Next generation molecular profiling of malignant rhabdoid tumours

Malignant rhabdoid tumour is a very aggressive form of childhood cancer which typically occurs in the kidneys, but can also occur more rarely in other soft tissues or the brain. Current treatment for rhabdoid tumours cures only around a quarter of affected children and often results in life-changing side-effects in survivors. Little is understood as to why treatment is effective in some children but not in others, or why the effect of initially beneficial treatment quickly wears off. There is an urgent need to discover new treatments with greater effectiveness as well as design molecular tests that can identify which children will benefit from existing drugs. Dr Huang’s research aims to identify which are the cellular molecules that drive growth and spread of rhabdoid tumours, and to use this information to nominate new treatment strategies and develop real-time diagnostic tests for personalising treatment in patients.

Dr Huang’s study will use tissue samples obtained from patients with rhabdoid tumour as well as from a group of closely related paediatric and adolescent cancers (which share a key genetic mutation, SMARCB1). By conducting comprehensive molecular profiling of these tumour samples, the team will generate a greater understanding of the ‘protein-specific molecular drivers’ which are specific to this tumour type. This information will allow the team to identify which drug treatment options are likely to work for this form of cancer, and develop new ways to predict patients’ responses to therapy. Dr Huang’s team anticipate that their research will provide a more personalised approach to treating children with rhabdoid tumours, by providing a new way to estimate the sensitivity of each individual patient to the available drug treatments ahead of starting their treatment. Going forward, the team hope that their research will provide the basis for new anticancer drug trials in rhabdoid tumours (and other SMARCB1 mutant cancers) that aim to establish effective and safe treatments for children with these devastating diseases.