Helene Decaluwe
Credit: CHU Sainte-Justine Research Center
Led by clinical researcher Hélène Decaluwe, from CHU Sainte-Justine, a team has developed a treatment that slows the progression of a rare disease that attacks children’s immune systems. The scientific paper describing this new treatment was published in early January on Journal of Allergy and Clinical Immunology.
The success of the dref. Decalogue, also a professor in the Department of Pediatrics at the University of Montreal, is due to the combination of two classes of drugs that work synergistically to reduce the progression of a rare immune disease. Currently, about a third of patients fail to respond adequately to treatment and die within five years of diagnosis.
“We have to try to do better. It is a very serious disease which is one of the immunological emergencies to be treated as quickly as possible ”, explains the doctor.
Familial hemophagocytic lymphohistiocytosis (FHL) affects children from birth. It is an immune system abnormality that takes the form of a cytokine storm. A severe inflammatory reaction ensues, because cytokines, these messenger proteins, send messy signals to the immune system.
Curbing LHF to transplant stem cells
A stem cell transplant is needed to cure LHF. However, it is essential to slow the progression of the disease before proceeding with a transplant. For some patients, traditional treatments to bridge the graft only partially work and do not prevent a flare-up of the disease. Furthermore, these treatments create a significant toxicity problem.
Thanks to that new studythe Dref.Decaluwe achieved complete disease control in a mouse model with no apparent toxicity. The cytokine storm was calmed by the use of the new drug duo.
“These are very promising results, which could eventually save and cure more children affected by this immune disease,” says Hélène Decaluwe.
What’s even more exciting is that these drugs (emapalumab and ruxolitinib) have already been individually approved for use in humans. The next step will be to study the effectiveness of the ‘bridge treatment’ in a clinical trial. “This confirms the leading role of the CHU Sainte-Justine in the management of this disease,” he adds.
The results of this study offer the hope of greatly improving the cure rate of children with FHL by allowing them to wait until stem cell transplantation.
About this study
The article «Combined inhibition of IFNγ and JAK for the treatment of hemophagocytic lymphohistiocytosis in mice»by Josée-Anne Joly, Alexis Vallée, Benoite Bourdin, Sara Bourbonnais, Natalie Patey, Louis Gaboury, Yves Théorêt and Hélène Decaluwe, was published on January 4, 2023 in Journal of Allergy and Clinical Immunology.
Funding for the study was provided by the Charles-Bruneau Foundation and the Canadian Institutes of Health Research.
