Steroids were probably the only cure for recurrent inflammation and disease progression in patients with muscular dystrophy. However, according to the recent study published in American Journal of Pathology a new way of treatment was discovered.
Scientists found out that selective estrogen receptor modulators (SERMs), namely Tamoxifen and Raloxifene, can greatly improve the function of respiratory, skeletal and heart muscles. These drugs also enhanced bone thickness in mice of both sexes having the same genetic deviations as in patients with muscular dystrophy.
According to Dr. Qi Long Lu, director of the Muscular Dystrophy Research Laboratory, there is an advantage of SERMs treatment comparatively to steroids treatment. Firstly, steroids make improvement of muscular functions to a lesser extent than SERMs do. Secondly, SERMs increase bone thickness, meanwhile steroids may cause osteoporosis and increase bone fragility.
During this study a group of mice with genetic defects identical to human muscular dystrophy (including dystroglycanopathy) was treated with Tamoxifen (2, 10, or 50 mg/kg), Raloxifene (50 or 100 mg/kg). The experiment lasted for one year. Researchers found out that these SERMs delay or even stop the progress of this disorder.
In one month, Tamoxifen and Raloxifene significantly reduced muscular pathology and the number of degenerating fibers. In one year, control group displayed great variety of muscle fiber size with multiple inflammatory foci. Whereas the treatment group had much less damage and inflammation.
Besides, SERMs also reduced collagen accumulation in limb muscles and increased the function of all types of muscles including cardiac and respiratory ones.
Additional benefits of Tamoxifen and Raloxifene
Among other SERM benefits one should mention:
- Elimination of focal infiltration in the diaphragm
- Improvement of breathing ability.
- Enhanced bone density in the tibia and femur,
- Improvement of grip strength of both forelimb and hindlimb muscles
- Increase of running ability on a treadmill test depending on the dose
“As we can see, SERMs therapy has high therapeutic potential and can be a very efficient alternative to steroid treatment,” noted Dr. Qi Long Lu.
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