At first glance, Liam MacGregor seems like any other 12-year-old, enjoying computer games, visiting amusement parks and playing the occasional hockey game. But for a long time, he watched his friends run around outside and while he wanted to join in the fun, being physically active caused him pain.
Diagnosed at age three with juvenile rheumatoid arthritis, Liam wasn’t able to participate in a lot of physical activities. “I would try and keep up with the other kids, but at night I would pay for it,” Liam recalls. “I couldn’t move.”
After exhausting a variety of treatment options without significant improvement, Liam’s doctor at The Hospital for Sick Children (SickKids) invited him to participate in a clinical trial in 2008. It didn’t take long before Barbara noticed a major transformation in her son.
“Nothing was working for my son. The first day of the clinical trial he came into the hospital in a wheelchair and was running later that same day,” says Liam’s mom, Barbara, adding that today, Liam can be active with “zero to minimal pain.”
Success stories like Liam’s are the goal for many families who hope research will have an impact on the participating child’s care. Liam is one of hundreds of SickKids patients who has played a role in helping advance patient-based research, which may involve drug interventions and monitoring of the patient’s response to treatment, physiologic measurement or asking parents to complete questionnaires.
Every other week, Liam spends a day at the Physiological Research Unit at SickKids, where he has blood tests, waits for test results and has an infusion of medication. He and his mom pass the time by playing games and interacting with his nurses.
“Many families are willing to join our studies, not just for the benefit of their child, but for the benefit of the larger patient population, and that’s nice to see,” says Dr. Felix Ratjen, Medical Director of the Physiological Research Unit, a dedicated space for clinical research that was launched last fall. “It’s a very natural concept to them because they can see that there are limits to what we can do right now in terms of treating their children. We want to make sure that 10 years from now, we are not at the same level in terms of what we do, that we’ve learned a lot and that we have new and better therapies.”
Families’ willingness to join studies creates “a positive feeling of moving beyond what is possible right now,” he says. “If there are options, there’s always a chance for new hope and an outlook into a brighter future, which is also what drives many of us to do clinical research.”
For some patients, clinical trials can mean more than an improved quality of life. In cystic fibrosis patients, for example, patients who participate in clinical studies have been shown to have better survival rates than those who do not take part.
In addition to patients, healthy siblings and other volunteers are also an important contingent of participants, as they enable researchers to compare children with specific conditions to those in the general population. To engage families in research activities, SickKids recently launched the web-based Research4kids Clinical Studies Recruitment Database.
Dr. Colin Macarthur, who recently returned to SickKids as the Associate Chief of Clinical Research, has a clear vision: to use research findings to inform practice and policy.
“This is an exciting time for clinical research,” he says, noting the explicit role of clinical research in Ontario’s provincial Excellent Care for All Act and the recently-released national Canadian Institutes of Health Research strategy for patient-oriented research.
Clinical research in children has historically been overlooked in the scientific community, but that is changing, Ratjen says. “There is more and more recognition that research needs to happen in children. We want to make sure that whatever we do in the clinical setting is based on good evidence that it is safe and effective.”
Bringing paediatric clinical research to the forefront is one of SickKids’ priorities; in fact, one of the organization’s strategic directions, to “foster clinical research excellence,” was one of the factors that attracted Macarthur to his new role.
The SickKids Research & Learning Tower, which is currently under construction and slated to be completed in 2013, is expected to contribute to enhancing clinical excellence through research. The Tower will be the new hub for research activities, encouraging interaction and collaboration between scientists. Macarthur sees the Tower as a place where basic and clinician-scientists will be able to interact and collaborate more easily, paving the way for new discoveries.
“Quite often, breakthroughs are made when people work together who don’t normally collaborate,” he says. “This will be a major competitive advantage.”
According to Macarthur, here is a natural link between SickKids’ dual roles as a world-class paediatric health-care facility and one of the top child health research institutes in the world. “A research hospital has a culture of inquiry with an expectation of clinical and research excellence,” he says, adding that the large and diverse patient population helps drive research to new heights.
This relentless pursuit of clinical excellence through clinical research can make all the difference to families like Liam’s. “To me, clinical research means that there is an opportunity for kids – an extra step that might not otherwise be available,” says Barbara.