DMD: Canakinumab reduces certain blood markers but not IL1b

Canakinumab (Ilaris®) is a monoclonal antibody that neutralises interleukin 1 beta (IL1β), an inflammatory marker that is highly expressed in Duchenne muscular dystrophy. It is an immunosuppressant that is already on the market.

A US phase IAu cours d’un essai clinique de phase I un médicament dont l’intérêt thérapeutique a été montré sur des modèles animaux et/ou cellulaires (essais précliniques) est administré pour la première fois à un petit groupe de volontaires sains, plus rarement à des malades, afin d’évaluer leur tolérance à la substance en fonction de la dose (Comment le futur traitement est-il absorbé et éliminé ? Comment se fait sa répartition dans les organes ? Est-il toxique et à quelles doses ? Existe-t-il des effets secondaires ?)./II trial evaluated the efficacy of 2 mg/kg of canakinumab in three boys with DMD (two aged 4 and one aged 5), without additional corticosteroid therapy, four weeks after subcutaneous injection.

• The product was well tolerated, with no reaction at the injection site.
• IL1β levels did not decrease significantly in the three patients;
• Plasma levels of serine protease inhibitor and the alpha subunit of the interleukin-6 receptor (IL-6 sRa), lymphocyte antigen 86 (LY86) and immunoglobulin D (IgD) decreased significantly;
• Myostatin levels also fell.

Further studies using canakinumab over a longer treatment period may demonstrate increased benefits for some of these blood protein markers.

Pilot study of canakinumab (Ilaris) in steroid naïve children with Duchenne muscular dystrophy demonstrates safety and exploratory changes in potential serum protein response biomarkers. Spurney CF, Chong J, Gordish-Dressman H et al. J Neuromuscul Dis. 2025 May