The pioneering Zayed Centre for Research into Rare Disease in Children (ZCR) is celebrating its fifth anniversary at Great Ormond Street Hospital (GOSH), with its ground-breaking achievements including the treatment of “incurable” leukaemia.  

Since its opening in 2019, the Zayed Centre has united hundreds of clinicians and researchers under one roof to advance tests, treatments, and cures for children with rare and complex diseases. The Centre is driven by a mission to offer hope to families and change lives globally. Each year, thousands of sick children attend appointments at the Centre, including 300 outpatients from the United Arab Emirates among the 1,800 international outpatients treated in the last year.

The Zayed Centre for Research is a partnership between GOSH, University College London and Great Ormond Street Hospital Children’s Charity (GOSH Charity), and was made possible thanks to a generous £60 million gift in 2014 from HH Sheikha Fatima bint Mubarak, Chairwoman of the General Women's Union, President of the Supreme Council for Motherhood and Childhood, and Supreme Chairwoman of the Family Development Foundation.

Professor Maha Barakat, Assistant Minister for Medical Affairs and Life Sciences at the UAE Ministry of Foreign Affairs, remarked: “In this short time, the Zayed Centre for Research has demonstrated the effectiveness of a pioneering model that combines world-leading research expertise with advanced clinical care, all within state-of-the-art facilities, transforming young lives. The Centre’s work truly embodies the humanitarian vision of the late Sheikh Zayed bin Sultan Al Nahyan, which is faithfully upheld by Her Highness Sheikha Fatima bint Mubarak. By combining world-leading research with advanced clinical care, the Zayed Centre is transforming the future of children’s health and providing hope to families around the world.”

In conjunction with its fifth anniversary, the Zayed Centre launched its annual impact report, highlighting notable achievements for the year 2023/2024.  These include major breakthroughs in gene-editing therapies, advancements in epilepsy diagnosis, and innovative work using organoids for disease modelling. The report underscores the Centre’s vital role in pioneering cutting-edge treatments and shaping the future of paediatric medicine.

 Key Achievements from 2023/24:

  • Breakthrough in Leukaemia Treatment: Researchers successfully treated three children diagnosed with ‘incurable’ T-cell leukaemia through an innovative base-edited T-cells clinical trial.
  • Revolutionary Epilepsy Diagnostics: The Centre introduced new approaches for faster, more accurate diagnosis of epilepsy, potentially improving outcomes for young patients.
  • Organoid Research: The Centre advanced its work with organoids—miniature, functional versions of organs—which are expected to revolutionize disease modelling and potentially enable new treatments, such as repairing organs with biologically accurate patches.
  • MHRA authorisation to manufacture viral vectors: The Centre earned a Manufacturer's Authorisation Licence to produce viral vectors, a key development that will accelerate clinical trials and expand personalised treatments for children with cancer and other serious conditions. With this licence, ZCR can accelerate the development of life-saving therapies.

Louise Parkes, Chief Executive of GOSH Charity, said: “Over the past five years, the Zayed Centre for Research has been at the forefront of medical innovation and the fight against rare paediatric diseases. It is incredible how many ground-breaking advances have been made by our experts in that time, from gene-editing therapies to rapid genome sequencing and so much more.

“For many children with rare diseases, research is their only hope of treatment or a cure. At GOSH Charity we stop at nothing to help give seriously ill children the best chance possible and a huge part of that is funding ground-breaking research to offer hope for countless young people around the world.”

In addition to offering world-class medical treatment, the Zayed Centre is a hub for collaboration and research. It provides valuable opportunities for medical students and organizations from the UAE to work alongside leading experts at GOSH. This year, the Centre has also expanded its partnerships with key UAE entities, including an education agreement with Danat Al Emarat Women and Children’s Hospital for Intensive Care, as well as a collaboration with M42 to enhance paediatric critical and complex care across the Middle East.

A recent delegation from the Department of Health – Abu Dhabi (DoH) visited the Centre this month, further underscoring the importance of strengthening ties between the UAE and GOSH in areas of medical research, genome therapy and latest innovative technologies. This collaboration aligns with the shared goal of improving healthcare outcomes for children worldwide.