Why study Brain tumour Biology??? Richard Grundy

Overview Paediatric oncology is one of the medical success stories of the 80’s-90’s Steady improvement on the overall survival of children with cancer BUT not CNS tumours – Only 50% survive 10,000 life years lost and considerable morbidity Plateau and Stasis : More recently small advances only are being made and for some tumours little progress at all Fight Fire with Fire

Children’s Brain Tumours : The Issues Brain tumours are relatively rare

Children’s Brain Tumours : The Issues Poor understanding of disease biology Brain tumours are relatively rare

Children’s Brain Tumours : The Issues Poor understanding of disease biology No accurate disease stratification Broadly based treatment strategies Brain tumours are relatively rare

Children’s Brain Tumours : The Issues Poor understanding of disease biology High Mortality/ Morbidity No accurate disease stratification Broadly based /aggressive treatment strategies Brain tumours are relatively rare

Children’s Brain Tumours : The Issues Low Priority for Molecular research Poor understanding of disease biology High Mortality/ Morbidity No accurate disease stratification Broadly based treatment strategies Brain tumours are relatively rare

Children’s Brain Tumours : The Issues Lack of molecular targets for new treatment approaches Low Priority for Molecular research Poor understanding of disease biology High Mortality/ Morbidity No accurate disease stratification Broadly based treatment strategies Brain tumours are relatively rare

Children’s Brain Tumours : The Issues Lack of molecular targets for new treatment approaches Low Priority for Molecular research Poor understanding of disease biology High Mortality/ Morbidity No accurate disease stratification Broadly based treatment strategies Brain tumours are relatively rare

Key Hypotheses Tumours arising in different anatomical locations are genetically distinct entities. Tumours arising indifferent age groups have distinct genetic aberrations. Tumours of different histological grade have distinct genetic fingerprints. Genetic markers can predict outcome in childhood CNS tumours. Tumourigenesis arises from disordered neural development and involves distinct tumour specific pathways. Development of large data sets linked to clinical parameters will enable the discovery of novel targets for therapy.

Biological studies Using Biological studies to better understand or find : ‘Markers’ that help us predict outcome or best treatment pathways involved in the development of brain tumours Where Brain tumours come from New Drug Treatments

Children’s Brain Tumour Research Centre Improved Patient Management & Outcome High resolution Molecular analysis of tumour samples Identification of molecular ‘fingerprint’ International & National Clinical Trials & Biological studies Understanding ‘Why’ in the context of the Normal development of the Brain Children’s Brain Tumour Research Centre Improved Diagnostic & Prognostic Imaging Fingerprint’ provides Novel prognostic Markers & targets for therapy Why Does Treatment Fail ? Need New Drug Development and Delivery