Pioneer - Summer 2022 edition

GOSH CELEBRATES ITS 170TH ANNIVERSARY

MEDICINES GIVING NEW PURPOSE

Exploring how existing medication can be repurposed to treat rare childhood conditions.

THE POWER OF PROGRESS

Celebrating the incredible breakthroughs at GOSH throughout its 170-year history.

TRANSFERRING FROM GOSH TO ADULT CARE

Discovering how GOSH supports teens as they move into adolescent and adult care.

Great Ormond Street Hospital Children’s Charity

22 MEDICINES GIVING NEW

Discovering new treatments and cures into rare childhood conditions takes time; repurposing existing drugs can help speed the process along. Researchers explain why this is crucial in child healthcare.

26

TRANSFERRING FROM GOSH TO ADULT CARE

What happens to a young person’s care at GOSH when they reach their late teens? Pioneer explores the process of moving from child to adolescent adult services.

30 WHY I SUPPORT THE CHARITY: FAIZA SETH

Entrepreneur and philanthropist

Faiza Seth tells Pioneer why GOSH will always be special to her family.

32 HELPING IN THE FIGHT AGAINST COVID-19

A GOSH-led international research team discovers further breakthroughs against COVID-19.

34 PARENT’S PERSPECTIVE

Angus, born with a congenital heart defect, spent the first nine months of his life in hospital.

Dad Jason shares his story.

Welcome to the Summer 2022 issue of Pioneer and another edition packed full of hospital news and inspiring stories of children and young people and the staff at Great Ormond Street Hospital (GOSH).

This year is an extra special one for the hospital as it celebrates its 170th birthday, in the same year that Her Majesty The Queen – GOSH’s patron – marks 70 years on the throne. To commemorate this incredible milestone in the hospital’s history, Pioneer shines a light on some of the major breakthroughs that have taken place at GOSH since it opened its doors in 1852. From page 12 you’ll read how GOSH has been at the forefront of countless advances across all types of medical specialties, from immunology and cancer care to regenerative medicine and neuroscience, and how charitable donations have always been an integral part of the hospital’s extraordinary care.

Innovations continue across the board at GOSH today. Turn to page 18 to discover how charity support enabled the GOSH Learning Academy, which trains and develops staff at the hospital, to continue offering and developing its programmes during the pandemic and beyond. Plus, we talk to GOSH’s newly appointed Chief Nurse, Tracy Luckett, on page 20, about her vision for the hospital’s nursing teams and how charity support demonstrates how much they’re valued.

And, as always, at the very heart of this issue we share some of the most incredible stories from the children treated at GOSH. Thirteen-year-old Kian tells us, on page 6, about his journey at GOSH, which began when he was just three days old, and how he and his family love taking part in the RBC Race for the Kids event to raise money for Great Ormond Street Hospital Children’s Charity (GOSH Charity). Turn to page 26 to hear from 17-year-old Conor, who speaks movingly about how his GOSH team helped him through some challenging times and how the hospital is now helping him make a smooth transition from his care at GOSH to another hospital for adult care.

I leave you now with this collection of personal and inspirational stories, which I hope will encourage you to continue supporting the incredible work that takes place both at GOSH and GOSH Charity.

Louise Parkes CEO Great Ormond Street Hospital

Charity

All photographs included in this edition were either taken before the Coronavirus crisis, or were taken adhering to all government guidelines.

IN REVIEW

STORIES FROM AROUND THE HOSPITAL AND CHARITY

1 CHARITY FUNDING BUYS MEDICAL EQUIPMENT

Generous support from our donors and supporters has meant the charity has been able to grant recent funding towards new medical equipment at GOSH. For cardiac patients, a new cardiac output monitoring kit will help enhance monitoring of those affected by paediatric multisystem inflammatory syndrome (also known as PIMS-TS), a rare syndrome associated with COVID-19.

Alongside this was investment into a lung function measurement system, which is vital in the diagnosis and surveillance of respiratory patients.

2 GREEN VISION FOR GOSH

GOSH has recently launched several short-term environmental initiatives as part of its vision for environmental development in and around the hospital.

A parklet funded by GOSH Charity is being installed on Great Ormond Street and, on 16 June, Play Street will take place, when Great Ormond Street will close to traffic so children can play outside.

Magali Thomson, GOSH Project Lead for Placemaking, explains why these small-scale initiatives are important steps towards the hospital’s long-term vision for the environment.

“Parklets [a seating area surrounded by plants] are a way of temporarily removing parking bays and evaluating the impact before permanent measures are implemented,” Magali says. “It is possible to experience at a smaller scale the potential effect of a greener, healthier and more people friendly street, which is our ultimate aim.”

Play Street, which coincides with Clean Air Day (the UK's largest air pollution campaign), presents the hospital with the opportunity to promote clean air and demonstrate how it’s tackling air pollution alongside the importance of play for children. The charity funded Play team and GOSH Arts team are also part of the team organising Play Street.

“If we’re treating children with respiratory conditions in the hospital then they go out on to a polluted street, that’s not right. Introducing greenery and cleaner air is advocating a much more preventative approach to health,” Magali says.

The hospital also received Steris Eschmann operating tables. These tables will help enable plastic, ear, nose & throat, neuro and cardiac surgeons, as they allow for surgical procedures to be carried out in a variety of positions.

And, in the radiology department, funding has provided a portable ultrasound machine for interventional radiology. This equipment provides a ‘one-stop shop’ experience for patients, allowing them to have their ultrasound scan performed in clinic and have their medical plans made immediately.

GOSH Head of Sustainability and Environmental Management Nick Martin adds that GOSH now has four new electric ambulances – also known as Peter Panbulance – the first-such paediatric ambulances in the UK. “We’ve also got green champions groups around sustainable care,” he says. “For example, we’ve been reducing anaesthetic gas use that contributes potent greenhouse emissions and our ‘sustainable medicines’ and ‘transport’ groups are looking at where we can improve on plastics, medical packaging and transportation. We’ve also been consulting with GOSH’s Young People’s Forum (YPF) for their ideas.”

Magali adds: “The young people treated at GOSH have already given us many sketches and diagrams and they overwhelmingly wanted to see more nature, more trees and less cars on the street.”

3 GOSH WELCOMES NEW FAMILY ACCOMMODATION

New family accommodation has opened next to Great Ormond Street Hospital (GOSH), thanks to the incredible support of Great Ormond Street Hospital Children's Charity (GOSH Charity) partners McColl’s and Royal Bank of Canada. Each has raised £1 million, which funded the refurbishment of this vital family accommodation.

Named McColl’s House and Royal Bank of Canada House in recognition of their support, the accommodation is housed in a pair of late 18th-century terraced houses, located close to the hospital’s main entrance. They will provide accommodation for up to 40 people, supporting, in particular, parents with children in intensive care units at GOSH. The facilities will include a mixture of family rooms and single rooms, all of which have their own en-suite facilities.

The communal facilities will be shared by the residents of both McColl’s House and Royal Bank of Canada House; these facilities include a kitchen, sitting and dining areas, laundry and a small quiet room. The family rooms enable parents and siblings to be together at weekends. They will also offer family members somewhere to take a much-needed break and to look after their own wellbeing while being there for their child. It’s a real lifeline during the most worrying of times for many families.

4 COVID-19 VACCINATION STUDY OPENS AT GOSH

Immuno-compromised patients at GOSH and Southampton Children’s Hospital, aged 12-17 years, are, for the first time, taking part in research to work out how they respond to the COVID-19 vaccination.

In 2021, a national study published early results suggesting some immuno-compromised adults had a lower response to the COVID-19 vaccination than healthy adults. This study helped inform the UK vaccination and booster programmes, but more needs to be understood about how these vaccines perform in younger immuno-compromised patients.

In September 2021, all four UK nations approved COVID-19 vaccinations for children in high-risk groups, aged 12 and over. While trial data showed the vaccine is safe for this age group, there was little evidence as to how much protection children with a weakened or altered immune system would get once vaccinated, and how many ‘boosters’ they might need.

This is because the body typically relies on a wellfunctioning immune system for a good response to vaccination. Some young people don’t have that because they are ‘immuno-compromised’; for example,

if they’re receiving cancer treatments or received a new vital organ and require immuno-suppressants to limit organ rejection.

The study will recruit up to 160 children between 12 and 17 years old who are already immunocompromised patients, with the first set of results expected by Autumn 2022.

“We are so excited to see this study open for the over 12s. Young people are often missed out as part of the research process, as they are deemed ‘too old’ for childhood research but ‘too young’ for adult research. This is a huge step forward to meet that area of unmet research need,” says Professor Lucy Wedderburn, GOSH Consultant Paediatric Rheumatologist and UCL Great Ormond Street Institute of Child Health Professor in Paediatric Rheumatology.

1934

This time in GOSH’s history

GOSH was the first hospital in the UK to obtain the Drinker Respirator (also known as an ‘iron lung’) to treat polio, helping to expand children’s lungs, allowing them to breathe and providing an alternative to frequent surgery. It was subsequently loaned out to other hospitals.

Much-needed new family accommodation.

Top right: When Great Ormond Street turns into Play Street for the day.

Bottom right: Artist impression of a greener, future Great Ormond Street.

Immuno-compromised patients at GOSH, aged 12–17 years, are taking part in an important COVID-19 vaccination study.

I HAVE... BLADDER EXSTROPHY

Thirteen-year-old

Kian from

Fife

in Scotland was born with his bladder outside of his abdomen. After many surgeries at GOSH, he’s now doing well and shares his story with Pioneer.

When I was born in 2009, I had a birth defect that meant my bladder sat outside my tummy and wasn’t formed properly. I came to Great Ormond Street Hospital (GOSH) when I was just three days old for an operation to put my bladder back inside my body and stayed at the hospital for 16 days. The surgery was successful. Although I can’t remember the early days at GOSH, my mum and dad told me that we were able to go back home to Fife after the first operation. We then returned regularly to GOSH for check-ups and to have cystoscopies [a procedure to look inside the bladder using a thin camera called a cystoscope] to work out what the next steps in my treatment would be. I had my next operation when I was 21 months old, which was a reconstruction of the bladder and getting a belly button made. My mum was able to stay on the ward with me and we were cared for by the same staff as when I was born and first treated at GOSH. Unfortunately, my bladder wasn’t growing properly with me so, in 2015, I had my next procedure – a bladder sling operation and more bladder reconstruction. Then in 2018, doctors decided I needed a big operation –called a bladder augmentation with Mitrofanoff –to enlarge my bladder.

When I visit GOSH now, I attend the urodynamics department, to make sure I can still pass urine without any problems through my specially made Mitrofanoff using a catheter. All being well, I won’t need any further surgery on my bladder for the rest of my life.

MEMORIES OF GOSH

I have lots of fond memories of GOSH. From playing on the big red bus in reception to being able to get out of bed after operations and head to the play room to go on the games console, from attending all the summer and Christmas parties to visiting the Disney Reef [a colourful, underwater-themed outdoor play area at the hospital]. Another favourite memory was when I drew a picture of a giraffe doctor, which was then made into a huge piece of art and displayed on the wall at the hospital. All the staff I’ve met over the years have been so supportive. But Karen and her team in urodynamics are the best. They’ve seen me grow up.

Nurse Donna and her team on Panther Ward are the same, they’re amazing and so nice. And my mum knows I am always in the best hands with my consultant and surgeon. He’s the man!

LIFE TODAY

I now come to GOSH annually for blood tests and an ultrasound to make sure my kidneys and bladder are doing well. I do have to choose hobbies that won’t involve much contact just in case it leads to a serious injury and causes me problems. I used to do acro gymnastics for years, which I loved, but in 2020 I decided gymnastics was no longer for me and joined a football team. I’m now a striker for the Hillfield Swifts, which I really enjoy. I love football; my favourite Scottish team is Dunfermline Athletic FC and my favourite English team is Manchester City – I would love to meet Kevin De Bruyne one day. Although the London cab drivers we talk to when I’m visiting GOSH tell me this is wrong and I should support Arsenal, which makes me laugh! When I leave school, I’d really like to play professional football, hopefully for either Dunfermline or Manchester City.

FUNDRAISING FOR GOSH CHARITY

Me and my family all took part in Great Ormond Street Hospital Children’s Charity’s (GOSH Charity) Race for the Kids in 2014, when the event was held in London’s Battersea Park. My favourite part of the event was – as it was a really hot day – my mum pouring cold water over my head to cool me down! I also liked how there were people standing at the side of the route just to give you a high five as you went past.

My parents also took part in the 2018 Race for the Kids when I was having one of my surgeries at GOSH and we took part again as a whole family in the first-ever virtual RBC Race for the Kids in 2020 and then again in 2021, in a local country park. I love taking part in this event and think it’s important to, as GOSH has been in my life since I was born. The hospital has done so much for me and has helped me lead as normal a life as possible. I want to give back to them for everything they have done, not just for me but for my family too.

THANK YOU TO GOSH

I’d like to say a big thank you to all the staff at GOSH, including the medical teams, cleaners, cooks, kitchen staff, everyone, as they are all amazing, funny and friendly. I cannot thank GOSH enough for everything the hospital has done for me and my family. When I’m 18 I’m going to get a GOSH tattoo just like my mum, to show my gratitude to this amazing place.

I cannot thank GOSH enough for everything it has done for me and my family.

GABRIEL’S CARE AT GOSH

Gabriel was under GOSH’s care for a form of acute lymphoblastic lymphoma for just over three years and recently finished his treatment. Mum Seyrah shares their story.

Gabriel, now 11, had a cough and cold that wasn’t getting better, after we returned from a holiday in October 2018. His face was swollen and he was more tired than usual. He progressively felt worse and, after a few weeks of being off school and numerous trips to A&E, he was admitted to our local hospital. Through a scan, doctors found something in his chest that they suspected was cancer. By the next morning, Gabriel had been rushed to Great Ormond Street Hospital (GOSH).

A PLACE IN GOSH’S INTENSIVE CARE UNIT

When we arrived at GOSH, Gabriel had to be put on a life-support machine. Because the tumour was on his chest and obstructing his airways, he couldn’t breathe on his own.

He was taken to theatre to try and find out exactly what it was. If it was cancer, they were trying to work out what kind of cancer it was and what stage it was at. When Gabriel returned from theatre, doctors explained everything clearly to me. He had T-cell lymphoblastic lymphoma, which was in his chest and was the size of a fist. Gabriel was then taken to intensive care and put on life support for six days. It was a difficult and traumatic time.

STARTING INTENSE CHEMOTHERAPY

The team looking after Gabriel initially tried to remove the cancerous mass, but it was too close to his heart. He was then recruited onto an ongoing clinical trial and his initial treatment was followed by chemotherapy, to try and flush it out of his system. Thank goodness this worked. We then spent 10 days on Giraffe Ward, where I was trained how to administer Gabriel’s medication. I was taught how to insert his nasogastric (NG) tube [a flexible tube passed through the nose, down through the oesophagus and into the stomach] making sure it was in the right place. He wasn’t eating well, so he had to have his medication in his drink. He would have milk to ensure he had all the nutrients, so his body could fight the disease. From then on, for six months, we visited GOSH every day. At the beginning it was quite intense. We’d go every day and quickly established a routine. It would either be a dose of his chemotherapy (it was different kinds at the beginning) or he’d have a lumbar puncture. For this, they would put Gabriel to sleep and take a sample of fluid from his spine, to check if the chemotherapy was working.

We were able to stay in charity funded family accommodation; the support GOSH gives is fantastic.

Some chemotherapy lasted around three to four hours and, in between that, Gabriel would get angry. He was so unwell at the beginning, he would just cry because he didn’t understand the way he was feeling. The team at GOSH just kept having to reassure him that “it’s just so you can get better”. It became frustrating for him as we were there for a long time. We kept on encouraging him to “do good waiting”, as he’d get bored.

This is where the Play team [fully funded by Great Ormond Street Hospital Children’s Charity], and particularly Play specialist Jennie, were amazing. Jennie and Gabriel would sing nursery rhymes, play games and dance. At one point the Play team even brought musical instruments; a drum and a guitar for Gabriel to play, as he loves his music. They would sing and Gabriel would get right into it! It made him so happy. The team made our time there a bit more enjoyable and a little less scary. He had something to look forward to, it wasn’t just medicine and treatment. The support GOSH staff gave to us while Gabriel was being treated was fantastic. We were able to stay in charity funded family accommodation next to the hospital and stayed there for three weeks. As I needed to be there for both Gabriel and his little brother, I was back and forth in my car. I was given a permit to park outside the hospital for the entire time Gabriel was in intensive care. Being a single parent, the team at GOSH made it easier for me to be there for my family. I could focus on Gabriel as we had a place to stay nearby.

MOVING INTO “THE MAINTENANCE” STAGE

Gabriel finished the intense chemotherapy midway through 2019 and was transferred to Safari Ward for outpatients. After that we went to GOSH every 10 weeks for the lumbar puncture – to make sure the chemo was doing what it was supposed to be doing. Every week we also went to our local hospital – for another part of his chemotherapy – and then he also took the oral chemotherapy at home.

At GOSH, Gabriel’s consultant, Professor Ajay Vora, would come and spend time with us – to see how Gabriel was doing. Gabriel has autism and ADHD, and there were some side-effects of the medication (specifically the steroids), which were affecting his behaviour, making him more emotional. It became quite a lot for me – like a mountain to climb.

So halfway through the trial, after almost two years, Gabriel’s team said let's try and stop it and see if he still responds to the treatment. They were understanding and very thorough – they listened to me and my worries and my concerns. They were adjusting the treatment to fit Gabriel and his needs.

BITTERSWEET ABOUT LEAVING GOSH

Gabriel ended his treatment at GOSH in March 2022. Before the end of it, I was both excited and anxious, as was Gabriel. The hospital has been such a big part of his life – he spent three and a half years there!

As he was finishing treatment he felt like “oh I’m not going to see my friends (at GOSH) again”. We kept reassuring him we’ll go back to GOSH for check-ups, that our contact with the hospital isn’t going to be just cut off. I’m hoping that, as time goes on, he’ll see this isn’t just goodbye. But I’m very happy and very grateful we don’t have to have any more lumbar punctures. Right now, Gabriel is doing really well. We’d been looking forward to ringing his end-of-treatment bell, all our friends and family were too.

HOPES FOR THE FUTURE

Gabriel’s funny, kind and very sweet. He’s affectionate and loves his cuddles and kisses. Gabriel and his little brother play in their boyish ways, throwing each other around. But they do need each other. They miss each other when they’re not together.

I hope Gabriel can lead a normal life and that he’ll be happy and healthy. This is what I hope and pray for him. I hope he’ll grow into a handsome, good young man, continue with his education and become something. I’d want him to be a good person and look out for other people.

SUPPORT FOR THE CHARITY

I’m so grateful there’s a place like GOSH for my Gabriel. The support the doctors and nurses give is amazing, they do everything to calm him down: singing nursery rhymes, watching trains on his iPad and just talking. If there wasn’t a place like GOSH, Gabriel would not be here, I would not be ok.

I’d encourage the supporters to please keep on giving. Nobody wants this for their child, but it happens. It’s just enough to know there is help and support. GOSH gave us hope, nothing is too challenging for them and they were always looking for ways to help us. Everyday I’m thankful these services are available as it’s helped Gabriel come a long way.

GABRIEL’S CONSULTANT, PROFESSOR AJAY VORA

A consultant paediatric haematologist at GOSH, Professor Vora cares for children with a variety of malignant and non-malignant blood disorders. He has a special interest in childhood leukaemia and cord blood transplantation. He talks to Pioneer about Gabriel’s time at GOSH.

“Gabriel had a less common version of acute lymphoblastic lymphoma (ALL). He had T-cell lymphoblastic lymphoma, which is a form of leukaemia that presents with lumpy masses. When Gabriel arrived at GOSH, he had a mass in his chest with some disease in the bone marrow.

"There are different combinations of chemotherapy for different types of cancer and even for different types of leukaemia. We have got standardised protocols for treating each type, as well as clinical trials.

“Gabriel was recruited to an ongoing clinical trial, for which I was the chief investigator (this means I had national responsibility for it).

“Trials like this – which we call Phase three trials – usually involve what’s called randomisation, where you are comparing different approaches to treatment. Future patients will benefit from the learnings of the trial Gabriel was part of.

“There are various factors that determine the chances of cure (for leukaemia) and, one important factor is a patient’s response during the first month of treatment.

“Gabriel responded really well, so we carried on with the chemotherapy. If he hadn’t, then he’d have been a candidate for either a bone marrow transplant or CAR-T cell therapy – where a patient’s immune system cells, known as T-cells, are modified to attack cancer cells.

“Instead, Gabriel continued the chemotherapy according to the trial schedule. The only challenges we had with treatment were around the side-effects of the steroids. But with Seyrah's help and support, we were able to deliver the treatment.

Gabriel is a lovely boy, and his mum is very good with him.

“Children with ALL do tend to respond well and have a good prognosis; we’re seeing more than 90% – approaching 95% – of children with this type of leukaemia cured. With T-cell lymphoblastic lymphoma the risk of relapse is primarily in the first two years after diagnosis. Gabriel is now three years post-diagnosis, so his risk of relapse is now very small, less than 1%. He’s got a good chance of being cured from this point. Not 100%, as nothing in life is 100%, but as good as.

“In the future, GOSH will look at developing new pathways for delivering cancer treatment for patients like Gabriel.”

“If there wasn’t a place like GOSH, Gabriel would not be here, I would not be ok.”

THE POWER OF PROGRESS

As GOSH marks its 170th anniversary in 2022, Pioneer celebrates the breakthroughs and innovations that have changed the lives of thousands of children.

Trailblazers in children’s healthcare, Great Ormond Street Hospital (GOSH) has, for almost two centuries, been the home of vital advances in paediatric medicine and care, the impact of which has reverberated across both the UK and the globe. The hospital has been at the forefront of countless innovations: from developing nursing training in the late 19th century and opening the UK’s first Leukaemia Research Unit in the 1960s, through to today’s breakthroughs in gene therapy and its pivotal role in the national management of COVID-19.

THE ROLE OF CHARITY

This extraordinary hospital has always depended on charitable support, since it opened its doors in 1852, to give seriously ill children the best chance to fulfil their potential. GOSH has a long history of fundraising, with restrictions on private fundraising introduced when the hospital became part of the newly established NHS in 1948. Now, while the NHS meets the day-to-day running costs of the hospital, GOSH relies on support above and beyond this to provide the best care, expertise and facilities. Great Ormond Street Hospital Children’s Charity (GOSH Charity) is younger than the hospital – it became a separate legal entity in 1998 – and supports the hospital and its patients by funding several key areas. These include state-of-the-art medical technology; pioneering research programmes into treatments and cures for rare conditions; patient and support services; staff support and education; and creating new child-focused environments that help children feel safe and calm during their appointments, treatments and recovery.

GOSH continues to forge ahead with pioneering breakthroughs, bringing millions of seriously ill children life-saving and life-changing treatments and cures for generations to come.

Here, Pioneer highlights just a snapshot of the remarkable progress GOSH has made in 170 years. You can find out more at gosh.org/what-we-do/ research/breakthroughs-childrens-medicine

BREAKTHROUGHS IN REGENERATIVE MEDICINE

1883 Sir William Arbuthnot Lane joined GOSH, establishing the ‘no touch’ technique, in which only sterilised instruments were used and fingers were kept at least four inches away.

1950s Richard Bonham Carter and David Waterston establish the GOSH Thoracic Unit, the UK’s first joint medical and surgical ward devoted to diagnosis and treatment of children with chest and heart diseases.

1970s GOSH Immunologist Professor Roland Levinsky developed a technique to isolate vital immune cells in the blood, extracting working immune cells from a healthy donor and transplanting them into a child. Professor Levinksy carried out the UK’s first successful bone marrow transplant in a child in 1979.

2001 Immunologists Professor Bobby Gaspar and Professor Adrian Thrasher sparked a groundbreaking programme of research into gene therapy, a technique where a faulty section of DNA (a gene) is replaced with a working copy. The team began a trial that would become the second-ever successful trial of gene therapy for any disease, anywhere in the world. Their patients were children born with a genetic fault that left them without a functioning immune system.

GOSH patient Rhys becomes the first child in the UK, and one of the first people in the world, to be treated with revolutionary gene therapy in 2001. Since then, GOSH has become one of the world’s leading centres for gene therapy.

GOSH celebrates its special anniversary in the same year that its royal patron, Her Majesty the Queen, celebrates 70 years on the throne.

2018 GOSH and University College London Hospital conducted the UK’s first pioneering surgery to help reduce the long-term symptoms of spina bifida, operating on a baby while still in the womb.

BREAKTHROUGHS IN CANCER

1958 Dr Roger Hardisty joins GOSH, a driving force behind remarkable changes in children’s cancer care. In his three decades at the hospital, he was pivotal in reducing the 100 per cent death rate for children with leukaemia to 70 per cent.

1961 The UK’s first leukaemia research unit opens at GOSH.

1973 Dedicated cancer inpatient unit established at GOSH.

1977 Hospital consultants set up the first UK-wide Children’s Cancer Study Group to promote research.

1999 Dr Paul Veys develops a kinder bone marrow transplant with less intense chemotherapy, helping children too sick for standard doses of drugs.

2013 Launch of one of the first European CAR T-cell studies, pioneered by Professor Persis Amrolia. It paves the way in this rapidly emerging field, but this type of CAR T-cells have now been superseded by ‘next generation’ cells that are more effective.

2015 GOSH immunologist Professor Waseem Qasim used CAR T-cells to treat a one-year-old patient with ‘incurable’ leukaemia. His incredible world-first sparked a new wave of CAR T-cell research around the world.

2018 An 11-year-old boy at GOSH becomes the first NHS patient to receive CAR T-cell therapy.

BREAKTHROUGHS IN NEUROSCIENCE

1928 Denis Browne appointed as UK’s first consultant paediatric surgeon, inventing a child-friendly mechanism to deliver anaesthetics, known as the ‘top hat’. It allowed surgeons to deliver sufficient anaesthetic to carry out operations, while crucially not covering the patient’s eyes. Tailoring medical care to children’s specific needs would be the driving force for significant further breakthroughs over the years.

1959 UK’s first children’s neuroscience unit established at GOSH under neurosurgeon Kenneth Till’s lead. Thanks to Till’s commitment, children with brain tumours, lesions, severe epilepsy or hydrocephalus could receive life-saving surgical care for the first time.

1977 Billy Butlin helps to raise funds to purchase the UK’s first paediatric CT scanner and GOSH is the first in the UK to have its own CT scanner suite, opened by the Queen in 1977.

1994 The hospital pioneers new investigation of brain damage, using MRI, ultrasound and spectroscopy.

BREAKTHROUGHS IN IMMUNOLOGY

1960s GOSH appoints John Soothill as the UK’s first dedicated paediatric professor in the relatively uncharted area of immunology. Of particular concern to Professor Soothill were children who had little or no resistance to germs. Soothill was the first to name these devastating conditions as severe combined immuno-deficiency syndromes (SCID); diseases arising from the body failing to produce the white blood cells required to recognise and attack germs.

1979/ Continuing Soothill’s work, 1980 in 1979 Professor Roland Levinsky attempted the UK’s first operation to transplant cells from a healthy donor into the bone marrow of a patient with SCID. Though not without serious complications, the procedure was ultimately a success, curing fatal immuno-deficiency with the UK’s first paediatric bone marrow transplant.

1992 GOSH researchers help identify the first genetic cause of lifethreatening immune diseases.

2000 World’s first gene therapy trials begin for children with severe combined immuno-deficiency syndromes.

2011 Gene therapy cures 14 children with previously fatal forms of SCID.

BREAKTHROUGHS IN HEART AND

LUNG

1947 The hospital’s heart and lung unit opens, the first of its kind in the UK.

1957 Mr David Waterson set up the heart and lung unit’s first research programme, funded with a charitable donation of £25,000.

1962 The hospital pioneers the first heart and lung bypass machine for children to help repair heart problems. By 1967, 60 per cent of infants with severe heart and lung problems were surviving.

1988 Professor Marc de Leval set up the transplant unit at GOSH thanks to a £200,000 fundraising appeal. It was one of the first centres in the UK to carry out life-saving transplants on children with heart failure. Today, the programme is one of the largest in the world, performing around 20 heart and lung transplants a year.

2000 Professor Martin Elliott launched a tracheal service (treating children with very narrow windpipes) to save the lives of children born with this condition. The multi-disciplinary team has since initiated an international training programme for hospitals around the world.

2010 GOSH pioneered a new treatment to save the life of a patient, who was born with a life-threatening narrowing of his windpipe. It was the world’s first trachea transplant in a child, grown from a patient’s own stem cells.

LISA’S STORY

I had a kidney disease/condition where the nasty toxins our kidneys help to excrete from the body were going back into both my kidneys and eroding them away. I’m left with half a kidney on one side and just under three quarters of a kidney on the other. Without specialist treatment at GOSH I may have lost one or both kidneys and needed transplantation.

I was treated at GOSH for around ten years – regularly to begin with but during my mid to late teens it was an annual check-up – and I remember my nurses being lovely. They would play cards or board games with me and, when I was more able to walk around, I used to sit at their desk on the ward and read a book or talk with them.

Back in 1997 when I was 28, I worked as a Float Clerk at GOSH, as I thought it might be a way for me to help 'pay back' for all the care I'd received as a child. The role was an amazing introduction to working at the hospital. I did this for two years before I went on to work in a variety of other administrative roles at GOSH.

I fondly remember the 150th anniversary celebrations when the hospital’s royal patron, Her Majesty the Queen, came and toured the hospital. I was lucky to be in the receiving line to meet her. Such a great day – a service at St Paul's Cathedral, lunch at the Guildhall and then meeting the Queen.

During my 18 years at GOSH I’d always taken an active interest in the charity’s work and had volunteered at charity events, but I heard there was a new Executive Assistant role in the Communications team working for the Director, a role I then worked in for six years. Getting the role felt like my journey with GOSH had come full circle. I’d once been a patient benefitting from the skill, care and excellence of the hospital, whose work is part-funded by money raised by the charity; now, I was going to help that charity raise funds to help patients like me.

GOSH means everything to me. It meant a second chance at a healthy life, an opportunity to do worthwhile work, be part of a team of caring, passionate experts and help play a small part in a much-loved, much-needed and much-respected organisation that helps children in need.

BILL’S STORY

Bill got in touch with GOSH Charity before he sadly passed away in September 2021, aged 96, thanking GOSH for looking after him as a baby and young boy.

Born in 1925, Bill arrived at GOSH two weeks after he was born, to be treated for a condition called pyloric stenosis. Pyloric stenosis develops after birth and is caused by overgrowth of the muscle controlling drainage of the stomach (the pylorus). It typically affects babies around six weeks old. Pylorus overgrowth causes the outlet of the stomach to be blocked, meaning milk cannot drain effectively. As all the feed is vomited, babies with the condition can become dehydrated.

In the early 20th century, a procedure called pyloromyotomy emerged; as a baby, Bill was likely one of the first generation to have this operation.

GOSH Neonatal and Paediatric Surgeon Mr Simon Blackburn describes a pyloromyotomy as a procedure where the muscle layers of the pylorus are split, without entering the stomach itself. Today, pyloric stenosis is still treated by a pyloromyotomy.

“Although we now perform the procedure using laparoscopic (keyhole) surgery,” Mr Blackburn says.

Bill’s pyloromyotomy during the 1920s would have been significantly riskier than it is today. “Open abdominal surgery on a six-week-old baby then would have been a big deal,” Mr Blackburn explains.

Bill's operation was successful and he remained an outpatient for five years.

Right up until Bill passed away in September last year, he could remember some of his time at the hospital, such as from when he was around five years old, he recalled standing outside GOSH and thinking it seemed like a “great big house”.

ANT’S STORY

Ant, now in his 30s, was treated at GOSH as a teenager in 2006 after he contracted three viral infections. Thanks to staff at GOSH and the life-saving ECMO machine (extracorporeal membrane oxygenation, a machine delivering oxygen into the blood) Ant made a full recovery.

Years later, he played a major role in winning a £7.5m charity partnership between Whitbread PLC and GOSH Charity, spending four years travelling the UK inspiring people to raise funds for the charity.

Bill said his parents often spoke of his early years and would say how impressed they were that GOSH saved his life. He also recalled his parents talking about a Dr. Schlesinger and how this doctor had played an important part in Bill’s early years. This would have been Dr. Bernard Schlesinger (1896-1984), who had a long career at GOSH as a general paediatrician.

On becoming a young man Bill joined the navy, where he stayed for 12 years. Later, he became an international diving instructor and swimming association teacher, marrying Norah in 1950. They were together for 57 years, until Norah sadly passed away. Norah and Bill had two children, both of whom had pyloric stenosis as babies.

From his home in Lyme Regis, Dorset, Bill still checked in on GOSH. He thought highly of the hospital and was grateful it enabled him to live a long, fulfilled and happy life. For many years prior to his passing, he donated to the charity every month.

“GOSH is absolutely marvellous. It’s great so many children get so much help these days,” he told us. “Thank you GOSH for saving me."

His optimism and drive to help others have been so inspiring that, in 2015, he was named one of the happiest people in the UK by the Independent on Sunday “Being an ex-ECMO patient has given me a second chance at life. Without it, I wouldn’t be alive. It took more than 100 people to help me survive. Families’ lives can change for the better thanks to the funds raised for the charity, allowing the hospital to truly provide the best care and the best medical equipment for patients and their families. Life is full of ups and downs but through the power of teamwork, amazing things can happen.”

Some of the hospital’s past patients share why GOSH means so much to them.

TAKING LEARNING TO NEW HEIGHTS

Pioneer reveals how the GOSH Learning Academy tackled the challenges of the past two years, pivoting education and training programmes to maximise the impact on staff and the children at GOSH.

Innovative and ambitious, the launch of the GOSH Learning Academy (GLA) at the tail end of 2019 aimed to move Great Ormond Street Hospital’s (GOSH) training, development and education into the same esteemed space the hospital is known for its care and research. With investment from Great Ormond Street Hospital Children’s Charity (GOSH Charity) and from GOSH, the GLA pooled undergraduate and postgraduate Medical and Nursing education, as well as Allied Health, Healthcare Science, and Non-Clinical Education under one umbrella.

Then, just a few months after the GLA’s launch, the UK went into lockdown.

Despite the challenges that lay ahead, the GLA revisited its approach to training and development in a multi-professional way, using its funding from GOSH Charity to the fullest effect.

“GOSH has become a safe haven for paediatric education over the last couple of years,” GLA Director of Education Lynn Shields (pictured right) says.

“Many things we’d planned had to change but we were well positioned to deliver education and support to staff using the investment from the charity, kickstarting the COVID-19 upskill training for GOSH staff and across the wider healthcare system. And, as part of their training, medical students, nurses and apprentices must work on wards but, as paediatric wards were full of adult patients in other hospitals, we welcomed everyone here so they could continue their training. We flexed to become a seven-day-a-week learning service.”

Lynn explains how charity funding allowed the GLA to prioritise systems safety simulation training, giving teams the opportunity to rehearse process, protocols and transfer of COVID-19 positive patients, shaping how the hospital kept patients, families and staff safe.

“This shift had a huge impact on our Just in Case, Just in Time training with the resuscitation team,” Lynn adds. “That included everyone in the team, from the desk administrators to the porters – they’re all needed as much as the clinical team. From running 30-minute education sessions at the bedside, we ensured the right patient was in the right bed and treated by the right team at the right time, potentially preventing an emergency and resuscitation call going out.”

EXTENDING THE GLA’S REACH

As face-to-face education was suspended during the pandemic, unless deemed essential around clinical competence and skill, the GLA utilised charity funding to launch its Digital Education Network (DEN) – slightly earlier than planned –so training sessions could be delivered virtually.

“A positive by-product of the DEN has been the extension of the GLA’s reach,” Lynn says, “and we now have candidates from across the world who are studying with us. Furthermore, as restrictions were placed on everyone, even children’s families visiting during the pandemic, so were work experience placements for medical and healthcare students. The solution? Using our virtual learning platform.

“The sessions were held at weekends using a blended online learning approach, with the added technology of remote simulation using cameras and Go-pros. Five hundred students signed up the first session and, because we can now work in a hybrid way to deliver education, our aim this year is to host even more students in this way.”

EDUCATION FOR ALL

On its launch in 2019, the GLA’s ambition had been to offer up to 20 specialist academic modules and postgraduate qualifications in its first three years. It’s already smashed that target, offering 36 academic modules at degree and Masters’ level in more than 19 paediatric specialties and is running the first national paediatric infection, prevention and control academic module.

“As part of the charity’s support of the GLA we were asked how we could use our voice at a national level and how it will positively impact the patients and their GOSH journey,” Lynn adds. “So, we’re running some modules in partnership, including the first paediatric bone marrow transplant module, in partnership with University College Hospital and the Royal Marsden; an orthopaedics and spinal module with the Royal National Orthopaedic Hospital; and a heart module with the Royal Brompton and St Bartholomew’s.”

Apprenticeships also remain high on the GLA’s priority list, with more than 200 now offered, across both clinical and non-clinical roles.

“We’ve been able to provide people with a career pathway and give them the confidence and support to study,” Lynn says. “Of the GOSH staff that embarked on the two-year nursing associate role that launched in 2017, the majority of them have stepped up to the nursing degree apprenticeship. We want to support healthcare professionals and their future, as this also ultimately benefits our patients.”

WHAT THE FUTURE HOLDS

The GLA continues its mission to ensure GOSH has the right staff with the right skills to care for GOSH patients, but also aims to enable others to care for GOSH’s patients locally. “For example, there’s a new national standard for paediatric chemotherapy so we’re asking, what sort of education is needed to deliver this?” Lynn explains. “Our lead educator, part-funded through the GLA, has written the only paediatric course around this subject, and we’ll be running the course next year. But it’s not just for GOSH staff; we’re looking at where children have chemo in their community – if we can train people local to them, they won't have to travel, and we enable further shared care. To use our voice at national level means we need to have all the right training in place to push forward.”

The GLA’s original plans may have had to alter direction slightly in the last two years but the principal one – to further cement GOSH as a highperforming, high-achieving educator on the national stage – continues to be a priority; a positive impact for the children and their families is the ultimate aim.

“To use our voice at national level means we need to have all the right training in place to push forward.”

THE PIONEER INTERVIEW

Pioneer talks to new GOSH Chief Nurse Tracy Luckett about her vision for the hospital’s nursing teams and what charity support means to them.

What inspired you to join the nursing profession?

My sister was a nurse and my godfather was a doctor so, when I was in Sixth Form, I decided nursing was the area I wanted to go into and trained in my home region of north Wales. I gained experience as a nurse in various clinical roles across the UK and my first role in London was at the Homerton University Hospital, followed by 13 years at Moorfields Eye Hospital, where I became Chief Nurse.

Why did you choose Great Ormond Street Hospital (GOSH) for your next role?

Having worked in a specialist organisation previously, you recognise the high quality of care and calibre of staff that work in them, and I was very aware of GOSH and all the wonderful work done here. GOSH has the fantastic combination of dedicated paediatric staff mixed with excellent academic and research work, all under one roof.

You started at GOSH just a few months ago; what are your ambitions for nursing here?

My role is to help make sure everyone here reaches their full potential and make informed decisions for the greater good of the organisation. I’m the accountable director for professional nursing and patient experience, so I’ll ensure we’re always part of the conversation to improve patient care and offer the training and development staff need to give the best care possible. It's still early days and nothing stands still but I’ve arrived at an organisation where nursing is already high performing and I have fantastic individuals working with me.

One area I’d like to explore opportunities in is the possibility of patient support officers, to help bolster communication on the wards between families and staff when it’s extremely busy. It could be a way to enhance what we already offer, for example, with our volunteers’ service.

Staff education and training influences every stage of the patient journey. What do you hope to see over the next year from the GOSH Learning Academy (GLA) for nursing staff?

I’m working closely with GLA Director of Education Lynn Shields, as well as with the apprenticeship lead, and we’re talking about the importance of allowing more individuals to gain access to leadership. There are numerous clinical programmes of work and education that clinical professionals can apply for and I’m keen to see whether there’s more we can do with the current leadership programmes, looking at how we work in the broader system of learning from each other and working collaboratively. Overall, this can only help patients and their families. Having great leaders who care, are passionate and who go the extra mile, who give more inclusive decision-making to the families; that should or will lead to better patient care.

I’ve been overwhelmed by the compassion I’ve witnessed walking around the wards at the hospital, as well as the kindness, excellent listening skills and professionalism of staff – all this, combined with good leadership, means you give the children and their families confidence in their care.

GOSH has the largest breadth of nursing specialties under one roof –what does that mean for the children and families who come here?

All these specialties mean they’ll receive expertise from committed people – when a child or young person comes to GOSH, they’re going to get the best care. And having such a vast organisation specifically dedicated to children, with 63 specialties under its roof, means staff don’t have to work just in one area forever. There are many initiatives around the retention of nurses, apprenticeships and newly qualified nursing programmes, and GOSH has a great internal transfer scheme that helps retain skilled staff. For example, if someone’s worked on one ward for 10 years, but wants to learn another branch of nursing, they can apply elsewhere in GOSH and the organisation can keep a good nurse. Rotational posts like these are critical. Although specialties are quite different there’ll be elements where pathways cross. So, if some children stay at GOSH for a long time, by retaining good staff you’re giving those children important continuity in their lives.

How do you see Great Ormond Street Hospital

Children’s Charity’s (GOSH Charity) support positively impacting the nursing teams?

Even though I’ve only been at GOSH for a few months so far, I’ve already noticed how some of the services I’m responsible for have benefitted from charity support, such as with the Chaplaincy and the wellbeing initiatives, elements of the Hospital School at GOSH – all these are fundamental to the hospital so we’re incredibly grateful GOSH Charity supports them. When the nurses see that support, it gives them some insight into how important nursing is at GOSH. Nursing is so much more than a skill; nurses see the wider picture and they are always putting themselves in the families’ shoes. The commitment from the charity to fund important work, whether it’s wellbeing, Chaplaincy, the most advanced medical equipment on the ward or training opportunities, that’s saying to nursing staff, we see you, we hear you, we know you are an integral part of this hospital.

I’ve already noticed how some of the services at GOSH have benefitted from charity support.

MEDICINES GIVING

NEW PURPOSE

Unfortunately, there are currently no treatments or effective cures and the care for children with this condition involves support from different clinical teams to help affected children with breathing, feeding and movement. There’s been ongoing research looking at gene therapies to correct the genetic mistake causing the disease, but it’s in the early stages and may not be suitable for everyone with the condition.

How did you and your team become involved with this global trial?

The principal investigator and site for the trial is SickKids, the Hospital for Sick Children in Toronto, Canada, and they invited the ICH to be part of the study. We’re the only site involved from Europe (two other sites are involved in the US). And, thanks to the support of the grant from GOSH Charity, we’ve been able to set up a collaboration with the team overseas.

Why is it important to research whether existing drugs for other conditions could be used to treat rare conditions in children?

It’s one of the fastest ways to develop new treatments, as the drug is already safely used in humans. Speed is crucial when new treatments are needed for severe and currently untreatable conditions.

In this case, tamoxifen is a well-known medication. Promising studies in mice with XLMTM found it may have significant benefits in slowing down the rate at which the disease progresses, improving survival rates and muscle strength. The drug seems to have an effect at both low and high doses. As the safety of the drug is widely known, plus the fact it’s relatively cheap, it’s an exciting potential treatment for the condition.

However, as this is the first time it’s been tested in this population of patients, there’s still a lot to be learnt, so this study has been designed to involve patients in a staggered way.

Discovering new treatments and cures into rare childhood conditions takes time, yet repurposing existing drugs can help speed the process along. Pioneer talks to two researchers about why this approach is crucial in child healthcare.

Dr Giovanni Baranello (pictured right), Great Ormond Street Hospital (GOSH) Consultant in Paediatric Neuromuscular Diseases and Associate Professor at UCL Great Ormond Street Institute of Child Health (ICH) – is leading the UK arm of a global trial looking at if the breast cancer drug, tamoxifen, could help children with a rare muscle disorder. His team was awarded a grant from Great Ormond Street Hospital Children’s Charity’s (GOSH Charity) National Call to fund this research.

What condition are you researching?

X-linked myotubular myopathy (XLMTM), a severe neuro-muscular condition that affects mainly male children. In most cases they show muscle weakness in the first few months after birth, have difficulty in achieving major growth milestones, may require respiratory support and the condition can lead to a shortened lifespan.

We’ve recently received approval from the MHRA [the UK government agency responsible for ensuring medicines work and are acceptably safe]. When we first applied for a research grant for this trial, the gene therapy trial had just started and we felt that, in addition to it, there were patients who could benefit from other ways of treating the condition, either as a combination or stand-alone treatment. As the gene therapy trial is currently on hold, there’s a higher expectation for results from this study with tamoxifen.

Why is charity funding crucial for this kind of research?

The conditions we’re researching are rare; there will only be a small number of patients we can consider for trial and there’s not always funding available for small groups from other areas. So it’s important to receive money from charities who want to help this specific population of patients.

Charity funding can often provide a bridge between pre-clinical and clinical research. When you’re collaborating with international teams and testing new medications in the lab, it may not always translate to patients because there isn’t funding for a clinical counterpart. Fortunately, GOSH and ICH have strong teams of pre-clinical researchers and clinical researchers; we already have that interface with patients so we’re in an ideal position to promote this medication and offer new treatment to children.

What are the next steps for the trial?

GOSH will hopefully soon recruit four patients. The entire process, from recruitment to trial completion, is likely to last a couple of years.

What are you hoping the outcomes will be?

The patients’ safety is paramount; ideally, we’d like to stop the decline of the respiratory function as well as providing some benefit to patients in preserving their motor abilities. For most patients and their families, even having something to stabilise their condition is perceived as a great advantage.

If this study is successful, tamoxifen has the potential to be one of the first effective therapies for XLMTM. This would give hope for children currently affected by the condition and future generations who develop it.

Speed is crucial when new treatments are needed for severe and currently untreatable conditions.

Professor Juan Pedro Martinez-Barbera, Professor of Developmental Biology and Cancer at UCL Great Ormond Street Institute of Child Health (ICH), is working across two research projects to discover treatments for a rare childhood brain cancer, diffuse intrinsic pontine glioma (DIPG). The research has been part-funded by GOSH Charity.

How does DIPG affect children?

Unfortunately, it’s an aggressive tumour found almost exclusively in children. The disease affects about 40 children a year in the UK and spreads so rapidly that, after diagnosis, the average survival is less than a year. The disease is caused by a faulty gene in the child’s DNA and the tumour grows in a deep part of the brain, the pons – this is part of the brainstem that helps transfer messages from the nervous system to various parts of the brain and the spinal cord. It's a critical part so, when the tumour grows there, many bodily functions are affected, such as breathing, sleeping and eye movement. The faulty gene removes a vital instruction that normally prevents cells from multiplying out of control. And, while we’re more accustomed to tumours that have a dense centre, with DIPG the cells are extremely mobile and migrate long distances from the pons.

Are there any treatments currently available?

As the pons is such a deep part of the brain, surgery is not possible. Chemotherapy doesn’t work with this tumour, so the main treatment is radiotherapy, which stops the tumour growing but only temporarily. Unfortunately, after three to six months the tumour will regrow, which is then fatal.

What is your research programme focusing on?

There are two different strands of research for this project. The main objective for the first, which started thanks to funding from GOSH Charity, is simple; as we know the faulty gene, we’re now looking at how we can counteract the actions of it. To that, we are growing DIPG tumour cells in the lab and exposing them to hundreds of different compounds – drugs – that have already been approved by regulatory agencies. If one of those drugs, or a combination of drugs, disrupts the gene’s cancer-causing ability there’s a chance of moving on to clinical trial.

How do you select which approved drugs to use on the cells?

We obtain libraries of compounds – there are around 500 in each library – from commercial suppliers and then screen them. DIPG is a brain tumour and, as the brain is protected from the rest of the body by the brain blood barrier [which protects the brain from toxic substances in the blood and supplies brain tissues with nutrients] we needed to source a library with permeable compounds. These allow us to test drugs that can get through the brain blood barrier and to the brain tumour.

Why is it important to consider existing drugs for complex conditions such as DIPG?

One of the main reasons is speed. But the advantage of redirecting "old" drugs to "new" uses is that a great deal is already known about the drugs, including their side effects and dosing requirements. This makes it much cheaper and faster to get them into the clinic than if they were new drugs. If we can successfully identify new drugs that could improve survival and quality of life for children affected by DIPG, Professor Darren Hargrave, GOSH Consultant Paediatric Oncologist and Clinical Professor of Paediatric Neuro-oncology, and his team, will take these discoveries forward into clinical trials.

How advanced is the research around DIPG? Alongside testing approved drugs on the cells grown in vitro, we want to identify a complementary treatment that will enhance radiotherapy given to children with DIPG. We know it works and now would like to find out how to prolong its positive effects. We know that radiotherapy induces tumour cells to enter a different cellular state called senescence. Senescent cells do not divide but secrete many factors that can accelerate tumour growth and tumour recurrence. Our aim is to identify drugs that kill these senescent cells in DIPG. Both strands of research are in their advanced stages; now, we are testing the efficacy of the approved drugs we have identified on mice, which are implanted with human DIPG cancer cells in the pons of their brain.

When we perform this operation on the mice we treat them like patients, giving them analgesia and ensuring they don’t have any other problems. We’re able to see how the tumour grows in the mice brains because it produces light, which we can measure with a specific machine. In addition, we can also irradiate the brains of these DIPG-bearing mice to simulate the treatment used in children with DIPG.

How important is it to researchers at GOSH and ICH that the charity funds research like this?

It’s critical. The funding from GOSH Charity allows for initial stages of research, meaning you can take a risk and initiate an approach where, if you see solid preliminary data, you can make your application for funding from other areas much more competitive. The funding from the charity is instrumental in the initial stages of research. For instance, previous GOSH Charity funding allowed work on a different childhood brain tumour (craniopharyngioma) and the preliminary data was used to attract significant funding from several other funding bodies, resulting in a clinical trial.

“ The funding from the charity is instrumental in the initial stages of research.”

Professor Martinez-Barbera (pictured third from the right) and his team.

CONOR THE PATIENT

Conor has developed a close bond with staff at GOSH over his 13 years being treated at the hospital.

“I’m reluctant to leave, it’s the place I’ve known my whole life,” he says. “I know all the faces; I know everyone there. I enjoyed my time in hospital, chatting with the nurses. At nights, if I was up and couldn’t sleep, one of them would play Uno with me.”

Conor’s mum, Jules, shares a similar sentiment.

“We have so many memories from GOSH. As we’ve been going there for so long, the staff are like family. It’s a home away from home. Any issues we’ve had, I always know I can ask anyone to help. I know Conor is in really good care.”

Moving from GOSH into adult care can be a challenging time for patients and their families.

“I have mixed emotions about the transition in the sense that he’s growing up now,” Jules adds.

TRANSFERRING FROM GOSH TO ADULT CARE

What happens to a young person’s care at GOSH when they reach their late teens?

Pioneer explores the process of moving from child to adolescent adult services.

Diagnosed with a form of cancer, acute lymphoblastic leukaemia, at just four years old, Conor, now 17, was referred to Great Ormond Street Hospital (GOSH).

Conor was put onto a treatment regime that lasted for three years, which included daily doses of chemotherapy. In March 2016, Conor relapsed and was told the cancer had spread to his central nervous system and his treatment was adapted to include chemotherapy and cranial radiotherapy.

In September 2017, Conor relapsed again. He was offered a new treatment, a clinical trial involving CAR-T therapy.* Unfortunately, this treatment wasn’t successful, which led to the family’s decision to try a bone marrow transplant. Thankfully, despite some complications, the transplant was successful.

Conor has had a remarkable journey at GOSH and now he’ll be embarking on another big change in his care; transitioning to adult services. Conor and his mum Jules share their thoughts on his next steps and how GOSH has been supporting them.

*CAR-T therapy involves a specialist collecting and making a small change to T cells. The cells are then re-entered into the bloodstream with the aim that they will recognise and attack cancer cells.

“Before his transplant, doctors said there might not be anything else they could do, but always adding ‘you’re not quitting and we’re not quitting’. I’m going to miss the bond we all developed. Knowing we’ll have to fit into a new hospital with a new team and try to find that same familiarity and rapport is a bit daunting. With the team at GOSH, every doctor in the hospital knows who Conor is.”

Despite some difficult times, Conor credits his support team with getting him through.

“At one point, I wanted to stop treatment. I’d had enough of seeing everyone sad. But the people around me were what changed my mind.”

The process of transferring Conor’s care to his new hospital, University College London Hospital (UCLH) began in January this year, kickstarting with Conor attending a joint clinic with his GOSH team and the team from UCLH.

“GOSH arranged it so we could meet the team at UCLH and they made sure they were with us for introductions. If anything arises ahead of the move, we know we can always contact the team at GOSH.”

“GOSH arranged it so we could meet the team at UCLH, and they made sure they were with us for introductions.”

ROCHELLE ADVANCED NURSE PRACTITIONER IN HAEMATOLOGY

Rochelle has been at GOSH for nearly 22 years, working closely with haematology patients who have malignant and nonmalignant conditions. She’s cared for Conor since he came to the hospital and tells Pioneer about how GOSH helps patients move on to adolescent adult services.

“We support patients the whole way from diagnosis, treatment and follow-up through to when they leave GOSH. As the outcome of leukaemia has greatly improved, the vast majority of our patients will have treatment and follow up and then will switch to adolescent or adult care.

“At GOSH, we’ve looked after our patients since they were little, so they’ve grown up with us talking to their parents. Once they move to adolescent care, they can take more control of their treatment plan. The support they will have around them in adolescent care is incredible; psychologists, activities co-ordinators, clinical nurse specialists, all experts at dealing with all the complexities of being a teenager.

“We’re lucky in our team because we don’t have to transition straight from paediatrics to adult, we have an adolescent service set up specifically for teenagers with cancer. This means patients don’t go straight into an overwhelming adult world.

“The medical and nursing care at GOSH is incredible but when patients reach their teenage years, we don’t have the support that the teenage unit has. Teenage patients might come into the clinic and be sitting in a waiting room with two-year-old patients versus the teenage unit where there are computers with internet, music rooms and other teenagers. Medically we give great care, and we can continue to give that, but we don’t have the adolescent peer support teenagers give each other.

“Conor had CAR-T cells and a bone marrow transplant meaning his move was delayed from our general pathway. He came back to our haematology team after his transplant was stabilised and we then started talking about the next steps, organising a joint clinic with everyone key to Conor’s treatment at GOSH and the team taking over his care at UCLH.

Q&A GRACE YOUNG PEOPLE’S FORUM

Have you discussed transitioning to adult care at the YPF?

“It was an opportunity to talk about any worries they had prior to that first appointment at UCLH.

“We’re always thinking about whether the services we offer meet our patients’ needs. We created the Joint Transition Clinics because the existing pathway setup for straightforward patients with one line of treatment wasn’t fit for purpose for patients having multiple lines of treatment or with complicated histories. We have a yearly team review where we look at the service, how many referrals and transitions we’ve had and whether we need to think about different ways of working.”

ON CONOR LEAVING GOSH

“We’ve looked after Conor since he was four years old through multiple lines of treatment. Now he’s outgrown us and we are helping him transfer to his new hospital. I know Conor and his family really well; we’ve been through so much together. We’ve supported them through lots of ups and downs, so understand there’s a sense of fear about leaving a team that knows him so well and has brought him back to a healthy place.

“For Conor, who has been coming here since he was four and is now 17, he can walk through the hospital and he’s a minor celebrity; everyone knows him. It’s not going to be the same straightaway at a new hospital; those relationships will be developed over time. We feel very confident and lucky because we know he’s going to an amazing hospital.

“So many times I’ve sat with Conor and his family where we’ve talked about another relapse or whether we think the next line of treatment will work. To see him now, able to move to the next step, is incredible. It’s something he, his parents and us at times, didn’t think would happen. That’s down to him, his family and us working as a team, so it’s tough to say goodbye. And, even though it’s worrying to move to a new hospital, it’s really positive that we’re in this position.”

Grace is 17 years old. She was born with a hole in her heart and has been a patient at GOSH ever since. She is also a member of the Young People’s Forum (YPF) at GOSH and will soon be moving over to adult care.

Why did you join the YPF?

I’ve been a patient at GOSH for a long time and have experienced many changes. The YPF looked like an interesting opportunity to get my voice across about my care and I felt I could share what I think needs to be changed.

Have you moved to adult care?

I’m still actively going to GOSH, I’ve got upcoming appointments this year and will continue to have them until I officially transfer to adult services at Chelsea and Westminster Hospital.

How do you feel about this next step?

I’m a bit nervous. I’m getting older and I knew it would come in the next couple of years, but I still feel a bit unsure.

We have. People have brought up that they’ll be moving and we’ve discussed how they felt. Some people knew what was going to happen and others were about to start so there were many different perspectives coming together to support each other.

From your experience, how might patients feel about transitioning from GOSH to adult services?

For people who have been at GOSH for tens of years or their whole life, it’s a big change. People wonder whether or not they’ll like it, whether it will be easy or a bit stressful.

When you get to around 17-18 years old, you are growing up in other ways as well. I’ve joined college in the last couple of months and got my provisional driving license. So, there are all these big life changes and a transition to a different hospital is on top of that.

Why is it important for GOSH to offer transitioning to adult services?

It shows they care and that you’re not left on your own. Some people are more ready than others to move across. It’s important that whatever stage you’re at – whether you’ve known about it for a while or whether you don’t know much about it yet – you are supported in a way that’s easy and fair.

It can take a while to process, but it makes it easier when GOSH offers the support to guide you and make sure you’re on the right path.

What advice would you give to a patient going through this change?

It doesn’t have to be this big and scary situation. It can be easy, friendly and quite a nice time. Talk to someone you trust in your team about everything that will happen during the transition.

“To see Conor now, able to move to the next step, is incredible.”

“GOSH offers the support to guide you and make sure you’re on the right path.”

WHY I SUPPORT THE CHARITY

What is your connection with Great Ormond Street Hospital (GOSH)?

Flying back from San Diego to London, in 2016, our daughter Aria, who was then two years old, was crying excessively, being sick and running a fever, so we knew something was wrong. It transpired that she had a hernia in her small intestine that had ruptured. We ended up at GOSH where she had emergency surgery. It was such a frightening time but Professor Paolo De Coppi [GOSH paediatric surgeon and Head of Stem Cell and Regenerative Medicine at the UCL Great Ormond Street Institute of Child Health] and his team saved her life.

Did this inspire you to begin your support of Great Ormond Street Hospital Children’s Charity (GOSH Charity)?

I grew up in the USA so, when we were leaving the hospital after Aria’s treatment, we were expecting to pay – yet this wasn’t the case at GOSH. We really wanted to do something to give back to the hospital, which is how we became involved with the charity. But we have been inspired by so much more than that. They saved our daughter’s life and we experienced, first-hand, the incredible way the hospital is set up. Aria was at GOSH for two months and, in that time, when I spent nights there by her bedside, I saw many other children who have spent so much of their lives there. Families would be having dinner together with their children, it was a real community. And making it so much easier in this difficult situation was the care the hospital staff gave to the children and their families. Everyone that works at GOSH wants to make things okay for the children, they have incredible patience, which, in turn, reduces anxiety.

How did the teams at GOSH reduce Aria’s anxiety?

Aria was very young at the time and had so many things to deal with, one of which was the blood tests, which she was frightened of. However, the patience and the playfulness that the nurses brought to their care for her made such a difference. I also remember the Play team at GOSH – they were amazing! And now, Aria is thriving, she’s at school, she has a lovely personality, cheeky with lots of energy and she knows she had surgery and that it was at GOSH.

How do you support the charity?

When Aria was being treated at GOSH we were so fortunate to have Professor De Coppi caring for her; he is an inspiration! This led us to supporting a PhD student in his hugely important programme of regenerative medicine research.

We also support GOSH Charity through event sponsorship such as the Valentine’s Party and encourage our businesses to take part in fundraising events. It’s important to us to donate to the charity and to support PhD programmes and research but we are also keen to raise awareness of GOSH and the charity, to highlight the incredible work they do and show that, as the hospital grows, so does the need to support it.

You supported GOSH Charity during the COVID-19 pandemic too?

Yes, when COVID-19 hit, we were based in Hong Kong and we saw there was a capacity issue with face masks in the UK and we knew we could support GOSH through our businesses.

We donated 235,000 medical-grade face masks to GOSH and we continue to support GOSH Charity through a partnership with our business called Three Masketeers, which we set up with our three children. Three Masketeers has one core product –a set of three reusable organic two-ply cotton face coverings with a cotton pouch. There are four designs available one of which, the ‘NHS We Appreciate You’ trio, incorporates the rainbow design that became synonymous with support for the NHS during the coronavirus pandemic. The children wanted to create fun face coverings ‘by children, for children, to help children’ which could be viewed as accessories and not as something frightening.

Why will you continue to support GOSH?

I come from a family of doctors so have had exposure to hospitals all my life and I’ve never seen one like GOSH anywhere. One where the care is exceptional but also where the doctors and nurses make things so much easier during a difficult time. The people I met – and that includes everyone that works there, the cleaners, the people working in the cafeteria, everybody – were so passionate and the positivity they brought to work every day was out of this world.

What I also love about GOSH is its holistic approach to caring for children. It’s not just physical health they take care of, it’s emotional and mental health too. The importance of the latter has been brought to light more since the pandemic yet GOSH was ahead of its time, because it was already providing this kind of care.

We fully appreciated this when Aria was there, it’s a rare thing. Children are the most vulnerable members of society and to know that, when they are sick, they get treated by people who are so passionate about healing them, holistically, makes it better for everyone.

The people I met at GOSH… were so passionate and the positivity they brought to work every day was out of this world.

Entrepreneur and philanthropist Faiza Seth shares with

Pioneer

why GOSH will always be special to her family.

HELPING IN THE FIGHT AGAINST COVID-19

A GOSH-led international team has recently discovered how the placenta acts as a protective shield against the COVID-19 virus. Pioneer finds out more about the work of GOSH researchers.

From the beginning of the pandemic, Great Ormond Street Hospital (GOSH) played an integral role in the national management of COVID-19. Among other projects, the hospital was part of rapid initiatives to study and track the genome of SARS-CoV-2, helping to guide the Government’s response to the pandemic; it explored the first human challenge studies to find a vaccine and manufactured a batch of the virus at the Zayed Centre for Research into Rare Disease in Children; and clinicians and researchers at GOSH and UCL Great Ormond Street Institute of Child Health (ICH) led the world’s largest study on Long COVID in children. Now, a new international study, led by doctors and scientists at GOSH and ICH, has revealed how unborn babies may be protected from the virus. The study, which was funded by the Medical Research Council (MRC) and UKRI COVID-19 rapid response initiative, provides the most definitive information, to date, regarding the susceptibility of the human foetus to COVID-19 infection.

It has shown that babies in the womb are mostly protected from COVID-19, via the placenta, but may become infected if their gut is exposed to the SARS-CoV-2 virus. Although the study did not look specifically at mothers with COVID-19 and whether their infection was transmitted to an unborn baby, it found certain foetal organs, such as the intestine, are more susceptible to infection than others.

However, researchers say opportunities for the COVID-19 virus to infect the foetus are extremely limited, as the placenta acts as a highly effective and protective shield. Evidence suggests foetal infection, known as vertical transmission, is extremely uncommon.

GOSH FACILITIES SUPPORTING THE STUDY

Professor Paolo De Coppi, GOSH paediatric surgeon and Head of Stem Cell and Regenerative Medicine at the ICH, and co-senior author of the study, led the international team. The research took place at the hospital’s Zayed Centre for Research (working with collaborators at UCL and UCL Hospital). Opened in late 2019, the Zayed Centre for Research was designed to offer a unique multi-disciplinary approach to paediatric research and care in a state-of-the-art facility.

It’s here that pioneering research and clinical care come together under one roof, helping drive forward new treatments and cures for seriously ill children in the UK and across the globe. A partnership between GOSH, UCL and Great Ormond Street Hospital Children’s Charity (GOSH Charity), the Zayed Centre for Research was made possible thanks to a transformative £60 million gift from Her Highness Sheika Fatima bint Mubarak, wife of the late Sheikh Zayed bin Sultan Al Nahyan, Founder of the United Arab Emirates. Major contributions were also made by Research England, The Wolfson Foundation, John Connolly & Odile Griffith and the Mead Family Foundation.

PIVOTING INTERNATIONAL RESEARCH IN A PANDEMIC

Due to the unique collaborative set-up at the Zayed Centre for Research, Professor De Coppi’s team was able to refocus its research into regenerative medicine towards infectious diseases, during the height of the COVID-19 pandemic.

Professor De Coppi explains why having the facilities to conduct the research under one roof was crucial to the study. "Being able to run the study at the Zayed Centre for Research allowed us to access all stages of research in one building, from cell selection to cell analyses in world-class facilities,” he explains.

“This work shows the power of collaboration between leaders in research – for the first time we are starting to understand how the placenta might ‘protect’ the unborn child from COVID-19 in the mother and how children can build up their own antibodies to coronavirus in the womb. Understanding the role of the placenta as the ‘filter’ to avoid the foetuses getting infected helps to reveal how mother and baby’s bodies ‘interact’ during pregnancy.

"We are focusing on this interaction at various levels and these laboratory-based studies have helped inform other clinical areas like surgery, where we are breaking new ground in foetal surgery, where we operate without delivering the baby.”

Recent advances in lab-grown mini organs, also known as organoids, can provide scientists with invaluable tools to study how human organs function, both when healthy and when impacted by disease. For the first time, an international team has used these advances to develop a lab-grown model of the human stomach, which will be used to study how infections in humans impact the gastrointestinal system. Researchers isolate stem cells from patient stomach samples and grow them under special conditions in the lab. This creates mini stomachs in a petri dish that can mimic the behaviour of a human stomach.

This development was pioneered by an international collaboration between GOSH, ICH and the Istituto Zooprofilattico Sperimentale delle Venezie in Legnaro, Italy. Much of the work was carried out in the Zayed Centre for Research.

As the COVID-19 pandemic progressed and gastrointestinal symptoms began to be reported, particularly in children, the team pivoted its research to focus on the impact of the virus on the gastrointestinal system, realising their mini-stomach could be a vital tool for studying this.

“This study has highlighted that SARS-CoV-2 infection may begin to infect the gastrointestinal system via the stomach in children and young babies,” Professor De Coppi says. “We hope this adds another piece to the puzzle as we try to build our understanding of the impact of the virus across the body. As a research team, we’re proud to have been able to contribute to the global fight against coronavirus in this way.”

The team now plans to continue its work with the mini stomachs, aiming to study how the stomach develops from early in pregnancy through to adulthood. It also hopes to look at the effects of other common gastrointestinal infections.

Running the study at the Zayed Centre for Research allowed us to access all stages of research in one building.

PARENT’S PERSPECTIVE

Angus was born with a congenital heart defect and diagnosed with two additional conditions within a month of his birth, which meant he spent the first nine months of his life in hospital. Dad, Jason, shares his son’s story.

Before Angus was born in September 2016, his mum Oona had become unwell with a life-threatening pregnancy complication that affects blood pressure and the liver called HELLP syndrome. We were aware prior to Angus’ birth that he had a congenital heart defect – called Tetralogy of Fallot (TOF) – but he was also diagnosed with two different conditions: tracheal stenosis and a pulmonary artery sling. Tracheal stenosis meant his windpipe was too narrow to function properly and wouldn’t continue to grow. A pulmonary artery sling meant one of his arteries connecting heart to lung was twisted the wrong way around his trachea, which wasn’t allowing enough blood from the heart to the lungs. The team in the

hospital he was born at in Glasgow placed Angus on continual oxygen support and recognised he would need to go to Great Ormond Street Hospital (GOSH) for life-saving surgery. GOSH was the only hospital in the UK, and one of few around the world, that could perform the necessary procedure. The operation was developed at GOSH and would include shortening and widening his trachea to help him breathe.

AN ANXIOUS JOURNEY TO GOSH

We readied ourselves for travelling to GOSH; we knew it wasn’t going to be easy as Angus was completely dependent on oxygen support to stay alive. He was sedated and intubated to allow for safe transport

down to London but there were complications and Angus suffered a cardiac arrest. He received CPR and the team managed to stabilise him. The next day he was placed on ECMO (extracorporeal membrane oxygenation), a machine delivering oxygen into the blood. Unfortunately, he’d also suffered a pneumothorax (a puncture in the lung) and this accelerated the need to get him to GOSH.

Once he was stabilised on ECMO, the immediate sense of panic was gone but the overall worry was heightened because we realised just how sick our son was. Travel had to be delayed as the weather in Scotland was terrible. It was the worst week of our lives.

Angus was flown down to London from Glasgow while Oona and I travelled by train. Seeing the number of people involved in his care brought home that we were in a unique situation. Two consultants and several nursing staff accompanied him on the flight. The plane couldn’t go too high; with the pneumothorax in his lung, the pressure would have been a problem. Halfway through our train journey we received a call to say he’d made it to GOSH via blue-light and was stable.

We arrived on Flamingo Ward where Angus was still on his ECMO machine and were able to hug him. That night we stayed in charity funded family accommodation, barely sleeping but so grateful to have somewhere to stay very close by the hospital.

SURGERY AND RECOVERY

The next morning, Angus had the eight-hour operation. His Tetralogy of Fallot (TOF) was repaired first, followed by replumbing the pulmonary artery sling and then his trachea was widened through the tracheal slide procedure. He had an amazing surgeon in Mr Muthialu and the operation was a success.

CHARITY SUPPORT

Angus remained in GOSH for a further three and a half months. He’d suffered major damage to his lungs and we experienced some worrying moments during this period. Oona stayed in the family accommodation the whole time he was in GOSH and I stayed there for two and a half months. To stay so close to Angus, to look out for him and be his biggest cheerleaders, was completely invaluable. I don’t know what we would have done if we hadn’t had somewhere to stay.

We also visited the GOSH chapel. The Chaplaincy team, also funded by the charity, would come and see us regularly. I knew the eight hours Angus was in theatre were going to feel like a lifetime and there’s little else you can do in that time but think, wish, hope and pray. It was nice to have somewhere I could go to do that.

In April 2017 we were eventually able to move back to Glasgow and Angus continued his recovery there, getting stronger the whole time. Finally, in June 2017, he came home, six months after the operation and nine months after he was born.

“ The Chaplaincy team, funded by the charity, would come and see us regularly.”

ANGUS TODAY

Despite still being on oxygen support and treated at our local hospital in Glasgow, Angus has vastly improved. He is now five and was due to start school last August, but we decided to defer him for a year to give him a chance to get stronger.

He’s a smart cookie and loves Hot Wheels, Brio trains and Transformers: Rescue Bots. He likes going on trains and driving my car – we can’t get in the car now without him jumping in the front seat and pretending to drive. He also loves cheese – we need cheese with at least two meals a day!

Our prognosis going forward is not straightforward and I’m sure there’ll be more challenges we have to face, but he has got a sense of normality about his future now, which is the best we could have hoped for. GOSH has given my son a life and changed the lives of everyone who knows him.

Great Ormond Street Hospital Children’s Charity 40 Bernard Street London WC1N 1LE 020 3841 3131 gosh.org

Thank you to all the patients, families and staff who took part in creating this edition of Pioneer.

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