$8 million in grants brings precision medicine to children suffering from arthritis

More than 20,000 children suffer from childhood arthritis in Canada; Kayla Baayens is one of them. The 10-year-old from Red Deer, Alta. has an aggressive form of the disease. It progressed to the point that her neck was stuck at a 15-degree turn. Unable to lie down comfortably in bed or brush her own teeth due to joint inflammation, Kayla and her family were desperate to find a treatment.  

Kayla, pictured above with Dr. Susanne Benseler, was referred to the Alberta Children’s Hospital. With a team of clinician researchers, Kayla received a diagnostic evaluation based on her own biological profile. “From a blood sample, we were able to examine Kayla’s biomarkers to identify her specific biological subtype of disease and choose the best method of treatment,” says Benseler, a rheumatologist and professor in the Department of Paediatrics at the Cumming School of Medicine and member of Alberta Children’s Hospital Research Institute and the McCaig Institute for Bone and Joint Health. 

Thanks to the precise diagnosis and targeted drug program, Kayla has gone from being bed ridden to being a typical kid without pain. “Compared to where she was, it’s like night and day,” says her father, Dean Baayens. “Kayla has even ridden a bike for the first time in her life!”

Stories like Kayla’s are rare. Most children with arthritis don’t have access to precision medicine diagnostics and targeted treatment. They are placed in conventional care involving a trial-and-error drug treatment plan with no specific biologic guidance on which therapy to choose and when to safely stop therapy.     

The importance of diagnosing and treating early

Childhood arthritis is an inflammatory disorder. There is a narrow window of opportunity to treat affected children before their joints are irreversibly damaged by the disease. “We’re incredibly thankful to the Alberta Children’s Hospital and Dr. Benseler,” says Dean Baayens. “Arthritis is not an easy thing to deal with; it’s a horrible thing for a parent to see their child go through. We’re incredibly thankful to the people who worked to develop these methods to treat Kayla appropriately.”

$8 million in grants to further the research

Now an international research network working to provide the precision medicine approach that Kayla experienced is getting a huge boost.  The network, called Understanding Childhood Arthritis Network, or UCAN, is receiving a total of $8 million in research funds from the Canadian Institutes of Health Research (CIHR) and corresponding organizations in the Netherlands. 

“We will use the investment to develop novel tests of specific immune and genetic markers and to determine individualized therapy for children living with arthritis in an attempt to treat children more rapidly, efficiently and with fewer side effects,” says Benseler, a co-principal investigator.  Benseler and Rae Yeung, rheumatologist and senior scientist at The Hospital for Sick Children, will lead the Canadian arm of the study.

Already, researchers have discovered five distinct subtypes of the disease, which activate particular proteins in the body that can be detected through an inexpensive biomarker kit. Benseler believes finding more subtypes will ultimately improve the care and quality of life for children with arthritis.

In brief, the funding will provide:

• a diagnosis determined by the biology of their disease for children in Canada and the Netherlands
• a protocol for treatment that is accessible to clinicians and families in these countries
• intensive research to find the inflammatory pathways that cause childhood arthritis

Large UCalgary interdisciplinary team involved

The team members on this grant include an interdisciplinary group of researchers from the Cumming School of Medicine and the joint pediatric and adult care team of the Division of Rheumatology in the Department of Paediatrics.

• Marvin Fritzler, MD, PhD, is a professor in the departments of Medicine and Biochemistry and Molecular Biology, and director of Mitogen Precision Diagnostics. His research team will provide the identification of specific biological subtypes of the disease.  
• Deborah Marshall, PhD, is a professor in the departments of Community Health Sciences and Medicine, Canada Research Chair in Health Services and Systems Research, Arthur J.E. Child Chair of Rheumatology Outcomes Research, and member of the McCaig Institute of Bone and Joint Health, the O’Brien Institute for Public Health and the Alberta Bone and Joint Health Institute. She is the Canadian health economic leader of the project and will measure the economic impact of precision medicine for childhood arthritis and develop mathematical models that will guide other countries.
• Paul Kubes, PhD, is the director of the Snyder Institute for Chronic Diseases and a professor in the departments of physiology and pharmacology, medicine, critical care medicine, and microbiology, immunology and infectious diseases. He is also a member of the Hotchkiss Brain Institute and holds a Canada Research Chair in Leukocyte Recruitment in Inflammatory Disease.  
• Marinka Twilt, MD, PhD, is an assistant professor in the Department of Paediatrics, a rheumatologist and clinician scientist and member of the Alberta Children’s Hospital Research Institute. She is the nominated Canadian Young Investigator on the project. 

The University of Calgary is uniquely positioned to find solutions to key global challenges. Through the research strategy for Infections, Inflammation, and Chronic Diseases in the Changing Environment (IICD), top scientists lead multidisciplinary teams to understand and prevent the complex factors that threaten our health and economies.