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Breast cancer drug tamoxifen can help to slow down the incurable hereditary muscle disease. Researchers at the University of Geneva report to colleagues that the drug prolongs the life of mice with this disease.
Myotobular myopathy is a serious hereditary disease that affects one in 50,000 newborns. The genetic defect causes muscle atrophy. In particular, due to the weakness of the respiratory muscles, this rare disease usually leads to death in the first two years of life.
Healing still does not exist, but at least it can slow down the progression of the disease: Researchers from the universities in Geneva and Strasbourg have been promising results in experiments with animals with tamoxifen.
Tamoxifen has been used in breast cancer treatment for years and has some interesting properties regarding the protection of muscle fibers, as announced by the University of Geneva. In a previous study, the researchers had already successfully tested the drug for its effect on more frequent muscle disease, muscular dystrophy of the Douche. This refers to one in 3,500 boys; life expectancy is 30 years. Clinical trials are underway now.
Based on these promising past results, scientists around Leonardo Scaposa have now also tested the efficacy of tamoxifen in models of myotbubular myopathy. The results are published in the journal Nature Communications.
However, both diseases lead to muscle weakness in various ways: in myotobular myopathy there is no enzyme called myotobularine. Without it, it accumulates a protein called dinitin 2, causing the muscles to be atrophied. Tamoxifen starts with Dynamin 2.
Mice with the same symptoms as babies with myotopular myopathy lived twice as long with the lowest dose tested, with the highest dose even seven times longer than untreated mice. The highest dose was – equivalent to the organism of mice – used in breast cancer.
In addition, the drug has slowed progressive muscle paralysis or even led to a slowdown. Muscle strength also increased significantly with treatment.
Early access prevents paralysis
The researchers started treatment with tamoxifen when the first symptoms appeared in mice for about three weeks. The Canadian research group from Toronto began earlier, and mice did not develop signs of paralysis, writes the University of Geneva.
In the meantime, there are research efforts to treat a serious hereditary disease through gene therapy. Its efficiency and reliability, however, should last for years, speculators study study author Olivier Durchis of the University of Geneva.
Because tamoxifen is already approved for human use, and as clinical trials of Douche muscle dystrophy are already under way, researchers hope that tamoxifen treatment will receive much greater approval in myotobular myopathy.