Our Funded Research
Brain/CNS
How do childhood brain tumours start?
With Dr Anestis Tsakiridis
Many early brain tumours contain cells that shouldn’t be there, mixed in alongside the expected brain cells. When brain tumours contain these cells, they can be a lot more difficult to treat and also to diagnose. Dr Anestis Tsakiridis, at the University of Sheffield, is trying to grow cells from these mixed brain tumours in the lab. He hopes to create a new ‘cell line’ of mixed tumours. Other researchers can use the ‘cell line’ to test new treatments and learn more about mixed brain tumours so that we can find better treatments. Anestis’ lab hopes to continue this work in a later project to look at the cause of these tumours.
Project title: Defining the cellular origins of neonatal and paediatric brain tumours
Lead investigator: Dr Anestis Tsakiridis, The University of Sheffield
Funded by: The Little Princess Trust
Funded: November 2016
Award: £24,990
Could not eating carbs help treat childhood brain tumours?
With Dr Lisa Storer
Brain tumours in children are one of the most difficult types of cancer to treat. When treatments are effective, there are serious side effects that affect the child’s quality of life for years to come. This is especially true for ependymoma and high-grade glioma brain tumours.
Dr Lisa Storer and her team at the University of Nottingham are researching whether limiting the amount of carbohydrates available to cancer cells could help fight these hard-to-treat brain tumours. Unlike most healthy body cells, cancer cells are very dependent on carbohydrates for energy and growth. This means that a carbohydrate restricted diet could be a better and safer treatment for ependymoma and high-grade glioma.
Project title: In vitro evaluation of the potential of glucose restriction as an adjuvant therapy for paediatric brain tumours
Lead investigator: Dr Lisa Storer, University of Nottingham
Funded by: The Little Princess Trust
Funded: November 2016
Award: £24,989
Finding the ‘Achilles Heel’ of childhood brain cancers
With Dr Ruman Rahman
Brain tumours are the second most common type of childhood cancer, and can be very hard to treat. Only one in ten children with a brain tumour have a high-grade glioma tumour, but most of these children don’t have many successful treatment options. Dr Ruman Rahman, at the University of Nottingham, is looking inside high-grade glioma cells to find out which building blocks, like proteins, they need to grow and survive. His team will try growing glioma cells without some of the building blocks to try to find potential targets for future treatments.
Project title: Identifying the metabolic ‘Achilles Heel’ of childhood brain cancers
Lead investigator: Dr Ruman Rahman, The University of Nottingham
Funded by: The Little Princess Trust
Funded: November 2016
Award: £24,806
Investigating a new way of delivering medicine for childhood brain tumours
With Dr Ruman Rahman
Treating childhood brain tumours can be difficult because many medicines don’t travel into the brain well. This means that chemotherapy doses have to be high, which can damage healthy body parts. If we could deliver the medicines directly to the right place in the brain, without having to subject the rest of the body to chemotherapy, it could help many children with brain tumours. Dr Ruman Rahman’s lab at the University of Nottingham have created a chemotherapy paste that can be applied around a tumour site after it has been surgically removed. In this project, he is looking at a way to find out whether the medicines in the paste are spreading into the surrounding tissue properly, so the team can see if this is a good treatment for children with brain tumours.
Project title: Brain distribution models to select polymer-delivered drugs for the treatment of childhood brain cancers
Lead investigator: Dr Ruman Rahman, The University of Nottingham
Funded by: The Little Princess Trust
Funded: November 2016
Award: £99,901
Improving our understanding of ependymomas in the spinal cord
With Dr Anbarasu Lourdusamy
Ependymoma is a type of childhood cancer which grows in the brain and spinal cord. It is more unusual for children to have ependymoma in their spinal cord, but it can be much more difficult to treat. Currently children will have surgery to remove the tumour and radiotherapy or chemotherapy. However, the surgery can be dangerous and the medicines don’t work for some children. Dr Anbarasu Lourdusamy from the University of Nottingham will use tumour samples from children with spinal ependymomas to find out more about this cancer. He will look at the genetic and molecular makeup of the tumours to improve our understanding of ependymoma. He hopes that his findings will indicate new ways that medicines could be used to treat spinal cord ependymoma.
Project title: Comprehensive molecular characterization of paediatric spinal ependymomas
Lead investigator: Dr Anbarasu Lourdusamy, University of Nottingham
Funded by: The Little Princess Trust
Funded: July 2017
Award: £99,961
Using modern scanning techniques to diagnose brain tumours faster
With Professor Andrew Peet
Brain tumours in children are normally diagnosed with an MRI scan, after a parent has noticed concerning symptoms. The MRI scans can show all of the structure and detail of the brain and easily shows if there is a lump. However, doctors often can’t tell what the lump is from a scan – whether it is a tumour or what type of tumour it is. For 15 years, Professor Andrew Peets team at the University of Birmingham have been developing advanced MRI scans called ‘functional imaging’. These scans can give details about the lump like how the blood flows inside it and whether it is starting to grow into the rest of the brain. These are good indicators of whether a lump is cancerous. Using their huge database of scans, Andrew’s team will try to use artificial intelligence to create the best possible non-surgical tool for diagnosing brain tumours.
Project title: Improving the Diagnosis of children’s brain tumours by Functional Radiomics
Lead investigator: Professor Andrew Peet, University of Birmingham
Funded by: The Little Princess Trust
Funded: July 2017
Award: £99,027
Understanding what makes up some types of glioma tumours
Dr Farhana Haque
Despite advances in treating and curing childhood cancer, paediatric high-grade glioma and diffuse intrinsic pontine glioma are still very difficult to treat. These brain tumours are caused by changes to essential genes. These genes help co-ordinate the rest of the cell’s genetic activity, so can cause a lot of problems. Dr Farhana Haque and her team at the University of Nottingham plan to investigate how these genes work so that they can progress their research into the next stage, where they will try to find medicines that will fight these mutated genes
Project title: Molecular pathophysiology of histone G34R mutated childhood brain tumours: towards the development of novel targeted therapies
Lead investigator: Dr Farhana Haque, University of Nottingham
Funded by: The Little Princess Trust
Funded: December 2017
Award: £99,837.30
Improving our understanding of brain tumours in teenagers and young adults
With with Dr Anbarasu Lourdusamy
In addition to the normal changes of growing up, teenagers and young adults have very different cancers to either adults or children. These young people don’t fit into the adult or childhood cancer groups, either in terms of how they would like to be treated or in terms of what medicines would work best. A common type of cancer for young people is brain cancer. However, there isn’t currently any research that can fully describe the ways teenage and young adult cancer is different to adults or children’s. Dr Anbarasu Lourdusamy, at the University of Nottingham, is looking at the genetic code of young people’s brain tumours which will be the first in depth assessment of teenage and young adult brain tumours. He hopes that this will suggest new treatment approaches that are personalised to young people.
Project title: Teenagers and young adults with primary CNS cancers: a systematic biological characterisation
Lead investigator: Dr Anbarasu Lourdusamy, University of Nottingham
Funded by: The Little Princess Trust
Funded: July 2018
Award: £99,911.31
Electrotherapy for childhood brain tumours
With Dr Stuart Smith
Brain tumours are one of the biggest problems in childhood cancer, and current treatments often have serious side effects. Research has shown that brain tumour cells rely on electricity, generated normally by brain cells, to spread and grow. There are clinical trials for adults using an electric field to stop brain tumours from growing, but this approach doesn’t work well for children.
Dr Stuart Smith and his team at the University of Nottingham will investigate the levels of electrical activity in different types of childhood brain tumours and see whether unusual amounts of electrical activity are linked to tumours being harder to treat. The team will then look at whether medicines that affect electrical activity in cells could help treat brain tumours.
Project title: Electrotherapy for childhood brain tumours
Lead investigator: Dr Stuart Smith, University of Nottingham
Funded by: The Little Princess Trust
Funded: £95,696.31
Award: August 2019
Understanding the biology of relapsed ependymoma brain tumours to help improve treatments
With Dr Anbarasu Lourdusamy
Ependymoma is the second most common childhood brain tumour, and is treated with surgery, radiotherapy or chemotherapy. It can be difficult to treat, and often returns after treatment. There is no standard treatment for ependymoma that has come back, and we need to understand ependymoma better to find the best treatments.
Dr Anbarasu Lourdusamy at the University of Nottingham will use existing tumour samples to look at what features are important in ependymoma. The team will be using genetic and molecular testing to find out more about ependymoma. The team hope this will help develop more effective treatments that stop the cancer from returning and treat ependymoma brain tumours better.
Project title: Deciphering the New Molecular Landscape in Paediatric Recurrent Ependymoma: Implications for Molecular Targeted Therapy
Lead investigator: Dr Anbarasu Lourdusamy, University of Nottingham
Funded by: The Little Princess Trust
Funded: August 2019
Award: £99,942.64
Starving brain tumours of what they need to grow
With Dr Madhumita Dandapani
Childhood brain tumours can be difficult to treat, especially high grade gliomas and ependymomas. Treatments can also have serious side effects, meaning we need better and kinder treatments.
Dr Madhumita Dandapani at the University of Nottingham is trying to find targets for medicines which are small enough to enter cancer cells and change the way the cancer cell works. She wants to stop the cancer cell from making specific amino acids, which are the building blocks for proteins. Without the proteins, the cell cannot survive. In this project the team will be exploring how amino acids are made and used in brain tumour cells, seeing where it is different from healthy cells, to hopefully find ways to kill the cancer cells without harming healthy cells.
Project title: Exploring alterations in amino acid metabolism as novel therapeutic targets in paediatric glial tumours using advanced metabolomics methods
Lead investigator: Dr Madhumita Dandapani, University of Nottingham
Funded by: The Little Princess Trust
Award: £96,449.07
Using artificial intelligence to diagnose brain tumours
With Professor Andrew Peet
When doctors think that a child has a brain tumour, doctors will send them for scans (like MRI scans) to see where the suspected tumor is. However, the scans typically cannot tell whether the lump is definitely a tumour or provide more information about what type of tumour it is. That usually requires an operation to remove part of the lump.
Professor Andrew Peet at the University of Birmingham and his team have shown that a new type of scans, called functional imaging, can answer these questions without surgery. However, functional imaging scans are very difficult to read so they aren’t used much. The research team have already created an app that uses artificial intelligence to help doctors interpret the results of functional imaging scans, by comparing the scans to a national library of scan images and results. In this project, the researchers will be further developing the app to tell the difference between different types of tumour and say how likely the tumour is to respond to treatment.
Project title: Moving Artificial Intelligence of Functional Imaging for children’s tumours into a multi-centre environment through a Clinical Decision Support System
Lead investigator: Prof Andrew Peet, University of Birmingham
Funded by: The Little Princess Trust
Funded: August 2019
Award: £99,959.67
Developing a paste to deliver chemotherapy directly to brain tumours
With Dr Ruman Rahman
It is difficult to treat childhood brain tumours at the back of the brain, because many treatments also damage healthy brain cells. It is also hard for medicines to get into the brain, because the brain is protected by a barrier that stops things like bacteria and viruses from entering the brain.
Dr Ruman Rahman at the University of Nottingham has created a paste which contains chemotherapy medicines and can be applied in the brain during surgery, bypassing the barrier. The paste is applied to the space left after a brain tumour has been removed, which kills any remaining cancer cells. This project will investigate how brain cancer cells behave and who is different in their DNA to healthy cells to find potential targets for medicines. The researchers will then assess which chemotherapy medicines would be best to use in the paste.
Project title: Neurosurgically-applied chemotherapy for childhood brain tumours arising in the posterior fossa using a biodegradable paste
Lead investigator: Dr Madhumita Dandapani, University of Nottingham
Funded by: The Little Princess Trust
Funded: December 2019
Award: £626,229
Fighting brain tumours with the Zika virus
With Dr Rob Ewing
It can be very difficult to treat childhood brain tumours because they can be very aggressive and the treatments have serious side effects. We need new ways to treat these cancers without harming healthy brain cells.
Dr Rob Ewing at the University of Southampton has discovered that the Zika virus could be used to fight brain tumour cells, without hurting healthy brain cells. The researchers will now investigate why some brain tumours are susceptible to the Zika virus, and how the virus can fight cancer cells. They then plan to assess whether the Zika virus could be used as treatment for brain tumours. This project will also generate a lot of detailed information about aggressive brain tumours that other researchers can use to inform their research.
Project title: Towards a new therapy against childhood brain cancer: How does the Zika virus kill aggressive brain tumour cells?
Lead investigator: Dr Rob Ewing, University of Southampton
Funded by: The Little Princess Trust
Funded: December 2020
Award: £34,765.74
Repurposing antihistamines to reduce treatment-related toxicity for children with WNT-medulloblastoma
With Dr Jessica Taylor
Medulloblastoma is a common childhood brain tumour that has four subtypes. One subtype is called WNT-medulloblastoma and has a good outlook. However, there are a lot of late effect which can mean that children have a poor quality of life after their treatment ends.
Dr Jessica Taylor at the University of Cambridge wants to find kinder treatments for WNT-medulloblastoma. All cells have a biological ‘kill switch’ for when there are problems, or the cell is no longer needed. This is in the form of tiny sacs filled with enzymes – if the sac bursts the cell dies. Research has shown that the sac in cancer cells is a lot thinner than normal cells. The researchers plan to use normal antihistamine medicines to overload the sacs, causing them to burst, without having an effect on healthy cells. They hope to show that using antihistamines can reduce the amount of chemotherapy and radiotherapy, improving quality of like for childhood cancer survivors.
Project title: Repurposing antihistamines to reduce treatment-related toxicity for children with WNT-medulloblastoma
Lead investigator: Dr Jessica Taylor, University of Cambridge
Funded by: The Little Princess Trust
Funded: December 2020
Award: £71,334.49
Developing new treatments for difficult to treat types of medulloblastoma
With Professor Steve Clifford
In the last 50 years, advances in treatment have meant that around seven in ten children with medulloblastoma can be treated successfully. However, there are groups of medulloblastoma that don’t respond to current treatments, such as MYC-driven medulloblastoma which has problems in a gene called MYC.
Professor Steve Clifford and his team at Newcastle University think that combining two types of treatment could help treat MYC-driven medulloblastoma with a lower risk of it becoming resistant to treatment. They have combined a type of immunotherapy called CAR-T with medicines that target MYC. In this project, the team will test their treatment in the lab to see how tumours respond to the treatment and checking whether tumours could become resistant to it. They hope their research will help progress this new treatment into a clinical trial where it can help children with medulloblastoma.
Project title: Developing and delivering small molecule drug and immunotherapy combinations for MYC-driven medulloblastoma: Efficacy, evolution and exploitation
Lead investigator: Prof Steve Clifford, Newcastle University
Funded by: The Little Princess Trust
Funded: August 2019
Award: £1,053,351.30
Designing better treatments for paediatric ependymoma
With Dr Timothy Ritzmann
Current treatments for ependymoma brain tumours don’t work very well and the cancer comes back after treatment about half of the time. Immunotherapy could be an exciting new treatment option, but scientists need to understand more about how ependymoma works first.
At the University of Nottingham, Dr Timothy Ritzmann and his team are investigating what cells are in different types of ependymoma and how this affects a child’s chance of survival. They are collaborating with experts in Denver and will use different stains to mark all of the different cell types and then analyse the results. Their findings will help researchers develop new treatments for ependymoma.
Project title: Understanding the immune environment in paediatric ependymoma in order to deliver effective immunotherapy and improve patient outcomes
Lead investigator: Dr Timothy Ritzmann, University of Nottingham
Funded by: The Little Princess Trust
Award: £50,010.33
Developing and understanding new treatments for craniopharyngioma
With Dr John Apps
Craniopharyngioma is a type of childhood brain tumour that is difficult to treat. It is in an area of the brain that makes the tumours hard to remove with surgery, and radiotherapy doesn’t work fully in a quarter of children. New treatments are needed to improve the future for these children.
Dr John Apps and his team at the University of Birmingham have found a group of medicines that could help stop tumours from growing. They are called MEK inhibitors and IL6 inhibitors, and will be tested in an international clinical trial for regrown (relapsed) craniopharyngiomas. The research team will be analysing tumour samples from the trial to find out more about what goes on inside the tumour and why they relapse. They will also look at the large cysts that often occur when craniopharyngiomas regrow to find out what they are and what treatments could help.
Project title: Biological Response to Novel Treatments in Adamantinomatous Craniopharyngioma and Exploratory Profiling of Cystic Fluid
Lead investigator: Dr John Apps, University of Birmingham
Funded by: The Little Princess Trust
Funded: July 2021
Award: £39,510
Developing new treatments for Medulloblastoma
With Dr Maria Victoria Niklison Chirou
Medulloblastoma is the most common brain tumour in children. Because there is a risk of it spreading to other locations in the body, treatment is very aggressive. This can leave children with serious side effects like development problems and changes in personality.
Dr Maria Victoria Niklison Chirou at the University of Bath wants to find kinder treatments that could improve quality of life for patients. The researchers will look at whether treatments from other diseases, such as high-cholesterol, could also help children with medulloblastoma. They think that lipid inhibitors could stop cancer cells from producing fats and therefore could starve the cancer cells. Dr Niklison Chirou hopes that using existing medicines means that patients could benefit from any new treatments sooner.
Project title: Repurposing Lipid Inhibitors for the Treatment of Aggressive Medulloblastoma
Lead investigator: Dr Maria Victoria Niklison Chirou, University of Bath
Funded by: The Little Princess Trust
Funded: July 2021
Award: £104,155
Developing a new test for ependymoma
With Dr Madhumita Dandapani
Around forty children in the UK are diagnosed with ependymoma every year. Ependymoma is a type of brain tumour that is difficult to treat, and often comes back. Researchers think that this means some of the cancer stays in the brain, even after surgery and treatment.
Dr Madhumita Dandapani and her team at the University of Nottingham want to create a test to see how much ependymoma is left after treatment. This could help show which children need to continue their treatment to reduce the amount of cancer remaining and prevent it coming back. To do this, the researchers will look at how ependymoma cells behave differently to healthy brain cells so that they can find chemicals that would only be present if ependymoma was. As well as helping to create the test, finding these chemicals could suggest new ways to treat ependymoma
Project title: Developing a biomarker for minimal residual disease in ependymoma
Lead investigator: Dr Madhumita Dandapani, University of Nottingham
Funded by: The Little Princess Trust
Funded: July 2021
Award: £87,678.73
Using man-made antibodies to treat brain tumours
With Professor Sergey Piletsky
Unfortunately, patients with diffuse midline glioma (DMG) often have no good treatment options, even with new advances like immunotherapy. Immunotherapy has been a game-changer for many cancers, using tiny proteins called antibodies to target cancer cells.
Professor Sergey Piletsky at Leicester University wants to create an immunotherapy treatment for DMG. His team have created man-made antibodies which are more reliable than the ones which naturally occur in your body. These antibodies will carry medicines straight to the DMG cells in the brain without harming as much healthy brain tissue. In this project, the team are assessing how well the antibodies work so that they can continue to work on this brand-new treatment.
Project title: Innovative Treatments of Diffuse Midline Gliomasbased on Synthetic Antibodies, A Pilot Study.
Lead investigator: Professor Sergey Piletsky, Leicester University
Funded by: The Little Princess Trust
Funded: March 2022
Award: ££112,514.60
Repurposing existing medicines to treat incurable childhood brain tumours
With Dr Dr David Michod
Diffuse midline glioma is a very aggressive tumour that mostly affects young children. Chemotherapy does not work to treat diffuse midline glioma, and radiotherapy can often only be used palliatively. Therefore, there is an urgent need for new treatment options.
Dr David Michod and his team at University College London has found that an existing medicine called disulfiram is very good at killing diffuse midline glioma cells. In this project they want to understand how disulfiram attacks diffuse midline glioma cells, and to gather data to show whether it is an effective treatment. Dr David Michod hopes that disulfiram could be introduced for use in real patients faster than usual because it has been already shown to have minor side effects and is already approved.
Project title: Repurposing the alcohol-abuse drug disulfiram to target a key epigenetic hallmark of diffuse midline glioma
Lead investigator: Dr David Michod, University College London
Funded by: The Little Princess Trust
Funded: July 2022
Awarded: £200,980.16
Delivering medicines into children’s brain tumours using ultrasound
With Dr Antonios Pouliopoulos
There is currently no effective treatment for diffuse midline glioma, a very difficult to treat childhood brain tumour. This is mainly because of the blood-brain barrier. This barrier protects our brains from pathogens in the blood, but also blocks most medicines from entering the brain. Whilst other brain tumours can break this barrier, diffuse midline glioma cannot. This means that nearly all medications can’t get into the brain, so only a tiny percentage of any medicines given can reach the tumour cells.
Dr Antonios Pouliopoulos and his team at King's College London aim to pack combinations of strong medicines into tiny particles, called liposomes. The researchers will design the particles to react to heat so that, when heated up, they will melt and release the medicines. They will combine ultrasound with another type of special particle, which can temporarily open the blood-brain barrier, to get the medicines into the brain. Dr Antonios Pouliopoulos hopes that this new method can treat the tumour without exposing the rest of the body to lots of chemotherapy.
Project title: Focused ultrasound and thermosensitive liposomes for paediatric brain tumour treatment
Lead investigator: Dr Antonios Pouliopoulos, King's College London
Funded by: The Little Princess Trust
Funded: July 2022
Award: £850,658.89
The glowing tumour trial for children with aggressive brain tumours
With Dr Madhumita Dandapani
Whether or not all of a brain tumour can be removed with surgery is the main factor that affects a child’s chance of survival. Unfortunately, tumours can often grow back even when MRI scans show that the whole tumour has been removed. This suggests that there are still some tumour cells left behind.
For some brain tumours in adults, surgeons use a special dye that makes brain tumours glow pink under a special light. This shows the difference between tumour cells and healthy brain, helping the surgeon accurately identify the edges of the tumour and remove all tumour cells.
Dr Madhumita Dandapani and her team at University College London plan to conduct a clinical trial to prove that the dye works for children as well as adults. They will examine the safety and feasibility of using it in children with aggressive brain tumours. They will also see what differences there are between the edges and the centre of the tumours. Dr Madhumita Dandapani hopes that this will improve our understanding of what makes tumours invade into normal brain and help develop new targeted treatments.
Project title: A Phase II multicentre trial for the use of 5-ALA in paediatric high grade brain tumours
Lead investigator: : Dr Madhumita Dandapani, University College London
Funded by: The Little Princess Trust
Funded: July 2022
Award: £542,667.32
What makes ependymoma in infants different?
With Dr Rob Dineen
Whilst ependymoma brain tumours can happen at any age, it is especially difficult to treat in children under three years old. The standard treatment of surgery followed by radiotherapy can have serious long-term side effects such as learning difficulties and hormone problems.
Dr Rob Dineen and his team at the University of Nottingham want to find out what makes ependymoma in young children different. They will look at what makes up ependymoma, such as what cells are present, what’s inside the cells, and their genetic code. Dr Rob Dineen hopes that a better understanding will help patients into clinical trials or future combination therapies, and improve the overall survival of young children with ependymomas.
Project title: A comprehensive molecular and MR imaging characterization of ependymoma in infants
Lead investigator: Dr Rob Dineen, University of Nottingham
Funded by: The Little Princess Trust
Funded: March 2022
Award: £196,346.87
Blocking chemical reactions which keep ependymoma cells alive
With Dr Ruman Rahman
Not much is known about how ependymoma cancer cells grow. We know that they use energy for their own growth and survival in a process called ‘metabolism’. This process also produces ‘metabolites’ – small substances that can be detected by scientists.
Dr Ruman Rahman and his team at the University of Nottingham have found that some types of metabolites were found in many patients’ ependymoma tumours. This could mean that these metabolites are important to the cancer’s survival. They will now test four medicines to see if they can shut down the metabolism of ependymoma brain cancer cells grown in the lab, starving them. They will then select the two medicines which work the best and test whether the medicines improve the chance of survival in models of ependymoma.
Project title: Blocking chemical reactions which keep cancer cells alive – a new therapy for childhood ependymoma
Lead investigator: Dr Ruman Rahman, University of Nottingham
Funded by: The Little Princess Trust
Funded: July 2022
Award: £196,492.08
Creating a new combination treatment for children's brain tumours
With Professor John Anderson
Brain tumours are the most common type of solid tumour in children, but are sadly very difficult to cure. In recent years, immunotherapy has become a treatment option, which can be a lot kinder than traditional treatments. CAR-T cell therapy is one of the main types of immunotherapy. Doctors take a patient’s own immune cells, called T-cells, and train them to hunt down specific molecules on cancer cells. When the CAR-T cell encounters a cancer cell with the specific molecule it is trained to recognise, it will kill the cancer cell.
Professor John Anderson, with his team at University College London Great Ormond Street Institute, wants to create a new type of CAR-T therapy that will work for childhood brain tumours. His new version of CAR-T therapy will target a molecule that is common on brain cancer cells. He also has found a medicine that can turn the CAR-T cells on and off, which will allow the CAR-T cells to rest and recover so that they don’t wear out. This project will test the combination of this medicine with the new CAR-T cells in the lab.
Project title: Combination therapies of switchable B7H3 CAR-T cells with immunomodulatory imide drugs for paediatric brain tumours.
Lead investigator: Professor John Anderson, University College London Great Ormond Street Institute
Funded by: The Little Princess Trust
Funded: March 2023
Award: £232,914.96
MonoGerm clinical trial for germinoma– is one chemotherapy medicine safer than three?
With Dr Matthew Murray
Germinoma is a type of brain tumour that mostly affects children and young people. Patients can be treated with a combination of three chemotherapy medicines, which is very successful but leads to lots of serious long term side effects. Finding a safer treatment is a top research priority.
In this clinical trial, Dr Matthew Murray at the University of Birmingham wants to find out whether the combination treatment could be safely replaced with just one chemotherapy medicine, and whether this causes fewer side effects. If the team find that using just one medicine is effective, they will continue to test the treatment with the goal of making this the new standard treatment for germinoma. Dr Murray hopes that this using just one type of chemotherapy will means that patients have a better quality-of-life, with fewer side effects, and spend less time in hospital.
Project title: MonoGerm: A phase II trial of carboplatin or vinblastine monotherapy induction prior to radiotherapy for intracranial germinoma
Lead investigator: Dr Matthew Murray, University of Birmingham
Funded by: The Little Princess Trust
Funded: March 2023
Award: £623,737.53
Engineering an effective new immunotherapy treatment for paediatric high-grade glioma
With Dr Jun Ishihara
Paediatric high-grade glioma (PHGG) is a type of childhood brain tumour that is very difficult to cure. The cancer can stop responding to standard treatment, which is why doctors need new ways to fight it. Immunotherapy is an exciting treatment for other cancers, where it enables a patient’s own immune system to hunt down and kill the cancer cells. However, there aren’t currently any immunotherapy treatments that work well for PHGG.
Dr Jun Ishihara at Imperial College London has developed a new type of immunotherapy that he believes could be a breakthrough for patients with PHGG. It is able to target glioma cells by their excessive use of collagen. This means that his immunotherapy will be directed at just the cancer cells, to reduce side effects on healthy brain cells. In this project, Dr Ishihara plans to test his new treatment in model systems and improve how it gets into the brain. If successful, he hopes to bring his treatment to clinical trials within five years.
Project title: Tumour-collagen targeted IL-12 cancer immunotherapy for paediatric high-grade glioma
Lead investigator: Dr Jun Ishihara, Imperial College London
Funded by: The Little Princess Trust
Funded: July 2023
Award: £99,991.32
Investigating the role of an amino acid to find a better treatment for children with medulloblastoma
With Professor Daniel Tennant
Medulloblastoma, a type of childhood brain cancer, can be very difficult to treat. It is especially difficult to treat children whose cancer has too much of a gene called MYC. Their cancer is very reliant on glutamine, which is a building block cells use to make proteins, to survive treatment and repair damage.
If there was a way of stopping the medulloblastoma cells from using glutamine, it could slow down their growth and make them more susceptible to the normal treatment. In this project, Professor Daniel Tennant at the University of Birmingham aims to find out exactly how medulloblastoma cells use glutamine. His team will look at how the cancer cells support their growth, and how they use it to resist dying after chemotherapy or radiotherapy. They will also investigate which medicines could starve medulloblastoma cells of glutamine, and whether this could be part of an effective treatment strategy.
Project title: Investigating the potential of interfering with glutamine addiction to better treat MYC-driven Medulloblastoma
Lead investigator: Prof Daniel Tennant, University of Birmingham
Funded by: The Little Princess Trust
Funded: July 2023
Award: £269,247.54
Understanding how immune cells around the brain protect childhood ependymoma tumours
with Dr Elizabeth Cooper
Childhood ependymoma is a rare and aggressive cancer affecting the brain and spinal cord. We need more research to understand how to cure these children.
Dr Elizabeth Cooper's team at the University of Cambridge is studying how ependymoma can block the immune system’s response. Normally, certain immune cells collect waste from cancer cells in the fluid around the brain and alert the immune system to the threat.
However, ependymoma tumours seem able to stop this process, meaning the immune system doesn't attack the cancer. By investigating how these immune cells work with the tumour, the researchers hope to discover ways to help the body fight cancer more effectively.
Project title: Tumour defenders at the brain’s edge: Elucidating the contribution of brain-border derived antigen presenting haematopoietic stem and progenitor cells in immunotolerance of paediatric ependymoma.
Lead investigator: Dr Elizabeth Cooper, University of Cambridge.
Funded by: The Little Princess Trust.
Funded: July 2024.
Award: £44,936.56.
