Approved Projects 2026

Understanding RNA Changes in High-Risk and Relapsed Neuroblastoma - Pilot Study

Project Number : 26-VIVO-01a ; Jodie Bojko

1) What is this research about?

Every cell in the body contains DNA, which acts as an instruction manual for making the proteins needed for normal growth and function. Before proteins can be produced, DNA is copied into a related molecule called RNA. RNA carries these instructions and helps control how proteins are made. RNA molecules can contain small chemical changes, known as RNA modifications, which act like molecular "switches" that influence whether an RNA molecule is used to make a protein or broken down. These modifications are increasingly recognised as important regulators of cancer development and treatment resistance. This project will investigate how RNA modifications differ between low-risk, high-risk, and relapsed neuroblastoma, a childhood cancer of the nervous system. Using advanced sequencing technology, researchers will analyse tumour samples to identify patterns of RNA modifications associated with aggressive disease and treatment relapse.

2) Why is it important?

Children with high-risk or relapsed neuroblastoma often have poorer outcomes and fewer effective treatment options. While genetic changes in neuroblastoma have been extensively studied, much less is known about how RNA modifications contribute to tumour growth, survival, and resistance to treatment. Understanding these RNA changes could reveal new biological mechanisms that drive aggressive disease and help explain why some tumours return after treatment.

3) What are the researchers doing?

The team will use cutting-edge direct RNA sequencing technology to study RNA molecules from neuroblastoma tumour samples. This approach allows researchers to detect multiple types of RNA modifications while simultaneously measuring gene activity and RNA processing. By comparing samples from low-risk, high-risk, and relapsed tumours, including matched samples collected before and after chemotherapy, the researchers will identify RNA modification patterns associated with disease progression and treatment resistance.

4) How could this help patients in the future?

The study aims to identify RNA modification signatures that could serve as biomarkers for aggressive or relapsed neuroblastoma. It may also reveal new drug targets by identifying proteins that control these modifications. In the future, this could lead to improved risk stratification, earlier detection of treatment resistance, and the development of more effective, targeted therapies for children with neuroblastoma.

Developing Blood Tests for Earlier Detection of Ependymoma Relapse - Pilot Study 

Project Number : 26-VIVO-01b ; Alina Pandele 

1) What is this research about?

Ependymoma is the second most common malignant brain tumour in children and most often affects young children under the age of five. Although surgery and radiotherapy can successfully treat the disease, more than half of children experience tumour recurrence. Once the tumour returns, treatment becomes much more challenging and survival rates are significantly lower. Currently, children are monitored using regular MRI scans after treatment. While these scans are essential, they may not detect tumour recurrence at its earliest stages, particularly before symptoms develop. Earlier detection could allow treatment to begin sooner and improve outcomes for children whose tumours return.

2) Why is it important?

Cancer cells can release small fragments of genetic material into the bloodstream. One type of genetic material, known as cell-free RNA (cfRNA), provides information about which genes are active within a tumour at a given time. Because cfRNA reflects ongoing biological activity, it may offer an earlier and more sensitive way of detecting tumour recurrence than imaging alone. Although cfRNA has shown promise as a biomarker in several cancers, it has not yet been studied in children with ependymoma.

3) What are the researchers doing?

This study will analyse tumour tissue and blood plasma samples collected from children with posterior fossa ependymoma, the most common and aggressive form of the disease. Researchers will compare RNA signals found in tumour tissue with those detected in the bloodstream to identify tumour-specific cfRNA markers. Advanced sequencing technologies and computational methods will be used to determine which RNA signals originate from the tumour and could serve as reliable indicators of disease activity.

4) How could this help patients in the future?

The goal is to identify a panel of blood-based biomarkers that could help detect tumour recurrence earlier than is currently possible. In the future, these biomarkers could complement MRI scans, allowing clinicians to monitor children more closely and identify relapse before symptoms appear. Ultimately, this research could lead to more sensitive and less invasive monitoring of childhood ependymoma, helping doctors intervene earlier and improving outcomes for affected children.

Can an Asthma Drug Help Stop Childhood Leukaemia from Returning - Pilot Study

Project Number : 26-VIVO-01c ; Sophie Kellaway 

1) What is this research about?

Researchers are investigating whether a medicine currently used to treat severe asthma could help prevent a type of childhood blood cancer, called t(8;21) acute myeloid leukaemia (AML), from coming back after treatment. Previous research has identified a marker, called the Interleukin-5 (IL-5) receptor, on the leukaemia stem cells that can survive treatment and cause the disease to return. This project will explore whether the asthma drug benralizumab can help the immune system find and destroy these cells before they trigger a relapse.

2) Why is it important?

Although many children with AML respond well to treatment, around one in five will experience a relapse. When leukaemia returns, it is often harder to treat and can be devastating for patients and families. Current treatments rely on intensive chemotherapy, which can cause long-term side effects and does not always prevent relapse. Finding a targeted treatment that specifically removes the cells responsible for the cancer returning could improve outcomes while reducing the need for additional intensive therapies.

3) What are the researchers doing?

The research team will study samples from children with t(8;21) AML to understand how well their immune system can respond to benralizumab. They will focus on natural killer (NK) cells, a type of immune cell that can destroy abnormal cells when guided by certain antibodies. The researchers will examine whether NK cells are present in sufficient numbers and functioning properly, and then test whether benralizumab can direct these cells to kill leukaemia stem cells carrying the IL-5 receptor. They will also investigate how much of the receptor needs to be present for the treatment to be effective.

4) How could this help patients in the future?

If successful, this research could pave the way for a new targeted treatment designed to prevent AML from returning. As benralizumab is already approved for use in asthma and has an established safety record in children, it may be possible to move more quickly towards clinical trials in patients. Ultimately, this approach could help reduce relapse rates, improve long-term survival, and offer children with AML a safer and more effective way of staying cancer-free after treatment.

Can a Simple Blood Test Help Track Rhabdomyosarcoma More Effectively? - Pilot Study

Project Number : 26-VIVO-01d ; Supriti Ghosh 

1) What is this research about?

Researchers are developing a new type of blood test to help monitor rhabdomyosarcoma (RMS), the most common soft tissue cancer in children and young people. The test looks for tiny fragments of tumour DNA that are released into the bloodstream, known as circulating tumour DNA (ctDNA). By analysing both genetic changes and chemical markers on the DNA, the researchers hope to create a more complete picture of how a tumour is changing over time.

2) Why is it important?

Monitoring rhabdomyosarcoma currently relies on scans and, in some cases, tissue biopsies, which can be invasive and difficult to repeat regularly. Detecting when a tumour is responding to treatment, becoming resistant, or starting to return can be challenging. A sensitive blood test could provide a less invasive way to monitor the disease and potentially detect relapse earlier, allowing treatment decisions to be made more quickly and accurately.

3) What are the researchers doing?

The team will study tumour samples and blood samples collected from young people with rhabdomyosarcoma. They will use a new sequencing technology that can examine both genetic mutations and epigenetic changes—chemical markers that influence how genes are switched on and off. The researchers will compare the results from this new approach with existing testing methods to see whether it can detect tumour DNA more accurately. They will also investigate whether specific DNA patterns can be used to identify signs of disease progression or relapse.

4) How could this help patients in the future?

If successful, this research could lead to a more sensitive and comprehensive blood test for children and young people with rhabdomyosarcoma. Such a test could reduce the need for invasive procedures, provide earlier warning of relapse, and help doctors tailor treatments based on how a patient's cancer is evolving. In the longer term, this approach could support more personalised care and improve outcomes for young people affected by this challenging disease.

Testing New Immunotherapy Combinations for Childhood Leukaemia - Pilot Study

Project Number : 26-VIVO-01e ; Katrina Lappin 

1) What is this research about?

This project aims to develop a new way of testing potential treatments for childhood acute myeloid leukaemia (AML). Researchers will use patient samples to create laboratory models that closely mimic how the disease behaves in children. These models will be used to study new treatment combinations, including immunotherapies that help the body's immune system recognise and attack cancer cells.

2) Why is it important?

Although survival rates for childhood AML have improved, some children do not respond to treatment or experience a relapse. Current treatments are intensive and can cause significant long-term side effects. Developing new therapies is challenging because childhood AML is rare, patient samples are limited, and existing laboratory models can be slow, costly, or fail to reflect the complexity of the disease. Better testing models are needed to identify the most promising treatments more quickly and efficiently.

3) What are the researchers doing?

The research team will use leukaemia cells donated by children with AML to develop specialised models using fertilised chicken eggs, known as patient-derived xenograft CAM (PDX-CAM) models. These models allow human cancer cells to grow in a living environment that supports blood vessel formation and some aspects of immune activity. Once established, the models will be used to test combinations of standard chemotherapy and immune checkpoint inhibitors, a type of immunotherapy already used to treat several adult cancers. The researchers will assess how well the treatments control tumour growth and whether they stimulate anti-cancer immune responses.

4) How could this help patients in the future?

If successful, this project could provide researchers with a faster and more reliable way to identify promising new treatments for childhood AML before they move into more complex studies and clinical trials. By helping scientists prioritise the most effective drug combinations, this work could accelerate the development of safer, more targeted therapies for children with difficult-to-treat leukaemia. In the longer term, it may contribute to improved survival and a reduction in treatment-related side effects.

Understanding Why Neuroblastoma Returns and Stops Responding to Treatment

Project Number : 26-VIVO-02; Gudrun Schleiermacher 

Using samples from the international BEACON trial

1) What is this research about?

Neuroblastoma is a childhood cancer that develops in nerve tissue. While many children respond well to treatment, some experience a relapse, where the cancer returns or stops responding to therapy. Outcomes for these children remain poor. This study aims to understand how neuroblastoma changes over time, particularly between diagnosis, treatment, and relapse. By studying tumour samples and blood samples collected through the international BEACON clinical trial, researchers hope to identify the biological changes that allow cancer cells to survive treatment and return.

2) Why is it important?

Relapsed neuroblastoma is one of the most difficult childhood cancers to treat. Researchers know that cancer cells can evolve during treatment, but the reasons why some tumours become resistant are not fully understood. A better understanding of these changes could help doctors predict which treatments are most likely to work and identify new targets for future therapies.

3) What are the researchers doing?

The team will analyse tumour tissue and blood samples from children enrolled in the BEACON trial. They will study genetic and biological changes in cancer cells and track tumour DNA circulating in the bloodstream over time. By comparing samples taken at diagnosis, during treatment, and after relapse, the researchers will investigate how neuroblastoma evolves, develops resistance to treatment, and spreads. They will also look for biomarkers that may help predict how patients will respond to therapy.

4) How could this help patients in the future?

This research could reveal why some neuroblastomas become resistant to treatment and identify new ways to target these cancers. It may also lead to blood-based tests that help doctors monitor disease more closely and detect important changes earlier. Ultimately, the findings could support more personalised treatment approaches, improve outcomes for children with relapsed neuroblastoma, and guide the development of new therapies for this aggressive disease.

Understanding How Brain Tumours Interact with Their Surroundings

Project Number : 26-VIVO-03; Elizabeth Coyle 

1) What is this research about?

Brain tumours do not grow in isolation. They interact constantly with the surrounding brain tissue, immune cells, and other factors in their environment. These interactions can influence how tumours grow and how they respond to treatment. This project aims to create advanced laboratory models that closely mimic the environment surrounding childhood brain tumours. By studying these models, researchers hope to better understand the biological processes that drive tumour growth and treatment resistance.

2) Why is it important?

Many current laboratory models do not accurately reflect the complexity of real brain tumours. As a result, treatments that appear promising in the laboratory may not work in patients, while potentially effective therapies can be overlooked. More realistic models could help researchers understand tumour behaviour more accurately and improve the way new treatments are tested before they reach clinical trials.

3) What are the researchers doing?

The team will use patient tumour samples to study how brain tumour cells interact with surrounding tissues, immune cells, and other components of the tumour environment. Using advanced technologies, they will compare patient tumours with laboratory-grown models to ensure the models accurately reflect what happens in real tumours. They will then investigate the biological pathways that support tumour growth and identify potential targets for treatment.

4) How could this help patients in the future?

By creating more accurate models of childhood brain tumours, researchers hope to identify treatments that are more effective and cause fewer side effects. In the future, these models could help predict which therapies are most likely to work for individual patients and speed up the development of new treatments. Ultimately, this research aims to improve outcomes and quality of life for children with brain tumours.

AStudying How Neuroblastoma Evolves from Diagnosis to Relapse

Project Number : 26-VIVO-04; Andrea Sottoriva 

1) What is this research about?

This study will investigate how neuroblastoma tumours, a type of cancer that affects young children, change between the time of first diagnosis and when the disease comes back after treatment. We will analyse tumour samples taken at both timepoints from the same patients, using advanced techniques that allow us to examine not just what is in a tumour but where different cell types are located and how they are organised. This spatial information has not previously been studied in neuroblastoma in this way.

 2) Why is it important?

When neuroblastoma comes back after treatment, it is very difficult to treat and outcomes remain poor. We do not fully understand why some tumour cells survive initial therapy and drive relapse. Recent research, including a large UK study called SMPaeds in which our team participated, has shown that the genetic changes alone do not fully explain why relapse occurs, suggesting that other features of how tumours are organised and structured may play an important role. Understanding these mechanisms is essential if we are to develop better treatments.

3) What will the researchers be doing?

Researchers will analyse pairs of tumour samples, one taken at diagnosis and one at relapse, from the same patients. Using specialised laboratory techniques, they will examine how the tumour's structure, cell composition, and surrounding environment change over the course of treatment. These findings will be analysed alongside existing genetic data from the same patients to build a more complete picture of how neuroblastoma evolves.

4)  How could this help patients in the future?

By identifying how tumours are reorganised during treatment and which features are associated with relapse, this research may help guide the development of more effective therapeutic strategies and improve our understanding of why current treatments fail in some patients.