Recent development in chemotherapy of paediatric brain tumours

Chemotherapy has gained a larger importance in the management of brain tumours, especially in children.

Converging results were presented in 2005 by the German, French and North-American cooperative groups indicating that a subgroup of young children with medulloblastoma (i.e. those with desmoplastic histology) could be cured with chemotherapy only strategies. The usefulness of high-dose chemotherapy followed by stem-cell transplant was shown not only as salvage strategy but also upfront in high-risk patients with medulloblastoma. Diffuse pontine glioma remains a devastating disease despite numerous attempts to improve on the standard radiotherapy. Targeted therapies have entered the paediatric neuro-oncology field as well.

In the most frequent paediatric brain tumors (medulloblastoma and low grade gliomas), the improvements have been impressive in recent years. These patients still await new targeted therapies to lower the burden of treatments and their related side-effects. Most of the brain tumours, however, are rare and the development of specific protocols too slow. Likely, they may have very specific biologic abnormalities that could be efficiently targeted in the near future.