Tackling childhood brain cancer at the root to improve survival and quality of life

Brain cancer remains a lethal and disabling disease, the leading cause of cancer-related deaths among children under age 20 and the third-leading cause in young adults aged 20-39. This is in contrast to childhood leukemia and other blood cancers, where survival and quality of life have improved markedly based on improved classification and novel targeted therapies implemented at diagnosis. There are particularly aggressive forms of brain cancer, with barely 10 percent of children and young adults surviving three years after diagnosis, and other forms where those who do survive suffer severe lifelong disabilities due to the life-saving therapies they receive.

The research teams of Drs. Nada Jabado and Jacek Majewski of McGill University and Dr. Michael Taylor at SickKids have previously discovered that many pediatric brain tumours are driven by mutations in genes that play a significant role in brain development. They also provided tools to improve the diagnosis and better classify these brain cancers in children, promoting more effective treatments. To decrease the burden of survivorship and improve survival rates, this project will fast track the use of treatments targeting specific genetic alterations early at diagnosis. The team will also perform innovative investigations of the tumour genome and transcriptome, including at the single-cell level, to identify new alterations and specific vulnerabilities that can be targeted for therapy. The team will ensure treatments are validated through relevant disease models and fast-track meaningful clinical trials to tackle refractory brain tumours during this grant; the goal is to work closely with health-care providers and regulators to ensure the rapid translation of validated treatments to the bedside.

Ultimately, the team’s work will improve the survival rate and quality of life for children and young adults with brain cancer, both during and after treatment.

Short version                  

The research team will provide targeted treatments for children with brain cancer based on the genetic makeup of the tumours and work with health-care providers to validate and make these therapies rapidly available at the bedside following diagnosis. The team will also continue to chart new genetic alterations leading to brain cancer. This work will improve survival and enhance the quality of life of patients both during and after treatment, while allowing for a more effective use of health-care resources.

Lead Genome Centre: Génome Québec

Co-lead Genome Centre: Ontario Genomics

Co-project leaders:

Co-investigators and Users: