Canada redefining treatment options for children with brain cancer

In a groundbreaking advance for pediatric cancer research and care, the VICTORY clinical trial is redefining treatment approaches for children with brain tumours. The VICTORY clinical trial will investigate the safety and efficacy of a combination of treatments, intravenous Vinblastine (a chemotherapy drug) with oral Tovorafenib (a targeted therapy). This trial aims to improve outcomes for children with recurrent pediatric low-grade glioma (PLGG). This innovative trial places Canada at the forefront of pediatric neuro-oncology research, showcasing leadership in advancing evidence-based treatments.

The VICTORY trial emerged from insights gained through the TRAM trial, a pioneering national trial that tested a targeted oral therapy for relapsed brain tumours. While the TRAM trial showed promising tumour shrinkage, it also revealed a critical gap: tumours often returned once treatment stopped. Patients and families expressed a need for longer-lasting, more effective treatment options, sparking the development of VICTORY.

“After the TRAM trial, we knew we could think of better solutions. VICTORY combines targeted therapy with chemotherapy to not just shrink but potentially eliminate the tumours,” says Dr. Sébastien Perreault, the lead investigator of the TRAM trial. “This is about pushing the boundaries of what’s possible for our kids.”

The VICTORY trial is part of a broader ecosystem of innovative research happening in Canada supported by various funders and collaborators to accelerate novel therapies for childhood brain cancers. For example:

1. The Deciphering 3D Chromatin States in High-Grade Childhood Brain Tumors research project is investigating how cancerous brain cells manipulate their chromatin architecture to identify communication pathways within the tumour that maintain these abnormal chromatin states to identify effective therapies to disrupt them.
2. The MIRV trial is evaluating the safety and efficacy of Mirdametinib, a MEK inhibitor, combined with Vinblastine, offering hope for the upfront treatment of PLGGs.
3. The DECRYPT trial aims to evaluate the safety and tolerability of a unique radiation-free treatment for embryonal brain tumours.
4. The U-R-Immune Glioma trial is the first trial in humans to use immunotherapy without radiotherapy for patients with highly mutated and immunotherapy-receptive glioblastomas.

Dr. Uri Tabori, VICTORY trial co-lead investigator, emphasizes the significance of these combined efforts. “Canada is leading the way with good brain tumour science. These studies and trials are proactive steps in advancing research, increasing access for children with cancer in Canada, and offering a case for continued and increased funding in the pediatric cancer space. Without support from ACCESS and CIHR this work would not have been possible.”

“My son was under 12 months old when the doctors found a mass in his brain. He has undergone various treatments, but the tumour keeps recurring,” said mother of a child enrolled in the VICTORY trial. “Families just need a chance, and this is the only option we have. And maybe, just maybe, this is the chance he needs to be well and live a full life. Every child is different, and every child has different symptoms, so the least we can offer is experiential evidence for another family,” reflected Mustafa’s mother. “Parents are in desperate need of a support system while going through this journey. We are so focused on the treatment that often school, social circles, and other aspects of life take a backseat. We are grateful to have circles of friends, family, and professionals to help us through this time.”

The VICTORY trial represents more than just a clinical trial—it’s a source of hope. “There’s fear and uncertainty that go alongside any early-phase clinical trial, but honest and frequent communication can help families through this time. Knowing that clinicians and researchers are working on better treatments offers families hope,” shares Tara McKeown, nurse practitioner at SickKids.

“From the onset, our goal with VICTORY trial was to have an innovative trial that was accessible. By using satellite sites, we can bring the trial closer to the patient, reducing the need for long travel and increasing enrollment,” said Dr. Nirav Thacker, Principal Investigator on the VICTORY Trial. “With equity, diversity, and inclusion at the core of ACCESS’ values, this approach allows for greater diversity and inclusion in patient participation from the start, ensuring that those living outside urban areas can also benefit from the trial.”

These studies and trials reflect a broader commitment to advancing pediatric oncology research and care. Access to these cutting-edge therapies is equally important, as families in Canada often face barriers to obtaining novel treatments available elsewhere. Aiman Siddiqi, Program Manager highlighted, “Supporting this work is a privilege. There’s a lot of learning involved, and we hope to apply this learning to other projects to accelerate research and discovery, ultimately placing Canada on the map.”

As Canada makes some pivotal strides in pediatric cancer research, the impact of this work reaches far beyond its borders, establishing a new global benchmark for pediatric oncology care. By integrating cutting-edge scientific inquiry with a commitment to patient access, Canada is not only advancing treatments but also ensuring that all children facing cancer have access to the best possible care.  Innovation, compassion, and the pursuit of better outcomes intersect, offering hope for brighter futures for children worldwide.

Learn more about ACCESS research themes, groups, and projects here.