Embryonal tumours constitute the largest group of malignant paediatric brain tumours. Their origin and histological classification remain somewhat controversial. However, in recent years real progress has been made in our understanding of the molecular genetic abnormalities that govern the initiation and/or progression of these tumours. A number of these abnormalities appear to involve alterations in signalling systems that control normal cerebellar development. Increasing our understanding of both the biology and clinical relevance of these molecular defects is a major challenge to the field of paediatric neuro-oncology. However, it likely represents the only means by which we will advance the management of these tumours, significantly reducing disease-related morbidity and mortality. This review focuses on the principal molecular genetic abnormalities so far identified in embryonal brain tumours and discusses their biological and clinical relevance.