A $50 Million Investment in Children’s Health
Imagine a world where rare childhood diseases are rapidly diagnosed, and treatment options are precisely tailored to each child and their unique characteristics.
Thanks to revolutionary advances in paediatric medicine and a $50 million dollar grant by the Azrieli Foundation, two pioneering Canadian hospitals dedicated to children’s health are carving a more individualized approach to care that accounts for every aspect of a child, from their genetic code to their postal code.
Currently, three million people in Canada are affected by a rare disease such as Tay-Sachs Disease, a genetic disorder that causes progressive damage to nerve cells resulting in debilitating symptoms such as muscle weakness, motor issues and vision and hearing impairment. Like Tay-Sachs Disease, most rare diseases and conditions have a genetic origin, with 70% of them occurring during childhood. Considering that 50% of Canadian children with a rare illness currently live without a diagnosis or treatment, this investment is more important than ever.
Precision Child Health offers hope
The urgency to understand the underlying genetic cause of childhood-onset conditions has become an international biomedical research movement. The Hospital for Sick Children (SickKids) in Toronto, Canada and CHU Sainte-Justine in Montreal, Canada are looking beyond genetics — leading to the emergence of Precision Child Health (PCH). Leveraging advances in cutting-edge science, technology and data analysis, PCH aims to improve patient outcomes by integrating all important information about a child into their care: genetic, biological, environmental, social determinants of health and beyond.
“Precision Child Health extends beyond traditional precision medicine. It not only incorporates genetics and molecular biology but also integrates environmental and social determinants to provide truly individualized care. While conventional precision medicine primarily focuses on DNA, PCH embraces a more holistic perspective, accounting for the interplay between genetic makeup, epigenetics, and the exposome. The goal: to better understand, diagnose, and most importantly, treat children with tailored interventions that meet their unique needs—allowing them to grow up healthy and reach their full potential.” — Dr. David Malkin (Scientific Director of the Precision Child Health Partnership, SickKids) and Dr. Elie Haddad (Scientific Director of the Precision Child Health Partnership, CHU Sainte-Justine)
Two Canadian hospitals join forces to advance Precision Child Health
SickKids and CHU Sainte-Justine stand at the tipping point of a paediatric healthcare revolution in Precision Child Health. They are two of North America’s largest child health research hospitals with combined coverage of over 60% of Canada’s paediatric population.
Both hospitals have a strong history in genomic medicine, – and both are global leaders in delivering innovative treatments such as immunotherapy, a revolutionary treatment where a patient’s own immune cells are modified to recognize and destroy their cancer. Other shared strengths include patient-centred care and patient-oriented research that together close the loop between clinical research and bedside application, otherwise known as “translational research”. Independently, they lead the world in such fields as maternal-child health, childhood cancers and neurodevelopmental disorders.
Given each hospital’s strengths, their partnership will undoubtedly advance the vision of Precision Child Health across Canada.
A $50 Million investment from the Azrieli Foundation
With the belief that every child deserves the opportunity to reach their potential, the Azrieli Foundation made a joint $50 million grant in December of 2023 to SickKids and CHU Sainte-Justine. The intention of this historic grant was to allow researchers and clinicians at both hospitals to work together to advance discoveries and therapeutic innovation in precision medicine.
Naomi Azrieli, O.C., DPhil., Chair and CEO of the Azrieli Foundation, said of the partnership when it was announced, “This partnership is based on excellence, visionary leadership and courageous collaboration. Philanthropy has been essential as a catalyst for innovation and progress, but our commitment goes beyond funding: we have taken a hands-on approach to foster a healthcare revolution with the extraordinary potential to improve lives.”
Accomplishments at the one-year mark
Now 12 months into the partnership, we checked in with both hospitals to get an update on how the year has progressed and what lies in store for the future.
“As co-directors of the Precision Child Health Partnership between SickKids and CHU Sainte-Justine, we are thrilled to reflect on an incredible year. We have successfully launched our first major cross-institutional driver program and catalyzed over 15 additional research collaborations across our two institutions. It has been truly inspiring to watch these teams form and move PCH forward. We are excited for the future and the continued advancements in child health that our partnership will bring,” say Roselle Gélinas (CHU Sainte-Justine) and Tomasz Czarny (SickKids), Azrieli Co-Directors, SickKids-CHU Sainte-Justine Precision Child Health Partnership.
The Catalyst Program
Made possible by the Azrieli Foundation grant, the Catalyst Competition facilitates the formation of interdisciplinary collaborations between researchers from both SickKids and CHU Sainte-Justine. Together, they propose new research projects aimed at advancing Precision Child Health.
In 2024, fifteen innovative projects by multi-investigator research teams were awarded a total of $4.5 million over two years to work on a broad array of innovative projects spanning developing Artificial Intelligence models leveraging electronic health records to a study that examines the effects of combination therapies on Sanfilippo syndrome, a rare genetic condition affecting the brain and nervous system.
Another team’s work focuses on developing a drug screening system using zebrafish models to identify molecules that could one day lead to new therapeutics to treat congenital heart or vascular diseases. Researchers will leverage these models to better understand these complex genetic diseases and, hopefully, one day be able to identify personalized treatments.
The principal researchers on the zebrafish project, Drs. Ian Scott (SickKids) and Rubén Marín-Juez (CHU Sainte-Justine) said of the funding made possible by the Precision Child Health partnership: “We are excited at the potential of creating patient-specific genetic models in the lab and conducting rapid testing of thousands of molecules for precision therapies. Thanks to the commitment of the Centre de recherche Azrieli du CHU Sainte-Justine and SickKids, a new era of bold and ambitious research is beginning.”
“As co-directors of the Precision Child Health Partnership between SickKids and CHU Sainte-Justine, we are thrilled to reflect on an incredible year. We have successfully launched our first major cross-institutional driver program and catalyzed over 15 additional research collaborations across our two institutions. It has been truly inspiring to watch these teams form and move PCH forward. We are excited for the future and the continued advancements in child health that our partnership will bring.”
Roselle Gélinas (CHU Sainte-Justine) and Tomasz Czarny (SickKids), Azrieli Co-Director SickKids-CHU Sainte-Justine Precision Child Health Partnership
Paediatric Neurosurgeon and Catalyst Program grant recipient, Dr. Alexander Weil at CHU Sainte-Justine says, “The funding from the Azrieli Foundation really does have a catalytic effect by helping us to create the research, get the results and then apply for larger-scale federal funding which allows us to have more sustained funding for our programs.”
The DECODE(u)r Study
A second initiative made possible by the Azrieli Foundation-funded Precision Child Health Partnership between SickKids and CHU Sainte-Justine is the DECODE(u)R study.
Many rare diseases are caused by a single gene mutation. Identifying single gene variants is a central goal of precision medicine.
The DECODE(u)R study will take place jointly at SickKids and CHU Sainte-Justine over a period of four years, recruiting over 680 currently undiagnosed patients with rare diseases from across Ontario and Quebec. Their genomes will be sequenced in addition to profiling other molecular markers, aiming to uncover key insights into the genetic basis of their conditions.
Genomic sequencing is the process of determining the complete DNA sequence of an organism’s genome – almost like a set of instructions for how an organism is made and how it works. Through genomic sequencing, scientists better understand genes, variations and mutations that could impact health, development and disease. With insights into what is going on inside the cells, researchers can learn how variations and mutations may lead to the onset of disease.
The goal of the DECODE(u)R study is to help researchers pinpoint genetic markers that cause rare diseases in children—results that may have wider application to adult diseases. To accelerate the pace of therapeutic innovation, the anonymized data from these studies will be shared across the two hospital sites and more broadly with other research partners.
“The DECODE(u)r study will not only help identify DNA variants that current clinical genetic tests might miss but also enable the discovery and characterization of new rare diseases. This will bring an end to diagnostic odysseys for many families, guide their child’s care, and provide precise genetic counseling. It will also connect families with others facing the same genetic condition and open the door to future personalized treatments,” say Dr. Gregory Costain (SickKids) and Dr. Philippe Campeau (CHU Sainte-Justine), Medical Geneticists.
Philanthropy fuels new frontiers in paediatric health
Transformational philanthropic gifts are a result of transformational visions. Philanthropy is the spark and the catalyst that fund high-risk and high-reward ideas before they will be considered by government granting agencies.
“Philanthropy is more than just financial support—it’s the driving force behind innovation and hope. Thanks to the generosity of our donors, researchers at CHU Sainte-Justine and SickKids can push the boundaries of what’s possible, accelerate the discovery of life-changing treatments, and transform lives. Together, we are giving children the opportunity to grow up healthier and empowering families with hope for a brighter future.” — Colin Hennigar, Chief Development Officer, SickKids Foundation & Tiffany Belliardo, Vice President, Philanthropic Development – Major and Planned Gifts, CHU Sainte-Justine Foundation
Dr. Tamorah Lewis, a physician-scientist and Division Head for Clinical Pharmacology and Toxicology at SickKids, and first-year Catalyst Program grant recipient, has emphasized the critical role of philanthropy in advancing paediatric healthcare. “This generosity enables our two hospitals to come together, combining world-class treatments with ground-breaking research, ultimately leading to improved outcomes for children.”
For clinician-researchers like Dr. Alexander Weil at CHU Saint-Justine, whose work on epilepsy has changed lives for many children, philanthropy is a nod of encouragement that allows him to continue striving for better and possibly curative treatments for his patients. “At the end of the day, why do we do all of this? To improve the lives of children, and we cannot do it alone,” he says.
The teams at SickKids and CHU Sainte-Justine are incredibly grateful for the support of the Azrieli Foundation for their vision in helping them achieve their joint vision.
The science behind the miracle of life
When it comes to maternal health, Montreal’s CHU Sainte-Justine stands as a world leader. It is the largest mother and child university hospital in Canada and one of the four most influential paediatric centres in North America. The 115-year-old hospital specializes in a comprehensive range of maternal- and child-health services encompassing areas such as high-risk pregnancies, mother-to-child infectious diseases, perinatal medicine and neonatology (the advanced care of premature newborns).
In the fall of 2024, the Azrieli Foundation visited CHU Sainte-Justine to learn more about the breadth of the institution’s revolutionary advances in paediatric healthcare and research. The visit was a follow up to a $50 million grant made jointly to CHU Sainte-Justine and The Hospital for Sick Children by the Azrieli Foundation in 2023, to advance the field of Precision Child Health. While at CHU Sainte-Justine, hospital staff provided members of the Foundation with a special tour of the Neonatal Intensive Care Unit (NICU) and introduced us to a highly specialized nurse and respiratory therapist team.
Pierra Barker, nurse, and Virginie Tanguay, respiratory therapist, are two members of the team that answers the call – day or night – and boards an ambulance to perform life-saving procedures on premature babies across Quebec, from big cities to rural communities. In most cases, the babies’ only hope for survival is transport to CHU Sainte-Justine where they can receive the most advanced care available. Not only the “eyes and the ears” of doctors back at Sainte-Justine who support them by phone during resuscitation and stabilization, these unique, specialized NICU professionals are experts in their own right, adept at using the most advanced life support equipment, as pictured above.
These women are real life heroes, yet you would never know it from their humility and easy smiles. When asked how they separate their work from the rest of their lives, they replied, “It is not hard. It is just my job.” Truly inspiring.
Each year, the NICU transport team cares for as many as 500 babies. To ensure 24/7 service, the CHU Sainte-Justine transport team is made up of up to 10 dedicated transport nurses and 10 respiratory therapists. CHU Sainte-Justine’s neonatal unit welcomes 1,500 babies annually, of which approximately 130 are extremely premature. It is the largest neonatal unit in Canada.
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