The Zayed Centre for Research into Rare Diseases in Children will change the global face of children’s medicine.
Neuroblastoma, hypoplastic left heart syndrome, severe combined immunodeficiency disease, primary ciliary dyskinesia, long segment tracheal stenosis and hypertrophic cardiomyopathy are just some of the names of children’s rare diseases.
They are a mouthful, aren’t they? The easy part is trying to pronounce the names of these diseases; the really difficult and most unrelenting part is living with them.
“The work that will take place in the Zayed Centre for Research will enable us to make breakthroughs and develop cures for rare diseases quicker than ever before, giving children everywhere the chance of a longer and fuller life,” said Dr. Bobby Gaspar, professor of paediatrics and immunology at the University College of London Great Ormond Street Institute of Child Health in London, England, as quoted in The National Newspaper from Abu Dhabi.
“The Zayed Centre…will enable clinicians and researchers to work side by side in advancing the understanding of rare diseases, identifying new and better treatments and manufacturing innovative medical devices,” added Gaspar.
3.5 million people in the UK, 30 million in Europe, 30 million in the USA and just over 3 million in Canada have rare diseases.
Majority of Zayed Centre’s funding will go towards six priority areas: birth defects and tailor-made transplants, hard-to-treat childhood cancers, heart conditions, hormone and cell function disorders, immune system disorders and muscle wasting diseases. The Zayed Centre will manufacture specialist products for novel therapies, which will help develop the regenerative medicine programme for children with absent or damaged tissues.
The medical centre will concentrate on advanced gene therapies to treat diseases and not the use of external medicines such as bone marrow transplants.
6,000 rare diseases affect more than 300 million people worldwide.
75% of rare diseases affect children.
Diagnosed with Severe Combined Immunodeficiency (SCID) at the age of five weeks, Nina Warnell was treated by doctors at the Zayed Centre who re-engineered her bone marrow with a vital missing gene and then rebooted her immune system.
“The procedure worked out incredibly well for Nina,” said Gaspar, adding that, “the work at the Zayed Centre for Research will allow gene therapy to move away from its current niche application and be used for certain skin conditions in children born with HIV, and those with the hereditary blood disorders like Thalassaemia and Sickle Cell Anaemia.” (You can view Nina’s video at the end of this article).
Children with SCID have very little or no immune system and therefore, are unable to fight off infections and can become quite ill with pneumonia, meningitis or chicken pox.
SCID affects about one in every 250,000 children in the UK and is estimated to occur in approximately one out of every 50,000 births globally. If left untreated, children with SCID usually live a year or two.
One in 17 people will suffer from a rare disease at some point in their life. A rare disease is defined as affecting fewer than one person in every 2,000 people.
Professor Gaspar is the chief scientific officer at Orchard Therapeutics, a biotech company dedicated to bringing transformative gene therapies to patients with serious and life-threatening rare diseases including primary immune deficiencies and inherited metabolic and blood disorders.
Orchard’s autologous ‘ex vivo’ gene therapy uses a patient’s own blood stem cells to correct a genetic disorder. Stem cells are taken from the patient and modified with a viral vector that carries a functioning copy of the missing or faulty gene. The modified gene cells are then transplanted back into the patient’s body.
Facts about the Zayed Centre
Zayed Centre, located in Guilford Street, central London, will run in partnership with Great Ormond Street Hospital (GOSH) and the Great Ormond Street Hospital Children’s Charity in the UK.
The six-storey, 140,000 square foot research facility with 134 solar panels accommodates:
- 500 researchers, clinicians and allied health professionals from Great Ormond Street Hospital (GOSH), Institute of Child Health (ICH) and University of College London’s Institute of Cardiovascular Sciences (ICS) will work in the state-of-the-art facilities
- 200 outpatients a day
- A cardiology research suite with facilities for 3-D modelling
- 160 lab bench positions
- 10 rooms and 86 incubators for growing cells
- 21 consultation rooms and 8 clinical investigation rooms
- the largest dedicated clean room facility of its kind in Europe housing seven highly specialized labs where cell and gene therapy-based medicines will be developed, as well as the repair and growth of new organs, such as the windpipe and oesophagus.
- state-of-the-art stem cell facility that will develop new treatments for children with conditions from blindness to nerve disorders
- a sensory room for children to relax before appointments and educational play activities to help children understand what’s happening with their bodies and how research can help them
30% of patients with rare diseases will die before their 5th birthday.
“As I walk around the new facility, it is clear the potential it holds to change the lives of children with the rarest and most complex diseases, and the hope it will offer to their families. On behalf of everyone at GOSH, I’d like to thank all our partners and donors who have made The Zayed Centre for Research possible,” says Matthew Shaw, Chief Executive at Great Ormond Street Hospital for Children - NHS Foundation Trust.
The Zayed Centre for Research, a partnership between Great Ormond Street Hospital, University College London and Great Ormond Street Hospital Children’s Charity will unite pioneering research and clinical care under one roof to drive forward new treatments and cures for children with rare and complex diseases.
In Honour of Sheikh Zayed Bin Sultan Al Nahyan
The Zayed Centre is named after the late Sheikh Zayed Bin Sultan Al Nahyan, the founding father of the United Arab Emirates (UAE).
“Understanding the UAE is impossible without understanding the life of Sheikh Zayed and his deep religious faith, vision, determination and hard work; his generosity at home and abroad; and the way in which he devoted his life to the service of his people and the creation of a better world,” states the Embassy of the United Arab Emirates.
His wife, Her Highness Shaikha Fatima Bint Mubarak, initiated the Zayed Centre in 2014 donating over $86 million dollars towards the project.
A pioneering force for Women’s Rights, Sheikha Fatima bint Mubarak is the Chairperson of the UAE General Women’s Union, Supreme Chairwoman of the UAE Family Development Foundation and President of the UAE Supreme Council of Motherhood and Childhood.
In 1975, she founded UAE’s Women Federation and was profoundly instrumental in a nationwide campaign advocating girls' education.
Sheikha Fatima bint Mubarak is a pioneering force for women’s rights and she has contributed significantly to the foundation of the first women’s organization in 1976, the Abu Dhabi Society for the Awakening of Women.
Since 2005, The ‘Sheikha Fatima Award for Excellence’ award has been presented to female recipients for their outstanding academic performance and commitment to the environment and world citizenship. The reward includes a full-tuition scholarship that extends to schools across the Middle East and India.
UNICEF, WHO, UNIFEM, UNESCO and UNFP (United Nations Population Fund) awarded Sheikha Fatima bin Mubarak for her significant efforts towards women's rights, education and literacy.
Resources
For additional information, Diligencia Investigative Reporting recommends the following articles and websites:
GOSH – (Great Ormond Street Hospital) – Zayed Centre for Rare Diseases in Children
The National Newspaper – Abu Dhabi – Medical Research Funded to Help Doctors Make Life Saving Breakthroughs for Sick Children
General Women’s Union founded by Her Highness Sheikha Fatima Bint Mubarak
Canadian Organization for Rare Disorders (CORD)
Orphanet: provides information on rare diseases and orphan drugs
Additional Resources
For additional information, Diligencia Investigative Reporting recommends the following articles and websites:
GOSH News and Inspirational Stories – Click on this link to read about:
- Dr Owen Williams, Professor of Cancer Biology at the UCL Great Ormond Street Institute of Child Health and his team, who have identified an existing drug that shows great promise in helping children with acute myeloid leukaemia.
- GOSH mum, Flavia, whose son, Matei, has been on a clinical trial at the hospital for the last couple of years.
- Eva Bugallo Blanco and Alice Piapi, two researchers at UCL Great Ormond Street Institute of Child Health (ICH), who are working on the development of new CAR-T cell therapies for Neuroblastoma - the second most common solid childhood cancer.
Australia: Association of Genetic Support of Australasia (AGSA)
Australia: Rare Voices Australia (RVA)
Belgium: Rare Disorders Belgium (RDB)
Denmark: Rare Disorders Denmark
European Organization for Rare Diseases (EURORDIS)
France: Alliance Maladies Rares
Germany: Allianz Chronischer Seltener Erkrankungen (ACSE)
Greece: Greek Alliance for Rare Disease (PESPA)
Italy: Federazione Italiana Malattie Rare (UNIAMO)
Netherlands: Alliantie voor Erfelijkheidsvraagstukken (VSOP)
New Zealand: Organisation for Rare Disorders (NZORD)
Spain: Federacin Espaola de Enfermedades Raras (FEDER)
Taiwan: Foundation for Rare Disorders
United States: National Organization for Rare Disorders (NORD)
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