The first centre in the world dedicated to rare diseases in childhood will help researchers and doctors from Great Ormond Street Hospital (GOSH) to revolutionise the treatment of seriously ill young patients.
The Zayed Centre for Research into Rare Disease in Children, in Guilford Street, central London, will bring together doctors from GOSH and scientists from its academic partner, the UCL Institute of Child Health.
Bringing together hundreds of experts and allowing more staff to be hired will accelerate the discovery of treatments and cures and ensure young patients gain access to the latest therapies and clinical trials.
Professor Bobby Gaspar, director designate for the new centre, which is due to open in 2018, 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 GOSH to respond to “ever-increasing demand”.
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.
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 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.
Meet the patients and doctors of GOSH
Meet the patients and doctors of GOSH
1/9 Elliott Livingstone
Two-year-old Elliott is a “cheeky” little boy who has a Thomas the Tank Engine sticker on his Berlin heart machine, which has kept him alive since his own heart failed eight months ago. Elliott has two tubes pumping blood around his tiny body. It keeps him alive but the machine has left him confined to the wards of Great Ormond Street Hospital until a new heart is found
2/9 Melissa Strickland
As the ward sister on Koala Ward, Melissa Strickland leads a nursing team with the challenging job of looking after children with craniofacial and neurological conditions. “You have to have all the skills and knowledge to do this job but personally for me you cannot do it unless you have passion but also compassion,” she said. “You don’t get used to the sad side of things but you do learn to manage it.”
3/9 Amy Willis
Amy Willis carries a discreet black medical bag everywhere she goes. It contains the cutting-edge HeartWare device that is keeping her alive. A smaller, more advanced version of the Berlin artificial heart, it was fitted in April after she was emergency airlifted to GOSH from Alder Hey hospital in Liverpool. The device means that 14-year-old Amy can be home in Flintshire this Christmas while remaining on the heart transplant waiting list. She is doing well but 15 per cent of patients with a HeartWare device or Berlin heart die while waiting for a new heart, so money raised by the appeal will also go to help researchers identify ways to keep children alive while they await transplant
4/9 Myra Bluebond-Langner
Professor Myra Bluebond-Langner represents the vital work of the Louis Dundas Centre for Children’s Palliative Care, GOSH’s world-class centre dedicated to research and care for children with life-limiting illnesses. The LDC is named in honour of Louis Dundas, a four-year-old boy who died in “unspeakable pain” after suffering a brain tumour in April 2008. Its aim is to ensure that no child suffers unnecessarily in their final days. Money raised from The Independent’s Give to GOSH appeal will go to fund the team’s work to manage pain, and also fund vital research into palliative care in children across the whole of the UK. Professor Bluebond-Langner, who heads the research, said: “Paediatric palliative care is a relatively new field where practice has outstripped research. We look to change that.”
5/9 Finella Craig
Together with with Professor Myra Bluebond-Langner, Dr Finella Craig represents the vital work of the Louis Dundas Centre for Children’s Palliative Care, GOSH’s world-class centre dedicated to research and care for children with life-limiting illnesses. The LDC is named in honour of Louis Dundas, a four-year-old boy who died in “unspeakable pain” after suffering a brain tumour in April 2008. Its aim is to ensure that no child suffers unnecessarily in their final days. “One of the worst experiences for a family is to witness their child in pain and discomfort, and for them to feel totally powerless to do anything about it,” said Dr Craig, a consultant in paediatric palliative medicine at GOSH since 2002. Money raised from The Independent’s Give to GOSH appeal will go to fund the team’s work to manage pain, and also fund vital research into palliative care in children across the whole of the UK.
6/9 Rowan Pethard
Like most little boys, Rowan Pethard loves playing football. At the start of 2015 the seven-year-old Spurs fan baffled his doctors in Hemel Hempstead with a string of coughs, colds, tummy bugs, aches, pains and rashes. It wasn’t until quite late on that doctors discovered he had leukaemia. He spent two days in intensive care while he had emergency chemo. He has two years of follow-up treatment ahead. “He’s amazing, a little superhero,” his mum said. “It makes it easier for his father and I and his brother to cope.”
7/9 Martin Elliott
Paediatric heart and lung surgeon Martin Elliott, 64, is one of the longest serving doctors at GOSH, leading groundbreaking research and treating thousands of patients over the past 30 years. His work has bridged the gap between surgery and research with skills ranging from heart-bypass surgery to correcting congenital lung disorders.
8/9 Ralph Frost
For Ralph the hardest thing about having to live at GOSH while he waits for a new kidney isn’t missing his toys. He has plenty of those and can terrorise the nurses by pushing his little red motorbike down the corridors of Eagle Ward. The hardest thing for the six-year-old is battling not to cry out during his nightly dialysis sessions. “It really hurts,” he said. “But the other kids are sleeping and I don’t want to wake them up.” Ralph suffers from nephrotic syndrome and is currently waiting for a kidney from his father, Nick. He’s called the kidney “Chase” and his parents, who have been trained to operate his dialysis machine, hope to be home by Christmas
9/9 Lynsey Steele
The strongest praise for Lynsey Steele, 33, comes from the parents of the children she helps. “The children here wouldn’t get by without Lynsey,” said Ralph’s mother Amie Frost. “If she wasn’t here then we’d have cracked up.” Lynsey’s role, which is funded by the Great Ormond Street Hospital Children’s Charity and will be supported by The Independent’s Give to GOSH appeal, is to help children play and relax, but also to have the difficult conversations explaining their treatment
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, named after the late Sheikh Zayed bin Sultan Al Nahyan, who died in 2004, has been made possible by a donation of £60m from the family of the founder 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.”
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.