Developing novel drugs for medulloblastoma

My research focuses on the development of novel drugs for the treatment of childhood brain tumours. I currently work on medulloblastoma, the most common malignant paediatric brain tumour and a major cause of mortality in children. The need to reduce the risk of metastasis has driven the clinical development of aggressive treatment regimens that leave survivors with severe side effects. We all can empathise with the physical and emotional pain that children and families are going through when affected by a brain tumour.

MB survivors will suffer of different long-term side effects such as walking problems or changes in personality; weakness, vision problems or speech problems which will affect the development. Hence, more targeted, and less toxic therapies are vitally needed to improve the quality of life for patients. I am working to understand the chemical reactions that allow tumours to grow, trying to ascertain how the metabolism of medulloblastoma is regulated, and whether and how these tumours could be starved of essential components in order to strengthen the effect of chemotherapy or radiotherapy given to patients.

This project will allow me to investigate if lipid inhibitors, already use to treat other diseases, can be repurposed for the treatment of aggressive medulloblastoma. We all hope for the development of new and more effective treatments that are accessible to all. Rare diseases are often underfunded, so the work of the few research groups that dedicate themselves to these tumours becomes essential for finding new treatments and bringing new hope to the thousands of young patients diagnosed with a brain tumour each year, as well as their families.