£2.1million cash boost for child health research by leading children’s charities

Great Ormond Street Hospital Children’s Charity (GOSH Charity) and Sparks, the children’s medical research charity, have announced a £2.1million investment into 12 groundbreaking child health research projects across the UK. This marks the largest annual charitable call dedicated to funding research into child health conditions.  This injection of funds in to paediatric research will provide a huge boost to an area of research that is severely underfunded, receiving only five per cent of public and charitable research funding in the UK each year1.

GOSH Charity and Sparks invited researchers from across the UK to apply for funding as part of a process called the ‘national call’. The £2.1 million will support 12 pioneering projects researching some of the most difficult and hard to treat childhood diseases. Based at eight institutions across the UK from Sheffield to London, they aim to improve diagnosis and develop more effective and kinder treatments for children who desperately need them.

The successful projects included research into pioneering gene therapy for children with difficult-to-treat epilepsy, creating superpowered immune cells to treat a range of childhood tumours, finding new treatments for an aggressive childhood brain tumour (DIPG) and understanding the genetic causes of skeletal disorders in children.

The commitment to paediatric research funding reflects GOSH Charity and Sparks’ ambitions to help unlock breakthroughs in children’s medicines that will find treatments and cures for seriously ill children with rare and complex conditions.

This year, for the first time the national call also involves partnering with rare disease charities Krabbe UK and Dravet Syndrome UK, thereby increasing the funding available. Dravet Syndrome UK has jointly funded a research project aiming to unlock the potential of gene therapy to treat Dravet Syndrome, while Krabbe UK has provided funding to a project to explore the potential of a new stem cell treatment for Krabbe disease. This demonstrates the commitment from GOSH Charity and Sparks to boost funding into research into the most complex of conditions and highlights the importance of collaboration to fund the highest quality research. 
Trevor Smithers, whose daughters Elsie and Rosabelle died from Krabbe Disease, commented: “Sparks and GOSH Charity, and the research they fund, is so important because there are still so many conditions that we don’t know enough about. The cure is the goal but we will only get that by investing money into these vital research projects.”

Kiki Syrad, Director of Grants at Great Ormond Street Hospital Children’s Charity says: “We were delighted to receive a large number of high-quality applications from the UK pediatric research community on a range of diseases. For many children, research is their only hope. We look forward to seeing how these projects progress, and the call re-opening later in 2019.”

The projects supported also reflect the ambition of both GOSH Charity and Sparks to drive new tests and therapies from the lab bench to the patient’s bedside, speeding up the diagnosis and treatment of rare and complex conditions.

For further information, please contact Ruth Maurice in the Great Ormond Street Hospital Children’s Charity Press Office on 020 7239 3125 or Ruth.Maurice@GOSH.org 

About Great Ormond Street Hospital and Great Ormond Street Hospital Children’s Charity: 

Great Ormond Street Hospital is one of the world’s leading children’s hospitals with the broadest range of dedicated, children’s healthcare specialists under one roof in the UK. The hospital’s pioneering research and treatment gives hope to children from across the UK with the rarest, most complex and often life-threatening conditions. Our patients and families are central to everything we do – from the moment they come through the door and for as long as they need us. 

Great Ormond Street Hospital Children’s Charity needs to raise money to support the hospital to give seriously ill children, the best chance for life. The charity funds research into pioneering new treatments for children, provides the most up to date medical equipment, funds support services for children and their families and supports the essential rebuilding and refurbishment of the hospital. You can help us to provide world class care for our patients and families. For more information visit www.gosh.org 

Sparks 

Sparks raises money to fund pioneering children’s medical research. Since 1991, it has funded more than 285 ground-breaking research projects in over 90 hospitals, universities and research institutions across the UK and overseas. 

The National Call Research Projects:

• Professor Dimitri Kullmann, from UCL Institute of Neurology, is pioneering gene therapy for children with difficult-to-treat epilepsy. 
• Dr. Susan Campbell, from Sheffield Hallam University, is finding better ways to diagnose Vanishing White Matter Disease – a devastating brain condition with no cure. 
• Professor John Anderson, from UCL Great Ormond Street Institute of Child Health, is creating superpowered immune cells to treat a range of childhood tumours. 
• Dr Katie Gallagher, of Homerton University Hospital NHS Foundation Trust, examining the approach involved in the delivery of very premature babies. 
• Dr David Carmichael, of King's College London, is studying the potential of the latest 7T MRI imaging technology in children with epilepsy. 
• Dr Matthias Zilbauer, University of Cambridge Studying, is studying the behaviour of single cells and growing 3D ‘mini-guts’ in the lab to help understand inflammatory bowel diseases like ulcerative colitis and Crohn’s disease. 
• Professor Henry Houlden, from UCL Institute of Neurology, is studying a genetic link between epilepsy, autism and developmental disorders. 
• Dr Sara Benedetti, from University College London, is unravelling the potential of a new treatment for Krabbe Disease (co-funded with Krabbe UK). 
• Dr Owen Williams, from UCL Great Ormond Street Institute of Child Health, is driving a new leukaemia treatment towards clinical trial. 
• Dr Rajvinder Karda, from University College London, is unlocking the potential of gene therapy for Dravet Syndrome, a rare, inherited form of epilepsy (co-funded with Dravet Syndrome UK). 
• Professor Grant Stewart, from University of Birmingham, hopes to understand a genetic cause of skeletal disorders in children. 
• Dr Jasper de Boer, from UCL Great Ormond Street Institute of Child Health, is looking to repurpose existing drugs to treat an aggressive childhood brain tumour (DIPG).