Understanding the biology of malignant rhabdoid tumours to develop targeted treatments

Malignant rhabdoid tumours (MRT) are aggressive, frequently lethal tumours of early childhood and infancy. They can occur in any part of the body, although are most frequently found in the kidney and brain. Current treatments using surgery, chemotherapy and radiotherapy are often ineffective and frequently cause harmful long-term effects. New therapies are desperately needed.

This project aims to characterise in the biology of MRT in very precise detail. MRT are caused by a mutation in a single gene. If we replace the faulty gene in MRT cells grown in a laboratory with a working copy of the gene, the tumour cells stop growing and effectively become benign. This points the way to possible future biological therapies to combat this disease. By understanding the biological changes that occur in these cells, we are ultimately aiming to recreate the effect we see in the laboratory in patients, using novel combinations of drugs.

State of the art 'mass spectrometry' techniques will be used to measure thousands of small changes in the amount of proteins in MRT cells with the mutated gene. Using sophisticated statistical analysis, we will be able to work out which drugs may be most effective in these tumours, and identify new targets for novel immunotherapies - treatments which use the patient's own immune system to fight the disease.