Battling childhood cancer with Cancer UK

Meet the UK’s leading national children’s charity dedicated to the fight against childhood cancer.

Around 4,500 children and young people (according to the Office of National Statistics) are diagnosed with cancer each year in the UK and will undergo gruelling and lengthy treatments, with potentially life-altering side effects. That equates to an average of 12 families a day receiving the devastating news that their child has cancer.

Cancer is the most common cause of death for children under 15 years in the UK with around 250 children in the UK losing their lives to cancer every year. However, despite this, only around 3% of all cancer research funding in the UK focuses on childhood cancer research. It is important to note that children’s cancers are different to adult ones. This is because they occur in different parts of the body, they are different at a molecular level and they respond differently to treatment. The most common types of cancer in children are acute leukaemia and cancers of the brain and spinal cord. Rare cancers in children include neuroblastoma and Wilms’ tumours.

We spoke to Mark Brider from Children with Cancer UK about some of their current research projects from CAR T-cell therapy for leukaemia to neuroblastoma research.

What’s the work and role of Children with Cancer UK?

Children with Cancer UK is the leading charity dedicated to scientific research into childhood cancer. We raise and invest funds into specialist research to improve survival rates and quality of survival in young cancer patients and find ways to prevent cancer in the future. In the past 30 years, Children with Cancer UK has raised more than £250m and funded over 200 research projects looking into causes and treatment of childhood cancers.

Thanks to our ground-breaking research we have helped to improve children’s cancer survival rates from 64% in 1990 to 84% in 2017 (this is an estimated figure by the Office of National Statistics, exact figures will be known in 2023). However, there are still a core of high-risk cancers with poor prognosis which we need to continue to target.

We also support families affected by childhood cancer by funding free patient and family accommodation near hospitals around the UK to keep families together while children undergo treatment, free family days out so families can create special memories away from the hospital ward, and financial grants programmes to support families with hardship related to their child’s cancer treatment.

Can you tell us about some of the current research projects?

CARPALL TRIAL: CAR T-cell therapy for leukaemia

A clinical trial to investigate a new CAR T-cell therapy, designed to target cancer cells more quickly and cause less side effects, has shown very promising results for children with previously incurable acute lymphoblastic leukaemia (ALL). The CARPALL trial was funded by Children with Cancer UK and Great Ormond Street Hospital (GOSH) Children’s Charity and led by Professor Persis Amrolia and Dr Sara Ghorashian.

The trial tests a new kind of CAR T-cell therapy in children and young adults with relapsed ALL. CAR T therapy uses immune cells (T-cells) and chimeric antigen receptors (CAR) to recognise cancerous cells. The patient’s own T cells are genetically modified to contain a new type of CAR molecule known as CAT-19. The modified CAR T-cells were used to treat 14 patients with relapsed ALL at GOSH, Manchester Children’s Hospital and University College Hospital, London. After receiving the CAR T treatment, 12 out of 14 patients with otherwise incurable ALL, cleared their disease after three months and five patients remained leukaemia-free.

ALL affects around 400 children a year in the UK and, while most patients are curable with standard treatments such as chemotherapy and transplants, in some patients the disease comes back (relapses) despite maximal treatment. CAR T-cells have shown great promise for relapsed ALL – the most common cause of cancer death in children in the UK. The study, which opened in June 2016, enabled UK patients to have access to this ground-breaking new treatment before a commercial CAR-T cell therapy, Kymriah, became available on the NHS.

Sarcoma research

Children with Cancer UK invested a significant amount of funding into research to find a therapy for this group of cancers that is more powerful and less toxic. Sarcomas are rare cancers but are the second highest cause of cancer death in children and young adults. The research project ‘Exploring retinoblastoma protein loss as an actionable target in paediatric bone and soft tissue sarcoma’ was led by Professor Sibylle Mittnacht at the UCL Cancer Institute.

A specific group (roughly 30%) of these cancers, identified as having a mutation in RB1, lose a specific way to repair damage to their DNA. Cancers exploit DNA repair to survive cancer treatment, generally aimed at damaging DNA, and defective repair can make cancer cells more sensitive to anticancer medicines. The work proposed seeks to establish information concerning these issues, with the view to develop a therapy for these cancers that is more powerful, and less toxic.

Children with cancers in soft tissue and bone currently need to receive intense treatment in order to provide the best chance for them to be cured. While cure can be achieved today in more than half of the cases, there are severe and sometimes life changing side effects associated with the treatment, and survival has remained uncertain in too many cases.

The work proposed will provide information that can be used to improve therapy for a sizeable group of children and young adults with sarcoma. In the first instance, producing molecular evidence to identify optimal medicine combinations, and by identifying new routes based in specific dependencies that the team believe exist the cancer cells in order for them to survive molecular damage to their DNA.

Neuroblastoma research

A three-minute MRI scan could safely and effectively test new precision treatments for children with neuroblastoma, a new study largely funded by Children with Cancer UK has shown. Advanced MRI techniques could test the effectiveness of drugs for children with neuroblastoma without the need for invasive surgery for the first time. Neuroblastoma is the second most common solid tumour in children, affecting just under 100 children a year in the UK, a third of whom will die. It has one of the lowest survival rates of all childhood cancers, with only 67% of patients surviving to five years.

Children with Cancer UK’s Research Fellow, Dr Yann Jamin, is pioneering improved MRI scanning techniques, leading new research that shows the potential role of functional MRI imaging in delivering precision medicine to children with neuroblastoma. A simple non-invasive MRI scan could play a key role in guiding the delivery of precision medicine to children with neuroblastoma. Until now, testing and monitoring of new cancer drugs in children has been limited by the risk of performing invasive biopsies on young patients. Researchers call for advanced MRI scans to be incorporated into clinical trials for promising improved therapies for neuroblastoma.

His team of researchers at The Institute of Cancer Research, London, found that non-invasive MRI scans can map the network of both blood vessels and red blood cells within neuroblastoma tumours without the need for invasive surgical procedure. The study, published in Cancer Research, means that doctors could predict which child would benefit from new effective and safer treatments which attack neuroblastoma blood supply to cut access to oxygen and nutrients for the first time.

When it comes to the five main research topics (causes, prevention, diagnosis, treatment, survivorship), where have you seen the most innovation and development over the past couple of years?

There have been some positive emerging trends in childhood cancer treatment in recent years, notably Precision Medicine and Immunotherapy, both of which Children with Cancer UK has invested significant funding into. However, these treatment approaches are not yet widely available to children in the UK, meaning further research is vitally important to improving childhood cancer outcomes.

MRD test

We funded a new method of measuring minimal residual disease (MRD), which helps to predict how likely it is that a child with leukaemia will relapse. The resulting national clinical trial in 2003 was so successful that the NHS has now adopted MRD testing as standard – enabling doctors to better tailor each child’s treatment to their individual needs. This has helped to drive the survival rate for childhood acute lymphoblastic leukaemia above 90%, despite being largely incurable 50 years ago.

In 2017, we invested over £3.7m into a project aiming to make Precision Medicine a reality for all UK children. This is an approach to treatment that focusses on the individual genetics of the patient when deciding on treatment and has major potential to not only make treatment more effective, but safer and less harmful. Since 2014, we have also invested over £1.8m to a number of projects developing promising new immunotherapy treatments.

This is an increasingly promising approach to treatment that uses the body’s own immune system to fight cancer. Children with Cancer UK believes that treatment is one part of a bigger picture when fighting childhood cancer. We will continue to invest in research to improve our understanding of the causes of childhood cancer and potential preventative strategies, as well as research to improve diagnosis, helping us catch cancers earlier.

Where do you hope to see the position of childhood cancer in the UK in the next five years? What role do you hope to see Children with Cancer UK play in this?

Ultimately, our aim is a world where no child dies of cancer and we hope survival rates will continue to rise over the next five years. Although survival rates for children with cancer have significantly improved, there is still a long way to go in reducing the impact of childhood cancer. We want to continue to develop our role as a major fundraiser supporting research that has direct impacts on treatment, prevention and quality of life for all those affected by childhood cancer.

For those cancers where more children are surviving, it is crucial that we continue to invest in pioneering new and kinder treatments, like precision medicine, that reduce the burden of toxicity for young cancer patients and improve survival rates in even the hardest-to-treat cancers. We are still on the long journey towards a cure or non-invasive treatments for many forms of cancer. There is also only a limited understanding of prevention. Given this context, there will be a continuing need to provide care and support to children and their families after a diagnosis. As well as funding research into treatments and causes of cancer we aim to improve understanding of the broader health, social and emotional impact of childhood cancers for survivors, especially over the long term.

Mark Brider
Children with Cancer UK
Tweet @CwC_UK

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