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.
GOSH Charity and Sparks National Call 2020-21
This year, GOSH Charity has announced a £2.5 million investment into 11 pioneering child health research projects. The funding is the UK’s largest charitable grant-making scheme of its kind dedicated to paediatric rare disease research.
Of the £2.5 million committed to support research into some of the most difficult and hard to treat childhood diseases, Sparks contributed £900,000.
£112,500 has been made available by two condition-specific partner charities (Acrodysostosis Support & Research, and Dravet Syndrome UK) to help co-fund research into these diseases.
The GOSH Charity and Sparks National Call is part of an ambition to help unlock breakthroughs in child medicine by supporting researchers’ investigations into the causes of rare diseases in children, and conditions that start in childhood. The funding will also help supercharge their efforts to discover new and better ways to diagnose, treat, and ultimately cure these life-changing and life-limiting conditions.
Researchers based at six institutions across the country will benefit from the cash boost, including: The University of Manchester, University of Warwick, and University College London Great Ormond Street Institute of Child Health, University of Southampton, University of Oxford, and University of Cambridge
We received a total of 124 outline applications for the 4th joint GOSH Charity and Sparks National Call for research project grants.
Following a triage meeting held on 7 October 2020, 34 full applications were invited for full external peer review.
GOSH Charity’s Research Assessment Panel met on 4 March 2021 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 Raj Chopra, Professor Veronica Swallow, Dr Li Chan, Professor Caroline Wright, Professor Carl Goodyear, Professor Rafael Yáñez-Muñoz and Baroness Margaret Jay.
The following 11 projects were awarded grants, totalling £2,504,880.76.
| Name | Institution | Title of Award | Amount requested |
|---|---|---|---|
| Prof Mina Ryten | UCL Great Ormond Street Institute of Child Health | Leveraging transcriptomics to improve the diagnostic rate and understanding of neurometabolic disorders | £222,826.37 |
| Prof Matthew Wood | University of Oxford | uORF-mediated SCN1A up-regulation for the treatment of Dravet syndrome | £234,677.00 |
| Dr Christian Babbs | University of Oxford | Reversing Zeta-Globin Transcriptional Silencing: Towards Embryonic Globin Induction in Patients With Severe Alpha-Thalassemia | £241,915.74 |
| Prof David Rubinsztein | University of Cambridge | Identification of pathways that protect against Beta-propeller protein-associated neurodegeneration | £248,780.00 |
| Prof Paolo de Coppi | UCL Great Ormond Street Institute of Child Health | Dissecting the role of mechanical lung compression in Congenital Diaphragmatic Hernia on a single cell level. | £229,305.04 |
| Prof Christina Liossi | University of Southampton | Development and Validation of a Paediatric Breakthrough Pain Assessment Tool | £224,650.00 |
| Dr Paula Alexandre | UCL Great Ormond Street Institute of Child Health | Investigate the underlying causes of cerebellar developmental disorders | £249,375.48 |
| Dr Siddharth Banka | University of Manchester | Building iPSC-derived models to identify treatments for KMT2 chromatin disorders | £248,605.00 |
| Dr Beth Payne | University College London |
Targeting amino acid metabolism as a novel treatment avenue for Diamond-Blackfan anaemia |
£249,650.29 |
| Prof Patrizia Ferretti | UCL Great Ormond Street Institute of Child Health | Generating human models of acrodysostosis for testing mechanisms and pharmacological intervention | £245,028.40 |
| Dr Hayley Crawford | University of Warwick | A clinical checklist of causes of poor behavioural outcomes in children with moderate-profound intellectual disability and complex needs | £110,067.44 |
GOSH Charity and Sparks National Call 2019-20
We 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 |