£10 million boost for Centre for Children’s Rare Disease Research

A centre bringing together the country’s leading clinicians and researchers to find effective treatments for children’s rare diseases has received a £10 million funding boost

The Centre for Children’s Rare Disease Research will bring together expertise from Great Ormond Street Hospital and University College London, in particular the UCL Institute of Child Health, to advance understanding and find treatments for children’s rare diseases.

The £10 million award from the Higher Education Funding Council for England (HEFCE) is a significant step towards raising the £85 million needed to build and equip the Centre. Great Ormond Street Hospital Children’s Charity has undertaken to raise the remaining amount and the HEFCE funding was given as part of a governmental initiative to support projects which have private funding from industry or the charitable sector worth a minimum of double the public contribution.

Professor David Goldblatt is Director of Clinical Research and Innovation for Great Ormond Street Hospital and the UCL Institute of Child Health and an academic lead for the new Centre. He said: “This is fantastic news. The Centre will provide huge opportunities to advance research into children’s rare diseases and translate discoveries into effective treatment quicker than ever before."

“The work will not simply assist in finding treatments for rare diseases but will inform our understanding of generic and more common disease mechanisms.”

The new Centre, to be built adjacent to Great Ormond Street Hospital and the UCL Institute of Child Health, will work to find treatments and cures for rare diseases, of which there are 6,000 identified. Seventy five per cent of rare diseases affect children. These include rare cancers such as childhood cancers, and some other better known conditions such as cystic fibrosis and muscular dystrophy.

Central to the work of the researchers and clinicians working in the new facility will be finding ways to speed up the delivery of effective treatments, as 30 per cent of children with rare diseases will not live past their fifth birthday.

Great Ormond Street Hospital is uniquely placed to advance research to tackle these life-threatening and life limiting conditions, as it sees many more children with rare disease than any other hospital in the country and is already working with researchers from ICH, many of whom are also clinicians, on ground breaking research in this area.

Current rare disease research includes work looking at the molecular basis of rare cancers, ciliopathies and rare obesity syndromes as well as novel genetic therapies for diverse conditions including inherited muscular dystrophies and rare immune deficiencies.