UCL Institute of Child Health and Great Ormond Street Hospital for Children NHS Trust UCL Institute of Child Health 30 Guilford Street London WC1N 1EH Research Review 2007 Tel: 020 7242 9789 Great Ormond Street Hospital for Children NHS Trust Great Ormond Street London WC1N 3JH Tel: 020 7405 9200 Ayesha, age 8 Contents Overview 04 Dean’s report 06 Chief Executive’s report 08 Research and development report 10 In the news Research Genes, development and disease 14 Kidney chaos 16 Decoding disease 18 Syndromes – a complete picture 20 Reproductive development Cancer 22 Non-toxic treatment 24 The vaccine approach Biochemical and nutritional sciences 28 Paediatric nutrition and health prediction 30 Stem cell potential Neurosciences and mental health 32 Epilepsy – alternatives to drug treatment 34 Gene therapy for muscular dystrophy 36 Repairing the retina Infection and immunity 40 Gene therapy and the immune system Cardiorespiratory sciences 42 Heart vessel inflammation 44 Heart-valve replacement – sharing innovation Population health sciences 46 Childhood immunisation 48 Science-based policymaking People 52 Awards, honours and prizes 2007 55 Grants and donations 2007 65 Senior academic staff 2007 71 Administration 2007 Harry, age 5 weeks 01 | Overview The UCL Institute of Child Health, in partnership with Great Ormond Street Hospital, is the largest centre in Europe devoted to clinical and basic research and postgraduate teaching in children’s health. Jennifer Frost, PhD Student, Clinical and Molecular Genetics Unit Overview | Dean’s report Dean’s report For the UCL Institute of Child Health, Further awards were achieved under the became Professor of Clinical Epidemiology like all university departments across the Higher Education Funding Council for England for her research on childhood infection at UK, 2007 was the year of the Research Clinical Senior Lecturer Scheme. In 2006, a population level. Catherine Law became Assessment Exercise (RAE). The RAE Dr Shamima Rahman and Dr John Anderson Professor of Public Health and Epidemiology, evaluated all aspects of research obtained one of these prestigious awards, for her contributions to research-based performance during 2001–2007. The and this continued in 2007 with awards public health policy for children. Institute submitted 134 senior research to Dr Persis Amrolia, Dr Neil Sebire and staff to the RAE, including 14 consultants Dr Russell Viner. We were also delighted that Promoted to Reader during 2007 were: in paediatric medicine at Great Ormond Dr John Achermann was awarded a Wellcome Dr Persis Amrolia for immunology of bone Street Hospital who, despite heavy clinical Trust Senior Clinical Research Fellowship. marrow transplantation, Dr Michelle de Haan workloads, had achieved a particularly for memory and the psychology of childhood high level of research output. The Institute While continuing to pursue its research perception, Dr Jugnoo Rahi for childhood visual formed the largest part of an experimental interests throughout the paediatric impairment and prevention of blindness, medicine submission to the RAE, together specialties, the Institute has established an Dr Lesley Rees for growth and heart disease “ Postgraduate education with the UCL Institute of Ophthalmology academic unit of General and Adolescent in children with kidney failure, Dr Paul Riley for and UCL Division of Medicine. Paediatrics. Its leader, Professor Brent Taylor, stem cells that can repair heart blood vessels, in a range of child health moved to the Institute during the year, Dr Neil Sebire for pathology research into areas has long been a There were many other highlights during bringing with him the UCL undergraduate childhood and placental disease, Dr Andrew 2007. We welcomed Francesco Muntoni as medical degree module in Child and Family Taylor for heart imaging research, Dr Paul Veys strength at the Institute, Professor of Paediatric Neurology and head Health. A long-term strategic goal is to build for bone marrow transplantation and Dr Russell of the Dubowitz Neuromuscular Centre, a unit this new grouping into a significant element Viner for adolescent medicine and obesity. but during 2007 the with an international reputation for research of the Institute’s overall research portfolio, Promoted to Senior Lecturer were Dr Andrew and clinical practice in muscular dystrophies. providing a higher profile than before in the Cook for education in congenital heart disease Institute took a further Professor Muntoni is one of three leaders area of general and adolescent paediatrics and Dr Eleanor Main for education and step into undergraduate (together with Professor Mike Hanna at the in the UK and beyond. research in physiotherapy for lung disease. UCL Institute of Neurology, and our own education.” Professor Martin Koltzenburg) of the newly Postgraduate education in a range of child As the year closed, the Institute embarked formed Medical Research Council Centre health areas has long been a strength at the upon an in-depth re-evaluation of its research for Neuromuscular Diseases, which opened Institute, but during 2007 the Institute took and academic strategy, in conjunction with in early 2008. a further step into undergraduate education our clinical partner Great Ormond Street with the development of an intercalated BSc Hospital. Having surveyed and evaluated Also new to the Institute were Professor Tessa in International Health and Development, run research performance since 2001 in the RAE, Crompton, who established a research team by Professor Anthony Costello. This course it now seems appropriate to look forward investigating the development of the immune forms an important part of a new UCL over the next five years to determine the system, and Professor Peter Hammond, who strategic development in Global Health, which priority areas for research and development. joined us from the Eastman Dental Institute brings together a range of UCL departments As I write this report, we are in the final to pursue research on the analysis of facial for which international health is a common stages of developing our strategic plan, and features in genetic disease. research and educational interest. I will return to this topic in next year’s report. Sadly, Professor Robert Surtees, one of the During 2007, more of the Institute’s senior Institute’s longest serving and most gifted staff were successful in UCL’s promotions academic paediatricians, died during the exercise than ever before. Helen Cross summer. Professor Surtees was widely known became Professor of Paediatric Neurology and respected for his expertise in all aspects for research achievements in childhood Andrew Copp of paediatric neurology, but with particular epilepsy. Bobby Gaspar became Professor Dean, UCL Institute of Child Health reference to neurochemistry and movement of Paediatrics and Immunology, for his disorders. He is greatly missed by all his groundbreaking research in gene therapy colleagues both at the Institute and hospital. for immunodeficiency disease. Ruth Gilbert Hannah, age 20 months 05 Overview | Chief Executive’s report Chief Executive’s report As a hospital that values research as highly An example would be the renal and cancer as the quality of clinical care, the last year databases that, with consent from children has been particularly challenging. We have and families, consist of longitudinal data needed to start adapting to the loss of the collected on patients for many years. Such annual NHS Culyer research and development data provides invaluable information, for block grant of £34 million and to find new example on the natural history of a particular alternative funding opportunities. It has problem or the long-term effects of different been made more difficult because we face a treatment regimes. Fortunately Great number of other significant funding changes Ormond Street Hospital Children’s Charity which affect the income we get for treating is willing to fund this sort of work as one patients, all of which have had an impact of the ways it supports our research. on our financial position. Juggling all of this while applying to become a Foundation Trust We also need to make sure we have excellent and yet protecting the research which facilities for research. The opening of The “ Together the UCL benefits our patients, has not been easy Somers Clinical Research Facility later in 2008 and we don’t have all the solutions yet. will provide this, enabling children to be Institute of Child Health enrolled in pioneering research. Previously and Great Ormond The good news is that we now understand investigators have used any space they could what the opportunities are much more beg, borrow or steal around the hospital when Street Hospital form clearly and therefore should be able to they wanted to do a study, which meant a lot of maximise our chances of success in our time was wasted. The Somers Clinical Research the largest paediatric funding applications. We can also start Facility will provide robust systems, processes to see how this new system, which is and infrastructure to make research as easy as centre in Europe completely transparent, will more explicitly possible. Its staff will provide support to any dedicated to clinical benefit patients. The great disadvantage investigator wishing to do a study. Many of the of the old block grant system was that we studies conducted in this area will also bring and basic research.” couldn’t always be clear how the money in additional funding from the Department of supported specific research outcomes and Health and elsewhere. This development is outputs but these are totally transparent possible through the generosity of both the under the new system. J N Somers Charitable Will Trust and Friends of the Children of Great Ormond Street. The successful award of Specialist Biomedical Research Centre status has been a good start. Together the UCL Institute of Child Health In partnership with UCL Institute of Child and Great Ormond Street Hospital form the Health we are the only specialist centre largest paediatric centre in Europe dedicated dedicated to experimental medical research for to clinical and basic research.