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 I/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.