Our Funded Research
Other / Multiple Cancer Types
Using an antibody 'smart bomb' to help the body fight cancer
With Dr Francis Mussai
Our immune systems detect and fight abnormal cells, like cancer or cells infected with viruses, every day. A lot of childhood cancers can manipulate immune cells, called suppressor cells, so that the anti-cancer response is switched off, leaving the cancer to grow and multiply. If we could target these suppressor cells, it could be a safer treatment with fewer side effects.
Dr Francis Mussai at the University of Birmingham has developed a treatment that targets suppressor cells. The treatment uses antibodies that help the immune system recognise the suppressor cells and kill them, allowing access to the cancer cells. In this project, the researchers will investigate how well this treatment works, with the hope to use their data to move into clinical trials.
Project title: Targeting Myeloid-Derived Suppressor Cells (MDSCs) and Tumour-Associated Macrophages (TAMs) with the anti-CD33 immunotoxin Gemtuzumab ozogamicin to restore anti-cancer immunity
Lead investigator: Dr Francis Mussai, University of Birmingham
Funded by: The Little Princess Trust
Funded: January 2019
Award: £94,060.69
Reducing the side effects of chemotherapy
With Professor Lucy Donaldson
A chemotherapy medicine called Vincristine is used to treat several types of childhood cancer. However, there are side effects that come with this, including pain and pins-and-needles in the patient’s hands and feet. This is because the chemotherapy can damage nerves, as well as attacking the cancer.
Professor Lucy Donaldson at the University of Nottingham wants to find a way to prevent these side effects, as patients can struggle with pain from their treatment for years later. Her team have created new chemicals that could prevent the nerves being damaged. They plan to test whether these chemicals can stop the nerve damage and reverse long-term pain. This project is first step in developing completely new ways of stopping the long-lasting pain and nerve damage caused by chemotherapy. These experiments could contribute to new drugs that would improve the lives of people after cancer treatment.
Project title: Can we reduce or eliminate the sensory nerve damage caused by vincristine chemotherapy? Potential novel adjunct neuroprotective/analgesic therapies.
Lead investigator: Professor Lucy Donaldson, University of Nottingham
Funded by: The Little Princess Trust
Funded: August 2019
Award: £95,820.30
Testing a new treatment for hard to treat cancer and immune system over-activation conditions
With Dr Francis Mussai
It can be difficult to treat children with cancer that had come back after treatment, or whose cancer didn’t respond to treatment. Some of these children can also have problems with ‘immune system over-activation conditions’, which gives them dangerously high fevers and blood poisoning type reactions. Current treatment is with chemotherapy that supresses the immune system, but it is not always effective. New research is needed to provide better treatment options leading to a cure.
Dr Francis Mussai at the University of Birmingham has found that cancer cells sometimes have a marker that stops the immune system from attacking it. There is already a medicine that targets this marker. The researchers will be investigating whether this medicine can help children with hart to treat cancer or immune over-activation conditions.
Project title: GOTHAM - A phase II trial to assess the activity of Gemtuzumab Ozogamicin Therapy in Hemophagocytic lymphohistiocytosis (HLH)/Macrophage activation syndrome (MAS) or relapsed/refractory cancers
Lead investigator: Dr Francis Mussai, University of Birmingham
Funded by: The Little Princess Trust
Funded: December 2019
Award: £197,574
Learning how which children would benefit from PARP inhibitors
With Dr Susanne Gatz
When cancer returns after treatment, we say it has ‘relapsed’. It is now standard in Europe to look at the genetic makeup of relapsed tumours, with the idea that doctors can match specific genetic errors to medicines that counter the errors’ effects.
As part of a new clinical trial, Dr Susanne Gatz at the University of Birmingham is trying to find which genetic errors mean that a child would benefit from new medicines called PARP inhibitors. The researchers will look at tumour samples from over 100 patients to see if they can find any early indications that PARP inhibitors would be a good treatment for that child. They also hope to find markers that will tell doctors whether the treatment is working well and fighting the cancer.
Project title: Novel biomarkers for PARP inhibitor trials for children with cancer
Lead investigator: Dr Susanne Gatz, University of Birmingham
Funded by: The Little Princess Trust
Funded: December 2020
Award: £103,7773.99
Monitoring chemotherapy levels in childhood cancer patients
With Dr Gareth Veal
It is difficult to know how much chemotherapy to give to very young children who are only a few weeks old. Too little chemotherapy could not cure the cancer, but too much could cause serious side effects like organ damage and hearing loss.
Dr Gareth Veal and his team at Newcastle University found a new way to make sure patients are getting the right amount of carboplatin, a chemotherapy medicine. They measure the levels of carboplatin in a patient and then change the dosage to make sure the right level is achieved. Now they want to expand their research into other chemotherapy medicines in a research programme that is treating up to 200 patients a year. Not only will this help children treated in this study, it will improve treatment for years to come.
Project title: Optimisation of the Treatment of Childhood Cancer Patient Populations through the use of Therapeutic Drug Monitoring
Lead investigator: Dr Gareth Veal, Newcastle University
Funded by: The Little Princess Trust
Funded: July 2021
Award: £174,580.00
Exploring DNA to find undiscovered causes of cancer
With Dr Sarra Ryan
Your genetic code contains all of the information that makes you, you. It tells your body when to grow, decides your eye colour, and many other things. It is made up of genes, which code for specific proteins like enzymes or antibodies, with a lot of code in between scientists don’t fully understand. When the genetic code was first investigated, they only looked at the gene sections.
Dr Sarra Ryan and her team at Newcastle University think that there may be clues in the code between genes that could help us to understand the origins of some cancers. They have developed a way to easily measure how much variation there is in specific areas of the genetic code, so that they can look at what effect differences in these areas have on childhood cancer.
Project title: A new era of cancer genomics: Utilising the new reference genome to explore sequence variation and transcription within repeat domains in childhood cancer
Lead investigator: Dr Sarra Ryan, Newcastle University
Funded by: The Little Princess Trust
Funded: March 2022
Award: £196,110.00
Identifying new drugs for the treatment of retinoblastoma
With Professor Majlinda Lako
Retinoblastoma is a common type of childhood eye cancer. Many of the treatments for it have serious side effects, and if the cancer returns there aren’t many more options. This all shows that there is a clear need for new treatments with fewer side effects.
Professor Majlinda Lako and her team at Newcastle University have been looking at the genetics of retinoblastoma cells. They have found the genetic triggers that lead to healthy cells becoming cancerous and identified which drugs could target these triggers. Now, the researchers are ready to test the new drugs they found. Whichever drugs are the most successful will then be tested to see whether they can reach the retina of the eye through the bloodstream, and how long they work for once there. Professor Majlinda Lako hopes this project will revolutionise retinoblastoma treatment.
Project title: Harnessing the power of patient specific organoids to discover new therapeutic treatments for retinoblastoma
Lead investigator: Professor Majlinda Lako, Newcastle University
Funded by: The Little Princess Trust
Funded: July 2022
Award: £199,902.00
International clinical trial to test the use of dye during children’s cancer surgery
With Mr Max Pachl
Surgery is an important part of the treatment for many young people with cancer, but it does not always go as planned. Parts of the tumour can be left behind or missed, other organs can be damaged when the surgeon tries to get all the tumour out.
Mr Max Pachl from the Cancer Research UK Clinical Trials Unit at the University of Birmingham wants test the use of a special green dye in childhood cancer surgery. The dye glows when looked at with light from special cameras. This means the surgeon can see exactly where the tumour is, making it easier to remove all of it and to avoid damaging to healthy organs.
In this project, a computer will randomly choose which patients will have the dye used in their surgery and which will not. This is so that the results can show if there is a difference between using the dye and not, such as less damage to healthy tissue or more tumour successfully removed. Mr Max Pachl hopes that the results will show whether using the dye makes surgery easier and safer so that more children can be helped.
Project title: GLOSurgery: indocyanine Green and near infrared fluorescence in Paediatric Oncology surgery. A randomised, multinational, multicentre study.
Lead investigator: Mr Max Pachl, Cancer Research UK Clinical Trials Unit at the University of Birmingham
Funded by: The Little Princess Trust
Funded: July 2022
Award: £656,327.63
Protecting young hearts from chemotherapy-related heart problems
With Dr Roisin Kelly-Laubscher
Some types of chemotherapy, like doxorubicin, can cause damage to the patient’s heart – but doctors often don’t have a choice because they need to fight the cancer. However, if they could give another medicine at the same time which would protect the heart, it could help prevent long-term health problems for children.
Dr Roisin Kelly-Laubscher at University College Cork plans to find out whether a medicine, called ethanolamine, could protect the heart if given before doxorubicin treatment. Her lab knows that this drug can decrease damage caused by doxorubicin one type of heart cell in the lab, called fibroblasts. However, there is another type of cell, called cardiomyocytes which are more important to protect. In this project, Dr. Kelly-Laubscher will see how the medicine affects these heart cells, then find out what dose and timings would work best. She hopes that this project will help take this treatment one step closer to clinical trials, where it can help real patients.
Project title: Protecting young hearts from drug-induced cardiotoxicity.
Lead investigator: Dr Roisin Kelly-Laubscher, University College Cork
Funded by: The Little Princess Trust
Funded: July 2023
Award: £52,097.00
Investigating nanomedicines to make treatment safer for children with cancer
With Dr Marie-Christine Jones
Medicines which treat cancer can have a lot of side effects and even cause long-term health problems because they also damage healthy cells. We really need new and safer treatments. One way to do this could be with nanomedicines, which use tiny particles to deliver the anticancer medicines. These can be safer because they can slip through the gaps in tumour blood vessels, which are especially leaky – these tiny medicines struggle to get through healthy blood vessels to attack normal cells.
Dr Marie-Christine Jones at the University of Birmingham wants to look at the use of nanomedicines in children, as they are currently mostly used for adults. This project aims to understand how nanomedicines work for children, learn more about leaky blood vessels, and test nanomedicines in leaky blood vessel models to find out which ones would work best.
Project title: Nanomedicine stratification to decrease the toxicity of anticancer treatment in children.
Lead investigator: Dr Marie-Christine Jones, University of Birmingham
Funded by: The Little Princess Trust
Funded: July 2023
Award: £49,313.43
How does MYCN change the way genetic information is read in childhood cancer?
With Professor Karim Malik
Cancer cells grow out of control due to changes in their genetic instructions. These instructions tell a cell how to behave, so when there are changes it can cause serious issues that lead the cell to become cancerous. Sometimes parts of the instructions, called genes, are incorrectly switched on or off. This changes what proteins the cancer cell makes, which has a big impact on how it behaves. In some cancers, this is caused by there being too much of a protein called MYCN. Not only can this protein turn instructions on or off, but it can also change the way they are followed.
In this project, Professor Malik wants to find out how MYCN alters the translation of these instructions into proteins. He is focussing on a specific way that MYCN does this, which has not been studied before. Professor Malik believes that this work assessing how MYCN alters how genetic instructions are read and followed, will lead to new ways to diagnose and treat children with many of the different cancers that involve MYCN.
Project title: The tRNA epitranscriptome: the missing link in MYCN-driven tumours
Lead investigator: Prof Karim Malik, University of Bristol
Funded by: The Little Princess Trust
Funded: July 2023
Award: £49,916.00
Protecting young hearts from chemotherapy-related heart problems
With Dr Roisin Kelly-Laubscher
Some types of chemotherapy, like doxorubicin, can cause damage to the patient’s heart – but doctors often don’t have a choice because they need to fight the cancer. However, if they could give another medicine at the same time which would protect the heart, it could help prevent long-term health problems for children.
Dr Roisin Kelly-Laubscher at University College Cork plans to find out whether a medicine, called ethanolamine, could protect the heart if given before doxorubicin treatment. Her lab knows that this drug can decrease damage caused by doxorubicin one type of heart cell in the lab, called fibroblasts. However, there is another type of cell, called cardiomyocytes which are more important to protect. In this project, Dr Kelly-Laubscher will see how the medicine affects these heart cells, then find out what dose and timings would work best. She hopes that this project will help take this treatment one step closer to clinical trials, where it can help real patients.
Project title: Protecting young hearts from drug induced cardiotoxicity
Lead investigator: Dr Roisin Kelly-Laubscher, University College Cork
Funded by: The Little Princess Trust
Funded: July 2023
Award: £52,097
Investigating nanomedicines to make treatment safer for children with cancer
With Dr Marie-Christine Jones
Medicines which treat cancer can have a lot of side effects and even cause long-term health problems because they also damage healthy cells. We really need new and safer treatments. One way to do this could be with nanomedicines, which use tiny particles to deliver the anticancer medicines. These can be safer because they can slip through the gaps in tumour blood vessels, which are especially leaky – these tiny medicines struggle to get through healthy blood vessels to attack normal cells.
Dr Marie-Christine Jones at the University of Birmingham wants to look at the use of nanomedicines in children, as they are currently mostly used for adults. This project aims to understand how nanomedicines work for children, learn more about leaky blood vessels, and test nanomedicines in leaky blood vessel models to find out which ones would work best.
Project title: Nanomedicine stratification to decrease the toxicity of anticancer treatment in children.
Lead investigator: Dr Marie-Christine Jones, University of Birmingham
Funded by: The Little Princess Trust
Funded: July 2023
Award: £49,313.43
How does MYCN change the way genetic information is read in childhood cancer?
With Professor Karim Malik
Cancer cells grow out of control due to changes in their genetic instructions. These instructions tell a cell how to behave, so when there are changes it can cause serious issues that lead the cell to become cancerous. Sometimes parts of the instructions, called genes, are incorrectly switched on or off. This changes what proteins the cancer cell makes, which has a big impact on how it behaves. In some cancers, this is caused by there being too much of a protein called MYCN. Not only can this protein turn instructions on or off, but it can also change the way they are followed.
In this project, Professor Malik wants to find out how MYCN alters the translation of these instructions into proteins. He is focussing on a specific way that MYCN does this, which has not been studied before. Professor Malik believes that this work assessing how MYCN alters how genetic instructions are read and followed, will lead to new ways to diagnose and treat children with many of the different cancers that involve MYCN.
Project title: The tRNA epitranscriptome: the missing link in MYCN-driven tumours
Lead investigator: Prof Karim Malik, University of Bristol
Funded by: The Little Princess Trust
Funded: July 2023
Award: £49,916
Making an ‘off-the-shelf’ CAR-T cell treatment for children with solid cancers
With Professor John Anderson
CAR-T treatments use patients’ own immune cells to fight cancer. Doctors take the cells from a patient’s immune system and modify them so they can recognise and kill cancer cells. Early results show that CAR-T cells could be very effective in multiple childhood cancers.
However, the treatment has to be freshly produce for every patient and is difficult to make - this leads to treatment delays and increased costs.
In this research project, Professor John Anderson at UCL Great Ormond Street Institute of Child Health hopes to create a universal CAR-T cell treatment that can be made in bulk. This means the treatment could be stored, ready for use whenever needed.
The researchers will use a different type of T-cell to standard CAR-T cells, and a new technique to modify them. If Prof Anderson’s method is successful, it will allow CAR-T treatment to become more widely available to children with cancer.
Project title: Development of an allogeneic V-delta-1 gamma delta T chimeric antigen receptor cell product for childhood solid cancers
Lead investigator: Professor John Anderson, UCL Great Ormond Street Institute of Child Health
Funded by: The Little Princess Trust
Funded: July 2024
Award: £89,720.95
Understanding how childhood cancers spread
With Dr Madhumita Dandapani
Cancer cells grow faster than normal cells and often invade the tissue around the tumour, like muscles and blood vessels. Normal cells can’t do this, partly because of the extracellular matrix (ECM). The ECM is like a scaffold that holds cells in their proper place. However, cancer cells have found ways of altering or breaking the scaffold in order to escape.
In this project, Dr Madhumita Dandapani and her team at the University of Nottingham will look at the building blocks of the ECM scaffold.
This project will look at the scaffold’s composition in different types of healthy tissues (such as the liver, kidneys and brain), and explore how this is different in cancer. Understanding how the scaffold breaks could show how cancer spreads and highlight ways to prevent this.
Project title: Understanding how childhood cancers spread
Lead investigator: Dr Madhumita Dandapani, University of Nottingham
Funded by: The Little Princess Trust
Funded: July 2024
Award: £49,565.07
Understanding how a new drug called can selectively fight childhood cancer cells
with Dr Igor Vivanco
Cancer cells often have different amounts of certain proteins compared to healthy cells. This can help support their fast growth and survival. One of these proteins is something called a ‘growth factor’. When this protein sticks to its receptor in the cancer cell, it helps the cell grow more.
Dr Igor Vivanco’s team at King’s College London has found a medicine that can kill cancer cells with unusually high levels of a specific growth factor. The medicine causes the growth factor’s receptor to be relocated inside cancer cells.
When the receptor is moved from its normal location, it becomes deadly to cancer cells. In this project, Dr Vivanco will test this medicine as a new potential treatment and uncover how it works. Excitingly, evidence suggests it is effective at killing cancer cells across every type of cancer tested. This means it could be a useful treatment for multiple childhood cancers.
Project title: Evaluating the therapeutic potential of pharmacologically-induced oncoprotein mislocalisation
Lead investigator: Dr Igor Vivanco, King’s College London
Funded by: The Little Princess Trust
Funded: July 2024
Award: £74,458.32
