Can targeting RNA help treat children with neuroblastoma? 

Neuroblastoma is a childhood cancer that mainly affects babies and young children. Some children are cured with current treatments, but for others, especially when the cancer comes back or does not respond, outcomes are still very poor. This project will support the Nijhuis group at Imperial College London to explore a new way to treat neuroblastoma by focusing on RNA, the molecule that carries genetic instructions inside cells. Laboratory studies show that drugs which disrupts the way cells process RNA molecules to become proteins, the functional building blocks of cells, can kill neuroblastoma cells very effectively.

However, we still need to understand how these drugs work broadly, and how they might can make the cancer more visible to the immune system. If we can achieve this, we may be able to help the immune system recognise and attack neuroblastoma and lay the groundwork for future vaccines and immune-based treatments designed specifically for children. 

This project is exciting because it focuses on a completely new way to fight neuroblastoma, a cancer that has been very hard to treat with existing therapies. We seek to make therapy kinder and more effective for children with this highly lethal cancer who would otherwise have very few treatment options left. 

The potential benefits for children with neuroblastoma are: 

1) New treatments: drugs that directly kill cancer cells and activate the immune system could replace harsh chemotherapy, reducing side effects. 

2) Personalised vaccines: if we find cancer-specific “flags” created by these drugs, we could design vaccines to teach the body to attack tumours, possibly preventing relapse. 

3) Hope for high-risk cases: children with MYCN neuroblastoma, who currently face the worst outcomes, could gain access to therapies tailored to their cancer’s biology.