National network to accelerate research for rare diseases in kids

July 5, 2024 — 

A group of researchers from the Rady Faculty of Health Sciences and Children’s Hospital Research Institute of Manitoba (CHRIM) has leadership roles in a national initiative to accelerate research and treatment for rare diseases in children. 

RareKids-CAN: Pediatric Rare Disease Clinical Trials and Treatment Network received $20 million in funding earlier this year from the Canadian Institutes of Health Research. The funding is part of the government of Canada’s national strategy for drugs for rare diseases, announced in 2021. 

“The goal is 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,” said Dr. Lisa Knisley, assistant professor in the College of Nursing.  

Knisley is co-lead of the initiative’s Knowledge Mobilization Platform, along with CHRIM investigator Dr. Terry Klassen.  

“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. 

Dr. Lauren Kelly, associate professor of pharmacology and therapeutics in the Max Rady College of Medicine, is on the network’s executive team and leads training for the initiative.  

“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.” 

The group noted that 14,000 children under the age of 15 die from rare diseases in Canada every year. 

“There are over 8,000 different rare diseases, and over 50 per cent of those affect children,” said Dr. Cheryl Rockman-Greenberg, distinguished professor of pediatrics and child health at the Max Rady College of Medicine. 

Rockman-Greenberg has worked in rare disease research for her entire 50-year career. Her focus is on the regulatory affairs and advocacy aspects of the RareKids-CAN project.  

She said the ability to diagnose children with rare diseases has increased significantly over the last decade, thanks to advances in gene diagnosis technology. However, the cost for treatments can be very expensive. 

“Our ability to diagnose these disorders has increased, but access to new diagnostic techniques and new treatments has not kept pace,” she said. 

Under various platforms, the initiative brings together leaders in several areas, including clinical trials, pharmacology, drug development and data analysis. 

“There are many doctors involved. And, of course, the patient’s voice will play a major role in this strategy,” Rockman-Greenberg said. 

Alan MacKenzie