A new era of individualized child health
“What we learn from Michael’s trial will help carve out a path for SickKids to explore more precision-based treatment options for rare diseases, as well as the infrastructure, policies and processes that we need in place to ensure we can scale these options to other patients.”
– Dr. David Malkin, Co-Lead, Precision Child Health, Director of the Cancer Genetics Program and the CIBC Children’s Foundation Chair in Child Health Research
SickKids 2025 is a roadmap to achieving unprecedented outcomes and ushering in a new era in child health: Precision Child Health.
What is Precision Child Health? It’s a movement to deliver individualized care for every patient at SickKids – one that challenges a one-size-fits-all approach to medicine by using integrated data about each child’s unique genes, biology and environment to tailor care. Through novel technology and innovative precision approaches, and by building on the paediatric care excellence SickKids is already known for, we will diagnose diseases more accurately, provide targeted treatments faster and prevent disease sooner. We will also partner with patients, families and communities to ensure that every child has access to the best possible care and the opportunity to participate in research, regardless of where they live or their socioeconomic status.
To help bring this vision to life, SickKids’ Precision Children Health strategy is bringing together efforts from across the organization to develop and improve access to new and existing precision tools, technology and expertise and support a more systematic and centralized individualized care pathway for patients. A comprehensive five-year strategic plan is helping to build the foundations needed to advance this work, with clinical, research, education and operational teams working together to make Precision Child Health a way of life at SickKids.
While Precision Child Health covers the spectrum of child health and treatment, efforts to harness the power of genomics and research to make a difference in the lives of children are already underway. One example of these efforts happened in 2022, when SickKids delivered its first SickKids-sponsored individualized gene therapy in a single patient, only three years after four-year-old Michael Pirovolakis was first diagnosed with an ultra-rare neurodegenerative disorder – and with promising results. This achievement is not only a significant milestone for Michael but a marker for PCH and the potential of individualized care to improve health outcomes for children with rare diseases. It is also a testament to the dedication, hard work and collaboration of teams at SickKids.
To build on the momentum of cases such as Michael’s, an Interventional Clinical Trials Task Force was initiated with representation from across clinical divisions and departments to streamline processes and infrastructure that may reduce barriers to rapid initiation of clinical trials and use of novel therapies to support precision medicine.
Alongside the transformation of the clinical trial pipeline at SickKids, a Translational Genomics multidisciplinary group of experts is working to better understand the clinical utility of, and improve patient access to, precision diagnostics. One example includes the development of a new service, SickKidsSeq, that will enable access to genome-wide sequencing to patients and families who do not have access through standard care or research initiatives. This new service will help clinicians to identify the genetic causes of diseases, provide more precise diagnoses, and improve treatment options for children with complex medical conditions. This is another example of the many initiatives that teams are undertaking to advance the vision of Precision Child Health at SickKids.
Looking ahead, SickKids is also building partnerships as part of a Precision Child Health network, combining forces with other paediatric institutions to support the development of a systematic approach to individualized care to help enable the future of Precision Child Health beyond SickKids’ doors.