BC Children's Hospital has officially launched its Clinical Trials Super Hub, an initiative aimed at transforming pediatric healthcare through enhanced clinical research. The launch was made possible by significant community support, including funds raised at the Crystal Ball 2024, and is championed by families like the Hoskins, whose eight-year-old son Simon is living with mucopolysaccharidosis (MPS) Type IV, commonly known as Morquio syndrome.
Addressing a Critical Need in Pediatric Care
Dr. Quyhn Doan, senior executive director of BC Children’s Hospital Research Institute, emphasized the importance of this hub, noting that while children represent a significant portion of the population, pediatric clinical trials are rare and limited. "Clinical trials are very rare and limited in the pediatric population and we thought that’s not right," she stated. The super hub aims to address this disparity by creating a dedicated infrastructure for pediatric research.
Simon's Story: A Catalyst for Change
Simon Hoskins, an eight-year-old living with Morquio syndrome, has become a central figure in the campaign to establish the Clinical Trials Super Hub. Morquio syndrome is a rare, progressive, genetic disorder characterized by skeletal abnormalities and short stature. Simon undergoes weekly enzyme transfusions to slow the progression of the disease, but there is currently no cure. His family's Cure MPS campaign has been instrumental in raising awareness and funds for the hub.
Simon's involvement extends beyond fundraising. He participated in the Crystal Ball Designation Video and spoke at the Crystal Ball 2024, which raised over $4 million for the super hub. "I was so impressed that he took it on. He really, truly understood the idea behind the clinical trial super hub," said Dr. Doan.
Building a Foundation for Future Research
The Clinical Trials Super Hub has already established a foundational expert team, including specialists in quality and regulatory affairs, clinical trial nurses, pharmacy staff, and laboratory coordinators. The goal is to expand this team with dedicated technicians to further enhance the hub's capabilities.
Overcoming Challenges and Looking Ahead
One of the primary challenges in pediatric clinical trials is the expense, as the pharmaceutical industry often sees limited profit in this area. By building a robust infrastructure and expertise, BC Children's Hospital aims to make pediatric clinical trials a more viable option. "It’s about finding new treatments and cures for kids diseases so that’s where we put our effort," Becs Hoskins said, highlighting the family's commitment to the cause.
The establishment of the Clinical Trials Super Hub represents a significant step forward in pediatric healthcare, offering hope for improved treatments and cures for children facing a wide range of diseases.
