DM Pioneer Winter 2018 - single page edition

WINTER 2018 Great Ormond Street Hospital Children’s Charity

A PARENT’S PERSPECTIVE

Mum Viki talks about her son Ben being treated at GOSH when he was diagnosed with meningitis

A NEW EAR Mackenzie tells us how his new ear has given him confidence

FACE TO FACE The Craniofacial team use 3D technology to improve facial reconstruction

WELCOME We’ve had another remarkable six months at Great Ormond Street Hospital Children’s Charity (GOSH Charity).

Back in June we celebrated the opening of the hospital’s new Disney Reef, which offers patients and families a much-needed outdoor space to play, and time away from a clinical setting.

Also, if you happen to have been watching ITV on Wednesday evenings recently, you may have noticed that staff and patients at the hospital were featured in Paul O’Grady’s Little Heroes.

One of the stars of the show, 13-year-old Mackenzie, features in this edition of Pioneer. He tells us more about life with microtia, a condition that meant he was born with a very small left ear, and his personal reasons for wanting to have reconstructive surgery.

On the theme of reconstructive surgery, Consultant Plastic Surgeon David Dunaway and researcher Dr Alessandro Borghi tell us how 3D technology is transforming the way the Craniofacial team prepare for procedures. And how, thanks to GOSH Charity funding, they are embarking upon an exciting new project with the aim of printing a material that acts like a child’s skull.

In this edition we also speak to 18-year-old Annie who was treated and supported by Great Ormond Street Hospital (GOSH) for 15 years. She tells us what it’s like to transition from GOSH to adults’ services and how the hospital’s doctors and nurses have helped her along the way.

I hope you enjoy discovering more about GOSH’s pioneering research projects, and hearing from patients who are benefitting hugely from GOSH’s expertise and from your generous support.

Thank you so much for helping us to create a better future for seriously ill children.

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

“ I hope you enjoy discovering more about GOSH’s pioneering research projects, and hearing from patients who are benefitting hugely from GOSH’s expertise and from your generous support.”

gosh.org/pioneer On the front cover: Viki with her son Ben (read her story on page 30).

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CONTENTS 05 AROUND THE HOSPITAL The latest news from GOSH.

07 I HAVE…A NEW EAR Thirteen-year-old Mackenzie tells Pioneer why he chose to have surgery and how his new ear has given him new-found confidence.

10 BIOLOGY ON THE BRAIN Professor Darren Hargrave explains how a collaboration between the clinic and laboratory will bring about more life-saving treatments.

13 THE PIONEER INTERVIEW

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In 2017 Sir Michael Rake became Chairman at GOSH. Pioneer finds out why the hospital is such an inspiration to him.

15 TRANSITIONING TO ADULT CARE After 15 years at GOSH, Annie is transitioning to adult care. Pioneer speaks to Annie and some of the people who helped her along the way.

18 FACE TO FACE WITH THE CRANIOFACIAL TEAM Surgeons and researchers explain how 3D technology is helping patients needing facial reconstruction.

22 WHY I SUPPORT THE CHARITY: GARY AND CATHERINE GRANT

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Founders of toyshop The Entertainer, Gary and Catherine tell Pioneer why they’re such huge supporters of GOSH Charity.

23 PARTICLES WITH A PURPOSE The Nuclear Medicine team tells Pioneer how targeted radiation treatments could help children with blood cancer.

27 THE POWER OF DATA

ioneer finds out how innovative new systems P are revolutionising research and clinical practice, and helping to improve children’s lives.

30 A PARENT’S PERSPECTIVE Mum Viki tells Pioneer about her family’s experience with meningitis.

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AROUND THE HOSPITAL PAUL O’GRADY’S LITTLE HEROES Over the summer, Paul O’Grady’s Little Heroes was screened on Wednesday evenings.

The series provided viewers with a unique insight into what happens at GOSH. In the show, he visited patients and families visiting from all over the UK. Paul says:

Paul with Angel who had both kidneys removed at GOSH.

“It has been an absolute honour to film at GOSH, meeting the children, and their families as they go through some of their toughest days. They were truly inspiring.

I have so much admiration for the truly dedicated staff who treat and care for them.”

The new multi-faith room A new area dedicated to prayer and reflection has opened for patients, families and staff.

The multi-faith room was officially unveiled by Imam Qasim Rashid Ahmad, Chairman and founder of Al-Khair Foundation, which provided generous support.

It complements the work of the hospital’s charity-funded Chaplaincy team and helps to provide spiritual and religious support to families during tough times.

This area is accessible at all times and is also open to ambulance drivers and paramedics from across London.

Tess Daly at the opening with Elliot who received a heart transplant at GOSH.

THE DISNEY REEF OPENS In June, GOSH opened The Disney Reef, a colourful, underwater-themed outdoor play area at the hospital. Created by Disney, it combines special lights, sensory elements and Disney characters to provide a vital outdoor space where children can spend time with their families and make happy memories. Disney Imagineers and Store teams worked with GOSH’s Play Team and young people treated at GOSH, in the design and development of this unique space.

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AROUND THE HOSPITAL Josh has a condition that causes blindness and progressive hearing loss.

Pioneering foetal surgery Babies with spina bifida have a gap in their spine because their spine and spinal cord have not developed properly in the womb.

A team from GOSH and University College London Hospitals (UCLH) has recently carried out the first two surgeries in the womb for babies with spina bifida in the UK.

SIGHT AND SOUND CENTRE Construction has begun on GOSH’s new state-of-the-art centre for children with hearing and sight loss. The centre will offer a unique environment tailored to the largest outpatient group at GOSH. The Sight and Sound Centre, supported by Premier Inn, which is due to open in 2020, will be the first medical facility in the UK dedicated to children with sight and hearing loss. It will include soundproofed booths, an eye imaging suite, a dispensing opticians and other specialist facilities. It will also feature a sensory garden and specially commissioned artwork.

Fundraising towards the charity’s £25 million target has already begun, with an incredible £10 million pledge from Premier Inn and Restaurants.

SUPPORTER SURVEY WINNERS

Return Address: Great Ormond Street Hospital Children’s Charity, 40 Bernard Street, London WC1N 1LE Great Ormond Street Hospital Children’s Charity. Registered charity no. 1160024.

SO18

Thank you to everyone who completed the supporter satisfaction survey this summer.

We received over 9,000 responses with lots of useful feedback to help us improve the way we communicate with you.

ROOM TO HEAR APPEAL SUCCESS Last May we launched the Room To Hear Appeal, asking for donations to build a cochlear implant booth in the new Sight and Sound Centre, supported by Premier Inn..

You can help him hear,“I love you.”

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For a deaf child like Max, the secret to hearing lies in a silent room.

Thanks to your generous support, we raised £240,000 to pay for the room.

This new soundproofed room is vital for testing hearing devices and will help us to improve the lives of many more deaf children.

Thank you for your support.

1st prize One-night Luxury Hotel stay.

Mrs Silvester, Norfolk.

His & Hers Elemis Skincare Sets.

Mr K J Davies, Lancashire.

Mr Davies kindly donated his prize to a family in the hospital.

At the end of October, we launched the 2018 Christmas Stocking Appeal. For this appeal, we are asking supporters to send messages to the patients, families and staff who will 3 be in the hospital over the festive period.

EG+ Job No: 741799-2 Production:Clients:Proximity London - GOSH:741799:Artworks and Linked files:Studio:741799-2_GOSH_C5_162x229.indd Trim: 420x297mm | Visual Area: 400x277mm | Bleed: 0mm ARTWORK @ 100%

Export Option: EG+ Large Format 300 | Ratio: 1.414 | Page 2 of 2

The winners of the five survey prizes are below:

2nd prize

CHRISTMAS MESSAGES

Date: 16.04.18 Time: 16:31 GMT+01:00 DI: Kelly Burton

PRE PRESS

Bankside 3, 90 Southwark Street, London SE1 0SW | www.egplusww.com

We’ve sent out paper stockings in the post and are asking for supporters to send them back with messages to help brighten people’s day. If you haven’t received a stocking, you can send messages and make a donation online at gosh.org/stockingappeal.

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This pioneering surgery has been brought to the UK thanks to funding from GOSH Charity and UCLH Charity, which has been put towards research and training for the surgical team to specialise in the procedure.

3rd prize Special edition Merrythought Bear.

Ms M Johnson, Wiltshire.

4th prize 250-piece Jigsaw.

Mrs Clapham, Hertfordshire.

5th prize GOSH Bernard Teddy Bear.

Mr Dinwiddy, Suffolk.

I HAVE...

A NEW EAR Thirteen-year-old Mackenzie has microtia – a condition where you are born with a small or absent ear. Last summer, he was referred to GOSH and made the decision to have reconstructive surgery.

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“I was constantly in and out of my local hospital when I was younger. Eventually (and very luckily) in July 2017 I was referred to GOSH, where I was offered this amazing surgery where the surgeons told me they could create me a completely new ear!

“I’ve always been bullied because of my condition and I’ve had to move schools because of some particularly cruel comments. I live in a small place and I don’t think people have ever seen anyone with microtia before, so they find it difficult to understand.

One day last year, I’d had enough of people being mean to me and decided that something needed to change – I wanted to feel more confident in myself. That’s when I made the decision to have the reconstructive surgery.

“Mum and I travelled down to GOSH from our house in Norfolk the night before my operation. We needed to be there for a week, so it was great that mum could stay in the family accommodation so close to the hospital. I was a bit worried I would miss playing with my mates on my PlayStation 4 and Xbox, but mum ended up being good company!

“When I first arrived at GOSH I felt a bit nervous, but as soon as I walked through the front door I felt happy.

There were two people standing there wearing yellow. They had big smiley faces and told me where to go.

They’re all so friendly there – everyone made me feel calm and welcome the minute I stepped through the door.

Every time I’ve been back to GOSH since, they remember me. It’s like a home from home.

and release my ear, so it should look even better once that’s happened.

“As well as getting a new ear, I also had the opportunity to meet Paul O’Grady when he was filming Paul O’Grady’s Little Heroes for ITV. Being on the show was an amazing experience that I won’t forget. I really wanted to take part in it because it was a way for me to tell my story and raise awareness about microtia.

“Since my first operation I feel so much happier.

I now walk with my head held high and I don’t cover my ear anymore!”

“So far I’ve had part one of my twopart operation. In the first operation, my surgeon Mr Neil Bulstrode took cartilage from my rib and carved out a brand-new ear to match my other one. He then put my new ear under the skin on the side of my head and used suction to pull the skin tight around it.

“Since my first operation I feel so much happier. Mum says I now walk with my head held high and I don’t cover my ear with my hand anymore! I definitely smile a lot more too.

“I’m really excited about the second part of the reconstruction surgery.

That’s when my surgeon will unpin

And I got to meet Paul! He was so funny and always managed to put a smile on my face, even when I was in pain. He also looked after my mum when I went into surgery.

“I really hope that, by increasing people’s knowledge about rare conditions like microtia, people will become kinder and more accepting.

It’s hard being different and it’s not nice to be bullied. I hope that if anyone is in the same position as me, they won’t be too frightened to have surgery. It’s changed my life and I am so grateful to GOSH and my surgeon Neil Bulstrode. Now I’m back at school and enjoying playing football with my friends.”

Above: Neil Bulstrode is a reconstructive surgeon and created Mackenzie’s new ear.

Mackenzie (pictured with his parents at GOSH) is much happier since his operation.

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HOW DOES MICROTIA RECONSTRUCTIVE SURGERY WORK? Ear nose and throat specialists help with the internal and hearing element, and plastic and reconstructive surgeons, like Neil Bulstrode, create the outside of the new ear.

The new ear is constructed over the course of two operations.

In the first operation, the new ear is made by taking cartilage from the patient’s rib and carving it into a framework to match the other ear.

The chest wall is then rebuilt, and the new ear structure is placed beneath the skin on the side of the patient’s head. Using suction, the skin is pulled taught over the new ear structure.

Six months later, the second operation takes place, where the surgeon creates the gap behind the ear using a small skin graft. After that, there is a phase of splinting to keep the ear in place.

As well as improving patients’ hearing, surgery offers another important benefit. Neil explains:

“What is so rewarding, is seeing how a child’s confidence changes. I think we can change their whole trajectory in life – these children will feel more able to follow their dreams and to achieve what they want to. Helping people in that way is what makes mine a dream job.”

GOSH Charity is funding research into less invasive methods of ear reconstruction that don’t require surgery to remove rib cartilage.

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BIOLOGY ON THE BRAIN Brain and central nervous system tumours are the leading cause of cancer death in children, with survival rates significantly lower than other types of cancer. But a strategic initiative between GOSH, the UCL Great Ormond Street Institute of Child Health (ICH), the UCL Cancer Institute and GOSH Charity is committed to changing that.

Pioneer speaks to Professor Darren Hargrave, the newly appointed GOSH Charity Professor of Neuro-oncology, who explains how a collaboration between clinic and lab will bring about more life-saving treatments for seriously ill children everywhere.

Darren joined GOSH in 2011 as a consultant paediatric oncologist, having previously worked at The Royal Marsden in London. He specialises in brain tumours and the development of new anti-cancer drugs for children and adolescents.

As well as treating patients with life-threatening illnesses, he leads a research team that is working to better understand exactly how cancer affects the body, which will help to inform the development of more effective treatments.

“We’re trying to understand the biology of brain tumours,” Darren explains. “Many brain tumours arise as a child is growing. As the child is developing, something goes wrong in their nervous system that can cause brain tumours to occur.

We think that one of the key aspects of neuro-oncology is understanding a child’s normal development, so we can better spot and comprehend any changes or differences in that process.”

Currently, the more conventional methods of treating children with a brain or central nervous system tumour include surgery, chemotherapy or radiotherapy. For some tumours, 10 PIONEER

these treatments work well and lead to a full recovery. However, these treatments often come at a price, causing unwanted side effects and leaving some children with a poor quality of life afterwards.

But thanks to research being conducted by teams like Darren’s, many new, groundbreaking cancer therapies are proving just as effective, with potentially fewer side effects than their more conventional counterparts.

PRECISION MEDICINE Immunotherapy is an exciting area of research that is changing the way we think about cancer treatment.

It works by helping the body’s own immune system recognise and attack cancer cells.

“At GOSH, we’ve had a lot of successes in treating leukaemia in this way,” explains Darren. “We’re now looking to see if we can take a similar approach for brain tumour patients, by developing ways to ‘mark’ the cancer cells and encourage the body’s immune system to attack them, avoiding the normal, healthy cells.”

There are many different types of immunotherapy, and some work better in certain types of cancers than others. One kind of immunotherapy the team is currently studying is called a checkpoint inhibitor.

Cancer can sometimes push a ‘stop’ button on immune cells, called T cells, so the immune system can no longer recognise and kill them. Checkpoint inhibitors are drugs that work by blocking cancers from pushing the stop button.

“When I’m speaking to parents, I talk about a cloak that suddenly disappears,” says Darren. “Once the cloak is off, the immune system can see the cancer and hopefully, it will attack it.”

A large part of Darren’s research is also in the development of targeted drugs.

Cancer cells develop from normal cells, but with changes in their DNA that make them behave differently.

Targeted drugs work by targeting the differences that help a cancer cell to survive and grow.

This might stop the cancer cells from dividing and growing, stop cancers from growing blood vessels, or help carry other treatments, such as chemotherapy, directly to the cancer cells.

“Over the past few years, we’ve had real optimism that this is a strategy that can work. We’re starting to see results in the clinic, where patients are able to take oral medication and see shrinkage of their tumours. We’re going to take these treatments forward into new clinical trials, which will be global frontline studies, and compare them with more conventional methods, like chemotherapy, to see which is better and which has fewer side effects for children.”

In the future, successful cancer treatment could see us using a combination of immunotherapy and targeted therapies, all tailored specifically to respond to each patient’s DNA.

Although some of the studies are still in their early stages, Darren says that the outcomes of these new forms of cancer treatment already look positive: PIONEER 11

WORKING AS ONE TEAM While brain tumours are the greatest cause of death for children with cancer, some of the types that Darren and his team see are extremely rare. This means that clinical trials can often have a low number of participants, so collaboration on both a national and an international level is essential in order to gather a meaningful amount of data.

Darren says: “Internal links with UCL and the ICH are extremely important for our research. We also have excellent collaborations on a national level. We’re involved in a large grant programme called INSTINCT, supported by funding from GOSH Charity, the Brain Tumour Charity and Children with Cancer UK, which links us with the Institute of Cancer Research at The Royal Marsden, and Newcastle University.

We’ve developed collaborations with international colleagues, including another big research grant from The Brain Tumour charity called The Everest Centre, where we’re working with researchers in Germany across several projects that will help us understand more about childhood brain tumours.

benefit from research at an even faster rate. Several areas of research in adult neuroscience overlap with paediatrics. For instance, the problems with the blood-brain barrier, seen in dementia, are similar to some conditions in children. It makes sense for us to work more closely together.”

Exciting times lie ahead for Darren and his team as they pave the way for the treatments of the future.

Darren says: “I’ve dedicated my career to developing better and kinder treatments for children with brain tumours. And I passionately believe that by increasing our knowledge of the biology of brain tumours, we can translate this into the clinic to improve the length and quality of survival for these children.

If you can learn something from each trial, then together we can try and put this massive, complex jigsaw puzzle together and really make a difference to the people who need it the most – our patients.”

“A lot of the trials that we’re involved in are also run as global studies, so there will be children being treated in the UK at GOSH, but also children being treated in America and some parts of Europe. Because these therapies are targeting very niche groups, you actually need to have a very global network.”

Collaboration is becoming even more important not only on a geographical level, but also in terms of patient age. As Darren explains, the barriers between paediatric and adult brain tumour research don’t always make sense: “When I first started medicine, the gap between a drug entering an adult early-phase trail and a paediatric early-phase trial was 10–15 years. Although this has now dropped dramatically, greater collaboration will allow children to 12 PIONEER

Professor Hargrave in the neuro-oncology clinic with Advanced Nurse Practitioner, Renate Tulloh.

“I passionately believe that by increasing our knowledge of the biology of brain tumours, we can translate this into the clinic to improve the length and quality of survival for these children.”

THE PIONEER INTERVIEW Sir Michael Rake is no stranger to the boardroom. Over the last 10 years, he’s had an illustrious career chairing for the likes of EasyJet, Barclays, and the BT Group. And in 2017, he became Chairman at GOSH.

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“GOSH is a fantastic institution where, despite the complexities we deal with, the staff always provide outstanding care to patients and families. Even in difficult periods, everyone ensures that the child comes first and always.”

We speak to Sir Michael about his vision for the future, and to find out why GOSH is such an inspiration to him.

How would you explain your role as Chairman at GOSH? As Chairman, I am very focussed on the overall reputation and culture of the trust.

As well as being an ambassador externally, it’s my role to maintain open and constructive relationships with the Governors, Board and management across the hospital as well as the charity and UCL Great Ormond Street Institute of Child Health. I try to ensure that all our energies are focussed on the same overall objectives of providing the best possible care for our patients and their families, as well as supporting our dedicated and hard-working staff.

What skills do you think you need to be a good Chairman?

The ability to think strategically and to help people think and work as a team. The NHS is complex in its structure, therefore it’s vitally important that the hospital can work together as a team – that involves everyone from receptionists 14 PIONEER

and porters, to nurses, doctors, consultants, administrative staff and management.

What inspired you to work at GOSH? For a large part of my life, I led a very large professional services firm.

And for the past 10 years I’ve been involved in large public companies.

I was keen to use my experience in some degree of public service and it is very valuable in this role.

Sadly, I lost my granddaughter to a very rare condition called Alpers syndrome when she was 18 months old. It’s a neurological disorder that causes symptoms such as seizures, dementia and exaggerated reflexes.

The issues of having a child with a very severe condition of course resonates with me.

My family historically has a medical background and my father really wanted me to become a doctor. So, when GOSH approached me, it just seemed right. It’s a great honour to have been appointed.

What is your vision for GOSH and what would you like the hospital to achieve?

We have an extraordinary and powerful purpose – helping children with complex health needs fulfil their potential.

We now need to be sure that we have the people, culture and facilities to support our purpose and reputation for research and care, for the foreseeable future. All at a time of great political and economic uncertainty and complexity.

In your own words, why does GOSH so urgently need continued support?

It’s thanks to the generosity of our donors that we have become a leading hospital in research and the treatment of children with rare paediatric conditions. Thanks to them, we can improve our facilities and develop our capabilities. Without our donors, we simply could not be where we are today.

What has stood out to you about GOSH in the time you’ve been here?

It’s a fantastic institution where, despite the complexities we deal with, the staff always provide outstanding care to patients and families. Even in difficult periods, everyone ensures that the child comes first and always.

TRANSITIONING TO ADULT CARE In 2002, two-year-old Annie was diagnosed with pulmonary arterial hypertension – a rare heart and lung condition which meant her body had to work extra hard to pump blood around her body, resulting in severe shortness of breath.

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Shortly afterwards, her local hospital referred her to GOSH. Over the following 15 years, Annie underwent a double lung transplant, open-heart surgery to fix three holes in her heart, chemotherapy to tackle cancer, and a surgical procedure on her stomach.

Annie has had a long and remarkable journey at GOSH. During this time, she’s made many friends, watched new wards open and got to know her doctors and nursing team well. So, transitioning to adult services at the end of last year was always going to be a big change.

Pioneer speaks to Annie, now 18 years old, and some of the members of staff who helped her along the way.

2018

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Annie and her mum have visited GOSH since Annie was two.

She is now 18 years old and has transitioned to adult care.

ANNIE THE PATIENT Annie is at university studying Early Years in Education.

“There’s a lot of stuff I don’t remember from when I first arrived at GOSH because I was so poorly at the time.

I just remember it being different to other hospitals – it felt more like a second home.

“My family and I had so many good people around us who were all really helpful and there was always plenty to do.

“It was a year and a half ago when GOSH started my transition to the Royal Papworth Hospital in Cambridgeshire. The date was pushed back a few times because I was so unwell, and GOSH wanted to look after me before I left.

“Leaving GOSH has been difficult, but the staff were supportive throughout.

“Dr Helen Spencer had a meeting with the Papworth team before I moved and told them everything they needed to know. She then came with me for a consultation with one of my new doctors, and later talked me through what would be different.

This made everything a lot easier.

“The transition process was good because it broke everything down.

If we’d done it all in one go, I think I would have been a bit scared, but this was at my own pace.

“In March, to celebrate my 18th birthday, I held a fundraising event for GOSH and raised £1,225. The hospital has helped us so much in the past, it was like a big farewell.”

THE HEART AND LUNG TRANSPLANT TRANSITION PASSPORT Each patient receives this booklet, which explains the different stages of their transition to adult services. It includes key information about the patient and is used at all their clinic appointments at GOSH. Once the patient has transitioned, they take the passport with them to their first appointment at adult service, to help their new team get to know them better.

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allowing us to tailor the right service to each person.

“When transitioning to adult services, the biggest apprehension tends to be simply meeting a new team – the patients know us so well and trust their doctors and nurses, so it’s very difficult to picture that all changing.

“Working with the parents is also crucial. They have to trust an entirely new team with the care of their child, which can be challenging, as many of our families have with been us for over 10 years.

AMANDA HAWKYARD THE CLINICAL NURSE SPECIALIST Amanda Hawkyard is a Clinical Nurse Specialist and Recipient Transplant Co-ordinator. She’s worked at GOSH for 15 years.

“It’s hard to say goodbye to a family, but it’s such a positive and rewarding experience. It’s nice to know the patients are enjoying a good quality of life, as that’s what we hope for everyone who has had a transplant.”

“I co-ordinate their journey with the correct adult team, organise the transition day and transition clinics, and manage any other events surrounding transition.

“My job is to see very sick children who have end-stage lung disease and decide if those patients are sick enough to require lung transplants. I then look after them before and after their operation.

“We start the transition programme at the age of 15, and patients will generally transition when they’re between 17–18 years old.

“Our patients have a choice of adult hospitals and each patient has a transition passport that we work through over two years. We can speed up or slow down at any point,

“Post transition, our former patients often keep in touch. When Annie turned 18, we sent a video from the team to wish her happy birthday.

When you’ve had that close relationship with a family for so long, it’s always nice to hear from them – knowing they’re doing well is part of what makes the job so rewarding.”

THE DOCTOR Dr Helen Spencer is a Respiratory Paediatrician and has been the Clinical Lead for the Lung Transplant Service at GOSH since 2007.

“For the past three years we’ve also organised a transition clinic at the adult centre. The GOSH team will go and do a formal face-to-face handover, and families often find it very comforting to have a well-known GOSH face with them when they meet their new care team.

“We’re really proud of our transition process and it’s now much smoother for patients and families. We’ve also found it’s had a huge impact on reducing the number of children whose bodies reject the transplant.

DR HELEN SPENCER

“As transition lead, I ensure all patients around 15 years of age have begun, or are planning to start, the transition pathway.

“The transition day is an annual event where all the adult teams attend, as well as the families due to transition in the next year. It enables everyone to meet on an informal basis and is a chance to learn more about the transition process and hear from the adult centres.

“We make sure our patients understand their own transplant journey and what that means for the future. We also talk about problems such as getting through school or applying to college.

“With a lung transplant, you can have lots of problems and complications along the way, and Annie certainly did. She developed a type of cancer and needed chemotherapy.

“When Amanda and I looked at our transplant data, we found about one-third of patients had rejection within 18–24 months of transitioning into adult services. That’s unusual.

“ We developed a two-year pathway and a transition ‘passport’ that patients fill out themselves and take with them to adult services.”

“We examined the transition process and, rather than just us writing a letter to the adult centre saying, ‘Please take over this patient’, we developed a two-year pathway and a transition ‘passport’ that patients fill out themselves and take with them to adult services.”

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FACE TO FACE

WITH THE CRANIOFACIAL TEAM It’s been a while since Pioneer caught up with the Craniofacial team. And since we last spoke, they have been busy pioneering 3D technologies so GOSH can offer patients less invasive procedures and more tailored facial reconstruction.

ON THE SURFACE The Craniofacial team have the enormous task of reconstructing the faces and skulls of children with extremely complex conditions. In the not-so-distant past, this meant children needed long and complex surgery involving a big incision and lifting the bones of the skull off the brain, reshaping them and then putting them back. Surgeons would do it using a Rigid External Distraction (RED) frame, which helps to manoeuvre the bones and hold them in place. The old operation meant four or five days in hospital, a night in the intensive care or high dependency unit and a high probability of needing a blood transfusion.

Children needing this kind of treatment are affected by craniosynostosis, which is when one or more of the soft seams in a baby’s skull fuse together prematurely.

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When this happens, the skull can’t grow properly, and this can increase the pressure in the skull, hinder brain development and result in a variety of conditions.

Nowadays the team takes a holistic approach to treatment – psychologists, orthodontists, speech and language therapists, surgeons and researchers collaborate to offer patients the best treatment possible.

Thanks to an impressive dedication to research and to substantial technological advancements, things have been moving quickly in the area of craniofacial surgery over the past few years. Professor David Dunaway, who is a craniofacial consultant and plastic surgeon at GOSH, highlights some of the major changes: “First of all, we understand the RED frame much better and we have lots of other types of distractors – which are devices that expand skulls to a more ‘normal’ shape. An example of these is springs.”

The craniofacial research programme has been made possible by supporters of GOSH Charity’s Face Value Appeal.

A 3D-printed skull used to prepare for craniofacial procedures.

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“We now have a device that will change the shape of a person’s jaw in a pre-programmed way. It means we can insert the device and it will make the correction itself, rather than us having to take measurements and turn screws.

“Some of the projects we’ve been involved with are making more accurate devices using shape memory metal, which is what flexible glasses are made from. You can sit on them and they will always return to their original shape because they’re preprogrammed to do that. We’ve used the properties of that metal to make devices that remember their shape. At room temperature they’re floppy. Then when you put them into the body they heat up and make their new shape.

“We have developed a device that we hope to use in the jaw and have also made new springs that divert force in a much more accurate and consistent way. We have done lots of 3D imaging and statistical analysis to work out how those springs work, and we have a very clear idea about how they can change the shape of a skull, how strong they need to be and where they need to be. It has revolutionised the treatment of craniosynostosis.”

A 3D scan of a child’s face.

A SEA OF FACES In 2015 the team collaborated with the Science Museum on a project called Me in 3D. The project imaged the faces of 12,000 volunteers and over the last few years the team has used the data collected to establish ‘normal’ facial features. Professor Dunaway elaborates: “One of the great things about being in London is that we are such a cosmopolitan society. We have been able to build a world model of faces and understand the difference in your face whether, for example, you are Chinese or English. That’s really helpful for people who come from non-European ethnic backgrounds because nearly

Professors, consultants and researchers work together to ensure the best outcome for patients.

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all of the ‘normal’ data available is built on white, Caucasian people. I think the Me in 3D facial model has now been shared with about 50 other institutions around the world.

“We’ve also taken images of children with craniofacial conditions and, through machine learning, have developed a technique where we can diagnose problems just from their 3D shape. It’s in a very early stage of development, but we think it will be a useful diagnostic tool and one day mean that conditions can be detected in the womb. That would be great because the evidence shows if you can treat children early, you don’t get the associated problems.”

MAKING A FACE The Craniofacial team not only use 3D technology to determine ‘normal’ face shapes, they use it as a vital stage of preparing for surgery and even in surgery itself. Professor Dunaway says:

“For a jaw reconstruction we make 3D cutting guides, we plan the surgery in 3D and even make plates and screws to put everything together. You can print those so that they’re the perfect shape.”

“We put 3D printed implants in people to normalise the shape of their face. And for children who were born without noses, we have 3D printed frameworks to build new noses tailored to their faces. I think the real buzz in craniofacial surgery is doing things that are designed for the individual. They are patient specific and that makes it much better, it makes the shape much better and it makes the operation simpler. You know what you are going to do before you start which means that the operation is quicker, you lose less blood, you can do it through smaller incisions and your chances of complications are less. It is a win all round. This is really at the cutting edge of personalised medicine.”

RESEARCH IN 3D

followed up with the clinician to see how much the patient had improved.

“At the moment, clinicians have a lot of experience, but all of that knowledge and data about the surgery is in their heads. What we’re trying to do is create an algorithm to say, if we do this procedure this way, we are going to have this much improvement.”

Alessandro is also working on another new and exciting project.

Professor Dunaway tells us more: “With the 3D printing that we have, we can print in different materials and Alessandro and Plastic Surgeon Juling Ong have been looking to create a material that behaves exactly like children’s bone.

“When you do craniofacial surgery there is always a little bit of bone that you don’t need, and you throw away. Patients have consented for us to use that bone as part of a research project and we have learnt the mechanical characteristics of the child’s skull.

“With this research project that GOSH Charity have funded, our hope is that we will be able to print a skull in plastic that has the same mechanical characteristics as a child’s skull. What that means is we can do practice operations on the plastic, we can try it all beforehand and then we can get the ethical approval and process all sorted with really good information.“

CHANGING LIVES One of Professor Dunaway’s patient’s, Aleksandra, recently came to GOSH for a life-changing eight-hour operation to reconstruct the bone around her eye and cheek using bone taken from her leg.

When she was six-years old, Aleks was diagnosed with orbital chordoma – a rare type of bone cancer. She had specialist surgery to remove a tumour but her eye could not be saved. Now 10 years old, she came back to GOSH for specialist reconstructive surgery on her eye socket, a procedure using innovative 3D planning.

Professor Dunaway says: “Finding the right shaped bone needed for Aleksandra’s reconstruction was crucial. We needed to be very accurate, and innovative 3D printing and planning allowed us to do that.”

LOOKING AHEAD With a variety of research projects taking place and 3D technology transforming treatments, the future is increasingly hopeful for patients with craniosynostosis and other conditions affecting the shape of the face or skull. And Professor Dunaway predicts more positive change to come: “I think things will become safer still and much more routine. There will be less need for big operations, and procedures will be much more accurate, so the results will be better.

We are absolutely at a frontier. “

Professor Dunaway and Alessandro Borghi in theatre 10.

Researchers such as Dr Alessandro Borghi, who works closely with the Craniofacial team, are huge advocates of 3D technology and it has become an integral part of their work. Alessandro says: “I would say we base 99% of what we do on 3D. “Recently we carried out a study where we printed models of patients’ heads before their treatment and tried to determine the severity of their condition and understand what needed to be changed geometrically in the skull.

Then we printed a model of the patient after their treatment and PIONEER 21

WHY I SUPPORT THE CHARITY CATHERINE AND GARY GRANT Gary and Catherine Grant have supported GOSH Charity for over 10 years In 1981, the husband and wife team founded The Entertainer Ltd, which began as a single toyshop in Amersham. Today it’s the largest independent toy retailer in the UK and their family-run business puts children at the heart of everything it does.

CATHERINE I trained as a nurse at GOSH in the 70s and worked with some remarkable consultants and nursing staff. I love their pioneering work, confidence, bravery and courage. Technically, nursing has changed hugely since the 70s, but the compassion has and will always be there.

I’m a health visitor and work with disadvantaged families locally. I have a great group of friends from GOSH, and it’s great to still have that link, but I don’t work day-to-day in a hospital setting now. It’s a privilege to support those who are in clinical practice.

The hospital’s need for support will never end because technology keeps progressing, you need more space and different apparatus for such specialist patient care, and the buildings need to reflect current needs.

My most memorable moment at GOSH is sitting in the new Heart and 22 PIONEER

Lung Unit, the week after it opened, when our grandson was one of the first patients admitted for emergency treatment. It was completely unexpected. He was born four months early and came to GOSH for a heart operation. I was sitting in a building we had helped to fund, and Alfie was one of their first patients.

GARY You never can be sure when you’ll be on the receiving end of this kind of support. Never in a million years did we think the new Heart and Lung Unit, which we had supported with our first major gift to the charity’s Tick Tock Club, would be a facility we would use as a family. It’s easy to pay for things when you know you’re the beneficiary; it’s quite different when you’re doing it for somebody else and then you find yourselves as the beneficiary. It was quite humbling.

One of the ways we first became involved with the charity was by donating toys to the play therapists, because we’re in the toy trade and want to give back to children and families. These days, we invite the play specialists to our stores to select the specialist toys and equipment they need to deliver their work.

GOSH Charity’s Chief Executive, Tim, invested his time in us, showing us around the wards. Cath was familiar with the hospital, but it was new to me. I remember seeing parents sitting by their children, with their hope invested in the wisdom and skill of the

doctors and nurses, and we wanted to help. I couldn’t add to the expertise of the nursing skills, but I thought we could help to improve the hospital environment.

I can see that the hospital is more than medical procedures, it’s an experience. A lot of what the hospital does isn’t straightforward: medical procedures are advanced, intricate and specialist. Yet everything is made child-friendly to reduce the anxiety attached to the situation for children and families. Play has an important role in making hospital experiences easier for children.

Catherine and I have spent 25 years supporting charities. I think our Christian faith is one of the main drivers, but of course you don’t have to be a Christian to be generous.

We believe that business can be a force of good and we can facilitate generosity. We run a payroll giving scheme in our business, The Entertainer, where our staff can give to a charity of their choice and we match what they give, to encourage people to be generous. We run a scheme called ‘Pennies’ within our shops, which allows our customers to round their purchases up to the nearest pound, and that additional money is given to inspiring charities, like GOSH Charity. Catherine and I are also members of the charity’s Tick Tock Club, a ‘giving club’ for individuals, trusts, foundations and companies who are passionate about children’s health.

Aiden, age six, preparing for a nuclear medicine scan.

PARTICLES WITH A PURPOSE There’s a revolution happening in radiology. Guided by highly trained physicists, radiation treatment is being delivered directly to specific cells and tissues, avoiding many of the damaging side effects of total-body radiotherapy.

At GOSH, the Nuclear Medicine team is priming these targeted treatments with radioactive parcels designed to search-and-destroy unwanted tissue, from damaged joint cells to cancer.

In a quiet area of the hospital’s basement, Medical Physicist Leanne Price points at a pink blob on a computer screen. “That’s the kidney,” she says, before the blob starts to shrink, trickling down the screen one dot at a time to form a second, growing shape. “And that’s the bladder.”

We’re watching a radioactive substance travel through the body, as fluid passes between the organs. It’s nuclear medicine in action. The technique involves injecting a small amount of radioactive material into the body, then using special cameras to track it as it travels through tissues – in this case from the kidney to the bladder.

“We can see any blockages and whether the kidney is transferring urine into the bladder at a normal rate,” Leanne explains. “That’s the great thing about nuclear medicine. Rather than providing a snapshot of anatomy,

The Nuclear Medicine team track radioactive material travelling between a patient’s kidneys and bladder.

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like an X-ray or CT scan, it shows processes happening in the body in real time.”

The radioactive particles act like tiny tracking devices, following particular cells and highlighting any unusual activity, or hitching a ride by attaching to drugs so doctors can make sure they’re reaching the tissues they need to.

UNPACKING THE PARCEL While incredibly useful, radiation has a major downside: it can cause serious damage. Too much radiation could harm healthy tissue, so each nuclear medicine dose must be carefully calculated and controlled.

But what if damaging tissue could be a good thing?

“For years, fixed amounts of radioactive material have been used to destroy problematic tissue in the body, particularly tumours and other cancer cells,” says Leanne. “But now we are starting to investigate how we can tailor the amount of radioactive material given to each patient.

That’s especially important for children, because they’re more sensitive to radiation.”

GOSH is already beginning to offer treatments that send radioactive material to specific sites in the body to destroy problematic tissue. It’s a technique that could revolutionise radiation therapy, reducing the need for total-body radiation that can damage healthy tissues. But these search-and-destroy missions need careful controllers: physicists.

MOLECULAR MATHEMATICS “It’s our job to assess the exact amount of radiation delivered to patients,” says Leanne. “That includes doing careful calculations to convert red blobs on a screen to a measure of the actual radiation reaching each piece of tissue.

“We have to account for radiation travelling through different tissues at different rates, and the fact that some types of particle are more 24 PIONEER

Aiden has scans at GOSH to check for problems with his kidneys.

‘energetic’ than others. The radiation dose also depends on things like the individual child’s condition and body function, as well as how diluted the material is and where it’s injected.

“We can do these calculations even more accurately now that we have a brand-new scanner, known as a SPECT CT, which was funded by GOSH Charity. It shows the exact location of radiation in the body, while simultaneously overlaying a detailed CT scan of the child’s anatomy. I can use those images to calculate exactly how much radiation is travelling to the child’s tissues over time.”

Untangling this complex relationship is especially important when the radiation is being used as treatment, rather than for diagnosis or monitoring.

“For any kind of nuclear medicine, we always want to check radiation isn’t migrating to sensitive areas like the lung and kidneys, because it could cause damage,” says Elizabeth Morris, another of the hospital’s medical physicists. “But in cases where we’re actually trying to damage tissue, we want to know whether we could safely give the child more and do an even better job at helping them.”

By focusing radiation on the cells that need it, the treatment could vastly reduce side effects and offer children with life-threatening leukaemia a kinder alternative to total-body radiation.

RADIOTHERAPY WITH FEWER REPERCUSSIONS Elizabeth is working on a research project to optimise the dose of radiation given to GOSH patients with severe forms of leukaemia. She says: “Usually these patients would have total-body radiotherapy, which aims to kill their bone marrow cells.

That creates the ideal environment for the child to receive a bone marrow transplant – receiving healthy cells from a donor.

“Total-body radiotherapy can be really effective at killing cancer cells, but the radiation also hits other, healthy cells around the body. That’s why children often lose their hair and feel sick. It can make an already difficult experience that much harder on small bodies.

“These children have conditions that require aggressive treatment,” says Elizabeth. “I’m studying their SPECT CT scans to see how much of the radiation is actually reaching the bone marrow cells, while monitoring any damage to surrounding tissues.

The aim is to work out the maximum possible dose, while keeping it safe.”

By focusing radiation on the cells that need it, the treatment could vastly reduce side effects and offer children with life-threatening leukaemia a kinder alternative to total-body radiation. Not only that, but Elizabeth and the team hope that because the treatment is so targeted, they should

be able to apply even higher doses of radiation.

“If we can kill off more bone marrow cells using this technique,” says Elizabeth, “it should mean a bone marrow transplant is more likely to work. That could give children more chance of beating the cancer.”

The hospital’s brand-new scanner and its talented team of physicists, radiologists and consultants are helping GOSH expand an incredibly promising area of medicine. And by carefully policing radioactive particles, Elizabeth and Leanne are making sure more of them hit their targets.

Medical Physicists Elizabeth Morris and Leanne Price are helping to tailor radiation treatments to children.

“Instead, we’re proposing to send little packages of radiation into the bloodstream, with markers that encourage them to attach to bone marrow cells. They’ll decay there and deliver a dose of radiation directly to the cells. Hopefully that will destroy lots of bone marrow cells, but not a lot else.”

The treatment – known as radioimmunotherapy – is currently in the early stages of a clinical trial, led

by Robert Chiesa and Paul Veys in the Bone Marrow Transplantation Group at GOSH. Patients on the trial have the most difficult-to-treat kinds of leukaemia, and many are running out of other options.

Above and left: GOSH’s new SPECT CT scanner, funded by GOSH Charity, produces detailed scans.

“Rather than providing a snapshot of anatomy, like an X-ray or CT scan, nuclear medicine shows processes happening in the body in real time.”

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LUKE’S STORY

BY LUKE’S MUM, JULIA “When Luke was 21 months old, he woke up one day with a faint bruise under his eye. I didn’t think too much of it, but the next day he had a little swelling and soon he had a lump that kept getting bigger.

“We saw 12 different doctors, and the last one suggested a blood test.

When the results came back, I was told that my son had cancer.

“The next day, we were transferred to GOSH. A bone marrow test revealed that Luke had a type of high-risk leukaemia that’s known to have a poor outcome.

“Our whole world was turned upside down. You hear about children having cancer, but you never think it’s going to be you, especially when you have a little boy running around with so much energy.”

A gift from big sister Lauren “Halfway through Luke’s second cycle of chemotherapy we were told that he was likely to relapse. Doctors decided that he should have a bone marrow transplant and thankfully his big sister Lauren was a ten out of ten match, which was amazing.

“Luke had his transplant in December 2015, so we spent Christmas in isolation. But the nurses on the ward made it very special for us. When we came home in early 2016, we started to build our lives back up again.”

Coming back to GOSH “We had two wonderful years in remission until I found another lump.

The cancer had come back, in a very rare way. While Luke’s bone marrow was still in remission, he had 26 PIONEER

leukaemia cells growing elsewhere in his body. We were told that standard chemotherapy would be too toxic and there wasn’t much else that could be done.

“But we kept on going and in February Luke had localised radiotherapy on the mass. It was successful, but after some tests they found two other lumps. The first was too close to his kidney for radiotherapy so he had to have chemotherapy. It shrank the mass, but unfortunately the disease had returned to Luke’s bone marrow.“

Hope through a new research trial “We heard about a new radioimmunotherapy trial through Luke’s consultant, Dr Rao. Total-body radiation is quite damaging to your organs, especially for a child like Luke who has already had so much treatment. This trial uses a drug designed to seek out certain cells and release radiation in specific areas where leukaemia cells are likely to reside, like in the spleen and bone marrow.

“Luke recently received the treatment directly into his blood stream. It has been designed to move through his entire body without causing damage to his heart or lungs. And that means they were able to use higher doses of radiation to destroy more bone marrow cells. Now that the he has received the treatment, Luke is

Luke and big sister Lauren, who donated her bone marrow for Luke’s treatment.

going to have another bone marrow transplant.“ “Luke knows that he’s at GOSH to have medicine to treat his ‘naughty lump’, but he doesn’t see coming to the hospital as a negative thing. He loves the play room and going to the music club on a Wednesday. He chats to everyone and is always full of energy.

“We don’t know whether the trial will work for Luke, because it’s never been done on a child with this kind of leukaemia before. But it’s our only hope. We have to try, because I refuse to not do anything. Things can change through research.

“Without people supporting the hospital, this trial wouldn’t be happening, and we wouldn’t get the chance to try and save him again.

It’s so important.”

THE POWER OF DATA In the 1930s, a GOSH surgeon removed a young patient’s tumour and kept the sample in wax. This tumour sat in the archives for decades, until a researcher found it, linked it up with other records of the patient, and published a paper that helped to shed light on the genetic mutations responsible for some of the rarest childhood cancers.

That researcher’s name is Neil Sebire, Professor of Paediatric Pathology and Chief Research Information Officer at GOSH. “When the disease is so rare that you’d only have three or four cases a year, it might take 50 years to get enough cases to determine which mutations are important,” Neil says.

Instead of waiting 50 years, Neil turned to historical data for an answer.

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INTRODUCING THE DIGITAL RESEARCH ENVIRONMENT In a world where we rely on data, apps and technology, it’s staggering to think that data science is still a relatively new idea in a healthcare environment.

That’s why GOSH Charity is investing £37 million in systems designed to collect, store and analyse patient data. One of those systems is the Digital Research Environment (DRE).

“The DRE will organise and share data across specialties, linking data in ways that people might not have thought of before. And who knows what those links will show.”

The DRE has been created to optimise the use of clinical data for research in a cloud-based research and innovation platform. Its role is to combine data from multiple sources, improve cross-organisational and multi-institutional analysis, and enhance the quality of healthcare research, ultimately improving clinical practice and patient care at GOSH. John Booth, Senior Data Steward for the DRE, explains: “The more clinicians you talk to, the more projects you realise have been going on in their own little bubble within each specialty. One of the remits of the DRE is to organise this data into a structure shared across specialties, linking data in ways that people might not have thought of before. And who knows what those links will show.”

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Sota is a storytelling robot that uses data from children’s responses to tailor the story to each individual.

USING DATA TO PRE-EMPT PATIENT NEEDS

USING DATA TO HELP SAVE LIVES

Richard Issitt is a Senior Clinical Perfusionist and the DRE Clinical Engagement Lead. He explains that a method used to combine data from multiple sources to identify patterns that suggest threats, allowing you to respond quickly.

The DRE platform has recently helped researchers discover a new way that patients can receive heart transplants from donors with different, and ordinarily incompatible, blood types to their own.

“One thing I’m working on is measuring the concentration of proteins in the blood. Initial results show that it might be indicative of renal failure,” Richard says.

“We could measure this at the start of an operation and determine whether the patient may need help.

We can predict what intervention they’ll likely need and put it in place during their operation. This allows the patient to start treatment almost pre-emptively and reduces the risk of permanent damage.

“We also hope that by combining data from multiple sources, including past patient data, we will help to develop an app that links to machines that take on the role of the heart and lungs when they aren’t functioning properly. It will calculate patients’ requirements, such as how much oxygen or fluid they need.

Hopefully centres outside of GOSH will be able to add data, to increase the cohort of patients to a scale that has previously not been observed.

“The more we learn, the more we can do for patients. And the less time they spend in intensive care, the better their outcome.”

Previously, patients receiving a heart with an incompatible blood type would need to have their blood replaced, otherwise antibodies in their own blood would reject the transplanted heart. This was an expensive procedure and meant that patients required a long stay in Intensive Care. However, the DRE platform has allowed researchers to analyse multiple data sets and investigate a new process known as immunoadsorption. This is a blood purification technique that removes the antibodies that would reject the transplant, from the patient’s own blood. This has cut the need for blood transfusions by 50% and means that patients spend much less time in Intensive Care.

This new procedure has not only led to significant cost savings, it has also helped to free up more hospital beds for other patients.

This technique was recently published in The Journal of Heart and Lung Transplantation and GOSH is currently the only centre in the world using it.

USING DATA TO IMPROVE INFECTION CONTROL In summer 2018, Arm, a technology company, came to GOSH to propose testing WiFi technology called LoRa within a healthcare environment.

LoRa exchanges small packets of information across a large number of devices. Arm proposed a project in which hand gel dispensers would be the devices sending the information.

The devices would periodically broadcast how many times a hand gel dispenser had been used. As a secondary data set, it would transmit the battery life of the device, so people knew when to change the batteries.

As John Booth explains, the data could be used in two completely different ways: “We can use the data in the operational sense: is the dispenser empty? And does it need to be replaced? Clearly, if you’ve got gel dispensers sitting there empty, that’s a bad thing.

“But the patterns of usage interest me more. As mobile devices, we can place them in different positions, relative to the door, for example, and with signage. We can then begin to explore that data at a very fine level of detail, finding out what does and doesn’t work and adjusting our practices accordingly.

Altogether, this data provides us with valuable insight into a vital part of infection control.”

FRESH PERSPECTIVES Once the Arm project is successfully completed, more cross-discipline collaborations could follow, with companies from completely different industries providing fresh or alternative ways of contributing to projects.

A technology consultancy called NTT Data distribute a robot called Sota.

GOSH and UCL students use Sota as a “storytelling robot”. It collects data, such as a child’s reaction, and tailors the story using that data or reaction.

John says: “We’d like to proactively go out to companies and say: ‘here’s the issue we’ve got, how could you help with this?’ Collaborators might be able to add value in areas we’ve not yet thought about.”

WORKING TOGETHER Data is important but, as Richard Issitt explains, having the right people in place to interpret that data is vital: “From a clinician’s point of view, you’ve got the question and the data, but you don’t know what to do with the data. And from the data scientist’s point of view, you’ve got the data analysis, but you don’t know about the clinical outcomes.

“Having one data area to combine everything means you can consolidate expertise. And in the research environment, if you need a certain expertise, you can invite a collaborator and it doesn’t matter where they are in the world.”

THE START OF A LONG JOURNEY Although the DRE team is embarking on an exciting journey, collecting more data than ever, the road ahead is long.

“These projects are interesting and important, but we’re taking baby steps,” says John. “The real value of what we’re doing will be seen in the years to come. Data science comes into its own with a wealth of linked information, by bringing data together over time and with a wide selection of usage.”

As part of our investment in digital systems, GOSH Charity aims to fund the recruitment of a new generation of clinical informaticians. They will have data and clinical expertise to help analyse and apply data, to support the DRE team.

“The real value of what we’re doing will be seen in the years to come.”

The data being collected today may one day be the equivalent to the 1930s tumour sample preserved in wax – providing answers that could help to save the lives of seriously ill children across the world.

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A PARENT’S PERSPECTIVE

BY BEN’S MUM, VIKI

Before Ben got sick, he was the more boisterous twin. He liked to explore, was mischievous, and took the lead when he played games with his brother James.

Then in December 2015, when he was 17 months old, he came down with what seemed to be a cold. After a few days, it became clear he wasn’t his usual self and he quickly became very ill. He was unresponsive and couldn’t wake up. And when he was taken to the local hospital he had a seizure.

The doctor called GOSH for advice and they sent an ambulance to collect him. A couple of days later the doctors told me that he had meningitis.

Ben went into an induced coma and when he came out I was told there was damage to his brain.

He developed cerebral palsy – a movement disorder that affects his ability to control his muscles and speech. He also lost his vision and hearing and developed epilepsy. He became a child I didn’t recognise, and I had to keep reminding myself that he was the same little boy. I desperately wanted to see him smile, to show me that my adventurous and funny little boy was still there.

When we got home, Ben often cried but showed no positive emotion.

Then after six months, I began to see a few little twitches and one day he rewarded me with a huge smile. It completely changed how I felt about our family’s future.

Ben’s vision gradually began to return and now, when he wears glasses, his vision is very good. Though he’s still profoundly deaf. He has a cochlear implant to help him hear better, but it’s difficult to know how effective it is because communicating isn’t that straightforward. When we communicate at home, we sign and speak at the same time, so he has both visual and sound cues.

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Every hour, Ben’s epilepsy causes around 15 seizures – some visible, others brief absences where he appears to be staring into space – but he recovers quickly.

So far, Ben’s biggest development has been his desire to engage with everything and everyone around him.

He’s a social little boy – he tries hard to communicate verbally, which has been great for his relationship with James. They’ve grown so much closer.

It was difficult for James to see Ben so poorly in hospital – he couldn’t understand why he wouldn’t react like he used to. Now, there’s more interaction between them and Ben will smile or giggle at James.

Ben responds best to faces and facial expressions. He likes seeing people up close. When we go out, he’ll be busy observing things around him, but as soon as someone zooms in close to him they’re met with a great big smile.

He’s well known around school for being so smiley.

Ben receives ongoing support from GOSH, who are the reason we still have him. We meet with the Speech and Language, Audiology and Cochlear teams every few months.

They continue to be the driving force behind his development and provide invaluable support. My hope is that one day all the support Ben gets from school, GOSH and his family will provide him with new opportunities.

He’ll never be able to fully look after himself, but it would be incredible if he developed more independence.

If he could make choices, choose an activity he’d like to do or where he’d like to go, that would be a big step.

“ Every hour Ben’s epilepsy causes around 15 seizures – some visible, others brief absences where he appears to be staring into space – but he recovers quickly and is soon back to being himself. “

Ben at home with his mum Viki and twin brother James.

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Great Ormond Street Hospital Children’s Charity 40 Bernard Street London WC1N 1LE 020 3841 3131 gosh.org/pioneer Great Ormond Street Hospital Children’s Charity. Registered charity no. 1160024.

Six-year-old Aiden preparing for his scan in the SPECT CT suite.

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

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