GOSH Charity and Sparks award over £1 million for child health research at the ICH

Great Ormond Street Hospital Children’s Charity (GOSH Charity) and Sparks, the children’s medical research charity, have announced funding for five projects at the UCL Great Ormond Street Institute of Child Health (ICH), as part of the UK’s largest funding scheme dedicated to child health research.

Projects include pioneering research to save the hearing of children who are born without sight, supporting the UK arm of a global clinical trial to test whether the breast cancer drug tamoxifen could help children with a rare muscle disorder, and creating an implantable device that supports liver function and could hold the key to reducing toxic chemical levels in the blood and avoiding the need for liver transplant.

This year, the charities’ annual funding scheme will invest £2.3 million in 11 pioneering child health projects around the UK. After an open call for applications in 2019, each proposal was rigorously assessed by expert committees and researchers around the globe. Those selected, including the five projects at the ICH, were rated extremely highly and recognised as having the potential to significantly impact child health.

• Professor Nicholas Greene will test 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’s project will support 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 (pictured) will investigate whether replacing the faulty gene that causes deafness in Norrie Disease could save the hearing of boys with the condition, who are born blind. This is a continuation of the pioneering work done by Professor Maria Bitner-Glindzicz, who sadly passed away in 2018.
• Professor Paul Gissen aims 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. The condition affects the liver, kidneys and bone marrow cells. Most patients die in early childhood so new treatments are desperately needed.
• Dr Hassan Rashidi hopes to develop an implantable and removable ‘liver patch’ to provide liver support to patients with the metabolic condition non-ketotic hyperglycinemia, helping them reduce the toxic levels of glycine without the need for liver transplant.