National calls

GOSH Charity has a passion and commitment to support paediatric research nationally. Each year we invite researchers across the country to apply for funding for their child health research projects through the National Call. We review these applications and fund the highest quality research most likely to have life-changing benefits to children.

This year, GOSH Charity and Sparks have committed to fund £2.3 million into 11 new child health research projects. The research will take place at 5 institutions across the UK, including 3 of the country’s leading universities.

The national call projects aim to tackle the understanding and treatment of rare diseases in children. The enthusiasm for this research is reflected in the rare disease community, with 3 of the projects being co-funded by their respective rare disease charities, including the Norrie Disease Foundation, Dravet Syndrome UK and Myotubular Trust.

All of this year’s projects are extremely exciting, offering the potential for life-changing treatments for seriously ill children and their families. The research spans a range of disease areas, with a key focus around novel treatment options for currently incurable diseases, kinder treatments to reduce side effects in already vulnerable children, and new methods to better understand and diagnose some of the most complex diseases.

Below we describe the 11 projects being funded – all of which we believe will bring us one step closer to improving the lives of seriously ill children and their families.

GOSH Charity and Sparks National Call 2019-20

We have received a total of 82 outline applications for the 3rd joint GOSH Charity and Sparks National Call for research project grants.

Following a triage meeting held on 5 September 2019, 30 full applications were invited for full external peer review.

GOSH Charity’s Research Assessment Panel met on 28 January 2020 to review these applications. Present at the meeting were Professor Charles French-Constant (Chair), Professor David Edwards, Professor Eamonn Maher, Professor David Goldblatt, Associate Professor Tim Collier, Professor Anne-Marie Rafferty, Professor Raj Chopra, Baroness Margaret Jay, and Ms Yvonne Parry.

The following 11 projects were awarded grants, totalling £2,273,124.

Lead Investigator Host Institution Total Grant Title
Dr Manavendra Pathania University of Cambridge £249, 945 Identifying drivers of aggressive childhood brain tumours that could hold the key to new treatments.
Professor Nicholas Greene UCL GOS ICH £249,741 Testing a new compound derived from cinnamon that could help lower toxic levels of ammonia and glycine in children with the rare metabolic diseases non-ketotic hyperglycinemia and Urea Cycle Disorders.
Dr Giovanni Baranello UCL GOS ICH £232,432

(£58,108 from Myotubular trust)
Supporting the UK arm of a global trial understanding if the breast cancer drug tamoxifen could help children with the muscle disorder X-linked myotubular myopathy.
Professor Jane Sowden UCL GOS ICH £251,332

(£62,500 from Norrie Disease Foundation)
Aiming to replace the faulty gene that causes deafness in Norrie Disease, saving the hearing of boys who are born blind.
Professor Paul Gissen UCL GOS ICH £228,757 Aiming to tackle all symptoms of a complex multi-organ disorder, Arthrogryposis Renal Dysfunction and Cholestasis Syndrome (ARC), with two types of gene therapy at once.
Professor Andrew Wilkie University of Oxford £169,449 Aiming to unravel why the activity of a gene called FOXD3 is affected, and whether it could offer a diagnostic tool, in some children with craniosynostosis - a condition which causes the bones in the skull to fuse too early.
Professor Nicola Dawes Oxford Brookes University £122,771 A lifestyle weight management program for children with conditions that strip the outer protective layer of their nerves, like MS or Neuromyelitis Optica Spectrum Disorder (NMOSD).
Dr Rajvinder Karda UCL £249,271

(£62,318 from Dravet Syndrome UK)
Driving a new approach to gene therapy for a rare, inherited and aggressive type of epilepsy called Dravet Syndrome.
Dr Victor Hernandez Hernandez Brunel University of London £220,191 Using gene therapy to the eyes and brain to correct the most common genetic mistake that leads to Bardet Biedl Syndrome – a condition that leads to blindness, learning disabilities, and weight gain (and other symptoms).
Dr Hassan Rashidi UCL GOSH ICH £101,394 Developing an implantable and removable ‘liver patch’ to provide liver support to patients with the metabolic condition non-ketotic hyperglycinemia.
Dr Kate Baker University of Cambridge £197,841 Understanding more, through gene and brain activity testing, about a range of conditions that affect the chemicals that allow nerves to communicate – Synaptic Vesicle Cycling Disorders.

GOSH Charity and Sparks National Call 2018-19

We received a total of 83 outline applications for the 2nd joint GOSH Charity and Sparks National Call for research project grants.

Following a triage meeting held on 6th September 2018, 31 full applications were invited for full external peer review.

GOSH Charity’s Research Assessment Panel met on 23rd January 2019 to review these applications. Present at the meeting were Professor Charles ffrench-Constant (Chair), Professor David Goldblatt, Professor Rosalind Smyth, Professor Anne Marie Rafferty, Professor David Edwards, Professor Raj Chopra, Professor Brian Bigger, Professor Ruth Newbury-Ecob and Ms Yvonne Parry

The following 12 projects were awarded grants, totalling £2,174,694.

Lead Investigator Host Institution Total Grant Title
Professor Dimitri Kullman UCL Institute of Neurology £190,404.38 Gene therapy for epilepsy & focal cortical dysplasia
Dr Susan Campbell Sheffield Hallam University £189,024 eiF2B bodies in association with the human disease Leukoencephalopathy with Vanishing White Matter -a novel diagnostic tool
Professor John Anderson UCL Great Ormond Street Institute of Child Health £212,589.30 Development of B7-H3 targeting chimeric antigen receptors for cellular therapy of childhood solid cancers
Dr Katie Gallagher Homerton University Hospital NHS Foundation Trust £23,209 The attitudes of health professionals involved in preterm delivery towards the treatment of extremely preterm infants
Dr David Carmichael King's College London £224,981.90 Realising the potential of 7T MRI as a clinical tool for paediatric neuroimaging
Dr Matthias Zilbauer University of Cambridge £238,250 Investigating intestinal epithelial cell biology in paediatric Inflammatory Bowel Disease
Professor Henry Houlden UCL Institute of Neurology £196,734.36 AMPA Receptor Mutations are important causes of epilepsy, autism spectrum and developmental disorders
Dr Sara Benedetti UCL Great Ormond Street Institute of Child Health £96,085.94* Targeting the blood-brain barrier endothelium to increase hematopoietic stem cell engraftment for lysosomal storage disease gene and cell therapy
Dr Owen Williams UCL Great Ormond Street Institute of Child Health £232,782.24 Pre-clinical evaluation of mebendazole in AML therapy
Dr Rajvinder Karda University College London £75,725.17** AAV gene targeting of long non-coding RNA in a mouse model of Dravet Syndrome
Professor Grant Stewart University of Birmingham £246,993 Investigating the pathogenic impact of mutations in TONSL as the genetic cause of a novel skeletal disorder in children
Dr Jasper de Boer UCL Great Ormond Street Institute of Child Health £247,914.21 Exploiting therapeutic targets in paediatric high grade gliomas with the H3K27M mutation

*co-funded with Krabbe UK

**co-funded with Dravet Syndrome UK

GOSH Charity and Sparks National Call 2017-18

We received a total of 142 outline applications for the 1st joint GOSH Charity and Sparks National Call for research project grants.

Following a triage meeting held on 28th September 2017, 36 full applications were invited for full external peer review.

GOSH Charity’s Research Assessment Panel met on 9th January 2018 to review these applications. Present at the meeting were Professor Stephen Holgate (Chair), Professor Maria Quigley, Professor David Goldblatt, Professor Rosalind Smyth, Professor Jill Clayton-Smith, Professor Anne Marie Rafferty, Professor David Edwards, Professor Jonathan Grigg, Professor Raj Chopra, Professor Brian Bigger, Professor John Anderson, Baroness Margaret Jay and Ms Yvonne Parry

The following 14 projects were awarded grants, totalling £2,156,225.

Lead Investigator Host Institution Total Grant Title
Dr Ming Lim Oxford University Hospitals £45,898 UK Multicentre Study of Children with Opsoclonus Myoclonus Syndrome (UMSCOM) Study
Dr Heidi Fuller Keele University £192,945 Therapy development for children with motor neuron disease
Professor Mary Rutherford King's College London £203,084 Investigating early brain development in Down Syndrome: new therapeutic windows for intervention
Professor Tessa Crompton UCL Great Ormond Street Institute for Child Health £146,243 Induction of tolerance by thymic epithelial cell transplantation
Professor Andrew Copp UCL Great Ormond Street Institute for Child Health £200,139 Chiari II brain malformation: association with spina bifida and response to fetal surgery
Professor Celia Moss Birmingham Children's Hospital £33,151 BPSU study: The incidence, management and early outcome of congential ichthyosis
Dr Alessia Cavazza UCL Great Ormond Street Institute for Child Health £41,589 Targeted gene correction for the treatment of Severe Combined Immunodeficiency caused by mutations in the IL7R gene
Professor Christina Liossi University of Southampton £214,821 End-of-Life pain management by carers and healthcare professionals in infants, children and young people in out of hospital settings
Professor Michael Duchen University College London £194,675 From the biology of EPG5 to the pathophysiology of Vici syndrome
Professor Chris O'Callaghan UCL Great Ormond Street Institute for Child Health £186,044 Restoring ciliary function in Primary Cilia Dyskinesia
Dr Thomas O'Brien Liverpool John Moores University £148,573 An intelligent ultrasound-based diagnostic tool to decouple neural and structural contributions to reduced joint range of motion in cerebral palsy
Professor Giulio Cossu University of Manchester £235,664 A pre-clinical investigation on cell pharmacodynamics after intra-arterial systemic delivery in a rat model of Duchenne Muscular Dystrophy
Dr Karin Straathof UCL Great Ormond Street Institute for Child Health £201,324 T-cell immunotherapy for childhood brain tumour DIPG
Dr Sylwia Ammoun Plymouth University £112,071 Investigation and targeting cellular prion protein PrPC in Neurofibromatosis type II related tumours schwannomas, meningiomas and spinal ependymomas

Joint call for project grants from across the UK from Action Medical Research (AMR) and Great Ormond Street Hospital Children’s Charity 2015–2016

We received a total of 115 outline applications in response to the charities' joint national call.

Following triage, 59 outline applications were invited to submit full applications with 54 being received and put forward for full external peer review.

AMR's Scientific Advisory Board met on 8 June 2015 to review these applications. Two of Great Ormond Street Hospital Children's Charity's Research Assessment Panel members provided written comments in advance of the meeting (Professor Stephen Holgate and Professor David Goldblatt) and one member was in attendance of the meeting (Dr Colin Michie).

The following 13 projects were awarded joint funding, totalling £2,050,839.

Lead investigator Host institution Total Project title
Professor John Anderson UCL Institute of Child Health £197,027 Optimising cellular immunotherapy for neuroblastoma
Professor Georgina Jackson University of Nottingham £97,651 Learning and breaking habits in tourette syndrome
Professor Judy Breuer University College London £62,911 Use of target enrichment deep sequencing and data modelling to improve the management of cytomegalovirus in immunocompromised patients at GOSH
Professor Lyn Chitty Great Ormond Street Hospital £198,634 Developing safe, early non-invasive prenatal diagnosis for autosomal recessive conditions: cystic fibrosis and spinal muscular atrophy
Professor Bobby Gaspar UCL Institute of Child Health £165,929 Adoptive transfer of autologous gene corrected T cells to treat X-linked lymphoprofilerative disease (XLP)
Professor Deborah Tweddle Newcastle University £196,219 Clinical and biological factors associated with relapse and length of survival following relapse in UK neuroblastomas
Professor Mark Johnson Birkbeck University of London £199,987 Identifying early infant treatment targets for neurocognitive development in neurofibromatosis type 1
Dr Antonio Valentin Kings College London £164,253 Brain electrical stimulation for the treatment of severe epilepsies in children
Professor Nick Greene UCL Institute of Child Health £177,915 Disease of the glycine cleavage system: understanding the causes and development of novel therapies
Dr Michael Clarke University College London £88,438 Development of an eye-pointing classifcation scale for young non-speaking children with severe cerebral palsy
Dr Pablo Lamata Kings College London £102,593 Improving surgical decisions in hypoplastic left heart syndrome (HLHS) through computational cardiac models
Professor David Sharp Imperial College London £199,814 Network dysfunction following paediatric traumatic brain injury
Dr Owen Williams UCL Institute of Child Health £199,465 Harnessing TEL-AML1-induced signalling pathways for targeted therapy of t(12:21) leukaemia

Translational paediatric rare disease research 2014–2015

We received a total of 43 outline applications in response to the charity’s national call for outline proposals in November 2013.

Following a triage meeting held on 19 December 2013, 11 full applications were invited to be submitted in February 2014 for full external peer review.

A National Assessment Panel met on 12 May 2014 to review these applications. Present at the meeting were Professor Timothy Barrett, Dr Colin Michie, Dr William Van't Hoff, Professor Phillip Beales, Professor David Jones, Professor Jill Clayton-Smith, Professor Andy Copp and Dr Sahar Mansour.

The following seven projects totalling £1,000,000 were awarded grants.

Lead investigator Host institution Total Project title
Professor John Anderson UCL Institute of Child Health £33,000 Exploring gene therapy approaches for neuroblastoma using a patient's own gamma delta T cells: Application for running costs
Dr Philippa Mills UCL Institute of Child Health £110,197 Rapid identification of children whose epilepsy can be treated with vitamin B6
Professor Philip Stanier UCL Institute of Child Health £134,026 Clinical and molecular characterisation of a new intellectual disability-cerebellar ataxia syndrome
Dr Brian Bigger University of Manchester £358,626 Development of myeloid driven autologous haematopoietic stem cell gene therapy for Sanfilippo disease
Professor Philip Beales UCL Institute of Child Health £30,506 Evaluating alternative genetic therapies for Bardet-Biedl Syndrome
Dr Hannah Mitchison UCL Institute of Child Health £133,489 Next generation sequencing to stratify and personalise the genetic basis of motile cilia disease
Professor Fenella Kirkham UCL Institute of Child Health £200,156 Potential Reversibility of structural brain abnormalities by preventing hypoxia in sickle cell anaemia

Biomarker research 2013–2014

We received a total of 43 outline applications in response to the charity’s national call for outline proposals in November 2013.

Following a triage meeting held on 4 December 2012, 16 full applications were invited to be submitted for full external peer review.

A National Assessment Panel met on 28 June 2013 to review these applications. Present at the meeting were Professor David Armstrong, Professor Bobby Gaspar, Dr Colin Michie, Professor Tim Barrett, Dr Peter Grabowski and Dr William V’ant.

The following six projects totalling £947,574 were awarded grants.

Lead investigator Host institution Total Project title
Dr Paul Humphries University College London £100,500 Whole body PET-MRI in paediatric and adolescent lymphoma
Professor Michael Levin Imperial College London £260,246 Development of a rapid diagnostic test to discriminate bacterial and viral infection in febrile children based on an RNA expression signature
Dr David Michod UCL Institute of Child Health £40,500 Phosphorylation of the H3.3 chaperone DAXX as a new biomarker and a new target for treatment of high grade gliomas in children
Dr David Long UCL Institute of Child Health £195,526 Angio-poietin-2 as a biomarker and mediator of cardiovascular disease in children
Dr Vivek Muthurangu UCL Institute of Cardiovascular Science £184,975 Magnetic resonance augmented exercise testing: a novel biomarker in paediatric pulmonary hypertension
Professor Kathy Pritchard-Jones UCL Institute of Child Health £165,827 Prognostic value of 1q gain in Wilms’ tumour