Embryonal brain tumors (EBTs) comprise the largest category of malignant central nervous system (CNS) tumors in children 0 to 14 years of age, and include a number of aggressive, deadly brain tumors that require timely, specific care. Approximately 60% of EBTs are considered high-risk, high fatality rare diseases of children younger than 3 to 6 years (“infants”). In fact, despite intensive chemotherapy and radiation, which cause devastating long-term physical and cognitive consequences, only a small fraction (10 to 35%) of patients with EBTs survive long term.

Canadian-led teams have been at the forefront of understanding and combatting these difficult cancers. Importantly, researchers from The Hospital for Sick Children (SickKids) have developed a novel radiation-free regimen (SickKids Standard of Care [SKSOC]). This regimen includes direct intrathecal therapy, during which chemotherapy is delivered into the fluids around the brain, and the addition of a metronomic chemotherapeutic regimen after high dose chemotherapy. The SOC treatment has improved survival without use of radiation in 60% of patients, which is significant due to the detrimental neurocognitive toxicity of radiation in young brains. This unique regimen has been adopted as best practice across Canada and used in many other centres outside of Canada. While this success is exciting, it is tempered by the lack of knowledge and tools to determine which biological or clinical factors are predictors of treatment success or failure and the absence of multi-institutional prospective data.

As such, the Canadian-led DECRYPT (Defeating Embryonal Cancer in Young People Together) – BABYBRAIN trial, with sponsorship by the C17 Council, will assess the safety and tolerability of SKSOC treatment regimen for patients less than 6 years old diagnosed with high-risk CNS-EBT across Canada. The trial will also establish a platform for a national tumour board discussion for all young patients with high risk EBTs, and the collection and analyses of tumour tissue and cerebrospinal fluids that will allow real-time return of information to tailor more effective and less toxic treatment for young patients diagnosed with high-risk EBTs in the future.

This trial represents the first step towards the development of a national precision medicine clinical trial platform for infant brain cancers driven by a multi-stakeholder group comprised of healthcare and research experts and patient partners. It is hoped that through the sharing of knowledge, resources and access to healthcare, that not only does pan-Canadian care and outcomes for patients with infant brain cancers improve, but that a powerful platform to launch similar future trials is established.

The trial originated from and is also supported by a coalition of Canadian charities and national research funders through the DECRYPT Research Grant. This unique grant was made possible through a partnership involving Kindred Foundation, Cancer Research Society, Phoebe Rose Rocks, Brain Tumour Foundation of Canada, The Miracle Marnie Foundation, Childhood Cancer Canada, and Tali’s Fund.

Adding to this continued support, the ACCESS Clinical Trials research theme is committed to facilitating the participation of every eligible child in this trial. This will be accomplished by supporting the trial investigators in the opening of this trial at as many of the 16 Canadian pediatric cancer centres as possible, as well as integrating the use of satellite sites, where possible, to minimize travel time for patients and families and enhance involvement from communities that have been historically underrepresented in clinical trials.

For more information, please contact Tricia Schneider.