Personalizing approaches to treating childhood arthritis

An international research network aimed at personalizing medicine for children with arthritis has received a multi-million dollar boost. Dr. Rae Yeung, Rheumatologist and Senior Scientist at The Hospital for Sick Children (SickKids) and her co-lead Dr. Nico Wulffraat from University Medical Center Utrecht are receiving $8 million over six years from the Canadian Institutes of Health Research (CIHR) and ZonMw (the Netherlands Organisation for Health Research and Development) and Reumafonds (the Dutch Arthritis Foundation).

The new network called UCAN CAN-DU, is part of the Understanding Childhood Arthritis Network (UCAN) founded by Dr. Yeung. UCAN CAN-DU includes all paediatric rheumatologists in Canada and the Netherlands and will use the investment to develop genomic tests and to determine individualized therapy for children living with arthritis in an attempt to treat children quicker, more efficiently and with fewer side effects.

Why is it so important for you to be able to provide personalized care to children with arthritis?

There is a very narrow window of opportunity to treat affected children before their joints are damaged by disease. The idea is, if we can catch them early enough, we can change the biology of disease and prevent damage. With genomic testing we want to be able to tell which child is at high risk or low risk for joint destruction. For the high-risk children we want to be able to identify them early and give them strong medication right away.  Similarly, we do not want to expose low-risk children to strong medications un-necessarily.

You have essentially already personalized approaches for children who have a particular subtype of arthritis. Can you do that for all children with arthritis?

There is a sub-type called systemic arthritis, which is the most severe form that not only causes inflammation of a child’s joints, but many parts of their body including their hearts and lungs. We have a wonderful success story in systemic arthritis. A molecule called IL-1Beta has been found to be responsible for this inflammation. There is already a drug that can block IL-1Beta. We have preliminary data from a genomic test that shows whether these children have active disease or not and will respond to blocking IL-1Beta or not. The medication to block IL-1Beta is often given by a needle and often for many years.  From these genomic tests we can determine whether a child will respond to this medication and when they can stop this medication.  With this funding we hope to be able to personalize treatment for all sub-types of childhood arthritis, not just systemic arthritis.

You have developed testing kit to make this testing accessible to all patients. How would these work?

These are easy to use kits that contain tubes to which a patient’s blood samples are added. These kits could then be sent to special labs in Canada and the Netherlands for analysis. Results will tell physicians which patients need aggressive therapy and which don’t; who needs strong medication and who doesn’t; who needs to be on these medications for life and who doesn’t.

Why have you partnered with the Netherlands?

I founded UCAN in 2009, at that time there were only a few researchers around the world studying basic research in paediatric rheumatology, we were asking the same questions for these exceedingly rare diseases. I proposed that we stop competing against each other and collaborate. Our initial partners included the Netherlands and the first stage of our UCAN network was launched in the Netherlands. Together with our partners, our UCAN family now has members from over 50 countries and over 300 sites. Canada and Netherlands combined will have more patients and biosamples than any other cohort in the world. By the end of this project, we will have over 3,000 new patients enrolled. Canada and the Netherlands also share many similarities in our health care system and consistently rank at the top of international surveys for arthritis care and research.  It was a natural partnership to bring our countries together.   

What are the next steps?

There are about 10,000 children affected with arthritis in Canada. The systemic arthritis sub-group is only one of seven subgroups and it represents only about 10 per cent of children living with arthritis. Next steps include identifying tests and drugs for the rest of the 90 per cent of children who live with arthritis, so that they have a better chance of receiving the appropriate treatment within the narrow window of opportunity.  

This new funding gives us the ability to generate the data we need to inform our decisions and ensure we impact policy decisions and treatment decisions at the bedside in order to make children better.