Pilot study in Becker and limb-girdle MD of prednisone given once weekly. Once a week treatment with a corticosteroid was seen to lower some biomarkers of muscle damage and inflammation, helping to improve muscle function in people with some forms of muscular dystrophy , according to a pilot study in the U.
Prednisone was given once weekly for six months to 19 people with limb-girdle muscular dystrophy LGMD , a disease type that primarily affects the hips and shoulders, and to one adult with Becker muscular dystrophy BMD , which most often affects the legs. A study aim was to investigate whether such low-dose treatment provides the benefits of corticosteroids without the side effects of long-term use. A group of diseases, muscular dystrophy is caused by mutations that disrupt the activity of genes important for muscle health, leading to muscle weakness and wasting that progressively worsen over time.
Patients are often treated with corticosteroids, or glucocorticoids, to reduce inflammation, but long-term use is linked to obesity, insulin resistance, and osteoporosis, among other side effects. Previous studies in mouse models of muscular dystrophies showed that once-weekly glucocorticoid treatment enhanced muscle strength and reduced tissue fibrosis, or scarring. Sex- and age-matched healthy adults were included as a control group for comparison.
A total of proteins considered muscular dystrophy biomarkers were identified, with found at higher baseline levels and at lower baseline levels in patients than in controls. Proteins at increased levels were mainly associated with muscle tissue function and differentiation when cells become specialized , in line with the leak of muscle proteins into the bloodstream due to muscle tissue breakdown.
Conversely, proteins at lower levels were associated with cell attachment to neighbor cells, organization and stabilization of cellular structures, and regulation of calcium levels inside cells. Six months of intermittent glucocorticoid treatment significantly increased the levels of 24 proteins and decreased those of others, largely in line with the known effects of glucocorticoids on the immune system.
