New clinical trial targets aggressive brain tumours in children and young adults

BRUSSELS - The first patient has been enrolled in the U-R-Immune Glioma trial, a clinical study testing a new immune therapy for children and young adults with high-grade glioma, a rare and aggressive brain cancer. This global trial, co-funded by the Anticancer Fund and the Canadian Institutes of Health Research, aims to explore additional treatment options for patients facing a disease with limited available therapies.
About this trial
U-R-Immune Glioma is an early-stage study investigating the use of nivolumab, an immune therapy, in patients aged 1 to 25 years with high-grade glioma that have specific DNA mutations. This approach involves administering the treatment before surgery and radiation therapy, with the goal of assessing whether it can delay or avoid the need for radiation therapy, which often leads to severe long-term side effects.
Immunotherapy uses the body’s own immune system to fight cancer by teaching the immune system to target cancer cells using immune checkpoint inhibitors (ICI).
Researchers will also study how the tumour cells react to the treatment, contributing to a better understanding of this rare and aggressive disease.
Up to 20 participants will take part in the trial at more than 10 hospitals across Canada, Australia, Jordan, and India.
A collaborative initiative
Led by Prof. Dr. Uri Tabori at The Hospital for Sick Children (SickKids) in Toronto, Canada, and Dr. Nirav Thacker from The Childrens Hospital of Eastern Ontario (CHEO), a paediatric health-care and research centre in Ottawa, Canada, the trial is part of the International Replication Repair Deficiency Consortium (IRRDC), a research group committed to understanding and addressing rare genetic diseases linked to damaged DNA.
The need for new approaches
High-grade glioma is the deadliest cancer in children, adolescents, and young adults (CAYA). Even with aggressive treatment—consisting of surgery, chemotherapy, and radiation—most patients survive only 9 to 15 months. Additionally, chemotherapy and radiation often lead to severe long-term effects that can affect quality of life. These include stroke, brain damage, hormonal issues and increased risks of additional cancers.
Recent research has identified that about 10% of high-grade gliomas in this age group are caused by defects in DNA repair mechanisms, leading to a high number of mutations within the tumour cells. These genetic mutations may make the tumours more responsive to immune therapies like nivolumab.
These findings inspired the investigators to test the use of immunotherapy without chemotherapy and radiotherapy for patients with highly mutated and immunotherapy-receptive glioblastomas.
“The possibility of being able to effectively treat glioblastomas without the use of chemotherapy and radiotherapy could revolutionize the way we treat these high-grade gliomas while having an incredible impact on the quality of life for these kids. The trial is the result of over 15 years of effort.The vision of this trial is to have impact not only locally, but globally,” says Dr. Nirav Thacker.
“Today, every patient with glioblastoma receives radiation and chemotherapy. This clinical trial will be the first time ever that patients – adults or children – receive a radiation and chemotherapy sparing approach to cancer treatment,” says Dr. Uri Tabori.
Why this trial matters
The Anticancer Fund chose to support this trial because it tackles gaps in the treatment of childhood cancers. Here are the key reasons why funding this trial is important.
- High unmet need: High-grade glioma is one of the most challenging cancers in young patients. Current therapies often result in severe long-term side effects. And, despite the aggressive treatments, the outcomes remain poor.
- Limited research funding: High-grade gliomas, classified as rare disease, receive limited attention from pharmaceutical companies and research institutions. By funding this trial, the Anticancer Fund is contributing to much-needed progress in this field.
- Global scope: The trial involves more than 10 hospitals and fosters international collaboration. This increases the potential impact of delivering research findings that could benefit patients worldwide.