Muscle damage in Duchenne muscular dystrophies (DMD) and Becker muscular dystrophies (BMD) and in congenital muscular dystrophy of the Fukuyama type, an alpha-dystroglycanopathy, is accompanied by inflammation of the muscle and cellular oxidative stress that contribute to muscle waisting and its loss of function. A Japanese team has just published the results of a phase IIa clinical trial evaluating in these three diseases, resveratrol, a natural antioxidant (polyphenol) found in grapes and red wine in particular.
Evaluated by a Japanese team in DMD model mice and Fukuyama disease model hamsters, resveratrol improved survival in treated animals and decreased muscle oxidation and inflammation. Based on these results, the same team set up an open-label phase IIa trial including 11 adolescent and adult patients (12 to 46 years old) with DMD (5, including one walking patient), BMD (4, including 3 walking patient) or Fukuyama’s disease (2, both are non walking patient). These patients were treated for 24 weeks with increasing doses of resveratrol: 500 mg / day for the first 8 weeks, 1000 mg / day for the next 8 weeks and 1500 mg / day for the last 8 weeks, a treatment which was well tolerated.
Mixed effects on motor function
Motor function and muscle strength were assessed at 0, 8, 16 and 24 weeks of treatment using the MFM Motor Function Measurement Scale and QMT quantitative muscle testing tests. At 24 weeks, motor function scores increased 10% from 34.6 +/- 29.3 to 38.4 +/- 31.3. A more detailed analysis shows that only some walking patients benefited from the treatment with a motor improvement. The assessment of muscle strength showed an improvement in 2 movements in 10 patients, including 3 who were not walking, for whom the measurement could be made. It shows a 2-fold increase in lifting strength in the shoulders and arms. For finer movements, differences in strength exist without being significant. Finally, the level of creatine kinase in the blood decreases by 34% with treatment.