Molecular approaches to ependymoma: the next step(s)

Effective treatment options for ependymoma apart from radical surgery and radiotherapy remain scarce, and the understanding of the molecular basis of ependymoma biology is crucial to the development of novel therapies. Comprehensive work revealing molecular pathomechanisms of ependymoma has been done; however, the elucidation of the processes underlying the origins of various clearly distinguishable ependymoma subgroups has proved to be difficult. The future challenges will be to reach consensus about molecular subgroups, to translate these into a clinical setting, and to use available models for drug screening and preclinical testing.

Ependymoma subgroups with clearly distinct biology have been delineated and novel mouse models generated, and the first high-throughput drug screens were successfully conducted leading to the identification of subgroup-specific active regimens.

Coordinated efforts to advance novel therapies into the clinic have led to breakthrough insights into the molecular biology of ependymoma. The next step will be the translation of preclinical findings into the clinical setting, and international study groups are starting to implement the most recent advances into clinical trials.