Paediatric Cancer Research

Developing a targeted therapy for paediatric sarcoma

Cancer remains the leading cause of death by disease in children and young adults. A major reason is the intrinsic ability of some paediatric cancers to become resistant and relapse, even after an initially successful response. Once relapse occurs, therapeutic options are scarce, and survival rates remain dismal.

But Dr Darrell Green at UEA thinks he can change that:

Unlike adult cancers, which often develop after years of DNA damage from smoking, diet, or ageing, childhood cancers are different. They usually carry few genetic mutations. Instead, they arise from developing cells that are naturally flexible - cells that “decide” what they will become as a child grows. In cancer, this flexibility becomes dangerous. Tumour cells can change their behaviour, become aggressive, spread to the lungs, or resist treatment, without obvious changes in their DNA. They are not simply “out of control” like adult cancer. They are making harmful decisions. We’re working to better understand this decision-making and develop new medicines that block it. Children deserve treatments designed specifically for them, not scaled-down versions of adult cancer therapy.

Around 1 in 5 childhood cancers are “sarcomas”, which are cancers of the bones, muscles and other soft tissues. Of these, around 80% will experience disease that has spread around the body and become resistant to chemotherapy. Long-term survival stands at just ~50%; that means half of children and young adults diagnosed will not live to see adulthood. However, Dr. Green and his team have developed a novel medicine that could stop the spread and the aggressiveness of sarcoma, without the side effects commonly experienced with chemotherapy.

UEA-522 (Bensofib) is ready to be tested

Preclinical studies have shown that UEA-522 is effective (and without side effects) in stopping the spread of sarcomas, but the next milestone is to prove this in clinical trials with patients. To do that, the team are looking for funding to produce the medicine on a mass scale and to carry out vital toxicology studies, to discover the safest and most effective dose of UEA-522. Sarcoma research is critically underfunded when compared to other types of cancer and receives almost no investment from traditional avenues of funding, so UEA is hoping to raise the money needed through the generosity of philanthropists.

The work that we have carried out so far was widely reported to be the biggest breakthrough in sarcoma treatment in 50 years (since chemotherapy was introduced) and it was only possible thanks to philanthropy. We hope that UEA’s generous supporters can help us reach our next goal and make a real difference to the lives of those children and young adults affected by sarcoma.

How you can help

Please donate now to UEA’s Paediatric Cancer Research. Your support could help fund the toxicology and clinical trials needed to test UEA-522 (also known as Bensofib in our project), or other paediatric sarcoma studies, and make this treatment available to start saving lives as soon as possible.

To find out more or to make a larger gift, please get in touch with the Development, Alumni and Campaigns Office by email at giving@uea.ac.uk or by telephone on +44(0)7990 438106.