Mohammed bin Zayed, King of Jordan attend a lecture on the future of pediatric medicine in the world

Mohamed Monir

During the course of the lecture, Profs Gaspar and Kurian, who is based at the UCL Great Ormond Institute of Child Health (ICH) in London, examined the changing role of medicine and the ways it will lead to a better future for seriously ill children with rare and complex diseases. Prof Gaspar also dwelt on how the imminent opening of the Zayed Centre for Research into Rare Disease in Children at the Great Ormond Street Hospital (GOSH) would help in developing ground-breaking new treatments for rare and complex diseases and "help children nationally and globally." The state-of-the-art center has been made possible thanks to a generous grant by Her Highness Sheikha Fatima bint Mubarak, Chairwoman of the General Women's Union and President of the Supreme Council for Motherhood and Childhood, "to improve the health of future generations". Together, GOSH and its research partner, ICH, are global leaders in pediatric medicine and have developed new treatments that have saved children’s lives around the world.

Prof. Gaspar said the Zayed Centre will bring together hundreds of experts to accelerate the discovery of treatments and cures for rare, childhood diseases, and would ensure young patients gain access to the latest therapies and clinical trials. He said the "cross-fertilization of ideas" was vital to maintain advances in cell and gene therapy. Prof. Gaspar is also Professor of Paediatrics and Immunology at ICH and has pioneered gene therapy research for the last 20 years, and is an expert on immuno-deficiencies. The professor said the increasing use of new gene therapies would be a "more fundamental" way to treat diseases - "you are not using external medicines. You are not doing bone-marrow transplants, using somebody else’s cells. This is using the individual’s own cells."

In 2001, Prof. Gaspar’s team conducted the world’s first gene therapy on a child born with SCID (Severe Combined Immuno-deficiency) whose immune system had not developed properly. The revolutionary new therapy involved inserting a working copy of the faulty gene into the child’s own cells. After just a few weeks, the boy’s immune system began working and he is now a healthy 17-year-old. Prof. Gaspar also spoke of his experience with patients like Nina Warnell, who was diagnosed with SCID when she was five weeks old. The Majlis heard how Nina’s treatment involved re-engineering her bone marrow to add a vital missing gene and "reboot" her immune system.

Prof. Kurian is UCL Professor of Neurogenetics, NIHR Research Professor at the ICH and Honorary Consultant Paediatric Neurologist at GOSH. She is an expert in childhood movement disorders including parkinsonism and early-onset dystonia - which can cause a wide range of disabilities, including painful muscle spasms, abnormal postures and can affect walking and speech. In 2017, Prof. Kurian discovered a gene that helped predict which children with dystonia could benefit from deep brain electrical stimulation, a therapy which targets the part of the brain responsible for the spasms. Her life-changing breakthrough has led to treatment so effective it can restore the ability to walk among some young patients.

Prof. Kurian’s team has expertise in neurotransmitter disorders, including AADC Deficiency where affected children have reduced levels of the important brain chemicals dopamine and serotonin. This life-limiting condition leads to severe difficulties in movement and painful body spasms. Her team is planning to take part in a clinical trial that could potentially slow down and even reverse the progression of this disease. The trial will see the delivery of gene therapy delivered directly into the patient’s brain. Over the coming years, Prof. Kurian and her team will be attempting to develop new gene therapy approaches for childhood neuro-degeneration and parkinsonism.

Revolutionary advances in genomics, gene, and stem cell therapies have made it possible to provide life-changing treatments at GOSH, one of the world’s leading children’s hospitals with the broadest range of dedicated, children’s healthcare specialists under one roof in the UK. The hospital attends to children from more than 90 countries, including children from the UAE who fly down to be treated for a range of diseases, including rare heart and neurological conditions.

Thanks to the pioneering work of Profs Kurian and Gaspar, new therapies have already been developed for children across the globe with severe immuno-deficiences, cancers, and neurological disorders. The future impact of their work is promising and will be underpinned by the Zayed Centre for Research. In addition, this world-class multidisciplinary center of excellence’s seven "clean rooms" will be used to repair and grow new organs, such as the windpipe and esophagus. These organs are taken from deceased donors and "reseeded" with cells from the new recipient.

Pediatric research is critical because early breakthroughs are likely to continue to come from the study of children with rare disorders. These findings have the potential to feed into treatments for more common conditions or those affecting adults. The Zayed Centre, the first in the world dedicated to rare childhood diseases, is set to revolutionize the treatment of seriously ill young patients once it opens in July.

H.H. Sheikh Rashid bin Saud bin Rashid Al Mu'alla, Crown Prince of Umm Al Qaiwain; H.H. Sheikh Hamdan bin Zayed Al Nahyan, Ruler's Representative in Al Dhafra Region; Dr. Amal Abdullah Al Qubaisi, Speaker of Federal National Council, FNC; H.H. Sheikh Nahyan bin Zayed Al Nahyan, Chairman of the Board of Trustees of Zayed bin Sultan Al Nahyan Charitable and Humanitarian Foundation; H.H. Lt. General Sheikh Saif bin Zayed Al Nahyan, Deputy Prime Minister and Minister of the Interior; H.H. Sheikh Hamed bin Zayed Al Nahyan, Chief of the Abu Dhabi Crown Prince's Court; H.H. Sheikh Abdullah bin Zayed Al Nahyan, Minister of Foreign Affairs and International Cooperation; H.H. Sheikh Khalid bin Zayed Al Nahyan, Chairman of the Board of Zayed Higher Organisation for People of Determination, ZHO; H.H. Dr. Sheikh Sultan bin Khalifa Al Nahyan, Adviser to the UAE President; Sheikh Nahyan bin Mubarak Al Nahyan, Minister of Tolerance; Jordanian Foreign Minister, Ayman Safadi; a number of Sheikhs; members of the diplomatic corps and key figures were also present.