Our Funded Research

Sarcoma Research Projects

What is important to rhabdomyosarcoma cells?

With Dr Yinyin Yuan

Rhabdomyosarcoma is a common type of childhood cancer. Treatment often goes well, but there are serious side effects, and there are types of rhabdomyosarcoma that don’t respond to treatment. This shows that we need new and more targeted treatments.

Dr Yinyin Yuan at the Institute of Cancer Research is looking for medicines that stop a protein from working. This protein, called PRMT5, has been shown to be important to another type of cancer’s growth and survival and the researchers think it that rhabdomyosarcoma cells also need PRMT5 to survive.  They will be assessing various medicines to find the best treatment against PRMT5 for rhabdomyosarcoma.


Project title: Deep learning: An integrated approach to define clinical significance to components of the tumour microenvironment of rhabdomyosarcomas
Lead investigator:  Dr Yinyin Yuan, The Institute of Cancer Research
Funded by: The Little Princess Trust
Funded: January 2019
Award: £98,292.50

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Repurposing existing medicines to treat Ewing sarcoma

With Dr Susan Burchill

Ewing sarcoma happens mostly in bones and is the second most common type of sarcoma. If the disease has spread to more than one location it can be difficult to treat, and the treatment can have serious side effects. Part of the reason for this is that current treatments aren’t able to fight the cells that are responsible for Ewing sarcoma not responding to treatment or coming back after treatment. These cells are called Ewing’s sarcoma cancer stem-like cells.

Dr Susan Burchill at the University of Leeds is studying these stem-like cells. Her research team wants to see whether their understanding of these cells could help diagnose and treat Ewing sarcoma. They will look at existing medications to see whether they can fight the stem-like cells, or whether a combination of them would work. The team will then see which medicines are the most unlikely for Ewing sarcoma to be resistant to.


Project title:  Repurposing of drugs targeting drug resistant self-renewing Ewing’s sarcoma cells to accelerate new treatments into clinical trials to improve outcomes.
Lead investigator:  Dr Susan Burchill, University of Leeds
Funded by: The Little Princess Trust
Funded: January 2019
Award: £111,246.07

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Improving treatment Ewing sarcoma that has spread to other parts of the body

With Dr Paul Huang

Ewing sarcoma affects the bones and soft tissue, like muscles and fat, in people under the age of 20. When the cancer has spread to other parts of the body, it is a lot harder to treat and there may not be any treatment options.

Dr Paul Huang at the Institute of Cancer Research wants to find a way to identify children who are more likely to have problems with treatment and would benefit from new medicines. In particular, he wants to find children who could be treated with medicines that stop blood vessels feeding the tumour. His team have found ways to identify children with other types of cancer that would benefit from the medicine and this project will investigate whether this can also be applied to Ewing sarcoma.


Project title:  Optimising tyrosine kinase inhibitor therapy in newly diagnosed metastatic Ewing sarcoma
Lead investigator: Dr Paul Huang, Institute of Cancer Research
Funded by: The Little Princess Trust
Funded: December 2020
Award: £199,372

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Re-purposing diabetes medication to treat Ewing sarcoma

With Dr Robin Rumney & Professor Dariusz Gorecki

Ewing sarcoma is one of the most common bone cancers in young people. When patients only have one Ewing sarcoma tumour there are effective treatment options, but if the cancer has spread it can be hard to treat.

Dr Robin Rumney and Professor Dariusz Gorecki want to investigate whether a molecule linked to Ewing sarcoma spreading could be stopped using a diabetes medicine. The molecule also causes problems for people with diabetes, so there are existing medicines that stop the molecule from working. The researchers will look at how the existing medicines work and try to learn more about the molecule and its effect on Ewing sarcoma growth.


Project title: Investigating repurposed drugs to decrease the progression of Ewing’s sarcoma
Lead investigator: Dr Robin Rumney & Prof Dariusz Gorecki, University of Portsmouth
Funded by: The Little Princess Trust 
Funded: December 2020
Award: £100,901

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Getting medicines for Ewing sarcoma into clinical trials

With Dr Susan Burchill

Around a quarter of Ewing sarcoma patients have tumours in multiple different places in their body at diagnosis. This shows how aggressive the cancer is, and many children relapse and have their cancer spread further after treatment. We need new treatment options so that more children can be successfully treated.

Dr Susan Burchill at the University of Leeds have identified a number of medicines that could treat Ewing sarcoma. With her team, she will investigate which medicines affect specific cancer cell activity and will test combinations of these medicines to find the best combinations that could be used in clinical trials. They hope to fast-track a targeted combination treatment that causes fewer side effects and helps more children.


Project title: Integrating multiple data to validate and prioritise lead-hit therapeutic combinations for acceleration into clinical trials to improve outcomes for patients with Ewing sarcoma
Lead investigator: Dr Susan Burchill, University of Leeds
Funded by: The Little Princess Trust
Funded: July 2021
Award: £198,979.45

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Blood tests to monitor rhabdomyosarcoma treatment

With Professor Janet Shipley

Most rhabdomyosarcoma patients can be successfully treated, but there are many children whose disease remains or returns after treatment. This can be difficult to monitor, but work in other cancers and in small studies for rhabdomyosarcoma have shown that blood tests looking for specific pieces of cancer DNA, called markers, can show how a child’s cancer is responding to treatment.

Professor Janet Shipley and her team at the Institute of Cancer Research will use blood samples already collected as part of an existing clinical trial. The team will measure the amounts and types of DNA pieces in patients’ blood samples as they go through treatment as part of the trial. They will use these measurements to see if the blood tests can show when treatments aren’t working, or when a patient has relapsed. Professor Janet Shipley hopes that this research could help reduce healthy tissue damage when treatments aren’t also killing the cancer cells and create options for earlier interventions with more effective and personalised treatments.


Project title: Prospective Assessment of Nucleic Acids in Liquid Biopsies from Frontline International Rhabdomyosarcoma Trial Patients to Determine Clinical Utility
Lead investigator: Professor Janet Shipley, The Institute of Cancer Research
Funded by: The Little Princess Trust
Funded: July 2022
Award: £210,885.00

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Discovering what makes teenagers and young adults with soft tissue sarcomas different

With Dr Paul Huang

Soft tissue sarcomas are a rare type of cancer that are more common in teenagers and young adults, with around 100 patients diagnosed every year in the UK. Young people are normally given adult cancer treatments, or sometimes treatments for children, which means that they might not be having the right treatment for their cancer. We need to learn more about teenagers and young adults with soft tissue sarcomas to find out what makes them different and how best to treat them.

In this project, Dr Paul Huang and his team at the Institute of Cancer Research will be looking at what is biologically different between young people’s tumours and adult tumours. The team will then look for new or existing treatments that would work well for these young patients. This will provide valuable new knowledge about soft tissue sarcomas in teenagers and young adults that will support the development of new treatments and help doctors choose the best treatments for their patients.


Project title: Accelerating TYA precision medicine in soft tissue sarcomas with integrative mass spectrometry based proteomics
Lead investigator: Dr Paul Huang, Institute of Cancer Research
Funded by: The Little Princess Trust
Funded: March 2023
Award: £249,942

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The MBE for voluntary groups was awarded to The Little Princess Trust by Her Majesty Queen Elizabeth.