Lab-Grown Tumors Spark Hope for Kids' Brain Cancer

Monash University

Inside a laboratory at Monash University, miniature replicas of the human brain are revealing secrets that could forever change how we treat childhood brain cancer. These tiny "organoids" – no bigger than a pea – represent a giant leap forward in the fight against medulloblastoma, the most common malignant brain tumour affecting children.

Children's Cancer CoLab has awarded $455,900 as part of its Next-Generation Therapies Impact Program to this groundbreaking research led by Dr Iman Azimi, recognising its potential to dramatically improve survival rates while sparing young patients from the devastating side effects of current treatments.

When current treatments hurt as much as they help

For children diagnosed with medulloblastoma, which accounts for 20% of all childhood brain cancers, the battle is twofold. First, they must fight the cancer itself. Then, they face a lifetime of managing the aftermath of harsh treatments.

Today's standard approaches – surgery, radiation, and chemotherapy – can leave survivors with severe physical and cognitive impairments that affect them for life. For children with the most aggressive forms of the disease, the odds are even more challenging, with 5-year survival rates below 50%.

Lead researcher, Dr Iman Azimi, Head of the Cancer Drug Discovery and Cellular Ageing Lab at Monash University in Melbourne, said medulloblastoma was one of the hardest-to-treat forms of childhood cancer.

"We urgently need treatments that target cancer cells more precisely while leaving healthy brain tissue unharmed. Our young patients deserve both the chance to survive and to thrive after treatment," said Dr Azimi.

Growing hope in the lab

Dr Azimi's research team's approach is as innovative as it is promising. They're developing sophisticated lab-grown models of medulloblastoma that precisely mimic the biology of real tumours, particularly focusing on Group 3 – the most aggressive and treatment-resistant type.

These models are created using induced pluripotent stem cells, allowing researchers to test many potential treatments quickly and accurately. What makes this work especially exciting is that the team intends to screen over 900 existing medications that can already cross the blood-brain barrier, potentially accelerating discoveries into clinical use.

"We're essentially building miniature versions of a child's brain tumour in the lab. This gives us an unprecedented window into how these cancers grow and respond to different treatments, without subjecting children to experimental therapies until we know they're likely to work," Dr Azimi added.

A future where every child with cancer survives and thrives

CEO of Children's Cancer CoLab, Dr Udani Reets, said this research offered hope for families facing a medulloblastoma diagnosis.

"We envision a future where no child dies from medulloblastoma, and where survivors can lead full, healthy lives without the burden of treatment-related disabilities. These lab-grown tumour models are bringing that future closer every day," Dr Reets said.

Children's Cancer CoLab's investment in this project represents a critical step toward personalised, less toxic therapies for childhood brain cancer – and potentially, a new chapter in how we understand and treat these devastating diseases.

In addition, a Monash University researcher working on the project, Dr Kaveh Baghaei, will receive a Future Leaders Fellowship. These fellowships aim to strengthen Australia's childhood cancer workforce to drive discoveries and clinical advancements.

Applying scientific rigour to drive impact and guide support

This project was thoroughly assessed by the Children's Cancer CoLab's Discovery and Translational Research Scientific Advisory Committee.

Children's Cancer CoLab Impact Program grants are supported by the Victorian Government's investment of $35 million and the Children's Cancer Foundation's commitment of $10 million to the Children's Cancer CoLab.

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