Pioneer Autumn/Winter 2015

Autumn/winter 2015

Clinical revolution Ordinary children are doing some extraordinary things to improve researchers’ understanding of treating young people

06 Welcome to the latest edition of Pioneer...

Supporting paediatric research is hugely important to Great Ormond Street Hospital Children’s Charity, and the groundbreaking research taking place at the hospital and its research partner the UCL Institute of Child Health is absolutely crucial to our ability to continue offering hope to the very sick children in our care. In this edition of Pioneer, we look at the impact of research – how Great Ormond Street Hospital is collaborating with institutions around the country and the world to ensure that the patients themselves are at the very forefront of researchers’ work.

We talk to young people and Great Ormond Street Hospital staff to find out more about Generation R and Young People’s Advisory Groups, which are giving our young patients a voice to help make paediatric research as relevant to them as possible.

We also meet Dr Karin Straathof to see how a clinical study is revolutionising the way we treat neuroblastoma in children – using the body’s own immune system to target the common but difficult-to-treat childhood cancer.

After another compelling and insightful series of BBC Two’s acclaimed documentary, Great Ormond Street, Pioneer also catches up with one of the stars of the series – 15-year-old Jessica. We find out what it was like to be followed by a camera crew at such a delicate time, and how her new set of lungs have transformed her day-to-day life.

Thank you for your continued support of Great Ormond Street Hospital, and I hope you enjoy these stories.

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Tim Johnson Chief Executive, Great Ormond Street Hospital Children’s Charity

Contents 04 Around the hospital

Latest news from the hospital

14 Feature: Ordinary children doing extraordinary things

Young people across the country are coming together to improve paediatric research for generations to come.

Pioneer finds out more

06 The Pioneer interview: Dr Peter Steer

Chief Executive Dr Peter Steer looks back on his first 10 months at the helm of Great Ormond Street Hospital, the unique challenges the hospital faces, and his vision for the future

17 Why I support the charity: Grahame Chilton

Tick Tock Club Chairman Grahame ‘Chily’ Chilton explains the unique rewards of supporting Great Ormond Street Hospital Children’s Charity

09 Me and my nurse

Three children introduce the nurses helping their recovery

22 Targeting tumours from within

10 I have new lungs

Fifteen-year-old Jessica talks lung transplants, cystic fibrosis and what it’s like to star in the BBC’s acclaimed Great Ormond Street documentary

12 Technology on the front line

Pioneer takes a closer look at the increasingly crucial role of technology at Great Ormond Street Hospital

Pioneer looks at how scientists are harnessing the power of the immune system to fight and destroy cancer cells

25 Shared outcomes, brighter futures

An ambitious programme is underway to transform medicine on a global scale, putting patients at the centre of reporting medical outcomes

26 A parent’s perspective: My Peter Pan

Danielle shares the story of her son, Dylan, whose rare genetic condition may never be diagnosed

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Around the hospital…

Topping Out A 3D first

A team at Great Ormond Street Hospital have used a desktop 3D printer to create an exact anatomical model of a six-year-old’s trachea. The model was created using a CT scan and allowed anaesthetists to practice a difficult surgical procedure prior to carrying it out in a child who had a rare respiratory disorder.

3D printing is already used at the hospital as a means of demonstrating conditions and procedures to patients and families in a visual way. The team of clinicians and researchers at the hospital have now demonstrated the potential of 3D printing technology to make complex surgical procedures safer for children.

“Each time a child comes in to have their procedure they will have grown, and so require different equipment to be used for their treatment,” said Colin Wallis, Respiratory Consultant Physician at Great Ormond Street Hospital. “A model that is tailor-made to the child each time they have treatment means that the right sized tubes can be identified prior to surgery and a child can potentially be under anaesthetic for a shorter period of time.”

The technique was successfully trialled in six-year-old Katie, whose lung condition requires regular ‘lung-washing’ treatments to clear dangerous build-ups of material. After CT scanning Katie, the Great Ormond Street Hospital team 3D printed an exact model of her trachea, and were then able to rehearse the procedure prior to surgery.

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Great Ormond Street Hospital marked a number of crucial developmental milestones in September, including the Topping Out of the Premier Inn Clinical Building – part of the Mittal Children’s Medical Centre.

The ceremony marked the completion of the building’s final concrete pour, signalling the start of construction on an interior that will include a new surgery centre, the Aghia Philothei Trust Respiratory Unit and the Khoo Teck Puat UK Foundation Centre for Children – a 16-bed isolation unit for children with dermatology and rheumatology conditions or infectious diseases.

The Topping Out event was celebrated with a traditional Scandinavian ‘flying of the fir’ in which a fir tree was flown over the building by tower crane, as well as a final laying of cement, in a rooftop ceremony.

The new facilities in the Premier Inn Clinical Building will provide much-needed space for a parent or carer to stay by the child’s bedside overnight, more room for children to play and eat together and areas for parents to have some space. Once complete, it is anticipated that the entire hospital will be able to treat up to 20 per cent more children with the most complex, rare and often life-threatening conditions.

Chapel’s 140th birthday

In November, Great Ormond Street Hospital celebrates the 140th anniversary of the iconic St Christopher’s Chapel with a week-long exhibition.

Once described by Oscar Wilde as “the most delightful private chapel in London”, the Grade II listed building has provided a place for prayer and contemplation since 1875.

Despite the continuing evolution of the hospital, the chapel has hardly changed in 140 years, even surviving the demolition of the old hospital building in the late 1980s.

In 1988, the chapel was moved brick by brick to its current home in the Variety Club Building – a hugely complex operation lasting almost six years.

Now, to mark the chapel’s 140th birthday, a week-long exhibition is being held to celebrate a wonderful and unique milestone in Great Ormond Street Hospital’s history.

Taking place from 14 to 23 November, the exhibition showcases a number of items, photos and archival materials documenting the chapel’s active role in the life of our busy hospital.

Unique new centre named The world’s first-purpose built centre dedicated to paediatric research into rare diseases was named the Zayed Centre for Research into Rare Disease in Children at a ceremony in September.

The Zayed Centre for Research is a partnership between Great Ormond Street Hospital, University College London and Great Ormond Street Hospital Children’s Charity. It will bring hundreds of clinicians and researchers together under one roof to drive forward new treatments and cures for children with rare diseases.

Charity named Independent and Evening Standard appeal partner We are delighted to announce that we have been chosen as the charity partner for this year’s Independent and London Evening Standard appeal.

Look out for coverage this winter, direct from the patients, families, staff and volunteers of Great Ormond Street Hospital.

Hospital team treats ‘incurable’ leukaemia

The centre will be the first of its kind in the world and has been facilitated by a £60 million gift made in July 2014 by Her Highness Sheikha Fatima bint Mubarak – the wife of the late Sheikh Zayed bin Sultan Al Nahyan – as well as Chairwoman of the UAE General Women’s Union, Supreme Chairwoman of the UAE Family Development Foundation and President of the UAE Supreme Council of Motherhood and Childhood.

Her Highness was represented at the event by HE Professor Maha Barakat, Director General of the Health Authority Abu Dhabi. HE Abdulrahman Almutaiwee, UAE Ambassador to the UK was also in attendance.

The name was announced at a family celebration at Coram’s Fields in Bloomsbury, London, opposite the site of the building, which is scheduled to open in 2018. The event was attended by dozens of young patients who celebrated this important milestone through arts and crafts and cultural activities.

HE Professor Barakat said: “By combining the expertise of Great Ormond Street Hospital and University College London, the Zayed Centre for Research will become a centre of excellence, tackling some of the most challenging children’s diseases. Through the treatment and cure of thousands of children, the centre will serve as a global research hub, benefitting children and families from around the world.”

A new treatment that uses ‘molecular scissors’ to edit genes and create designer immune cells programmed to hunt and kill drug-resistant leukaemia has been used at Great Ormond Street Hospital.

The treatment, previously only tested in the laboratory, was used in oneyear-old Layla, who had relapsed acute lymphoblastic leukaemia (ALL).

She is now cancer free and doing well.

This breakthrough comes from Great Ormond Street Hospital’s and the UCL Institute of Child Health’s pioneering research teams, who together are developing treatments and cures for some of the rarest childhood diseases.

Find out more about gosh.nhs.uk

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The Pioneer interview Dr Peter Steer, Chief Executive of Great Ormond Street Hospital, speaks to Pioneer

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r Peter Steer joined Great Ormond Street Hospital as Chief Executive in January 2015, following a distinguished international career as a clinician, senior medical administrator and academic. He arrived at Great Ormond Street Hospital having overseen the completion of the Lady Cilento Children’s Hospital – a state-of the-art facility in Brisbane, Australia.

Could you describe your career before joining Great Ormond Street Hospital?

I’ve had a fairly long career across clinical work, education and some research over in Australia, New Zealand and Canada.

I did my paediatric and subspecialty neonatal training in all three countries, spending two years each in New Zealand and Canada prior to going back to Australia where I was a neonatal consultant in a teaching hospital.

During my tenure there I became a director of post-graduate education, which led opportunistically to a temporary management position that I never managed to get rid of! It genuinely was not a specific and deliberate career move.

Immediately prior to this role, I was CEO of a children’s health service and tertiary teaching hospital in Brisbane. I had the privilege and pleasure of building a new A$1.2 billion children’s hospital there, bringing two hospitals together. It was an exciting project, and something that I’m very proud of.

Why did you decide to come here?

It was two things, really. A combination of the major Lady Cilento Children’s Hospital project coming to an end, but more 06 Pioneer

importantly, it was the extraordinary opportunity of joining an organisation as prestigious as Great Ormond Street Hospital. In lots of ways, it was a very easy decision to make.

I’ve never had a bad job, and I’ve always worked with good people. It’s difficult to leave good people. But when I was offered this role, it was simply too great an opportunity to pass up. This is truly, I believe, the premier leadership position in children’s healthcare in the world. It was an honour to even have been considered for it.

Was it difficult leaving your clinical practice behind to concentrate on management?

For many years I was able to juggle clinical work with my management roles, but as those roles became bigger it became increasingly difficult. The risk was that I would be spread too thinly, so I decided to stop clinical practice.

It was a difficult decision to make, but I do think that clinicians have something to offer leadership in healthcare.

What advantage does your clinical background give you as Chief Executive?

My perspective on leadership is that it’s about enabling people to perform at their very best, and I think that having been a clinician myself helps me to facilitate good work in other clinicians. That’s probably as worthy a calling as caring for one patient at a time, because it’s about helping to shape an entire system of care.

I think it helps me ask the right questions.

It also means that I’m comfortable when I do walk around the hospital – I understand the space and the context of the care. It gives me a shared language with our staff, particularly with the clinicians.

I also believe that people who go from a clinical background to chief executive have a head-start with credibility. One needs to build on that credibility, though, as we can quickly waste it if we don’t perform and deliver.

It’s great to have sweated over a difficult case at 2am, to have had those difficult discussions with parents. It keeps me grounded in my role, and helps me understand what people are doing on the front line and the challenges we face.

Could you describe a typical working day?

Probably 50 per cent of my week is quite structured – recurrent meetings with key stakeholders, funders, the government, and with departments and internal leadership. The other 50 per cent is wonderfully random – opportunistic discussions about everything from clinical opportunities to the next educational challenge, or research and the way we work with the UCL Institute of Child Health, right down to where the next child is going to go if we can’t fit them into our intensive care unit. Ironically, despite the fact that I’ve given up the clinical work, it’s never too far from the context of anything I do between Monday and Friday.

I’d like to think that, symbolically, I have an open door. I think I’m approachable.

“ Our children and families are going through the most incredibly tough times of their lives.

We have an opportunity to make a difference.

What more could you ask for...?”

The downside is that it makes a mess of the day. I have a very untidy diary, and a very tolerant and wearied assistant as a consequence!

It’s important that people have access to leadership. That can be one-to-one, but obviously not all the time as we have 3,500 staff. Every month we have open all-staff forums and I try to get out and about across the organisation as well as ensuring I keep in regular contact with staff through all-staff emails and newsletters.

Has the hospital lived up to your expectations?

It’s exceeded them. You always have an impression of a place before you go there, and Great Ormond Street Hospital is iconic. It’s fantastic, but being here and seeing the context, and being able to touch and affect that is quite extraordinary. There are incredibly smart and committed people working here.

The reality of engaging with that day-to-day is hugely rewarding, and has truly lived up to, and surpassed, my expectations.

There’s a particular case that stands out in my mind. It was really long and difficult but it highlighted the incredible efforts of our staff to help the child, and the teamwork at play. Our staff went above and beyond to deliver exceptional care as a team. But that’s normal here – people really do go the extra mile.

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“ We fit into a space that few other organisations do in the world.”

Given your academic background, do you take a special interest in the research being performed at the hospital?

Well, firstly, the researchers here are way out of my league! Although I’ve had a particular interest in research and have done some myself, the people we have here are just on another level. The research being produced by the partnership between Great Ormond Street Hospital and the UCL Institute of Child Health is better than any children’s institution or hospital in the world. These people are the very best at what they do.

So my job now is to see how we can provide these people with the right environment for them to do their best work. Then, it’s making sure that two things happen: one, that their work and the questions they’re answering are informed by real patient problems, and two, how do we find and take their discoveries back into medical care?

That’s the interface that I’m responsible for every day.

Research is core to the hospital’s future.

We fit into a space that few other organisations do in the world – from discovery to care, the full spectrum of paediatric health. That’s hard to do well, but we have such resource here: in our tradition, in our patient population and in our talented staff, so it is our responsibility to deliver.

How important is the relationship between the charity and the hospital?

It’s absolutely critical. Strategically, it puts us in fantastic position to care for children as expertly and efficiently as possible.

Thanks to the support of our generous donors through the charity, we have a balance sheet that isn’t burdened by debt for the new buildings. We’re incredibly fortunate for that, as it gives us a level of freedom that our colleague institutions around the world don’t enjoy.

But that’s the big picture stuff. The day-to-day stuff is the vital equipment that the charity buys for us, and the welfare it helps us provide for our patients every single day.

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There are tough times ahead for the NHS, and for all UK hospitals, but supporters of Great Ormond Street Hospital Children’s Charity are supporting something incredibly important. We need to be worthy of that support, by being the very best that we can possibly be.

What’s your vision for the future?

I don’t really do anything alone so, in terms of the hospital leadership, there are a number of parts to our vision for the hospital’s future.

Together, we are stewards of an extraordinary tradition and extraordinary work. So we have a great platform, and now we need to build on it. That means continuing to work with all our stakeholders, external and internal, to continue to grow the mission of being a cutting-edge provider of the most highly specialised children’s healthcare anywhere in the world.

We need to be well resourced, we have to have the right people, and they have to be

equipped to be innovative and to do research that always has patients and their families at its core.

In short, our vision is to grow both the substance and reputation of Great Ormond Street Hospital as the exceptional provider of healthcare to children - a national and international resource. We have to be both strategic and opportunistic, and ensure that the best interests of our patients are always at the heart of everything we do. We can’t do it alone, either, so our partnerships will remain key to our future.

Those of us who work at Great Ormond Street Hospital have a rare privilege. Our children and families are going through the most incredibly tough times of their lives. We have an opportunity to make a difference. What more could you ask for in terms of a rewarding role? To me, it’s the best motivation there is.

“Our vision is to grow both the substance and reputation of Great Ormond Street Hospital as the exceptional provider of healthcare to children.”

Me and my…

Nursing is at the heart of the exceptional care that Great Ormond Street Hospital provides to tens of thousands of children every year. The hospital employs more than 1,000 nurses in a wide variety of roles and, in partnership with the London South Bank University, is the largest trainer of paediatric nurses in the country. Here, three Great Ormond Street Hospital patients talk to Pioneer about their favourite nurses.

Matilda, 6, and mum Melanie, Somers Clinical Research Facility Matilda’s nurse is called Laura – she’s a Novel Therapy Research Nurse.

Every 10 days Matilda comes to the hospital as part of a clinical trial.

She’s the first child in the UK to take part.

Laura helps Matilda with a transfusion every two weeks.

Matilda loves Peppa Pig and fast roller coaster rides!

Stanley, 3, Bear Ward My nurse’s name is Tash.

She cheers me up by making me balloons out of rubber gloves.

I come to hospital because I have some problems with my heart and lungs.

My favourite thing about Bear Ward is the play room and the bears on the wall.

When I grow up I want to be Spider Man.

Her favourite food is sausages, mash and gravy, and she loves chocolate mousse too.

Alfie, 9, Giraffe Ward My nurse’s name is Charlotte.

Giraffe is my favourite ward at the hospital because of my nurses.

They help to make me better, and they sing and dance with me which makes me happy!

The best thing about hospital is the nurses and my doctors, they are fab.

I don’t mind being in hospital, as long as it’s not for too long.

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I have new lungs Fifteen-year-old Jessica was born with cystic fibrosis, and featured in the second episode of BBC Two’s Great Ormond Street earlier this year as she underwent a lung transplant 10 Pioneer

I was diagnosed with cystic fibrosis when I was still a baby, but I was very active when I was young. I led a normal life until I was about nine, when I had to go to hospital every few months. By the age of 11, I had deteriorated significantly. I was on oxygen all the time, and I couldn’t really do stuff – even walking from my bed to the toilet would leave me out of breath.

“It wasn’t much of a life – I felt like I didn’t really have a purpose or a reason for being. I like to keep busy, I always have, but trying to keep busy just made life even harder. I’d go to different places with my family, and my little sister Summer would run around and play, but I couldn’t do that.

It made me even more upset.

“I used to dream that I was healthy – that I was running and having fun. So I loved sleeping, because I dreamt that I was normal. It was a fantasy world where I could breathe. Some people’s fantasy world is being a princess or something – mine was just being normal. That was my dream.

“When doctors first offered me a place on the lung transplant list, I turned it down.

I just thought that there wasn’t any point having it then – I wasn’t even on oxygen at that point. But then, as I got more and more ill really quickly over the following year, I decided to go on the list.

“In the 10 months I was waiting, I got taken off the list three times because of infections. It really annoyed me when that happened, because I thought I might not survive long enough to get back on it. Life on the list is really hard. You can’t carry on as normal, because the call can come at literally any moment. You always have to be prepared for it. It’s always at the back of your mind.

“When the call eventually came, my transplant surgery lasted about eight hours. When I first woke up and they took my tubes out, I hated it! I was just so used to breathing with rubbish lungs – I’d never known anything else. It took a while to get used to. But when I started walking again, and I wasn’t getting out of breath – that was really crazy. After a few weeks I felt fine, it was so much better. Now I don’t get breathless at all – my legs give out before my lungs do!

like going out with my oxygen on and in a wheelchair, because people would stare at me. Now if people stare at me, it’s because they think I’m that girl from Game of Thrones!

“It was weird being filmed by the BBC while it all happened – I found it quite scary at first. But once I’d got to know the film crew, it was actually really nice to have so many familiar faces around at the hospital. All I kept thinking was that if the programme went out, it would show so much of the raw journey of transplantation. It’s not just being put on a list and then suddenly having new lungs.

There’s so much work that goes into it, so much waiting, so many people involved, and it can be incredibly hard at times – for everyone. I love that they showed the surgery and how hard it was for the surgeons – how tired they were afterwards. So I’m really happy with the way the documentary told my story, and my friends and family thought it was really good too!

“ The surgeon came through to the intensive care unit to see me, even though he was so tired… It shows the passion and the thought that they put into every single case that they have.”

“I still come to Great Ormond Street Hospital for check-ups, and will do until I’m an adult. It’s a really great hospital – the doctors here were always thinking of ways to improve my condition. After my transplant, the surgeon came through to the intensive care unit to see me, even though he was so tired. He did that because he really cared. It shows the passion and the thought that they put into every single case that they have.

“I think it’s a normal life ahead for me now – nothing’s going to stop me. I’m still hoping to go to university, and I love swimming and cycling and just seeing my friends out and about. It’s crazy to think that someone has done this for me – that they or their family gave me this chance.

But now I feel like I shouldn’t waste time sitting around and being lazy, I need to be out there living my life. There will be bumps in the road, but I know they won’t be anywhere near as bad as they were before my transplant.”

Jessica with mum, Cheryl, and sister Summer

“Having a transplant has completely changed my life. My outlook is so different now – I’m much more confident. I didn’t Pioneer 11

Technology on the front line With the use of technology becoming increasingly prevalent in global medicine, Pioneer speaks to medical innovators at Great Ormond Street Hospital to find out how technology is altering the traditional hospital environment

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re we on the cusp of the next truly great disruptive development in medical technology? With robots in operating theatres, 3D printing routinely used in hospitals around the world, and machines with the practical knowledge of a second-year medical resident, the signs are promising.

As recently as the turn of the decade, wearable technology was seen as the next big thing. Now, arguably, it’s the big thing. As the world’s biggest technology companies fall over themselves to dominate the market in wearable technology, the medical world is more than playing catch-up.

The unveiling of the Apple Watch in March this year was widely hailed as a landmark event in modern technology – one of the world’s information technology giants taking its first major step into wearables.

It reinforced the notion that wearable technology will play a significant societal role in coming years, and opened the door wider for medical pioneers in the field of wearables and big data.

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One such pioneer is Elin Haf Davies, co-founder of aparito – a smartphone app utilising wearable technology that is currently under development at Great Ormond Street Hospital. The app takes advantage of motion tracking in innocuous wristband devices to monitor the movement of patients with ataxia – the loss of control of bodily movements – and related ambulatory conditions.

“I worked at Great Ormond Street Hospital as a children’s nurse for 15 years, specialising in research,” said Elin. “When I started, a child and their family might have to go through a long and emotionally exhausting clinical trial of drugs using tests that weren’t designed for specific ages or diseases. So I wanted to find a way of measuring ataxia in children using ageappropriate, disease-specific tests for children, which were not painful or invasive, and didn’t take a lot of time.

“With aparito, a child wears a device that looks just like a normal wristband but transfers data via Bluetooth to a smartphone app. That data can then be

accessed by the patient’s doctor, who will have a complete picture of how that child has been getting on since their last visit.”

The benefits to patients and clinicians alike are clear. A patient’s normal activity can be analysed in a constant, non-invasive way, leading to more accurate measurements and fewer or shorter hospital visits. As well as recording more ‘normal’ ambulatory data, the app will also enable clinicians to precisely map the events leading up to a seizure or fall.

“ The appetite for technology, and the people who use it and want to use it, is definitely higher than average at Great Ormond Street Hospital.”

Elin Haf Davies

“There’s also the bigger picture to consider, and the societal value of the big data we can produce using wearable technology,” says Elin. “There is a clear benefit for the child and family, but in the wider spectrum of big data analytics, it’s going to offer doctors a better insight into individual patients and whole cohorts of patients with specific diseases. NHS service providers will then know what groups of patients need in terms of care and support management, and we’ll even see whether patients on specific medication or treatments are enjoying better quality of life or improvements in their conditions when compared to other patients.”

The wider implications of big data in medicine is something that Professor Martin Elliott, Paediatric Cardiothoracic Surgeon at Great Ormond Street Hospital, finds exciting. Ahead of a 2015 lecture on disruptive technology at Gresham College, Professor Elliott surveyed his peers across the globe, finding that wearables and big data are close to the top of the international healthcare agenda.

“Almost everybody around the world is imagining that we will be putting devices into our patients, and that patients will voluntarily be wearing things like the Apple Watch and collecting data about what they’re doing,” says Professor Elliott. “If you combine that data with the ability to do big data analytics, you can imagine machines helping you build algorithms to tell you whether a patient is inside a control range or not. Then, as they get further and further out of that range, you would see escalation policies determining whether you should see the patient or change their treatment.

“The really hard thing is that the data you would use to create that system is currently of questionable quality. Until electronic patient records around the world have really strong data quality and standardised data definitions, we simply

can’t rely on the data. You’re going to need really smart search engines that make inferences, rather than factual conclusions, based on what they see. And then the machines can teach each other – something that is happening already.

“One current example of that sort of search engine is IBM’s Watson, which is estimated to know as much as a secondyear resident in the United States. It’s really quite a remarkable thought – a machine fed with the answers to certain questions will come up with as good an answer as someone with a lot of training.

“If you imagine a piece of wearable technology transmitting data to a piece of software with that level of analytical capacity, it could completely change the management models of how we look after patients.”

“ It’s really quite a remarkable thought – a machine fed with the answers to certain questions will come up with as good an answer as someone with a lot of training.”

According to Professor Elliott, the argument that a combination of wearable technology and big data analytics – and how that data is presented – could lead to a radical shift in the hospital environment is persuasive.

“I think what could make this technology disruptive for us is the mode by which we make diagnoses and interrogate each other,” says Professor Elliott. “The ability to find the right treatment is not necessarily going to come from a doctor – it might come from systems that know better than we do. Learning to trust those and to work with them and let them start making decisions on their own is going to be hard, and will take time. A long time, perhaps, but the implications for future hospital staffing models are certainly there – the roles of care providers may become more prominent, for example.”

For now, wearable technology remains in its infancy in UK healthcare, despite significant recent advances in other

areas of medical technology, such as 3D modelling and printing. However, Professor Elliott believes that Great Ormond Street Hospital is perfectly placed to adapt to the future, thanks to its innovative and world-class staff.

“You’ve got a lot of visionaries at Great Ormond Street Hospital – a lot of people who know what the future might hold and will rapidly embrace it. They are already using absolutely frontline technology, and they are actually contributing to that technological development themselves.

“The appetite for technology and the people who use it and want to use it is definitely higher than average at Great Ormond Street Hospital. People here are constantly pushing for innovation.

“And the bottom line is that none of this is any good if it’s not of value to the patient. That’s the only way that you can make this work. Everything we do in technology must be judged first on its value to the patient. Nothing else. We work the rest out later.”

What’s coming next at Great Ormond Street Hospital?

3D printing and modelling

“We’re already seeing the benefits of 3D imaging and modelling at Great Ormond Street Hospital, and the further potential is clear. We’ll be rehearsing operations, designing procedures, playing with new materials, printing bespoke items for specific people – that’s all going to happen, and soon.”

Big data

“If we have all the data associated with a patient’s medical history and genome, we can analyse it and see why their condition developed. As our storage and analytic capacities become larger and cheaper, then we will have a fundamental role in that field because we see 85 per cent of rare diseases.”

Wearable technology

“We can already project CT scans onto patients on the operating table, but it’s only a matter of time until you can actually wear those images in a headset. You could then add information which would help you make your decision. For example ‘there’s a 47 per cent chance you’ll hit the liver if you take the 42-degree angle’. That’s just one example of how wearable technology can help us – there are thousands more.”

Professor Martin Elliott Pioneer 13

Clinical research Ordinary children doing extraordinary things

Pioneer takes a look at the extraordinary benevolence of ordinary young people who are helping to improve paediatric research for generations to come

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t any one time, there are several hundred active research projects taking place at Great Ormond Street Hospital, from questionnaire studies to clinical trials. This research is essential in order to understand more about childhood conditions. There is a misunderstanding that treatments that work on adults can be used on children by simply reducing the dosage, but this is not always the case. It’s thanks to the willingness of young people that researchers are able to test how these treatments affect children.

The millennials – that group of young people born between the early 1980s and early 2000s – are increasingly coming under fire in the tabloid press. It’s refreshing, then, to see proactive and engaged young people up and down the country taking ownership of their lives in order to help others like them by getting involved in research. They are becoming advocates for learning more about the effects of treatment on their health.

Eighteen-year-old Esme took part in a three-year clinical trial at Great Ormond

“ Research isn’t simply on children, it’s with children.”

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for Children Research Network. The London group was set up in 2008, and is now funded through the NIHR Biomedical Research Centre at Great Ormond Street Hospital and University College London.

Each of these groups comprises 10–15 members, aged between eight and 21.

Esme, now 18, took part in a clinical trial when she was eight years old Street Hospital for her condition, Alport syndrome – a rare, inherited disease that causes kidney inflammation – when she was eight years old. “It feels pretty awesome to be part of a clinical trial,” says Esme. “It’s an unusual thing for people to hear about. You become a lot more aware of the importance of medicines: at eight years old I would have just taken my tablets and not really noticed, but because of the trial I became more aware.

Meeting every six to eight weeks, the YPAGs work with researchers to help evaluate and guide the appropriateness of the information they deliver to children and their families when embarking on clinical trials and other forms of research.

They contribute to raising the profile and awareness of involving children and young people in research.

“Some researchers don’t have much experience of talking with children in this consulting capacity,” says Dr Erin Walker, Joint Lead for Patient and Public Involvement and Engagement in Research at the Biomedical Research Centre at Great Ormond Street Hospital and University

“When I first received the information leaflet for my clinical trial, I read through it, but I didn’t process much of it,” explains Esme. “My experience of participating in a clinical trial means I know what sort of information a young person might need at a particular age. Also, a lot of the members are children of nurses or are regular consultant patients, so, as a group, we have a bigger variety and range of ideas and experiences.”

Although feedback from researchers has been positive, it can take time to influence a culture shift among some of those individuals who are used to doing things a certain way and without the input of young people. “There are different kinds of ways that researchers have presented themselves,” says Esme. “Some come to the table and discuss without any presentations, and they speak to us normally. Then there are others who talk us through huge presentations about what they’re doing, and we have five minutes to

“Clinical trials are helpful for not only yourself – because you learn more about how your medicine works – but what you’re getting involved in is helpful for other people who have the same condition.

I think that’s why a lot of young people take part. You feel that what you’re doing is making a difference somehow, even if it might not be right in front of you. It’s about being aware of what your decision means for others.”

Participating in a clinical trial is an enormous undertaking, and speaks of a profound altruism embedded within the young people who take part, some of whom may not even have a condition at all. So it’s understandable that those who participate should be fully informed of what they are signing themselves up for.

As adults, many of us will have received medical information that is about as easy to decipher as the Enigma code: how can younger children be expected to fully comprehend what they’re signing up to if the families themselves struggle to overcome the seemingly jargon-riddled information leaflets given out by researchers? Enter the Young Person’s Advisory Groups (YPAGs).

Esme is a member of the London YPAG, one of six groups across the UK initiated via funding from the National Institute for Health Research (NIHR) and the Medicines

The London YPAG meets every six to eight weeks

College London, who facilitates the groups and acts as a conduit between the researchers and the young people.

“We’ve had some excellent feedback from the researchers who have brought work to the sessions. The young people will advise that information is conveyed differently depending on the target age.

They may even redefine the age ranges for information.

“We’ve seen researchers go away and say ‘wow, this is all helpful feedback. I’d never have thought about that’ to something the young people come up. That’s exactly why these groups exist. They have insights that are different from adults and crucial to success.”

discuss and contribute to their project.

We’re not able to help as much in that small timeframe.

“There’s a nice line we use at our events: ‘research isn’t simply on children, it’s with children.’ That not only talks about how you should treat children, but also that the involvement of young people throughout the whole process of the clinical trial is super important. You have to learn how to work with a young person.”

For Dr William van’t Hoff, Head of the Somers Clinical Research Facility and Consultant Paediatric Nephrologist at Great Ormond Street Hospital, the success of the YPAGs presented an opportunity.

Pioneer 15

He felt that the young people could demonstrate the impact they had made at a national meeting designed, run and delivered by the young people. In September 2013, London’s Science Museum played host to Generation R, the first event of its kind in the world. The young people decided to run the event in the format of a TV panel show, where they interviewed several high-profile delegates, including Chief Medical Officer for England, Professor Dame Sally Davies. More than 160 stakeholders attended, including parents, researchers, pharmaceutical company representatives, health professionals and funders.

Dr William van’t Hoff, local director for the MCRN and Consultant Paediatric Nephrologist

From that event came 11 recommendations of future work that are currently ongoing, including exploring alternative and innovative ways of engaging with more young people and families, and working with the education sector to promote clinical research education in schools.

Feedback post-event pointed towards more and more researchers wanting input on their research from young people.

Since then, Generation R has evolved from a one-off conference to a movement for change in the way researchers collaborate with young people. The Nuffield Council on Bioethics recently published a report on ethical issues around children and clinical research, which had significant input from the YPAGs. The group made recommendations and one member attended the launch to speak on their behalf. Sir Iain Chalmers of The James Lind Initiative, a partnership to improve clinical research, has strongly supported Generation R and is now heavily involved in its future work.

16 Pioneer

“We’re doing a lot to change the way research is done with children and young people, to make researchers more aware of young people and their importance and role within clinical trials,” says Esme. “The Nottingham and Liverpool groups went out to speak at a conference in the USA recently about our work. There are groups of young people like our YPAGs in different US states, and we’ve been the inspiration for young people to get together to actually involve themselves in the development of clinical trials.”

Away from the high-profile international collaborations, the UK YPAGs remain committed to their work where it all began.

For the London group, that means making an impact at Great Ormond Street Hospital.

Recently, they have been influencing the look and feel of posters that are being created as part of the hospital’s research awareness plans, suggesting the type of information and case studies that should be made available to patients, families and staff.

“There is no one better who communicates the point than a young person or patient in terms of helping the whole hospital to really understand the value and power of research,” says Dr van’t Hoff.

This voice is crucial to the future of collaborating with young people in research. Their opinions are delivered with authenticity and they are being trusted with the tools to proactively raise awareness.

A Generation R website, (generationr.org.uk) was launched on International Clinical Trials Day in May 2015, giving the YPAGs ownership of a cohesive voice, and providing visitors to the site – from researchers, to teachers, to parents – with appropriate content.

At the end of every session, each YPAG member offers their feedback

“Momentum is starting to build around young people in research,” says Dr Walker.

“Two of our young people delivered a masterclass to a packed room with adults and researchers at an event at a clinical research facility in May, and they Instagrammed throughout. That provided them with another channel through which to raise awareness, and one that an adult may not have even considered.”

Now that Generation R is paving the way for a new generation, what does Esme see for its future? “We’re creating a foundation to make all young people more aware of clinical trials. We’re teaching them research terms and giving them a greater understanding so that when a doctor approaches them or they are given a claim about a treatment, they are able to understand and decide whether that treatment is good for them or not. We want to be able to teach that in schools and workshops too, which is really exciting.”

Thanks to the extraordinary selflessness of ordinary young people like Esme, improvements are being made in the future of paediatric research around the world. Generation ‘R’ may stand for research, but for some, the impact these young people will make in health research will be revolutionary.

Why I support the charity

By Grahame Chilton

I

’ve always been aware of the wonderful work being done at Great Ormond Street Hospital, but I’m fortunate that I’ve never had to experience the hospital’s care first-hand.

My first direct encounter with Great Ormond Street Hospital Children’s Charity combined two of my passions – charitable giving and motorsports. It was a fundraising event, with drinks, canapés and an auction. I wanted to make a big contribution to the auction, so I bid myself silly and ended up with Kimi Räikkönen’s steering wheel from the Monaco Grand Prix. It still sits proudly in my home today.

A short while later, a new Chairman was needed for the Tick Tock Club – a ‘giving club’ founded in 2006 to support specific hospital projects – and I was approached to see if I’d be interested in taking on the role. It wasn’t the sort of thing I’d really considered previously, because I had so many other commitments. And my wife, Nadine, was particularly concerned about the health implications of me taking on too much. I said to her that it was something I’d love to do, but that we should walk around the hospital together first – if she said to me “you shouldn’t do this”, then I wouldn’t.

After we’d been to a single bedside and met a family, she took me to one side and said “you’re definitely doing this!”. She went from “you shouldn’t do this” to “you must do it” almost immediately, which is the impact that the hospital and its families have on you.

It was also on that trip that I met Mr Joe Curry, a Consultant Neonatal and Paediatric Surgeon at the hospital. He had a significant impact on me as well. He said to me, quite simply, that a better surgery unit would improve the outcomes of surgery for a greater number of babies and children.

Those patients may then go on to benefit from those outcomes for 70 – 80 years.

That’s a hell of a thing, to help give children the chance of living a full and normal life.

Grahame ‘Chily’ Chilton (left, with wife Nadine and Great Ormond Street Hospital surgeon Mr Joe Curry) is the Chief Executive of Arthur J. Gallagher International – the international wing of one of the world’s largest insurance brokerage and risk management companies. As well as maintaining strong business interests in insurance and reinsurance, he also runs the largest motorsport team outside Formula 1®.

So I’ve been Chairman of the Tick Tock Club for a year-and-a-half now, and thus far we’ve raised more than £6 million on our journey towards £10 million for a new surgery centre for Joe and his world-class colleagues. It’s such a rewarding thing to be involved with – trying to get like-minded, philanthropic entrepreneurs to join us and help the most vulnerable children in society.

“ There’s an immediate need, right here, right now, but it’s also for children who aren’t even born yet. It’s an investment in the future.”

I absolutely love it when the charity calls me up to tell me about a milestone that we’ve reached, and that we’ve edged closer to our target. Once we’ve reached that, we can find another Chairman who can raise another £10 million, or hopefully even more, to help with the next project. The quicker we can all do that, the better.

But make no mistake – I’ll be a significant supporter of the hospital long after I cease being Chairman of the Tick Tock Club.

Whether you have a direct and personal connection to the hospital or not, it’s clear how important your support is – it materially changes the life expectancy of children who won’t survive without our help. Children who are otherwise completely helpless, but in such great need. If we can make their experience better, if we can improve or add to the options available to them, then what better way to give back to society? I’ll continue to support Great Ormond Street Hospital until I pop my clogs.

Of that I have absolutely no doubt.

Pioneer 17

Shaping lives with the Plastic Surgery department The Plastic Surgery department at Great Ormond Street Hospital treats some of the world’s rarest and most complex cases. It includes services for ear, hand and cleft lip and palate reconstruction, as well as general plastic surgery.

Each has its own consultants, physiotherapists and occupational therapists.

18 Pioneer

Branavan Sivakumar Consultant Plastic and Hand Surgeon

I specialise in upper limb and hand abnormalities, such as missing fingers and thumbs. We try to correct those abnormalities, giving patients as good a function as possible and improving the appearance as well.

For example, if a child is born without fingers or thumbs, I can reconstruct them by transferring a toe or two from their feet up to create fingers on their hands.

It’s not done in many centres as it’s very specialist work, but toe to hand transfer is the gold standard in reconstruction for children missing fingers and thumbs.

One of the biggest challenges I face is that children adapt so quickly. A child born with an abnormality will quickly adapt to it, so I need to take that into account and make sure that I don’t make their function worse through surgery. Knowing when to operate, and when to just let them get on with things, is not easy.

The best bit of the whole job is that I will see a very distressed child, I’ll treat them, I’ll follow up with them, I’ll hopefully see their lives improve, and as I grow older they grow older with me. You almost feel like a family doctor, which is something you don’t get in many surgical specialties – particularly adult ones.”

Nicky Jessop

Clinical Specialist Occupational Therapist My role as occupational therapist in the congenital hand anomalies service involves providing hand splints, exercises and compression garments. It also includes assessment of patients’ strength, motion and functional skills, and support for families while deciding or going through surgery. I see children over many years to review their splints or reassess their hands.

In my 20 years at Great Ormond Street Hospital, I’ve seen patients grow from newborn babies to adulthood. It’s a rewarding job and families value the continuity of care. The team know what their child is like, and we can remember what difficulties they’ve had and what surgery they’ve been through, which is so valuable for them.

We see a very rare patient group – there are very few hospitals in the country that see the same number of rare and complex conditions. It’s also a centre of excellence – one of the great things about my job is that I work with highl expert colleagues, who I have learnt a huge amount from. And that’s not just from consultants, but nurses and therapists as well. That’s what attracted me to Great Ormond Street Hospital in the first place, and I haven’t been disappointed.

It’s why I’m still here two decades later!”

Neil Bulstrode

Consultant and Clinical Lead for Plastic Surgery One of the best things about plastic surgery is that we have links with so many other specialties.

We work all over the body, not just on one small area, so we work closely with ear, nose and throat, cardiothoracic and general surgeons, dermatologists, oncologists, interventional radiologists and more.

Because of that, there’s not really a typical day. Clinically, I have a number of specific interests, including ear reconstruction congenital melanocytic naevi (birthmarks) and chest deformities.

But the variety and complexity of the cases we see makes it such a privilege to work here. I really have my dream job – it’s so interesting and challenging, and I get to work with some really great people.

By performing reconstructive surgery on children, we hope to affect not only their health, but also their self-confidence and their self-belief. If we can do that in a positive way, we can change what they are able to achieve in their lives.

That’s the true value that we can add here – changing the whole trajectory of a child’s life in a positive way.”

Pioneer 19

Andrea White

Clinical Nurse Specialist for Craniofacial Surgery I’m one of two craniofacial nurse specialists. We work clinically on the wards, we run clinics with the children, we provide support for families at the end of the phone, and we generally help to run the service. We’re the advocates for the family.

Particularly in craniofacial surgery, where we have a number of specialised teams, it can be difficult for families to know where they are and what they’re doing. We can point them in the right direction, and reassure them about any anxieties they might have.

The biggest thing for us, in terms of our clinical work, is that we’re dealing with children with a facial difference. Supporting a family with a child who looks very different from what they were expecting is crucial, especially during the early years. It’s so rewarding seeing families love, accept and support their child, despite being initially overwhelmed.

We then provide life skills for teenagers helping them come to terms with the way they look, and giving them a toolkit of coping techniques. Sometimes, it’s as simple as saying ‘hello’ to someone who is staring at you. We try to give them control of the situation, to empower them.”

20 Pioneer

David Dunaway

Consultant and Clinical Lead for Craniofacial Surgery My main interest as a plastic surgeon is in craniofacial surgery, and I’m there to correct deformities in the face and head. Mostly, it’s about changing the shape of the bones of the face – cutting the bone and moving it, before screwing it back together in a better shape. It’s a specialty that, while I don’t think ‘artistic’ is quite the right word, is certainly a craft.

I’m in theatre for a full day once per week, often on all-day cases. We treat very rare conditions – very complex problems that require a big team to treat them.

A major challenge is that those conditions tend to cause not only a change in appearance but functional problems too – difficulties with speech, vision, feeding or breathing. So we have to tie everything together to deal with all of those problems.

Ideally, I like to see the main role of the Craniofacial Unit as giving everybody the opportunity to maximise their potential.

We change the shape of the face and the head so that they function better, but also to help people feel more comfortable with the way they look and more easily accepted in society. I take tremendous pride in that work.”

Pioneer 21

g n i t e g r a T ours n m i tu with m o fr

Our immune system is an intricate and sophisticated defence network, armed and ready to fight off the viruses and bacteria we encounter to keep us healthy. Now, scientists are finding ways to harness the power of the immune system to fight and destroy cancer cells

I

mmunotherapy is exciting cancer scientists across the world.

According to Dr Karin Straathof, Clinical Training Fellow at the UCL Institute of Child Health and Specialty Registrar in Paediatric Oncology at Great Ormond Street Hospital, the approach could soon be used to help children with neuroblastoma – a childhood cancer that is very difficult to treat.

Each year, around 1,600 children in the UK are diagnosed with cancer. Fortunately, advances in conventional treatments, including chemotherapy, radiotherapy and surgery, have dramatically improved survival rates over the years, with around three-quarters of all children with cancer now cured.

However, such intensive treatment can cause serious and long-term health effects. And, sadly for children with particularly aggressive and harder-to-treat cancers, despite all efforts and intervention, not all therapies lead to a cure.

Striving to develop new treatments and improve outcomes for childhood cancers, Dr Straathof is part of the team implementing a clinical study that uses the body’s own immune system to target neuroblastoma.

22 Pioneer

“Neuroblastoma affects around 100 children in the UK each year,” Dr Straathof explains. “The tumour normally develops as a lump in the abdomen, which can then spread to the bone or bone marrow.

Unfortunately, 40 per cent of patients have the ‘high risk’ condition, which means that intensive treatment is required.”

Currently the best available treatment is a combination of chemotherapy drugs, surgery, radiotherapy and antibodies. This treatment approach is intensive and can be problematic, causing an increased risk of infection and effects on the gut.

In addition, children can develop more long-term effects on the lungs, heart and Dr Karin Straathof

“ We aim to manage the children’s pain so that we can help them get better more quickly and avoid parents’ distress at seeing their children in pain.”

fertility. Immunotherapy stands to reduce these side effects by specifically targeting neuroblastoma cells sparing normal tissues.

The technique involves taking a patient’s own immune cells (T-cells) and engineering them so that they recognise neuroblastoma cancer cells. This type of T-cell therapy has already begun to show promise for treating blood cancers such as leukaemia and lymphoma, where children have achieved quite remarkable remissions (read Harry’s story on page 24).

Dr Straathof is keen to extend this progress to solid tumours such as neuroblastoma.

“Neuroblastoma lends itself very well to immunotherapy, because the tumour is coated with a molecule, GD2, which makes it easier for the immune cells to tell it apart from normal tissue. Scientists have already used this information and made antibodies that target GD2, which are given to patients as a drug. These antibodies then target and kill neuroblastoma cells, and are now part of the standard treatment for the disease.”

However, as is the case for over half of all children with the high-risk disease, neuroblastoma eventually returns.

Dr Straathof hopes that immunotherapy will offer a more potent and longer lasting treatment option.

“We’re engineering a patient’s own T-cells to target the cancer. T-cells are living cells – they are not a drug or antibody, which means they will divide and multiply and make the tumour-killing response bigger and bigger. The hope is that such a persistent immune response

will be successful in keeping the cancer away – a particularly important feature for neuroblastoma.”

Her team are planning to open an early phase clinical trial later this year, testing the effectiveness of this new treatment on children with neuroblastoma. But, for Dr Straathof, the work doesn’t stop there.

“What we have learnt about treating cancers in this way is that can be difficult to find something that is only present on a cancer cell and not on other parts of the body. We know that GD2 is expressed on nerve endings and children undergoing GD2-antibody treatment experience pain and have to be given morphine. Our team is looking at other things aside from GD2 for targeting neuroblastoma to really make it more specific to neuroblastoma and eliminate these side effects. We know that the best way to target cancer is using multiple approaches, and that’s probably true for immunotherapy as well. Eventually, what we will likely end up doing is giving T-cells that are able to recognise a number of different molecules on the tumour cells.”

Aside from neuroblastoma, Dr Straathof’s team are also looking at ways that immunotherapy could be used to treat other childhood cancers. “My overriding ambition is to improve the outcome for all childhood cancers, starting with neuroblastoma, because we have the tools available to do this. But if you look at childhood cancers and mortality, there is a great need for new treatments for brain tumours. So we’re currently looking at the differences between healthy brains and brain tumours to see what we can find and use to target.”

By boosting the body’s natural defences to fight cancer, immunotherapy is fast becoming a more targeted and personalised approach to treating childhood cancers. And as technology advances and scientists like Dr Karin Straathof gain a better understanding of the interplay between tumour cells and the immune system, immunotherapy promises to not only increase the number of children cured, but also reduce the number of side effects and improve quality of life along the way.

Pioneer 23

Leukaemia on trial – Harry’s Tale Harry and his dad, Steve

mentioned the trial that we’d seen, which was ideal!”

Harry went back onto the treatment he’d initially received as a two-year-old, which he again responded well to, before undergoing a gruelling second bone marrow transplant.

He then needed to receive the modified T-cells as quickly as possible, to avoid another relapse. The modified blood typically comes from the same donor as to the bone marrow, but attempts to generate the cells failed.

When Harry’s acute lymphoblastic leukaemia (ALL) returned for the second time, further conventional treatment offered little chance of success. Following a landmark clinical trial at Great Ormond Street Hospital, the future looks very different for the football-loving 10-year-old…

Making the decision to take part in the clinical trial was easy for us,” said Harry’s dad, Steve, “it wasn’t really a choice at all! We had two options. One was a total dead end, the other – hope. You’d take the second option every time.”

The road to taking part in the trial was a rough one for Harry and his family. It began with an innocuous trip to the doctors at the age of two, and went on to include an ALL diagnosis, one failed bone marrow transplant and two relapses.

At every stage Harry seemed to respond well to treatment, but the prognosis continued to get worse.

“Initially, his treatment was going brilliantly.

He was low risk, so the treatment was pretty mild. He sailed through it for three years, and then we got the all-clear in 2010.”

However, by the time he was down to monthly check-ups at the local hospital, blood tests revealed the worst – Harry’s leukaemia had returned.

24 Pioneer

“We started the relapse treatment, which was a lot more severe than the first time around. Harry didn’t respond brilliantly to the chemo, so we were put forward for a bone marrow transplant.

“He had the transplant, and it all went fine.

He was all clear. But, within seven weeks, he had relapsed again. At that point, we were told that there weren’t any more conventional treatments for him.”

It was at this time that Harry’s consultant at Great Ormond Street Hospital, Professor Persis Amrolia, was leading a pioneering trial to help children with ALL – specifically those not responding to existing treatments.

Professor Amrolia’s trial used genetically modified T cells, the body’s diseasefighting white blood cells, to target and attack ALL-carrying cells.

“We’d known people who had been on trials, and had actually seen Professor Amrolia’s trial during our own research.

When we came back up to see him, he

“By this point, we’d started to get a bit twitchy. But 12 weeks went by, and Harry was still 100 per cent, the transplant was working. So they decided to use Harry’s own blood instead.

Two years later, and Harry is still all clear “Professor Amrolia and his colleagues have been brilliant – they’ve been with us right from the start. And the nurses too. When we were first told we’d have to have a bone marrow transplant, it tore us apart, but the nurses were fantastic. They’re under so much pressure all the time, but they’re always there for you.”

Close to a decade of severe treatments have taken a toll on Harry, whose osteoporosis means he currently uses a wheelchair. But the future looks promising for a family left with few clear options before Professor Amrolia’s cutting-edge trial.

“When I grow up I want to be a footballer for Arsenal,” said Harry, grinning. “A striker or a goalkeeper. I’ve started playing wheelchair football for a proper team. I’ve only been playing a few weeks, but I’ve wanted to play for ages. Now I want to get into the first team and score my first goal.”

“He’s been nagging me for quite a while to find him a team,” added Steve. “We’ve found him one just down the road - it’s brilliant! “I don’t even really think about the leukaemia anymore. We’re just going to catch up, keep progressing, and see where life takes us.

That’s how life should be.”

Shared outcomes, brighter futures Great Ormond Street Hospital is at the forefront of a global initiative to improve healthcare outcomes – potentially changing the face of medicine

I

CHOM, the International Consortium for Health Outcomes Measurements, is an international academic thinktank based in the United States. Its ambitious aim is to transform healthcare on a global scale – measuring and reporting outcomes directly from patients in order to reduce the levels of variation in medical standards.

Based on a framework developed by Harvard Business School Professors Michael Porter and Elizabeth Teisburg, the theory of ICHOM is based on a simple principle. If patient-reported outcomes are measured and recorded throughout the world, then global benchmarks can be set. Those benchmarks can then be used to improve both care and, significantly, the value of care – highlighting where the most effective treatments are being used, at what cost, and how the patient has actually benefitted.

The first step in the process is to take a defined condition, and to ask an international team of experts and patients to determine a set of that which should be collected to best represent the outcomes of treatment. Following that, the data set will be collected from both developed and developing countries across the world.

At Great Ormond Street Hospital, Mr Loshan Kangesu led the charge to determine the standard data set for cleft lip and palate treatments – one of 50 Standard Sets that ICHOM hopes to have published by 2017. Mr Kangesu joined colleagues and patients from 23 institutions across 17 different countries, taking part in monthly telephone conferences, follow-up questionnaires and votes on contentious issues. After nine months of international collaboration, the Standard Set for cleft lip and palate was formed – including sample patient questionnaires and a timeframe by which the information should be gathered over the course of a patient’s treatment.

“The point is that it has to work around the world, it can’t just be a Western thing,” said Mr Kangesu. “If you compare results and outcomes around the world in a completely transparent way, you will see differences.

You can talk to each other to reduce those differences, leading to a narrower variation in healthcare standards. The simple truth is that, at the moment, we haven’t got a standard way of looking at medical outcomes across the world. We’ve never asked patients for their opinion.

“A classic example is prostate cancer.

Doctors and people in health authorities get lots of data on the cure rates for prostate cancer. But if you look at the symptoms of prostate cancer around the world, you’ll see vastly different rates of complication despite similar cure rates. For prostate cancer, the two main complications that men see are impotence and incontinence.

Those are symptoms that really matter to patients. So if we start asking patients how they feel about outcomes, it will refocus our attention in healthcare.”

“ There are few institutions around the world where you can do more to advance healthcare.

We are one of them.”

Now that the Standard Set for cleft lip and palate has been determined, Mr Kangesu and his international network of colleagues hope to have data from at least 100 patient interviews by summer 2016. Following analysis at McMaster University in Canada, what happens next is, according to Mr Kangesu, relatively simple: total transparency.

Mr Loshan Kangesu

“I really believe that by sharing data and by being transparent, you will have a narrower distribution of healthcare. If you went to your office and said to your colleagues ‘Don’t change your routine, but I want to know what time you arrive in the office.

Just write down what time you come in and we’ll put it up on a noticeboard in the kitchen,’ you’d find that the distribution of people’s arrival times would gradually narrow. Humans behave this way naturally – it’s a very gentle form of coercion.”

With 12 Standard Sets of data already completed, three in progress and another 16 under consideration, the burgeoning scheme is now well on its way. However, the work being done by Great Ormond Street Hospital and fellow pioneering institutions around the world should not be underestimated, insists Mr Kangesu.

“Great Ormond Street Hospital Children’s Charity puts us in an unusual position – we have that little bit of potential to do something more. Hospitals around the world are struggling to just break even and complete the day job, but there are a few institutions around the world where you can do more to advance healthcare. We are one of them. ICHOM is a fantastic thing that will affect everyone. I’d like to expand it to other medical specialties – we just need to spend time with our colleagues and show them the benefits.”

Pioneer 25

A parent’s perspective Simply put, my son is unique.

There’s no other Dylan. He’s undiagnosed, and has syndrome without a name, or ‘SWAN’.

“The first sign that something was wrong with Dylan’s development came at our 20-week scan, when we were told that his organs were reversed. They also revealed that he was a boy, and I don’t know which part shocked me more as I was convinced I was having a girl!

“Seven weeks later, I had a growth scan at our local hospital that showed that Dylan wasn’t growing. In fact, he was getting smaller. The registrar told us that he had a genetic condition, but that we wouldn’t know what that meant until he was born.

“He was born at 36-and-a-half weeks, and initially he didn’t need any special care at all. He was this perfect, tiny, little thing — he didn’t cry, he didn’t murmur. Until he reached eight weeks, when he had his first seizure. Then came ear infections and throat infections, and horrendous apnea attacks, when he would stop breathing.

He was finally rushed to Great Ormond Street Hospital after my mum noticed that one of his legs wasn’t developing properly, and was red-hot to the touch.

Dylan with his mum, Danielle

initially diagnosed as his worst problem.

He’s fantastic, as are the team of clinical nurse specialists who look after us when we’re at the hospital.

“Dylan has a very rare genetic disorder, which is undiagnosed. It means that he’s stuck in the body and mind of a baby. He’s developed up to about nine months in almost five years.

And he still has terrible apnea attacks – he stops breathing day or night, with no prior warning, and it can happen up to nine times a day. Despite full investigations, no one knows why it happens. He’ll go blue and stiff, and he needs resuscitating and then oxygen.

He’s immune-suppressed, he has arthritis in his knee, which is deformed, and he’s become steroid-dependent.

“Blue flashing lights carried us here five years ago in the middle of the night, and we’ve been looked after by Great Ormond Street Hospital ever since. Dr Paul Brogan from the Rheumatology department is our lead, purely because Dylan’s arthritis was

“His condition is labelled as ‘life-limiting’, firstly because of his breathing problems but also because doctors simply don’t know how, or if, he will grow up. Will he end up being a 30-year-old man in the body of a five-year-old? Nobody knows.

There’s no other child like him.

“Caring for Dylan these last five years has changed me. I’m not as selfish as I was.

He’s my priority, our priority, and he comes first, always.”

“Dylan has a nine-year-old sister called Scarlett, who he absolutely adores – she’s his tonic. But it’s becoming increasingly difficult for us to socialise as a family. The age gap between them is effectively widening every day. Another family whose children are five and nine can go to the cinema or a soft play area, but I can’t take him to anything like that because his immune system wouldn’t cope. So we mainly just go and eat, because then they both get to do something.

26 Pioneer

“So where do we go from here? It’s impossible to say. We’ve been given a walking frame, but Dylan refuses to use it. He’s very strong-willed! From my perspective, as long as he’s here, with us, and with his pain being controlled, that’s all that matters. If he walks, if he talks, whatever. I don’t care if Dylan doesn’t know his alphabet, and I don’t care if he’s not going to be a doctor or an accountant. I’ll carry him around forever if I have to, and I’ll do whatever I have to do to look after him. Because I love him.

“I didn’t realise what a long road it was going to be. Five years ago I thought we’d just be in and out of hospital – I had no idea that it would become such a hard journey. But Great Ormond Street Hospital is obviously the best place for us to be. We couldn’t ask for better care.

Now the hospital is bringing in a nurse specifically to deal with undiagnosed children, which is amazing. We’re hoping to be involved, but it’s the first position of its kind in the UK so it’ll take some time to establish.

“Caring for Dylan these last five years has changed me. I’m not as selfish as I was.

He’s my priority, our priority, and he comes first, always. He could take his last breath at any moment, so we have to cherish every second we have with him.

And it’s the love of our family that has got him this far – I’ve been told that by several doctors. He’s here today because of the love that we show him, and the care that we give him.”

My Peter Pan Pioneer 27

Great Ormond Street Hospital Children’s Charity 40 Bernard Street London WC1N 1LE 020 7239 3000 www.gosh.org Great Ormond Street Hospital Children’s Charity.

Registered charity no. 1160024.

Thank you to all the children at the hospital who took part in our photoshoot.