Making the future possible for more children with cancerChildhood cancer has been one of the success stories of modern medicine. Such have been the advances made over the past 30 years that eight out of 10 children diagnosed today will be cured. However, cancer in children and young people remains a serious issue.

Around 1,600 children are diagnosed with cancer each year in the UK and, despite all the progress, cancer remains one of the main reasons why children from one to 15 years of age die prematurely. We lack efficient therapies for some children who present with advanced stage disease or for those whose cancer has come back. There is also a downside to the improving survival rates, in that the side effects of current treatments are recognised as an increasing problem. With one in 700 young adults now a survivor of childhood cancer, many long-term survivors suffer from chronic health problems related to their original therapy.

Newcastle Centre for Childhood Cancer is a planned £5.5 million research facility, which will provide state-of-the-art resources to keep advancing children’s cancer research, and ultimately give more children a future to look forward to. The Future Fund, a collaborative project between Newcastle University, North of England Children’s Cancer Research (NECCR) and the Great North Children’s Hospital, aims to raise money to make the centre a reality.

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“Newcastle University has established itself as the leading centre for research into childhood cancer in the UK. It is one of the major centres for paediatric oncology research in Europe. The creation of a Centre for Childhood Cancer here in Newcastle will enable us to expand our activity, help us to retain key staff members, and to attract additional internationally recognised experts in the field. We are already working on exciting novel developments in targeted therapies, and we want to be one of the key players in the next step-change by continuing to develop treatments that are more effective and less toxic. This will help us to secure the future for more children with cancer.”

Professor Josef Vormoor, Director, Northern Institute for Cancer Research

Children’s cancer research at Newcastle University

At the Northern Institute for Cancer Research (NICR), which is part of Newcastle University’s renowned Faculty of Medical Sciences, we are working hard not only to understand more about childhood cancer but also to develop more efficient therapies for children and young people whose cancers are difficult to cure and, for all our patients, therapies with fewer side effects.

The NICR and the Great North Children’s Hospital have a strong track record in the fields of drug development, translational research and early phase clinical trials. This is recognised by many national stakeholders: we are a Cancer Research UK (CR-UK) centre, a Leukaemia and Lymphoma Research (LLR) Centre of Excellence and one of the paediatric Experimental Cancer Medicine Centres (ECMC).

The NICR hosts one of the leading research groups in children’s cancer and leukaemia in Europe, with 13 academic members of staff and a team of 51 laboratory staff and students, and we are partnered with a comprehensive clinical programme at the Great North Children’s Hospital. We are an NIHR Cancer Research Network principal treatment centre for children and young people with cancer, and we are an accredited member of the European Innovative Therapies for Children with Cancer (ITCC) network, which runs clinical trials exploring new drugs for the treatment of childhood cancer.

In the pages that follow, you will see how research carried out at Newcastle University and the Great North Children’s Hospital has already improved the lives of many children and young people with cancer. We will also tell you about our ambitious plans to grow our research programme further.

The establishment of dedicated research laboratories for childhood cancer will ensure that we are able to build upon our success and make a difference to even more children and young people affected by cancer in the future.

The fundraising campaign is a collaboration between Newcastle University, Great North Children’s Hospital and North of England Children’s Cancer Research (NECCR). NECCR does wonderful work in our region as well as supporting the University directly.

All children deserve a future free from cancer and the long-term side effects of treatment. We are committed to advancing research to make this possible. Our staff have been producing world-leading research for many years, and the time has come to create more space to enable the work done at Newcastle to have an even greater impact on children’s lives. The Newcastle Centre for Childhood Cancer will provide the facilities necessary for this research to move forward.

Photo © Northern Institute for Cancer Research

Photo © Mike Urwin

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“It is the worst feeling you can imagine, just such a shock. It was his brother’s birthday as well, so he had to open his presents in the hospital. It just transforms your life when you get a diagnosis like that.”

Stephen, Joseph’s Father

................StoryJoseph’s

TREATING LEUKAEMIA NOW

When Joseph Green woke up one day during November 2009 with a red, swollen toe, it looked to his dad, Stephen, like a simple infection. But just three days later, Joseph was beginning a gruelling three and a half years of chemotherapy after being diagnosed with leukaemia. He was then just five years old, and the diagnosis hit the family hard.

Stephen said: “It is the worst feeling you can imagine, just such a shock. It was his brother’s birthday as well, so he had to open his presents in the hospital. It just transforms your life when you get a diagnosis like that.” During periods of his treatment at the Royal Victoria Infirmary in Newcastle, Joseph was taking 17 different tablets and having injections in his spine every three months.

But then, in 2013, the family got the welcome news that Joseph was in remission. He is now in school and is able to get back to his Karate lessons. Stephen added: “He was so brave during the whole thing, he even stood in front of his class at school to tell them what was wrong with him.”

TREATING LEUKAEMIA IN THE FUTURE by Professor Christine Harrison

Joseph was diagnosed with acute lymphoblastic leukaemia (ALL), a cancer of the white blood cells, which is the most common childhood cancer. Thanks to improved treatments and disease monitoring, up to 90 per cent of children with ALL are now cured. Research at Newcastle University into the underlying genetics of ALL has contributed to improving the survival rate. Years of study, including research within our own group, the Leukaemia Research Cytogenetics Group, has shown that genetic abnormalities in ALL predict the way in which patients respond to therapy. As a result, these abnormalities are used to guide the choice of treatment for individual patients.

‘Good risk’ abnormalities are present in about half of children with ALL, and patients with these abnormalities have an excellent outcome on standard treatments. Unfortunately, a minority of patients do not respond to standard treatment, resulting in a high risk of relapse.

Much of our research has focused on improving outcome in these ‘poor risk’ patients. Ten years ago, we discovered an unusual genetic abnormality among poor risk patients, known as iAMP21. We found that this abnormality was present in around two per cent of children diagnosed with ALL and that it gave them a much greater chance of suffering a relapse and a much lower long-term chance of survival. Since 2003, every child diagnosed with ALL has been tested for the presence of this abnormality using a genetic test designed by our research group. Once identified, they are immediately recommended for very intensive treatment.

Recent results after long-term follow up have shown that children in this group treated with intensive chemotherapy had their risk of relapse reduced by 75 per cent,

and the proportion surviving for five years or more increased to nearly 90 per cent. These results demonstrate the huge potential of personalised medicine.

These discoveries have been possible because we maintain the largest cytogenetic database in the world, containing genetic data from more than 27,000 children and adults diagnosed with acute leukaemia treated on UK and international clinical trials.

The Newcastle Centre for Childhood Cancer will enable us to continue to develop our research in this area and will ultimately result in more lives being saved. It will also enable us to build on our equally important work into the reduction of the long-term side effects of treatment.

Photo © Mike Urwin

Photo © Mike Urwin

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................StoryJames’

“As a parent, when your child is diagnosed with cancer you are willing try anything. The clinicians and researchers did a great job and hopefully other children will be able to benefit in the future too.”

Katherine, James’ Mother

He also took part in a trial of new drugs, being run by the staff at Newcastle University and the Royal Victoria Infirmary. The new drugs were designed to help his body fight back against the cancer cells, and involved extra injections, on top of his chemotherapy. Two years after the first diagnosis, James went into remission and is now back in school with his classmates and able to work on his football skills.

TREATING NEUROBLASTOMA IN THE FUTURE by Professor Deborah Tweddle

Thanks to our research, we now understand more about the genetics and treatment of neuroblastoma, so the chance of survival has increased for many children with high-risk neuroblastoma like James, particularly through the introduction of immunotherapy. However, only around 50 per cent of patients with high-risk neuroblastoma – where the disease has spread to distant sites in a child over 18 months of age – are cured of their disease. In addition, those who survive often have long-term toxicities which cause problems such as hearing impairment and reduced fertility.

The neuroblastoma research group at Newcastle University is helping develop targeted and less toxic therapies to hit the genetic abnormalities present in neuroblastoma. These genetic abnormalities are also used to determine different levels of risk for different patients which in turn determines how much treatment they need. In addition we hope to discover new targets using novel cellular models of neuroblastoma.

Over the last 10 years, research undertaken by the Newcastle University team has suggested that inhibitors of certain proteins are beneficial for patients with this malignancy, and recently we have undertaken pre-clinical studies as part of a drug discovery programme. Our molecular diagnostic service continues to develop in response to the discovery of new treatments for neuroblastoma that are dependent on the presence of specific genetic abnormalities at either diagnosis or relapse.

We urgently need extra space and more researchers if we are to continue and develop our vital work on neuroblastoma. This will help us to further develop molecular diagnostics to identify the patients who are most likely to benefit from new treatments, as well as helping us to develop new treatments and identify new targets that can be exploited in the future.

TREATING NEUROBLASTOMA NOW

Watching 10-year-old James McDermott kicking a football around his garden at home in Chester-le-Street, County Durham, no one would ever guess that, just five years ago, he was diagnosed with cancer and given a 50 per cent chance of survival.

The Newcastle Centre for Childhood Cancer will enable us to fulfil our ultimate goal of curing more children with neuroblastoma in the future, with survivors having fewer long-term side effects.

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His mother, Katherine, explains that he was a happy, healthy boy until he suddenly fell ill and was hardly able to walk. It took just three weeks for James to be diagnosed with neuroblastoma – the most common malignant solid tumour of childhood outside the central nervous system. The cancer was wrapped around his kidneys, stopping them from functioning normally. Further tests revealed 12 other cancer hotspots around his body, so James started an intensive course of chemotherapy that lasted for around 18 months.

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....................StoryCharlotte’s

“I felt like I was getting my life back, I felt like I had a future again when I was told I was in remission. Without the other people who were being treated at the same time as me, many of whom didn’t make it, I wouldn’t be here.”

CharlotteTREATING BRAIN TUMOURS NOW

When Charlotte was 13 she developed neck pain, nausea and vomiting. Following an MRI scan, specialists at the Great North Children’s Hospital diagnosed her with a malignant brain tumour of the cerebellum. She underwent surgery to remove the tumour. The pathologist confirmed that the tumour was a medulloblastoma and Charlotte underwent a year of intense treatment.

She had radiotherapy of her head and spine daily for six weeks followed by chemotherapy for the rest of the year. Amazingly, though her schooling was hugely disrupted, Charlotte was able to get eight GCSEs and went on to study at Kirkley College while working part-time in Kirkley Zoo. Charlotte is now 19 years old and free of disease.

TREATING BRAIN TUMOURS IN THE FUTUREby Professor Steve Clifford

Advances in treatment over the last 20 years have led to improved survival for many children with medulloblastoma. However, not all children fare as well as Charlotte. Outcomes are difficult to predict – around 35 per cent of children with medulloblastoma still succumb to their disease, and many surviving patients suffer significant long-term side effects. The childhood brain tumour programme at Newcastle University is focused on understanding the biology of medulloblastoma and how it varies between patients. In turn, we are exploiting these discoveries clinically, with the aim of delivering individualised therapies more accurately to each child, based on the biology of their tumour. This approach holds the promise of improved cure rates and reduced side effects following treatment.

Over the last decade, our team has discovered critical biological features of medulloblastomas – or biomarkers – which can be measured in the laboratory and more accurately predict the course of a disease. These discoveries are now forming the basis of worldwide clinical trials, which are testing for the first time whether biologically-driven treatment selection leads to improved outcomes. Newcastle University is the international reference centre for biological testing in European trials, and undertakes analysis on all patients in the UK.

Despite these promising advances, we still need to understand medulloblastoma biology in greater detail, and apply these findings to benefit all children with this disease.

Such cutting-edge tumour investigations, as part of our ongoing clinical and biological research programmes, are entirely dependent on the development of the proposed laboratories and the resulting improvement in facilities and staffing.

For many children with brain tumours, the future is in question. With our research, we aim to offer every sufferer a life free of the disease and the side effects of medication. The Newcastle Centre for Childhood Cancer will help us to achieve this goal.

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................StoryJoe’s

“You are living on your nerves all the time. Joe is one of the lucky ones. Being part of the trial made such a big difference. There are so many of the young children who were on the ward with him who didn’t make it.”

Joe’s mother, Carla

CLINICAL TRIALS IN THE FUTUREby Dr Gareth Veal

Over the past several decades we have seen significant improvements in the treatment of childhood cancer, with overall five-year survival rates exceeding 80 per cent now being reported. Advances have largely been made through the development of new drugs and improvements in the use of established chemotherapy treatment. In the pharmacology group at the NICR, we are investigating how children’s bodies process cancer drugs, and how the actions of drugs are influenced by variation in a person’s genes. By investigating differences in the way that individual patients process drugs and respond to them, we can endeavour to improve the beneficial effects of the drugs for every patient.

In the study that Joe took part in, which was recently published in the journal Clinical Cancer Research, we found that in three quarters of cases the patients were achieving drug exposures which may not be beneficial. The study showed that, when given a standard dose, some patients were achieving levels of drug in the bloodstream that could be too low to be effective, and that this problem could be overcome by individually tailoring the dose of the drug to children taking part in the study.

A major challenge in giving the drug is that it only comes in capsule form. As most children with neuroblastoma are too young to swallow the capsules, they have to be opened up and the contents either put down the child’s feeding tube or mixed with food. This means it’s very hard to control exact doses, as some medicine can be left in the capsule or not transferred efficiently. This under-dosing could have a significant impact on a child’s recovery. The findings from the trial, which recruited patients being treated at hospitals across the UK, have resulted in patients being prescribed more appropriate doses Europe-wide.

It is essential that drugs like 13-cis-retinoic acid are used as effectively as possible to further improve the treatment of childhood cancer. In this respect, clinical pharmacology studies are central to determining the most appropriate selection of drugs and dosing regimens to further improve survival rates and minimise side effects in children with cancer.

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CLINICAL TRIALS NOW

Joe Johnson was transformed from a normal healthy boy almost overnight when he developed the childhood cancer neuroblastoma at the age of three. He suffered severe weight loss and developed a lump on his kidney. The cancer spread to his adrenal gland and down his legs. Doctors thought his chances of survival were not very good.

But thanks to a new trial at the Royal Victoria Infirmary involving chemotherapy, surgery, high dose chemotherapy, radiotherapy and further treatment, Joe, aged eight, is now in remission.

Joe was part of a clinical trial which investigated the treatment of neuroblastoma with the drug 13-cis-retinoic acid, which plays a key role in preventing the cancer from returning following surgery, chemotherapy and radiotherapy.

Photo © Mike Urwin

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The Newcastle Centre for Childhood Cancer will enable us to continue to run clinical trials which directly impact on patient treatment, helping us to make more discoveries that save the lives of children like Joe.

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Our plans for the future

We are committed to continuing to support our staff in their fight against children’s cancer. By creating the advanced laboratories needed for our world-class research in the heart of the Newcastle University campus, we will have the opportunity to build on our strong track record and achieve our goal of changing the lives of more children like James, Joe, Charlotte and Joseph.

The additional laboratory space will allow us to develop further as one of the leading centres for paediatric oncology research in Europe by helping us to keep key staff and attract additional internationally recognised experts in the field. With more laboratory space and more staff, we will be able to do more and better research to improve the treatment of cancer in children. The new centre will lead to more people working together to develop more effective and less toxic therapies and offer more children the chance of a future free of cancer and the side effects of treatment.

We hope that, with your support, Newcastle University will continue to develop as a leading centre for research in this area. This redevelopment is essential in helping us to achieve our goal of improving the lives of children with cancer, and you can be part of it. Donations from people like you can help us to save more young lives.

The Newcastle Centre for Childhood Cancer will comprise: Open plan Class 2 laboratory space for

over 100 staff (Approximately 65 are in post at present)

Modular office accommodation for 12 senior researchers

One large and several smaller meeting rooms, plus a social space to facilitate collaborative research

Open plan office accommodation for administrative staff and Laboratory personnel

Specialist facilities for:

• Tissue culture

• Fluorescence activated cell sorting

• Cryopreservation

• Bacteriology

• Microscopy

For more information, visit futurefund.co.uk

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If you would like to make a donation or find out more about the campaign, please contact:

Dr Katie Harland Senior Development Manager Newcastle University katie.harland@ncl.ac.uk 0191 208 6072

Christopher PeacockChairmanNECCR0191 2769631

or visit futurefund.co.uk

About the Great North Children’s Hospital

The Great North Children’s Hospital (GNCH) at the RVI in Newcastle is one of the largest in the UK, offering more services than any other children’s hospital outside of London. It is an internationally leading research centre into childhood diseases, to help improve treatments for young patients.

For further details, visit www.gnch.nhs.uk or call 0191 282 5261.

About North of England Children’s Cancer Research

The North of England Children’s Cancer Research fund was founded in 1978 by families affected by childhood cancer. Over the past 35 years the charity has raised over £20 million for research in Newcastle, helping to establish the Northern Institute for Cancer Research and cementing the region’s reputation as a leading centre for research into children’s cancer. The NECCR has partnered with Newcastle University to raise £5.5M for the Future Fund campaign.

For further details go to the NECCR website, www.NECCR.org.uk

futurefund.co.uk