• A Spanish study has tested the effects of the drug nintedanib, approved for idiopathic pulmonary fibrosis, on the muscle condition of mice model of limb-girdle muscular dystrophy related to SGCA (LGMD R3).
• For 10 weeks, the researchers gave 50 mg/kg of nintedanib orally twice a day to 14 diseased mice, and compared their progress with that of 14 untreated and six untreated diseased mice.
• The study showed that the treatment was well tolerated.
• At the end of the treatment, comparison of treated and untreated mice showed that:
- they ran 2.6 times farther and had 20% greater forepaw grip strength
- their quadriceps, gastrocnemius and triceps show significantly less fibrosis: the amount of collagen in these muscles is reduced and the expression of genes involved in the fibrotic process is decreased;
- the surface area affected by the dystrophic phenotype is smaller, the fibres are more homogeneous and smaller, and the pathological regeneration process is reduced;
- Macrophagic infiltration is reduced in all muscles, especially in the triceps. The expression of more than 80% of the cytokines analysed is decreased.
• These results suggest the potential usefulness of nintedanib in the therapy of muscular dystrophies, alone or in combination with gene therapy.
