Targeted Therapies in Paediatric Brain Tumours

Although improvements in survival have been observed in recent times, treatment outcomes for paediatric brain tumours still remain unacceptable; cure rates for certain tumour types remain dismal, while for others, success can be achieved but only with a significant impact to the survivor’s quality of life. It is likely that further manipulation of conventional multi-modality treatments will only confer more toxicity without improving prognosis. Biological research has tried to address this issue in recent years, generating numerous novel agents designed to target tumour-specific molecular pathways in the hope of improving antineoplastic effectiveness with minimal toxicity to the patient. This chapter reviews the current developments in such targeted therapies for common childhood brain tumours in the current era.