The first centre in the world dedicated to rare childhood diseases is to open in London after a record donation to Great Ormond Street Hospital Children’s Charity.
The family of the founder of the United Arab Emirates gave the charity £60 million – the majority of the consutuction costs – with construction work due to start in March.
Due to open in Bloomsbury in late 2018, it will be named the Zayed Centre for Research into Rare Disease in Children in honour of the late Sheikh Zayed bin Sultan Al Nahyan, who died in 2004.
The centre, in Guildford Street, will bring together doctors from Great Ormond Street Hospital and scientists from its academic partner, UCL’s Institute for Child Health. By co-locating hundreds of experts and allowing more staff to be hired, this will accelerate the discovery of treatments and cures and ensure the young patients gain the access to the latest therapies and clinical trials.
Professor Bobby Gaspar, director designate for the new centre, said the “cross-fertilisation of ideas” was vital to maintain advances in cell and gene therapy. The extra space will allow up to a 20 per cent increase in outpatients, helping GOSH to respond to “ever-increasing demand”.
Gene therapy offers new hope to children with rare diseases by effectively using their own bodies to repair the damage caused by faulty or missing genes, rather than relying on bone marrow donations from other people. This removes the difficulty of finding a match and the risk of the transplant being rejected.
There are more than 6,000 rare diseases, including childhood cancers, cystic fibrosis and muscular dystrophy. A rare disease is defined as affecting less than one person in 2,000.
“This is a more fundamental way to treat these diseases,” Professor Gaspar said. “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.”
One of his patients was Nina Warnell, who was diagnosed aged five weeks with severe combined immunodeficiency (SCID). The condition is sometimes dubbed “bubble baby” as patients need to live in a sterile environment.
Nina’s treatment involved re-engineering her bone marrow to add a vital missing gene and “re-boot” her immune system. SCID affects about one in 250,000 children in the UK.
Nina’s parents Graeme and Aga Warnell didn’t want to put her through a risky bone marrow transplant. She received the gene therapy in April 2013. Next month she will turn four.
“It’s worked out incredibly well for her,” Professor Gaspar said. “There were greater risks with using a bone marrow donor. The immediate risks of using her own cells were very low. What we didn’t know was whether it would work.”
Gene therapy has potential to move away from its “niche” application and be used for certain skin conditions, in children born with HIV and in children with the hereditary blood disorders thalassaemia and sickle cell anaemia.
The Zayed Centre for Research will have a key role in the interpreting the data received from the Prime Minister’s 100,000 Genomes project, which is sequencing DNA from patients with rare diseases or cancer and their families. The challenge is not in reading the code but interpreting what it means in terms of disease.
In addition, the Zayed Centre for Research’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 “re-seeded” with bone marrow cells from the new recipient.
The donation from Sheikh Zayed’s widow, Sheikha Fatima bint Mubarak, was inspired by a wish to support pioneering healthcare globally, including for patients from the UAE.
Professor Gaspar said: “They saw this as a big project, a big statement of what they wanted to do. It was very innovative. It involved both the care of children with very severe conditions, and most importantly, developing cutting-edge research that would impact on these children.
“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. It was incredibly generous, and allowed us to take this project forward.
“Medicine is moving so rapidly in so many kinds of different areas. We at the Institute of Child Health and Great Ormond Street have real expertise and innovation in so many areas that we have developed in the last 10 to 15 years. We want to take that to another level.”
He said the new centre would enable a huge increase in clinical trials. “At the moment we are treating 10 to 15 patients a year in gene and cell therapy. We hope to be treating 100 a year.”
The site, at 20 Guildford Street, was previously a UCL computer lab. It was bought by the Great Ormond Street Children’s Charity in 2010. It sits opposite Coram’s Fields, which has been dedicated to the wellbeing of children for 250 years.
The Zayed Centre for Research has been designed by Stanton Williams architects, which won the 2012 Stirling Prize for the Sainsbury Laboratory at Cambridge university. The outpatient space is expected to provide 21 consultation rooms, eight clinical investment rooms and other facilities. There will be tissue culture labs, a cardiology suite and a flow cytometry suite for cell analysis. It will train the next generation of doctors and scientists.
The centre’s work will be targeted in key disciplines for GOSH: cardiac, respiratory, immunology, solid cancers, bone marrow transplantation and neuro-muscular diseases.
The glass design of the building will provide a “window” to the research being carried out on in its basement floors. Scientists and patients will share the same entrance – a deliberate ploy to encourage one group to inspire the other.
“There is a real sense that these are two activities that are joined,” said Gavin Henderson, director of Stanton Williams. “The researchers will see patients and see the outcomes of what they are doing. The patients will engage with the research staff, and understand the work that is going on in the building is positively feeding back to the treatment they are receiving.
“It’s not something that is happening in an ivory tower research environment. It’s somewhere you can understand the importance of what you are doing, because you see patients who are benefiting on a day-to-day basis.”
The building’s exterior will feature vertical terracotta “fins”, with timber and other natural materials inside. The brief was to focus on medical research rather than “iconic” architecture, and to fit in with the Bloomsbury conservation area. The designs benefits from lessons learned from the Sainsbury Laboratory.
Mr Henderson said: “I think it’s going to make a powerful statement about the importance of the work done here. It’s not trying to symbolise in any obvious way the hospital nature of the building. Children are going to be treated here. It’s not about creating an aggressively clinical environment.
“This is a very unusual brief, and an exciting brief because of this combination of research and treatment in the same building. That makes it very complicated to design, but is at the heart of what made the project interesting.
“It’s tremendously exciting to be working on something of international significance and such importance to society as a whole, and to be working with the teams from GOSH and ICH, who are world-leaders in their field.”