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 to manage and cure. 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.

However, 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 addition of a metronomic chemotherapeutic regimen after high dose chemotherapy. This has resulted in significant improvements in radiation-free survival of up to 40%, 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. 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 success or failures and absence of prospective data.

As such, the Canadian-led DECRYPT (Defeating Embryonal Cancer in Young People Together) trial will assess the safety and tolerability of SKSOC treatment regimen in high-risk CNS-EBT patients under the age of 6 years. The trial’s primary objective is to evaluate the prospective feasibility of this treatment combination and capture unbiased safety data. The trial will also aim to report progression-free survival and overall survival. Trial participants will have the opportunity to provide biological specimens, importantly cerebrospinal fluid, as part of the study for biobanking for future research.

This trial also represents the first step towards the development of a national 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 ACCESS Clinical Trials research theme is committed to facilitating the participation of every eligible child in this trial. This will be accomplished by opening the trial at all 16 Canadian pediatric cancer centres, 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.