Why developing treatments into rare childhood cancers is a team effort

Sarcoma Molecular Pathology Team, Antonio Romo-Morales is pictured top-right.

You might not have heard of the childhood cancer Ewing sarcoma. Fewer than 30 children in the UK are diagnosed with it each year. It’s also highly aggressive, which means it can spread quickly to other parts of the body.

By studying the condition as part of my PhD, I’ve realised that the rarity and low profile of Ewing sarcoma, and other uncommon cancers, are often barriers to overcoming them. But families, touched by tragedy and inspired to make a difference for others, are helping us turn the tables.

Striking the right balance

In recent years there have been considerable developments in the diagnosis and classification of Ewing sarcoma, and in our understanding of the mechanisms driving this disease. However, these developments have not translated into significant changes in how patients are treated.

Unfortunately, this story is very similar across various other types of childhood cancers. For these reasons, the main objective of my PhD project is to find a realistic combination therapy that is ready to be introduced for this paediatric cancer as soon as possible.

A key challenge in finding this therapy is striking the right balance between eliminating the maximum number of cancer cells, while causing the minimum possible damage. This is particularly problematic in children since non-specific and toxic chemotherapy drugs can result in long-term side effects that seriously impact the child’s development. Drugs that were originally designed for adults are often repurposed for children and whilst some aspects of the disease may be similar, others differ greatly.

Whilst lifestyle and environmental stresses have been identified as factors increasing the risk of adults developing cancer, we know far less about the causes of most paediatric cancers. Over the years, cases of childhood cancer have been reported to increase. One reason explaining the rise in numbers may be the development of more precise diagnoses. The challenge remains to develop specific and improved therapies with children in mind, as well as investigating the causes of these less common cancers.

It is in this context where the efforts of my lab fit in, in which we seek to develop more effective and kinder therapeutic strategies to improve the outcomes for children with cancer.

The ICR's Sacroma appeal helps to support our ongoing work to improve treatments for children and young people with sarcoma, an aggressive form of cancer.

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An exciting time for my research

Currently, I’m exploring the role of a specific protein that drives the progression of Ewing sarcoma. This protein is one of many that controls how DNA is compacted and organised, which can influence whether a gene is switched on or off. In this way, the protein can stop the tumour suppressor genes from doing their job to prevent a cancerous growth, making it a crucial player in this type of sarcoma. 

It is now possible to target this protein with drugs and so I have started to assess how effective these drugs are at treating Ewing sarcoma cells in the laboratory. The aim is to introduce this strategy as a combination therapy along with the current standard care of chemotherapy and radiotherapy.

Given that a new treatment is most likely to be part of a combination therapy, I have looked into inhibiting our proposed target in conjunction with current treatments. It’s an exciting time for my research but I know it wouldn’t be possible without some very special support.

Making a real difference for children

Working in the Sarcoma Molecular Pathology Team in the Divisions of Molecular Pathology and Cancer Therapeutics at the ICR, has taught me a lot about how childhood cancer research is supported and the challenges that this field faces.

Research on cancers, such as Ewing sarcoma and rhabdomyosarcoma, is often funded through smaller charities set up by the families of children that have been affected by these rare types of cancer. 

We work in close collaboration with them, often having families visit our labs where we can share our findings with them. This has been a great opportunity to share our enthusiasm and improve how we communicate science as well as really appreciate, at a personal level, the importance of our research. 

Combining the ultimate objective of helping patients and making new discoveries that increase our understanding of cancer has been a huge motivation to me. I’m excited to continue learning from my team at the ICR and to have the opportunity to share my findings more widely.

Knowing that my PhD project is funded by a family touched by Ewing sarcoma really drives and motivates my research. I am confident that through our collaborative efforts, we will help overcome the barriers in childhood cancer research so we can make a real difference for children.