Zayed Centre for Research into Rare Disease in Children celebrates three years of progress and breakthroughs

Since opening in 2019, the Zayed Centre for Research’s Falcon outpatients unit has seen patients from more than 30 different countries. The Centre has welcomed almost 50,000 patients a year, seeing up to 200 patients daily.

Professor Maha Barakat, Director General of the Frontline Heroes Office, commented on the global research to have already come from the ZCR since it opened three years ago.

“The global research contributions generated by the Zayed Centre for Research over just three short years is a testament to the scientists and clinicians who have dedicated themselves to saving young lives, as well as, the cutting-edge facilities that make their work possible.”

As well as providing life-changing treatment for seriously ill children, over the past year, its state-of-the-art facilities have also supported complex, global health challenges.

In April, the UK Health Security Agency (UKHSA) called on world-leading genomics experts to help identify which virus was driving an unusual rise in childhood hepatitis cases.

The UKHSA approached an existing collaboration of UCL scientists from the Zayed Centre for Research and clinical scientists at GOSH. This group is led by Professor Judy Breuer, Professor of Virology and Consultant at GOSH.

The UKHSA recognised the group's expertise as one of the only teams in the world that could provide highly-specialised, clinically-guided metagenomics answers, bolstered by the cutting-edge equipment and expertise housed in the Centre.

By July, the World Health Organisation (WHO) reported more than 1,000 children across 35 countries had been affected. The Zayed Centre for Research and GOSH-based teams were able to report that co-infection with two viruses may have caused the onset of severe liver disease in affected children. A separate study in Scotland corroborated the UCL/GOSH results. The findings provide the world-first example of adeno-associated viruses (AAV) being implicated in disease.

In February, as part of UK Pavilion events at Expo 2020, GOSH’s Professor Claire Booth led a virtual seminar on her research in gene therapy. The seminar highlighted how modified harmless viruses can be used to treat patients with severe immune system diseases.

Professor Booth is Mahboubian Professor in Gene Therapy at UCL Great Ormond Institute of Child Health and Consultant in Paediatric Immunology at the hospital.

The Zayed Centre for Research’s third anniversary also coincides with the release of its first annual report.

As well as highlighting recent progress, the report provides insights into the work of some of the 33 independent research groups currently based at the Centre. For example, research into the use of innovative CAR T-cell therapy for childhood cancers.

The Zayed Centre for Research into Rare Disease in Children was made possible thanks to a transformative £60 million gift from Her Highness Sheikha Fatima bint Mubarak, wife of the late Sheikh Zayed bin Sultan Al Nahyan, founding father of the United Arab Emirates, in 2014. We are also grateful to Research England, The Wolfson Foundation, John Connolly & Odile Griffith and the Mead Family Foundation whose generous support contributed to the creation of the Zayed Centre for Research. The Zayed Centre for Research is a partnership between Great Ormond Street Hospital, UCL and Great Ormond Street Hospital Children’s Charity.