Approved Projects 2025

Leukaemia stem cell and biomarker study in children with acute myeloid leukaemia treated on Myechild 01

1) What is this research about? 

This research is focused on understanding a special group of cancer cells called leukaemia stem cells in children with acute myeloid leukaemia (AML). These cells act like "roots" of the disease, even when most of the cancer is destroyed by chemotherapy, the roots can stay hidden and grow again, causing the cancer to return. The study aims to identify these stem cells in different types of childhood AML and find new ways to remove them completely. 

2) Why is it important? 

Childhood AML is a very aggressive blood cancer, and even with strong treatment, some children relapse because current therapies do not fully eliminate the leukaemia stem cells. These cells are particularly tough and resistant to chemotherapy. By learning exactly what these cells look like, how they behave, and what makes them different from healthy cells, researchers hope to design treatments that target the disease at its source and reduce the chances of relapse. 

3) What are the researchers doing? 

The researcher team will study samples from children taking part in the interntional Myechild 01 clinical trial. Using advanced technologies that can study thousands of cells one by one, they will look at both of the outer features (proteins) and the internal workings (genes) of these leukaemia stem cells. A key focus is on a marker called CD180, which appears to be found on the surface of these stem cells but not on healthy ones. The researchers will also test new therapies, including immunotherapies and drug-linked antibodies, that specifically target CD180 to see if they can effectively destroy these harmful cells. 

4) How could this help patients in the future? 

By learning how to find and eliminate leukaemia stem cells, this project could lead to treatements that are both more effective and less harsh for children. Targeting CD180 could enable new, personalised therapies that stop AML from returning and improving long-term survival. Ultimately, the study aims to help children not only beat AML but stay free of it, offering a stronger foundation for lasting health.   

Nonomedicine stratification to decrease the toxicity of anticancer treatement in children

1) What is this research about? 

This research is exploring whether anticancer drugs can be delivered in a safer and more targeted way using nanomedicines, tiny particles designed to carry drugs directly to tumours. These treatments take advantage of a feature seen in many cancers, where the blood vessels around tumours are more "leaky" than normal. This leakiness allows nanomedicines to enter and build up inside the tumour more easily. The goal of this study is to understand how this process works in children and young adults, and to find out which patients are most likely to benefit from nanomedicine-based treatments. 

2) Why is it important? 

Many cancer treatments cause serious side effects that can affect children for the rest of their lives. Nanomedicines could make treatment more gentle by targeting cancer cells more precisely and sparing healthy tissues. However, because most nanomedicines research has been done in adults, we dont yet know how well these treatments behave in children, whose tumours and blood vessels may be different. By identifying biological markers that predict how nanomedicines work in young patients, doctors could tailor treatments to each individual child. 

3) What are the researchers doing? 

Researchers are studying blood samples from children and young adults with cancer to measure biomarkers, substances in the blood linked to how tumour blood vessels behave. At the same time, they are building a laboratory model that mimics the movement of nanomedicines through tumour-like blood vessels. Together, these studies will help reveal how nanomedicines act in different types of childhood cancers and which patients are most likely to benefit from them. 

4) How could this help patients in the future? 

This research could lead to a simple blood test that helps doctors decide whether nanomedicine-based treatments are suitable for a specific child. That would allow more targeted, effective, and less toxic treatments, improving recovery and quality of life for children and young people living with cancer. 

Using Imatinib to improve treatment for children with ABL-Positive Acute Lymphoblastic Leukaemia 

1) What is this research about? 

This study focuses on a particular subtype of childhood acute lymphoblastic leukaemia (ALL), a cancer of the blood and bone marrow. In this form of ALL, a change in the DNA caused two genes to join together (a "fusion"), creating a signal that tells the cancer cells to keep growing. This signal is controlled by a protein called an ABL-class kinase. The study is testing whether adding a targeted drug called imatinib, which blocks this signal, can improve treatment for these children. 

2) Why is it important? 

Children with this subtype of ALL often don't respond well to standard chemotherapy and have needed very intensive treatment, which can cause long-term side effects. Targeted therapies like imatinib act directly on the cancer-driving protein, which may allow doctors to use lower doses of chemotherapy while still keeping the treatment effective. This could make therapy both safer and more successful. 

3) What are the researchers doing? 

Researchers will study blood and bone marrow samples from children treated with imatinib as part of their standard care. They will look at how well each child's leukaemia responds during the first few months of treatment, using genetic tests to understand why some respond better than others. In the laboratory, they will also test how leukaemia cells react to different drugs that target the same protein. 

4) How could this help patients in the future? 

This research could help tailor treatments for children with this high-risk type of ALL, showing who benefits most from drugs like imatinib and why. The long-term aim is to improve survival while reducing the harsh side effects of chemotherapy, helping children not only beat cancer but also enjoy a better quality of life after treatment. 

Understanding why Immunotherapy works differently in children with leukaemia

1) What is this research about? 

This study is exploring why a treatment called blinatumomab, a type of immunotherapy used for B-cell acute lymphoblastic leukaemia           (B-ALL), works very well for some children and young adults but not for others. Researchers want to discover biological markers or "biomarkers", that can show early on who is likely to respond to this treatment and who may need additional therapies. 

2) Why is it important?  

Blinatumomab had helped many young people with leukaemia achieve long-term remission, but some patients still have disease that persists or returns. Understanding why this happens could help doctors tailor treatments to each individual, improving cure rates while reducing unnecessary side effects. This knowledge will also refine how blinatumomab is used in future clinical trials. 

3) What are the researchers doing? 

Researchers are analysing information from children and young adults treated with blinatumomab across the UK as apart of their regular NHS care. This includes linking genetic test results, treatments details, and measurable residual disease (MRD) results, a highly sensitive way of detecting any remaining cancer cells, with patient outcomes. As all data and samples have already been collected, no changes will be made to standard treatment. 

4) How could this help patients in the future? 

By finding the biomarkers that predict whether blinatumomab will work, this research could allow doctors to personalise therapy, giving each child the treatment most likely to succeed. This could lead to more effective care, fewer side effects and better long-term outcomes for children and young adults with leukaemia. 

Finding safer and kinder therapies for blood cancers 

1) What is this research about? 

This research aims to create safer and more effective treatments for clood cancers such as leukaemia and multiple myeloma. Researchers are developing "mini bone marrow" in the lab using human stem cells to recreate the environment where these cancers grow. By studying how cancer cells interact with their surroundings in the bone marrow, researchers hope to understand why treatments sometimes fail and how to stop cancer from coming back. 

2) Why is it important? 

Leukaemia is the most common cancer in children. While current treatments can be successful, they are often harsh and can cause serious long-term side effects. Sadly, some patients relapse when their cancer becomes resistant to treatment. A major reason for treatment failure is that leukaemia cells are protected by their surrounding bone marrow environment. Existing research models, often using mice, do not fully reflect what happens in the human body, meaning many promising treatments treatments fail before they ever reach patients. This project solves that problem by using human-based models, helping to speed up the discovery of therapies that work in real patients. 

3) What are the researchers doing? 

Researchers have developed a new laboratory platform that mimics the human bone marrow using human stem cells and advanced 3D bioprinting technologies. They will use this model to grow patient-derived leukaemia cells and observe how they survive, communicate and resist treatment. They will then test new drugs and treatment combinations to see which ones are most effective at stopping cancer cells, while being kinder to healthy cells. This approach removes the need for animal testing and allows scientists to conduct large-scale drug screening more accurately. 

4) How could this help patients in the future? 

This research could lead to treatments that are more targeted, less toxic, and more successful at curing blood cancers without causing lifelong side effects. By using models that closely represent real patients, researchers can identify the most effective therapies faster and with greater accuracy. Ultimately, this work could help prevent relapse. improve survival rates, and give children and adults with blood cancer a much better quality of life. 

Characterising and targeting amino acid dependencies of leukaemia cells in the bone marrow 

1) What is this research about? 

This research is focused on understanding how leukaemia cells survive inside the bone marrow by using nutrients, particularly amino acids, as fuel. Amino acids are the building blocks of proteins, and leukaemia cells may rely on certain amino acids to protect themselves from treatment. By studying these metabolic "weak spots," researchers hope to discover new ways to stop treatment-resistant cancer cells from surviving and causing relapse. 

2) Why is it important?

Although treatments for leukaemia have improved, many patients still experience relapse because a small number of cancer cells survive inside the bone marrow. These cells are often resistant to both chemotherapy and newer targeted therapies. One reason they survive is that they use specific amino acids to protect themselves from treatment damage. Currently, the models used in research do not accurately reflect the bone marrow environment in patients, meaning new drugs often fail when tested on real patients. This project will recreate a realistic bone marrow environment in the lab to identify and target the nutritional dependencies of leukaemia cells, leading to treatements that are more precise and less toxic. 

3) What are the researchers doing? 

Researchers will compare nutrient levels, especially amino acids, in the blood and bone marrow of healthy people and those in leukaemia. They will create a 3D laboratory model that closely copies the bone marrow environment by using real patient cells and specifically designed gels and fluids that mimic the body. Using these models, they will test how leukaemia cells respond when certain amino acids are removed or blocked, and they will investigate new treatment combinations that make cancer cells more vulnerable. 

4) How could this help patients in the future? 

By identifying which amino acids leukaemia cells depend on to survive, scientists can develop new therapies that "cut off the fuel supply" to resistant cancer cells. This could make existing treatments more effective, reduce the chances of relapse, and lower the need for harsh chemotherapy. Ultimately, this research aims to deliver more personalised and kinder treatments that improve survival rates and quality of life for patients with leukaemia. 

A HR-NBL2 assoicated biological study to link gentic studies performed at diagnosis and relapse with serial circulating tumour DNA (ctDNA) profiling 

1) What is this research about? 

This research is focused on high-risk neuroblastoma, an aggressive childhood cancer that often spreads and is difficult to treat. The study aims to understand how the cancer changes over time by analysing genetic information from both tumour samples and tiny fragments of tumour DNA found in the blood, known as circulating tumour DNA (ctDNA). By studying these changes at diagnosis, during treatment, and at relapse, researchers hope to identify which genetic features are linked to treatment success or resistance. 

2) Why is it important? 

Currently, all children with high-risk neuroblastoma recieve very intensive treatments, but not all patients respond in the same way. Some children relapse or become resistant to therapy, and doctors currently have limited tools to predict who will respond well. Understanding the gentic changes that drive treatment resistance could allow doctors to tailor therapies to each child, improve survival rates, and reduce unnecessary side effects from ineffective treatments.

3) what are the researchers doing? 

Researchers will analyse tumour tissue collected at diagnosis and at relapse, alongside multiple blood samples collected during treatment from 400 children taking part in an international clinical trial (HR-NBL2). Using advanced sequencing technologies, they will track genetic changes in the cancer by studying ctDNA in the blood. This approach allows them to detect aggressive cancer cells that may not be captured in a traditional tumour biopsy. They will also study how the cancer keeps growing by investigating telomere maintenance mechanisms, features that cancer cells use to become "immortal."

4) How could this help patients in the future? 

This research could lead to significant breakthroughs in how high-risk neuroblastoma is treated. By identifying the gentic markers linked to treatment resistance, doctors may be able to predict which therapies are most likely to work for each patient. This could enable more personalised, effective treatement plans, earlier detection of relapse, and new targeted therapies. Ultimately, the goal is to increase survival rates and improve the quality of life for children with high-risk neuroblastoma.    

Understanding why some children with B-cell Acute lymphoblastic leukaeima do no respond to Blinatumomab

1) What is this research about? 

This study investigates why some children with B-cell acute lymphoblastic leukaemia (B-ALL) do not respond fully to Blinatumomab, an immunotherapy that helps the immune system target cancer cells. Although Blinatumomab has improved outcomes for many patients, some still relapse or fail to respond. Researchers aim to uncover the biological reasons for this resistance.

2) Why is it important? 

Blinatumomab is increasingly used to treat childhood B-ALL because it is more targeted and less toxic than traditional chemotherapy. However, without understanding why some children resist treatment, they may continue recieving therapies that don't work. Identifying markers of resistance will help doctors choose the most effective treatments for each child, improving survival and personalising care. 

3) What are the researchers doing? 

Researchers will compare leukaemia and immune cells from children who respond well to Blinatumomab with those from children whose disease returns or resists treatment. Using advanced tools such as flow cytometry and single-cell sequencing, they will examine surface proteins and genetic changes to identify features linked to resistance. 

4) How could this help patients in the future?

Findings could help predict early which children are unlikely to benefit from Blinatumomab, allowing doctors to offer better treatment options sooner. This research may guide new strategies to overcome resistance and ensure each child recieves the therapy best matched to their cancer biology. 

Investigating Genetic Risk Factors for Leukaemia in Children with Down Syndrome

1) What is this research about?

Children with Down syndrome have a much higher risk of developing acute lymphoblastic leukaemia (ALL), the most common type of childhood cancer. However, most children with Down syndrome never develop leukaemia, and researchers do not fully understand why. This study aims to identify genetic differences that may increase the risk of leukaemia in some children with Down syndrome. By comparing the DNA of children with Down syndrome who developed ALL with those who did not, researchers hope to uncover new genetic risk factors.

2) Why is it important?

Children with Down syndrome who develop ALL often experience more treatment-related complications and may have poorer outcomes than children without Down syndrome. A better understanding of the genetic factors that contribute to leukaemia risk could help researchers understand how the disease develops and identify children who may be at increased risk. This knowledge may also provide new clues for improving treatment and prevention strategies.

3) What are the researchers doing?

The research team will analyse the complete genetic code of children with Down syndrome who developed ALL and compare it with that of children with Down syndrome who have not had leukaemia. Using advanced genome sequencing technology, they will search for inherited genetic changes across the genome, including changes on chromosome 21, which is present in an extra copy in people with Down syndrome.

4) How could this help patients in the future?

This study could reveal new genetic factors that increase the risk of leukaemia in children with Down syndrome. Understanding these risks may help doctors identify children who would benefit from closer monitoring and could support the development of more personalised approaches to treatment. In the longer term, the findings may improve our understanding of childhood leukaemia more broadly and contribute to better outcomes for children with and without Down syndrome.

Can Low Oxygen Levels Explain Differences in Survival in Childhood Brain Tumours?

1) What is this research about?

Childhood brain tumours can vary greatly in how they behave. Some respond well to treatment, while others are more aggressive and lead to poorer outcomes. This study is investigating whether low oxygen levels within tumours, known as hypoxia, could help explain these differences. Researchers will analyse tumour samples from children whose brain scans have already been studied to look for signs of hypoxia and other features of the tumour environment.

2) Why is it important?

Previous research found that certain patterns seen on MRI scans were linked to shorter survival in children with brain tumours. These patterns may indicate that some tumours have a poor blood supply and low oxygen levels. In adult cancers, hypoxia is known to make tumours more aggressive and less responsive to treatment. Understanding whether the same is true in childhood brain tumours could help identify new ways to predict outcomes and develop more effective treatments.

3) What are the researchers doing?

The team will examine tumour samples stored in the VIVO Biobank using advanced imaging techniques that allow multiple biological markers to be measured within the same tissue sample. They will look for markers linked to low oxygen levels, blood vessel growth, immune cells, and tumour growth. These findings will then be compared with MRI scan data and survival information to determine whether hypoxia is associated with poorer outcomes.

4) How could this help patients in the future?

If hypoxia is found to play an important role in aggressive childhood brain tumours, it could become a new target for treatment. The findings may also help doctors identify patients at higher risk of poor outcomes and improve the way tumours are assessed. Ultimately, this research could contribute to the development of more personalised treatments and better outcomes for children with brain tumours.

Improving Risk Assessment for Children with Neuroblastoma

1) What is this research about?

Neuroblastoma is a childhood cancer that can behave very differently from one patient to another. Some tumours may shrink on their own or respond well to treatment, while others are more likely to grow or return. This study aims to identify the best way to predict how a child's cancer is likely to behave. Researchers will compare three different molecular testing methods that analyse tumour samples for genetic and biological features linked to patient outcomes.

2) Why is it important?

Doctors currently use factors such as a child's age, the stage of the cancer, and certain genetic changes to decide how much treatment is needed. However, these methods do not always predict outcomes accurately. More precise risk assessment could help ensure that children receive the most appropriate treatment—avoiding unnecessary side effects for lower-risk patients while identifying those who may need closer monitoring or more intensive care.

3) What are the researchers doing?

The study will analyse tumour samples from around 200 children with low- and intermediate-risk neuroblastoma across several European countries. Researchers will compare three different approaches to risk assessment: analysing patterns of gene activity, studying how tumour cells maintain their chromosome ends (telomeres), and examining changes in chromosome structure. They will then compare how well each method predicts patient outcomes over time.

4) How could this help patients in the future?

By identifying the most accurate way to assess risk, this research could help doctors make better treatment decisions for children with neuroblastoma. In the future, more personalised risk assessment could reduce unnecessary treatment and its side effects for some patients, while ensuring that higher-risk children receive the care they need. The findings may also help standardise neuroblastoma care across Europe, giving more children access to the most appropriate treatment for their individual disease.

Understanding Why Some Children Develop Lymphoma After an Organ Transplant

1) What is this research about?

Children who receive an organ transplant need medicines that suppress their immune system to prevent rejection of the transplanted organ. As a result, some children develop a group of conditions known as post-transplant lymphoproliferative disorders (PTLD), which are often linked to infection with the Epstein-Barr virus (EBV). PTLD can range from a mild illness that resolves on its own to an aggressive lymphoma. This study aims to understand whether these different forms of PTLD are related and whether children who develop a mild form of the disease are at greater risk of later developing lymphoma.

Why is it important?

PTLD remains a significant cause of illness and, in some cases, death among children who have received organ transplants. Doctors currently have limited ability to predict which children are most at risk of developing severe disease. A better understanding of how PTLD develops could help identify high-risk patients earlier and improve monitoring and treatment strategies.

What are the researchers doing?

Researchers will study tissue samples and clinical information from children who experienced more than one episode of PTLD after transplantation. Using genetic sequencing, they will compare samples collected at different stages of the disease to determine whether later lymphomas developed from earlier PTLD lesions and identify the genetic changes involved in disease progression. The project forms part of a wider European collaboration studying these rare conditions.

How could this help patients in the future?

By understanding how PTLD develops and progresses, researchers hope to identify early signs that a child may be at risk of developing lymphoma after transplantation. This could lead to earlier diagnosis, closer monitoring of high-risk patients, and more timely treatment. Ultimately, the findings may help improve outcomes and reduce the impact of PTLD in children who have undergone organ transplantation.

Using Artificial Intelligence to Find New Treatments for Childhood Leukaemia

1) What is this research about?

Acute myeloid leukaemia (AML) is a type of childhood blood cancer that remains difficult to treat. Unlike some other childhood leukaemias, survival rates for AML have improved more slowly, and new treatment options are urgently needed. This project aims to create a detailed "knowledge bank" of AML cells using advanced genetic technologies and artificial intelligence (AI). By studying thousands of individual leukaemia cells, researchers hope to build a much deeper understanding of how AML develops and survives.

2) Why is it important?

Many of the treatments that have transformed outcomes for other childhood leukaemias have not had the same success in AML. Researchers believe that new breakthroughs may require a more fundamental understanding of the disease. By analysing AML cells in unprecedented detail, this study could uncover previously unknown biological processes that drive the disease and reveal new opportunities for treatment.

3) What are the researchers doing?

Researchers will analyse leukaemia samples from children with AML using cutting-edge technologies that measure gene activity, DNA changes, and other molecular features in individual cancer cells. They will combine these data with existing datasets and use AI to create a large-scale model of AML biology. The team will then use this model to identify new treatment ideas, which can be tested in laboratory studies. The data generated will also be made available to researchers worldwide.

 4) How could this help patients in the future?

This project could provide entirely new insights into how childhood AML develops and behaves. By identifying weaknesses in AML cells, researchers hope to discover new treatment approaches that could improve survival and reduce the burden of treatment. The resulting knowledge bank will also be a valuable resource for the wider scientific community, helping to accelerate future research into childhood leukaemia and ultimately improve outcomes for children diagnosed with AML.

AMonitoring Rhabdomyosarcoma Through Liquid Biopsies

1) What is this research about?

Rhabdomyosarcoma (RMS) is the most common soft tissue cancer in children and young people. Although many patients are successfully treated, some cancers do not respond well to treatment or return after therapy. This study is investigating whether “liquid biopsies” – blood and bone marrow samples that contain traces of cancer DNA and other tumour markers – can help doctors monitor the disease more effectively. Researchers will analyse samples from patients taking part in the international Frontline and Relapse-RMS (FaR-RMS) clinical trial and compare the results with scans and clinical outcomes.

 2) Why is it important?

Current methods of monitoring RMS mainly rely on imaging scans and tissue biopsies, which may not always detect changes in the cancer early enough. More sensitive tests could help identify patients at higher risk of relapse and detect when treatment is not working. Earlier detection may allow doctors to make treatment decisions sooner and improve outcomes for patients.

 3) What are the researchers doing?

The research team will first identify genetic changes in patients’ tumour samples. They will then look for these same cancer-specific markers in blood and bone marrow samples collected throughout treatment. By tracking these markers over time, the researchers hope to monitor treatment response, detect cancer returning earlier than current methods, and better understand how the disease changes as it progresses.

4) How could this help patients in the future?

This research could lead to more accurate and less invasive ways of monitoring rhabdomyosarcoma. In the future, simple blood tests may help doctors identify patients who need different treatments, detect relapse earlier, and tailor care more closely to each patient's disease. Ultimately, this could improve survival and quality of life for children and young people with RMS.