Written by Dr Iman Azimi, BDI Cancer Program

On 18^th February 2025, Associate Professor Raelene Endersby, Co-Head of the Brain Tumour Research Group at The Kids Research Institute Australia, presented a seminar titled "Reinventing Preclinical Approaches to Better Model Treatment Responses for Paediatric Brain Cancers" as part of the BDI Cancer Program / MPCCC Seminar Series at Monash University.

A/Prof Endersby focused on medulloblastoma, the most common malignant paediatric brain tumour. Despite intensive treatment with surgery, radiotherapy, and chemotherapy, the five-year survival rate for aggressive medulloblastoma subgroups remains below 50%. Survivors often experience severe and lifelong cognitive and physical impairments, highlighting the need for more effective and less toxic therapies. A/Prof Endersby’s research aims to identify and develop improved therapies that enhance survival while minimising long-term side effects for children with this disease.

A central focus of the seminar was the potential of ATR inhibitors to enhance cancer cell death in medulloblastoma by targeting the DNA damage response pathway. Preclinical studies demonstrated that combining these inhibitors with craniospinal irradiation significantly prolonged survival in high-risk medulloblastoma mouse models, highlighting their promise as a therapeutic strategy.

The seminar highlighted A/Prof Endersby’s leadership in developing innovative, clinically relevant laboratory models that closely replicate human paediatric medulloblastoma. These models have been instrumental examining the effects of radiation therapy in a preclinical setting. The research revealed that standard treatments deplete adaptive immune cells, emphasising the potential of immunotherapy as a complementary strategy. Notably, preclinical studies demonstrated that combining immunotherapy with craniospinal irradiation significantly improved tumour clearance and extended long-term survival, supporting its potential as a first-line treatment approach.

By working closely with clinicians, A/Prof Endersby and her team at The Kids Research Institute Australia are generating strong preclinical evidence to inform clinical trials, ultimately aiming to improve outcomes for children with medulloblastoma.

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