Call 2024

The fifth call dedicated to paediatric brain tumour led to the selection, by independent international experts, of eight innovative research projects for a total amount of 10,7 million euros. These projects include 6 translational projects and 2 clinical trials.

ELICIT: EarLy phase Immuno-oncology ClinIcal Trials platform for pediatric-type malignant glial tumors

ELICIT project aims to set up a platform to speed up the testing of new immune-based treatments for children and adolescents with aggressive brain tumours.

Brain tumours, especially aggressive types known as malignant gliomas, are the biggest cancer killers among children and teenagers. Every year, more than 500 young patients in Europe lose their lives to these cancers because current treatments simply aren’t effective enough. In recent years, treatments that harness the body’s own immune system to fight cancer have shown incredible results in blood cancers. However, when it comes to brain tumours, these treatments are still in very early stages. While initial small-scale studies have shown promising signs, moving to bigger studies that can truly prove whether these treatments work has been a major challenge.

ELICIT project focuses on creation of a platform that aims to close the gap between early promising results and getting these treatments to patients on a wider scale. The platform will select the most promising immune-based treatments and test them in larger, well-designed clinical studies. To ensure the best possible results, the platform will create a shared set of rules and guidelines for how patients are selected for each study and how their responses — whether medical, immune-related, or based on brain scans — are measured. This way, results from different studies can be compared and combined, giving researchers the strongest possible evidence.

By producing solid and reliable evidence, ELICIT platform will help convince drug companies and health authorities to approve these treatments for wider use – ultimately bringing new hope to children and adolescents battling aggressive brain tumours.

Financed: 738 000 €
Duration: 2 years
Countries: France, Switzerland, United Kingdom, Spain, Sweden, the Netherlands
Disease: Brain tumours
Status: Ongoing
Principal investigator: Jacques Grill

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REVIIH-BT: audiovisual telerehabilitation programme on visual field restoration in children with hemianopia consecutive to a brain tumour

REVIIH-BT project aims to test an innovative home-based virtual reality programme designed to help children and young people recover vision lost as a result of brain tumours.

Brain tumours and their treatments frequently cause vision loss in children. Depending on the type of tumour, between thirty and ninety percent of young patients experience some form of visual impairment – from blurred vision and difficulty seeing colours to the loss of part of their visual field (known as hemianopia). These vision problems significantly affect their ability to learn, develop, live independently, and enjoy a good quality of life. Despite the scale of this problem, there are currently no visual rehabilitation options available for children who lose part of their vision due to a brain tumour.


Building on promising early results showing that combining sound and visual stimulation can help patients recover some of their lost vision, this project will test an eight-week home-based rehabilitation program using immersive virtual reality technology. One hundred children, teenagers, and young adults aged 10 to 25 with hemianopia caused by brain tumours will be recruited from eight hospitals across seven European countries. Participants will follow a personalised program from the comfort of their homes, with the research team remotely monitoring their progress. Vision assessments will be carried out at regular intervals to measure improvements in visual field, day-to-day visual function, and quality of life.

This program is expected to help restore visual perception in the damaged visual field, ultimately improving participants’ development, independence, and quality of life. By enabling children and young people to receive personalised therapy at home – without the need for hospital visits or direct supervision – this approach ensures that even those living in remote or rural areas can access specialised care. The REVIIH-BT project has the potential to transform how we support young brain tumour survivors, offering them a real chance to regain lost vision and live fuller, more independent lives.

Financed: 1 226 000 €
Duration: 3 years
Countries: France, United Kingdom, Denmark, Italy, Austria, the Netherlands
Disease: Low-grade glioma
Status: Ongoing
Principal investigator: Michael Reber

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MiMiC-Kids: mirroring microglia-cancer cell interaction to enhance anti-glioma immune response

MiMiC-Kids translational project develops innovative lab models that replicate a child’s brain tumour and its surrounding environment to identify and test new immune-based treatments for one of the deadliest childhood cancers.

Diffuse midline glioma (DMG) is one of the most aggressive brain tumours in children, with fewer than 10% of patients surviving beyond two years after diagnosis. Despite significant advances in understanding the biology of these tumours, finding new treatments that can be brought to clinical trials has been incredibly difficult. One of the most promising treatment approaches – immunotherapy that harnesses the body’s own immune system to fight cancer – faces a major obstacle: the brain’s own immune cells, called microglia and macrophages, play a critical role in helping tumours resist treatment and continue growing. Yet current lab models used to test new therapies fail to account for these immune cells, meaning researchers are missing a crucial piece of the puzzle when developing new treatments.

To address this gap, the MiMiC-Kids project brings together experts to develop and use unique 3D lab models – known as « patient avatars » – that combine a patient’s own tumour cells, immune cells, and brain tissue. These avatars closely mimic what happens inside a child’s brain, allowing researchers to test treatments in a much more realistic setting. Using ten patient-derived models, the team will: first, test existing and new treatments that target the interaction between immune cells and tumour cells; second, identify markers that predict whether a treatment will work or fail; and third, develop and test new treatment combinations designed to boost the immune system’s ability to fight the tumour.

This project is expected to uncover new treatment options for children with DMG who currently have no effective therapies. If an existing drug shows promise, it could be fast-tracked into a clinical trial. If a completely new target is discovered, the team will collaborate with biotech partners to develop new drugs, which can then be tested on these patient avatars to speed up their path to the clinic. Ultimately, these models will be made available to the wider research community, creating a powerful shared platform for discovering and testing new treatments.

Financed: 1 805 500 €
Duration: 3 years
Countries: France, United Kingdom
Disease: Diffuse midline glioma
Status: Ongoing
Principal investigator: Florent Ginhoux

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RADIO-MEDSCREEN: dual chemogenomic screening to increase the efficacy of radiotherapy against medulloblastoma

RADIO-MEDSCREEN project aims to find ways to make radiotherapy more effective against childhood brain tumours while reducing its harmful side effects.

Medulloblastomas are the most common type of malignant brain cancer in children. Radiotherapy is one of the main treatments for this disease, but it comes with significant challenges. While some patients respond well, others do not respond at all or see their cancer return after initial improvement. On top of this, the vast majority of survivors face life-long side effects from radiation to the brain, including learning difficulties, memory problems, and difficulties with speech and movement. There is therefore an urgent need to find ways to make radiotherapy work better while using lower doses to reduce these devastating long-term effects.

The team will use advanced high-throughput screening technology – automated systems that allow the rapid testing of hundreds of potential treatments – to identify ways to boost the effectiveness of radiotherapy. They will test two large collections in parallel: a library of 220 approved drugs and molecules already in clinical development, and a library of more than 77,000 small DNA fragments that can individually switch off every gene in the human genome. By testing these both alone and in combination with radiotherapy, the team aims to identify drugs that can enhance the effect of radiation, as well as genes that play a key role in helping tumour cells resist treatment. The most promising findings will then be validated using clinically relevant models of the disease.

The RADIO-MEDSCREEN project will not only deepen our understanding of why some medulloblastomas resist radiotherapy but also open the door to new treatment combinations that could improve outcomes while reducing harmful side effects. The team’s strong network of paediatric oncologists involved in clinical trials will help ensure that promising results are quickly brought from the lab to the patients who need them most.

Financed: 365 000€
Duration: 3 years
Countries: France
Disease: Medulloblastoma
Status: Ongoing
Principal investigator: Eddy Pasquier

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ITCC BrainTAP: the ITCC Brain Translational Accelerator Platform

ITCCBrainTap project creates a systematic pathway to turn scientific discoveries about childhood brain tumours into new treatments that can be tested in clinical trials.


Over recent decades, our understanding of childhood cancer has been transformed thanks to advances in technology. However, this improved knowledge has largely failed to translate into new treatments for young patients. This gap between scientific discoveries and actual therapies is especially concerning for brain tumours, where treatment is made even more challenging by the brain’s natural protective barrier, which makes it difficult for drugs to reach the tumour. Nearly 2,000 children are diagnosed with brain tumours each year across the EU, with about half falling into high-risk groups. While overall survival across all brain tumours is around 75% at ten years, for many high-risk tumours, even five-year survival is tragically close to zero. And for those who do survive, many face lifelong side effects that severely impact their quality of life.

This project tackles the problem in three ways. First, working with leading experts, the team will create and regularly update a comprehensive map of the most promising treatment targets across all childhood brain tumours, ensuring no opportunity is overlooked. Second, they will establish a rigorous process for testing new treatments in the lab using multiple tumour models, generating the essential data needed to move towards clinical trials. Third, they will build strong links with clinical trial teams and pharmaceutical companies to ensure that the most promising treatments are actively pushed forward into well-designed clinical studies.

By creating a clear and structured bridge between laboratory discoveries and clinical trials, this project aims to significantly increase the number of new treatments reaching young brain tumour patients. This systematic approach will help ensure that promising therapies are identified, tested, and brought to patients faster — offering real hope to children and families facing some of the most devastating forms of cancer.

Financed: 2 000 000 €
Duration: 4 years
Countries: Netherlands, United Kingdom, Germany, Austria
Disease: All brain tumours
Status: Ongoing
Principal investigator: David Jones

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SOUP: scanning the liquids of paediatric brain tumour patients to personalize treatment

The SOUP project aims to develop simple, non-invasive tests using body fluids to improve diagnosis, treatment monitoring, and follow-up care for children with brain and spinal cord tumours.

Every year, more than 1,800 children across the EU are diagnosed with a brain or spinal cord tumour, making these the leading cause of cancer-related death in children. Survival rates vary hugely depending on the type of tumour – ranging from almost 100% to tragically close to zero. Throughout their journey, patients and their families face enormous challenges and anxiety: the risks of surgery, long waiting times for a diagnosis, uncertainty about whether additional treatment is needed, whether the tumour is responding, whether it will come back, and if it does, whether any further treatment options exist. Currently, monitoring relies heavily on brain scans, which cannot always detect subtle changes happening within the tumour.

This project focuses on advancing a technique called liquid biopsy – a simple, non-invasive method that analyses body fluids such as blood, urine, or cerebrospinal fluid (the water-like fluid surrounding the brain) to detect DNA shed by the tumour. Using this tumour DNA, the team aims to: enable faster diagnosis at or even before surgery; help surgeons better plan tumour removal while minimising damage to the brain; track how the tumour responds to treatment in ways that scans alone cannot reveal; adapt the treatment to each patient’s specific needs, reducing unnecessary side effects; detect tumour recurrence earlier; and analyse how tumours evolve during treatment to guide the choice of targeted therapies if the cancer returns.

This project has the potential to transform how childhood brain tumours are diagnosed, monitored, and treated. By giving doctors a more complete and real-time picture of each patient’s tumour, liquid biopsies could lead to faster diagnoses, more personalised treatments, fewer side effects, and earlier detection of recurrence – ultimately improving outcomes and quality of life for young patients and their families.

Financed: 1 998 000 €
Duration: 4 years
Countries: Germany, Netherlands, Denmark, France, Czech Republic, United Kingdom, Sweden
Disease: All brain tumours
Status: Ongoing
Principal investigator: Johannes Gojo

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EUROPE: exploring unknown relapse origins in paediatric Ependymoma

The EUROPE project seeks to discover new treatments for recurring childhood ependymoma by understanding how tumour cells survive initial therapy and become resistant to treatment.

Ependymoma is the third most common malignant brain tumour in children and adolescents, with 200 to 300 new cases diagnosed in Europe each year. Tragically, 50 to 60% of these children will die from their disease. Current treatment options are limited to surgery and radiation therapy, as standard chemotherapy has shown little effect against this type of tumour. Even when treatment is successful, it often comes with severe, lifelong side effects such as cognitive impairment, hormone deficiencies, and growth problems. The biggest challenge is that when ependymoma comes back after initial treatment, there are currently no effective therapies available – leaving patients and their families with very few options.

To address this critical gap, the team will study the tumour cells that survive initial treatment and go on to cause the cancer to return. They will systematically compare the genetic makeup of tumour cells taken from the same patient at two points — at first diagnosis and at recurrence. This will help them understand what genetic changes allow certain cells to survive treatment and become resistant. In addition, they will investigate how different cell types within the tumour communicate with each other and how normal surrounding cells influence tumour growth. By combining all of these insights, the team aims to identify biological weak spots that could be targeted with new drugs.

By revealing the mechanisms behind treatment resistance, this project has the potential to pave the way for the development of targeted therapies – offering real hope to young patients who currently have no effective treatment when their cancer returns.

Financed: 878 000 €
Duration: 3 years
Countries: Netherlands, Germany
Disease: Ependymoma
Status: Ongoing
Principal investigator: Kristian Pajtler

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FIGHT4MB: establishing first preclinical genetic mouse models of group 4 Medulloblastoma

The FIGHT4MB project aims to create the first lab and animal models of the most common type of childhood brain tumour to better understand the disease and find new treatments.

Medulloblastoma accounts for around 60% of all brain tumours in children, with approximately 650 new cases diagnosed in Europe each year. The most common form, known as Group 4 medulloblastoma (G4MB), makes up 40% of all cases. Despite being so common, the biology of G4MB remains poorly understood and no treatments have been developed specifically for this tumour type. Current treatment relies on radiotherapy followed by chemotherapy, but results are disappointing – around 40% of patients see their cancer return, which is almost always fatal. A major reason for the lack of progress is that researchers have had no reliable lab or animal models to study this disease, making it extremely difficult to test new treatments.

Recent scientific breakthroughs have identified the genetic changes that drive G4MB and pointed to a specific type of brain cell – called Unipolar Brush Cells – as the likely cell from which this cancer originates. Building on these discoveries, the team will take the crucial next step and develop the first ever models of G4MB. They will engineer both mouse models and lab-grown cell models by introducing the known genetic changes into Unipolar Brush Cells to see if this triggers tumour formation. Once created, they will verify that these models accurately mimic the human disease. Finally, they will use these models to test how tumours respond to current treatments and identify which genes could be targeted to prevent relapse.

By creating the first reliable models of G4MB, this project will provide the research community with essential tools to better understand the disease and test new therapies before they enter clinical trials. This work will open the door to new, more effective treatments for children with Group 4 medulloblastoma.

Financed: 1 678 000 €
Duration: 4 years
Countries: Portugal, Germany, Spain
Disease: Medulloblastoma
Status: Ongoing
Principal investigator: Adriana Sanchez Danes

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Contact

Programme support and management is provided by the European Science Foundation (ESF), which is the intermediary with the funding organisations.

For further information about the application, review, selection, and reporting process please contact FIGHT KIDS CANCER Secretariat at ESF: fightkidscancer@esf.org

For further information about the post-grant management please contact FIGHT KIDS CANCER directly at: ellina@fightkidscancer.eu

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