Canadian project aims to accelerate pediatric rare disease research
Nonprofit, hospitals lead $20M government-funded effort
A national research network in Canada is working to accelerate research and treatment advances for rare diseases that affect children, such as aromatic l-amino acid decarboxylase (AADC) deficiency.
RareKids-CAN: Pediatric Rare Disease Clinical Trials and Treatment Network aims to help streamline and support rare disease clinical research in order to improve health outcomes for children living with these conditions. It will bring together researchers, patients, caregivers, and clinicians to achieve that goal.
The effort was supported by $20 million in funding from the Canadian Institutes of Health Research (CIHR) as part of the country’s efforts to develop treatments for rare diseases.
The program is being led by the Maternal Infant Child and Youth Research Network (MICYRN), a nonprofit that links research organizations across Canada. Scientists at the University of Manitoba (UM) and the Children’s Hospital Research Institute of Manitoba (CHRIM) hold several leadership roles.
“RareKids-CAN will ensure that there are clinical trial leaders with expertise in rare diseases that are supported to lead life-changing research for generations to come,” Lauren Kelly, PhD, associate professor at UM and an executive team member for the RareKids-CAN network, said in a university press release.
Rare disease research to benefit kids
According to the Canadian Organization for Rare Disorders, a rare disease is one that affects fewer than one in 2,000 people in their lifetime. One such condition is AADC deficiency, an ultra-rare genetic disorder in which severe neurological problems begin to emerge in the first year of life.
About 14,000 children younger than 15 die from rare diseases in Canada every year, according to UM. “There are over 8,000 different rare diseases, and over 50 per cent of those affect children,” noted Cheryl Rockman-Greenberg, MD, a UM professor who is focusing on the regulatory affairs and advocacy components of the RareKids-CAN project.
While diagnosing rare diseases has drastically improved in recent years, aided by the availability of genetic testing, treatment costs can be debilitating for many families, Rockman-Greenberg said. “Our ability to diagnose these disorders has increased, but access to new diagnostic techniques and new treatments has not kept pace,” she said.
The RareKids-CAN network will bring together leads in clinical trials, drug development, and data analysis in an effort to accelerate progress.
“There are many doctors involved,” Rockman-Greenberg said. “And, of course, the patient’s voice will play a major role in this strategy.”
Another researcher taking a leadership role in the program is Lisa Knisley, PhD, assistant professor at UM. Knisley and CHRIM investigator Terry Klassen, MD, will co-lead the network’s Knowledge Mobilization Platform. “We will be working with experienced clinicians, researchers, parents and families living with rare diseases to share clinical trial opportunities and study findings, in ways that are meaningful, accessible and useful,” Knisley said.
The program aims “to provide the essential infrastructure and resources needed to give children with rare diseases in Canada the opportunity to participate in clinical trials closer to home,” Knisley said.
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