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CHU Sainte-Justine
Thursday, January 5 2023
Press release

New treatment offers hope for children with a rare disease

MONTRÉAL, le 4 janvier 2023 – A team led by clinical researcher Hélène Decaluwe of Sainte-Justine University Hospital Research Centre has developed a treatment to control the progression of a rare disease that attacks the immune system of children. The article is published today in the Journal of Allergy and Clinical Immunology.

Dr Hélène Decaluwe, who is also a professor at the Université de Montréal, has successfully developed a new treatment combining two classes of drugs that work synergistically to control a rare immune disease called familial hemophagocytic lymphohistiocytosis (fHLH). Currently, approximately one-third of patients do not respond effectively to treatment and die within five years of diagnosis. “We have to try to do better. fHLH is a very serious disease and is an immunological emergency that must be treated as soon as possible” explains the clinical researcher.

fHLH affects children from birth. It is a disorder of the immune system that takes the form of a cytokine storm, where cytokines, messenger proteins, send confusing signals to the immune system, resulting in a severe inflammatory reaction.

Cytokines storm illustration by Benoîte Bourdin, PhD

Stem cell transplantation is key to curing fHLH. However, the disease must be under control before proceeding with a transplant. For some patients, conventional treatments used as a bridge to the transplant work only partially, allowing the disease to recur. Furthermore, these treatments also cause significant toxicity.

In this new study, Hélène Decaluwe has successfully managed fHLH in a mouse model, without causing overt toxicity. The cytokine storm was reduced to zero using the new combined pair of drugs.

“These are very promising results, which could ultimately save and cure many more children afflicted by this immune disease.” Even more exciting is that these drugs (emapalumab and ruxolitinib) are already individually approved for human use. The next step will be to study the effectiveness of the bridge treatment in a clinical study. “This confirms the leadership role of CHU Sainte-Justine in the management of this pathology.”

The findings of this study offer hope of significantly improving the recovery rate of children with fHLH by allowing them to maintain their strength while waiting to receive stem cell treatment.

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Updated on 1/18/2023
Created on 1/4/2023
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