First-of-its-kind pan-Canadian trial to treat baby brain cancers without radiation

Paediatric centres nationwide have united to launch the DECRYPT-BABYBRAIN trial, a first-of-its-kind clinical trial that uses chemotherapy delivered to the brain to avoid radiation in treating babies and young children with aggressive brain cancer. The trial has recruited its first six participants, with enrollment now open at The Hospital for Sick Children (SickKids) in Toronto, BC Children’s Hospital in Vancouver, Alberta Children’s Hospital in Calgary, CHU Sainte-Justine in Montreal, Children’s Hospital at London Health Sciences Centre in London, and additional sites to follow. 

Short for Defeating Embryonal Cancer in Young People Together, the DECRYPT-BABYBRAIN trial is co-led by study chairs Dr. Annie Huang (SickKids, Toronto), Dr. Sylvia Cheng (BC Children’s Hospital, Vancouver) and vice chair Dr. Lucie Lafay-Cousin (Alberta Children’s Hospital, Calgary). The trial builds on two decades of research and clinical experience at SickKids where patients with rare brain tumours were first treated with a novel standard of care protocol known as SKSOC.  

“This trial is a powerful step toward harnessing the collective strength of Canadian-led innovation in paediatric brain cancer science and clinical care,” says Dr. Annie Huang, study co-lead, Staff Oncologist, Principal Investigator at the Arthur and Sonia Labatt Brain Tumour Research Centre, and lead of the global Rare Brain Tumor Consortium based at SickKids. “Building on a protocol developed at SickKids, we’re aligning diagnosis and treatment across centres and creating a real-time national dialogue so we can treat these aggressive tumours more quickly with precision medicine approaches.” 

The trial is supported by a unique coalition of Canadian charities and funders, who partnered to create the DECRYPT Research Grant, designed to drive transformative research in childhood embryonal brain tumours. Coalition members include Kindred Foundation, Cancer Research Society, Phoebe Rose Rocks Foundation, Brain Tumour Foundation of Canada, The Miracle Marnie Foundation, Childhood Cancer Canada and Tali’s Fund.Additional support from the ACCESS network is helping to support the trial’s pan-Canadian reach. 

A new approach for rare brain cancer patients 

Embryonal brain tumours are the most aggressive central nervous system (CNS) tumours in children under six, often spreading rapidly to the brain and spine. For babies diagnosed with atypical teratoid rhabdoid tumours (ATRTs) and other rare forms of brain cancer, there is no universally accepted treatment. Children often receive varying combinations of highly intensive chemotherapy as well as radiation, yet survival rates remain low —)and survivors face lasting side effects, including cognitive challenges linked to radiation exposure. 

The DECRYPT-BABYBRAIN protocol combines chemotherapy delivered directly into brain and spinal fluid with low dose oral chemotherapy, aiming to reduce long-term side effects by omitting or delaying radiation while maintaining effective tumour control. Inspired by childhood leukemia treatment regimens, this approach supported survival in nearly 60 per cent of ATRT patients previously treated at SickKids, most of whom avoided radiation.  

“The trial will seek to confirm the safety and feasibility of using the SickKids SOC for every child diagnosed with embryonal brain cancer across the nation,” says Dr. Sylvia Cheng, Investigator at BC Children’s Hospital. “This goal is to create national harmonized treatments for young children with high-risk brain cancers that is tailored to a specific child's tumour with the aim to reduce late side effects of radiation if possible.” 

Innovation and collaboration at its core 

A key innovation is the dual use of cerebrospinal fluid (CSF), not only for more direct delivery route for chemotherapy but more importantly for real-time sampling of disease status and treatment response. 

“Using CSF is a game-changer for infant brain cancer care. Inspired by leukemia protocols, this approach could make CSF sampling as routine and informative as blood tests, enabling more responsive, personalized treatment and laying the foundation for future precision medicine trials,” says Huang. 

The trial includes centralized diagnostics and case review, with tumour samples and imaging from all sites sent to a central hub for careful second review. Each case is also discussed at a national tumour board of multidisciplinary experts, ensuring every patient benefits from the collective expertise of Canada’s leading paediatric specialists. 

Led by a steering committee of long-standing patient partners and health-care and research experts, the platform is also designed to support future clinical trials and training for clinicians and researchers. By prospectively collecting tumour and fluid samples and fostering knowledge exchange across the country, the trial lays the groundwork for ongoing national collaboration to bring precision medicines and research to families affected by these rare diseases. 

To learn more about this trial, visit the Rare Brain Tumour Consortium website. 

Sponsored by the C17 Council, the trial is the next step in a research program built by Huang and a community of patients and families affected by rare brain cancers, including one of the program’s earliest donors, Tali’s Fund. The research and development was made possible through The Arthur and Sonia Labatt Brain Tumour Research Centre, the Garron Family Cancer Centre, Pediatric Brain Tumor Foundation of the United States, Inc., Hope4ATRT, the Meagan Bebenek Foundation, The Daniel Bertoia Family Fund, Entertainment One, Bunzl Canada and Family and Friends of Mary Jean de Jourdan and Andrew Pilat, together with the collective support of donors through SickKids Foundation.