Emerging treatments

Silmitasertib

The US Food and Drug Administration (FDA) has granted rare paediatric disease designation and fast track designation for silmitasertib in the treatment of recurrent sonic hedgehog (SHH) medulloblastoma. Silmitasertib is a protein kinase inhibitor of casein kinase 2.[63] One US clinical trial to assess the safety and tolerability of this drug in patients with refractory medulloblastoma is ongoing.​

Personalised treatment

Studies are beginning to utilise tumour biological profile to stratify patients according to risk criteria.​[64][65][66][67] Novel targeted therapies to personalise treatment and reduce toxicity have been of great interest since the four major molecular subgroups of medulloblastoma were identified, one of which is characterised by activation of the SHH pathway. Pre-clinical data suggest that inhibitors of the SHH pathway could become valuable treatment options for patients with this subgroup of medulloblastoma.[68] Much more work is required before broad-based clinical applications can be made toward the treatment of medulloblastomas.[69][70]

Pencil beam scanning proton therapy

Pencil beam scanning proton therapy, a more advanced type of proton therapy, has demonstrated good tumour control and low toxicity rates among children/adolescents with brain tumours.[71][72]​​

Chemotherapy intensification and/or radiation enhancers

Trials for high-risk patients include dose intensification of chemotherapy and/or the use of radiation enhancers in an attempt to improve survival. As of yet, only concurrent carboplatin with craniospinal irradiation (CSI) for high-risk group 3 medulloblastoma has shown to be of significant clinical benefit.[60]

Immunotherapy

A number of clinical trials investigating the safety and efficacy of immunotherapy for recurrent medulloblastoma are ongoing, including oncolytic virus immunotherapy, immune checkpoint inhibitor and other immunomodulatory therapies, vaccine or RNA therapy, and chimeric antigen reception (CAR) T-cell and other cellular therapies.

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