

NIHR BRC / CRF NIHR (RCF, CLRN, Grants) Research CouncilCommercial Charity Special TrusteesMEC EU Funding HEFCEOther

Figure 3: Research Funding to MEH and IoO combined 2009 – 2014.

• Together, MEH and IoO have attracted over £130 million in research grants in the past 5 years.

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Open Session MEH Board

b. Study number

2009-2010 2010-2011 2011-2012 2012-2013 2013-2014100

120

140

160

180

200

Growth in active studies

Figure 4: Increase in number of studies active at MEH.

• The total number of studies undertaken at MEH has increased by 25% since 2009/10.

2009-2010

2010-2011

2011-2012

2012-2013

2013-2014

2014-2015

05

101520253035404550

Growth in commercial study number

Figure 5: Commercial study number increase from 2009 – 2014.

• We have delivered a 6-fold increase in commercial study number since 2010-11.

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c. Patient recruitment

Figure 6: Increase in patient recruitment to MEH studies

• Patient recruitment to Moorfields / IoO research studies has increased year-on-year.

• Patient recruitment has increased almost 5-fold since 2010/11.

• We are performing a greater number of studies which is resulting in increased patient

recruitment at MEH.

• The proportion of observational studies and has increased which recruit relatively high

numbers of patients partly explaining 5-fold increase in recruitment over 5 years vs 25%

increase in study number.

• Patient recruitment increased significantly in 2012/13 and 2013/14 mostly due to six

projects, each of which have recruited at least 200 patients, with one recruiting over 500

patients.

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d. Quintiles / UCL Partners Prime Site performance

• UCLP Prime site was the world’s leading Quintiles prime site in 2013 based on recruitment of 792 patients to quintiles research studies.

Figure 7: 792 patients recruited onto Quintiles studies in 2013

• Moorfields is the top recruiter to Quintiles prime site studies in 2014, albeit to one high recruiting study.

Figure 8: MEH recruitment to Quintiles prime site studies in 2014.

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e. Publication volume

2010 2011 2012 2013 20140

100

200

300

400

500

600

Chart Title

Figure 9: Number of joint publications for MEH and IoO (2010 – 2014(to November 2014))

• MEH and UCL have the highest proportion of collaborative papers (RAND analysis 2002-2006) compared to all NHS Trusts in England.

• Our key strengths are in cataract, inherited eye disease, regenerative medicine, gene therapy and imaging research based on first author publication number.

• MEH and IoO are leading its competitors in inherited eye disease research.

• MEH and IoO are losing ground to its main competitors based on publication number in

cataract, glaucoma, retina and AMD research.

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4. Strategic areas

a. Major Research Themes

“It is the absolute peaks of excellence that create the reputation of the institution.”

• Endothelial cell biology: In this theme we are focusing primarily on how blood vessels

form in health and disease and how blood vessels interact with other pathological

processes, notably inflammation.

• Inflammation: Both at IO and in collaboration with colleagues at Bristol we have several

programmes looking at how inflammation in different parts of the eye drives disease and

how this might be blocked.

• Pathogenesis of retinal vascular disease: Here, the mechanism of age-related macular

degeneration, diabetic retinopathy and other retinal vascular diseases are being studied

with a view to developing novel approaches to diagnosis and treatment.

• Pathogenesis of inherited eye disease: As well as major programmes seeking the

genetic causes of a wide range of eye disease much activity focuses on understanding

how these gene defects cause disease.

• Gene therapy: The pioneering gene therapy programme has secured significant

research funding over the year but needs considerably more to move forward from

successful experimental proof of concept studies to clinical trials.

• Stem cell approaches to eye disease: Major programmes are addressing novel

approaches to treating inherited and acquired retinal degenerations, corneal disease

and glaucoma. In addition, we are leading in the use of stem cell technology and gene

editing to study mechanisms of disease.

• Visual neuroscience: Here we are interested in understanding the detail of how the brain

takes information from the retina and turns it into a visual percept of the world, how this

degrades with disease and what might be done to improve vision in those with

significant impairment.

• Tissue repair and Drug delivery: We are investigating mechanisms of tissue scarring

which are involved in many blinding diseases and new ways to deliver drugs to the eye

to prevent this scarring

• Ocular oncology: This is a relatively small theme which we seek to expand and that is

looking at mechanisms of ocular tumour development and seeking to identify new ways

of tackling this.

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Open Session MEH Board

b. Research Talent

Attracting, training and developing premier research talent, to drive research output, discovery and innovation in new treatments

Our reputation has been established through the work of the researchers who have been

and are based here. The intellectual capital of our staff is our most valuable R&D asset:

without researchers there is no research.

We face an ever more competitive international market for research talent. For us to retain

our top-ranking position, we need to identify the talent requirements for our chosen strategic

areas, and then attract, train and develop those select researchers. These plans will link to

the Moorfields education strategy and Institute talent management programmes.

• We have the opportunity to enhance investigator productivity.

• Detailed analysis to be presented later

c. Innovation through Integration

Goals & Progress:

• The joint management team has started the work up of materials designed to promote

what we do, the skills that we have, the impacts we deliver, the partnerships we develop

and the value we bring through the research work that happens. A wide internal

consultation has begun and co-creation workshops started to draft the initial documents

that will form the core of how we engage with our stakeholder groups. The

communications team, the Moorfields way team and a NED are involved.

• Cross Institution Events are on the work agenda for 2015.

• There are robust ties with the eye bank and other areas in UCL such as the translational

office, consulting and the EU Horizon 2020 office.

• The ACTIVETM team (Applied Clinical Trials in Vision & Eyes) led by Dr Jen Burr, Dr

Catey Bunce and Prof Richard Wormald, is a major partnership with the UCL CTU and

has made significant impact in the success of winning the large NIHR grants covered in

the performance section of this paper.

• Working with Mary Sherry's team in clinical operations, we have research participation

in the clinical transformation programme, which will develop the integration of research

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Open Session MEH Board

work happening at the heart of the clinical service in addition to the more specialist work

happening in the CRF.

• Prof Sir Peng Khaw's work as UCLP lead for Eyes and Vision is now supported by Dr

Julian Hughes, who recently joined research as Performance and Alliance manager to

strengthen the drive for maximising results.

d. World-leading partnerships

Goals and Progress:

• There are several areas within partnership development including:

• Quintiles Prime Site. As the largest global clinical research organisation we have

partnered with them to grow our portfolio clinical trials.

• Partnership with Roche, to strategically develop new therapies at fundamental science

level and pull them through to first in man studies in the CRF.

• Collaboration with a company called Bicycle Therapeutics (Cambridge) to explore

opportunities to develop novel compounds to treat ophthalmic disease.

• Existing long-term partnerships with Pharma and Biotech are ongoing. We are

maximising the translational potential of these partnerships by linking laboratory and

clinical research.

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• Developing work with the TEVA Corporation. They already have a partnership

agreement with the Cabinet office for £20M of investment for Chronic Illnesses of aging.

• The Ophthalmic Futures Event led by Keith Barton brings together key industry figures

to explore partnerships and development opportunities.

• Recruited a new member of the research team to manage these Partnership and

Alliance developments with the 'Health to Wealth' agenda as a key area of focus.

• Work with Queen Elizabeth Diamond Jubilee Trust on blindness in commonwealth

5. Enablers

Goals and Progress:

a. Attracting resources by delivering outstanding value for our funders:

• Appointment of Dr Jen Burr decision maker and panelist on the NIHR grants, who also

mentors researchers at the joint site for the development of high quality research

applications.

• Adnan Tufail leading on the EU grant partnership for 7M euros, Involving 17 countries.

• CRM database now live and ready for use as needed by research.

• PK & PL working in close partnership with MEC for the prioritisation of research

requirements in new funding awards.

• Working with an agency and in liaison with the MEH Communications team for the work

up of 'good news stories' to highlight research impact and success, due on stream by

February 2015.

b. Insights from Informatics:

• Research funded an Applications Engineer for the Open Eyes Team. Several meetings

with OE have outlined the requirements research have for OE and have co-developed

with the OE team to design of a module for the future.

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• We have created new links to the UCL Informatics Group.

• Full participation in the 100K Genome project and recent UCLP Genomics Medicine

Centre bid.

c. Discovery Science Headlines

• Several studies addressing how the permeability of blood vessels is controlled at the

molecular level. This is critical for the development of new approaches to controlling

oedema (excess tissue fluid) in the retina.

• Refinements to tissue culture of stem cell derived Retinal pigment epithelial (RPE).

• Investigations into the control of normal and pathological blood vessel development

involving Neuropilin 1 and Lrg1 and including major publications in Development,

Developmental Cell, Journal of Experimental Medicine and Nature. (high impact

journals).

• New insights into how mutations in genes controlling RNA processing cause dysfunction

of RPE.

• Elucidation of how mutations in cone pigments cause disease.

• Many studies on the genetics of glaucoma, corneal disease and retinal degeneration

including in Nature Genetics.

• Studies of how inflammatory signaling works in supporting (non-neuronal) cells of the

retina known as Muller cells.

• Key study using stem cell technology to elucidate the mechanism of a retinal

degeneration caused by mutations in the RP2 gene (Human Molecular Genetics). As

well as being of intrinsic interest this study shows the power of using cells from patients

with known mutations to understand disease processes in cell cultures.

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• The demonstration of how a specific protein links junctions between cells to an

intracellular system controlling cell survival. This is part of a large programme of work

that helps us understand RPE disease (notably AMD) and certain categories of tumours.

• A study providing novel insights into how RPE processes rod outer segments.

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