Researchers in Lyon have investigated, both in vitro and in vivo, the potential therapeutic synergy between the type 6 HDAC inhibitor (givinostat), which is already used to combat muscle atrophy in Duchenne muscular dystrophy, and nusinersen, which is already marketed for the treatment of spinal muscular atrophy (SMA):
- in the SMA cell models used (SMA myoblast cell lines), the addition of the HDAC-6 inhibitor to nusinersen had a positive impact on the development and differentiation of myotubes;
- these same beneficial effects were also observed in a mouse model of SMA (Taiwanese strain), which showed an increase in lifespan as well as an increase in muscle mass and strength.
This research could lead to a clinical trial, given that it involves the repurposing of givinostat and that nusinersen is already available on the market.
