GOSH Charity and Sparks funding for child health research at ICH

Four UCL Great Ormond Street Institute of Child Health researchers have been awarded research grants from Great Ormond Street Hospital Children’s Charity and Sparks, the children’s medical research charity as part of their annual National Call. The awards will support new research into advanced treatments for childhood cancers such as leukaemia and brain tumours, and into a cutting-edge new stem cell therapy for Krabbe disease – a rare lysosomal storage disorder helping to meet the charities united aim of aiming to improving diagnosis and treatment for children with rare and complex conditions.

Professor John Anderson, Dr Sara Benedetti, Dr Jasper de Boer (pictured above) and Dr Owen Williams have all received funding awards as part of the GOSH Charity and Sparks National Call – alongside eight other projects totalling a £2.1 million investment into child health research projects across the UK.

• Dr Owen Williams (Professor of Cancer Biology at ICH) and his team have previously identified an existing anti-parasitic drug (mebendazole) that shows great promise in helping children with a life-threatening form of leukaemia - AML. They will carry out further laboratory testing of the drug to prove its effectiveness alone, and in combination with other drugs, with the ultimate aim of initiating a trial of the most effective combination in patients. 

• Professor John Anderson (Professor of Paediatric Oncology at ICH) is developing a pioneering approach to treat a range of childhood cancers including sarcoma, neuroblastoma and brain tumours. This involves using CAR-T cell therapy to target and destroy cancer cells. CAR-T cells have been successfully used to treat blood cancers like leukaemia and now Professor Anderson’s team want to harness their potential to tackle solid tumours.

• Dr Sara Benedetti, (Lecturer in Gene Therapy at UCL and ICH) aims to maximise the potential of a new stem cell treatment that could stop the toxic build-up of a molecule in the brains of children with Krabbe disease, reducing symptoms and improving life expectancy. This project will be co-funded with Krabbe UK, as part of a new partnership which sees GOSH Charity and Sparks partnering with with rare disease charities including Krabbe UK and Dravet Syndrome UK.

• Dr Jasper de Boer (Senior Research Fellow in Cancer Biology at ICH) is looking to repurpose existing drugs to treat an aggressive childhood brain tumour (DIPG). Dr Jasper de Boer wants to screen thousand of existing drugs to see if they could target the genetic cause of this hard-to-treat childhood cancer. Dr de Boer said “I am delighted to have received funding from GOSH Charity and Sparks which will enable me to further my work in research for DIPG. It’s fantastic to know that these charities are making such a large amount available for child health researchers across the UK to bid for each year”

The other successful projects include research into pioneering gene therapy for children with difficult-to-treat epilepsy, understanding the genetic causes of skeletal disorders in children and increasing understanding of the causes of inflammatory bowel diseases.