Zayed Centre for rare disease in children to open in July

Professor Gaspar said the centre, which will change the global face of children’s medicine, has been made possible thanks to a transformational £60 million (Dh280 million) gift made in July 2014 by Her Highness Shaikha Fatima Bint Mubarak, Wife of the late President Shaikh Zayed Bin Sultan Al Nahyan, as well as 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.

“The Zayed Centre for Research into Rare Disease in Children 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,” said Gaspar, who is also chief scientific officer at Orchard Therapeutics, a biotech company dedicated to bringing transformative gene therapies to patients with serious and life-threatening rare diseases.

The Centre, located in Guilford Street, central London, will bring together doctors from Great Ormond Street and scientists from its academic partner, the UCL Institute of Child Health.

Gaspar said the “cross-fertilisation of ideas” was vital to maintain advances in cell and gene therapy.

The extra space will allow a 20 per cent increase in outpatients, helping Great Ormond Street to respond to “ever-increasing demand”.

He said rare diseases are not so rare.

“There are more than 6,000 rare diseases, including childhood cancers, cystic fibrosis and muscular dystrophy. A rare disease is defined as affecting fewer than one person in every 2,000.

He said Great Ormond Street was a world-leading paediatric centre for the treatment of rare diseases, 75 per cent of which affect children.

“One in 17 people will suffer from a rare disease at some point in their lives, and 30 per cent of patients will die before their fifth birthday,” Professor Gaspar said.

Professor Gaspar 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,” he added.

The new centre will be able to help patients like Nina Warnell, who was diagnosed with severe combined immunodeficiency (SCID) when she was five weeks old.

The condition is sometimes dubbed “bubble baby” because patients need to live in a sterile environment. Last year The Independent’s Give to GOSH (Great Ormond Street)appeal heard how Nina’s treatment involved re-engineering her bone marrow to add a vital missing gene and “reboot” her immune system. SCID affects about one in every 250,000 children in the UK.

Professor Gaspar said the procedure “worked out incredibly well” for Nina and that work at the Zayed Centre for Research would 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 thalassaemia and sickle cell anaemia.

The Zayed Centre for Research will have a key role in interpreting the data received from the 100,000 Genomes project, which is sequencing DNA from patients with rare diseases or cancer and their families.

In addition, the centre’s seven “clean rooms” will be used to repair and grow new organs, such as the windpipe and oesophagus. These organs are taken from deceased donors and “reseeded” with cells from the new recipient. The centre will also enable an increase in clinical trials.

The centre was named after the late Shaikh Zayed Bin Sultan Al Nahyan, the founding father of the United Arab Emirates.

Professor Gaspar said: “Right from the beginning, this was not just a centre that was there to help children at Great Ormond Street. It was there to help children nationally and globally.”

Central to the project is the desire to promote “bench to bedside” translational research by encouraging interaction between research staff and patients within a single building, and giving public expression to the important work being carried out. At street level the building is animated by views directly into the principal research laboratories on the lower ground floor. Research staff and patients share a single entrance which passes above these laboratories. Internally, extensive glazing creates strong visual connections between research and outpatient areas.

The building sits opposite Coram’s Fields, a space which has been dedicated for over 250 years to the wellbeing of children. A carefully articulated network of vertical terracotta fins gives a strong visual identity to the facades opposite Coram’s Fields, reflecting the public significance of the building and the unique relationship of its function with its context.

The Ramadan series of lectures is part of Shaikh Mohammad’s efforts to spread the spirit of knowledge and learning in the UAE, by inviting renowned scholars, experts, officials and entrepreneurs to speak at his majlis at Al Bateen Palace in Abu Dhabi. The lectures are attended by Shaikhs, senior government officials, diplomats, business leaders and others.