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Tamoxifen breast cancer drug could help to slow down at least incredible legacy muscle disease. Researchers at the University of Geneva report with colleagues that the drug extends the life of mice with the disease.
Myotoby myopathy is a national disease that affects one in 50,000 newborn boys. A genetic deficiency causes the muscle to perform. In particular, due to the weakness of respiratory muscle, this rare disease usually leads to death during the first two years of life.
Healing does not exist yet, but at least it could slow down the sequence of the disease: Researchers from the Universities of Geneva and Strasbourg report promising results in experiments with tamoxifen.
Tamoxifen has been used for years in breast cancer therapy and has some interesting property in terms of protecting muscle fibers, as announced by the University of Geneva. In a previous study, researchers had already successfully experienced the drug for its impact on more common muscle disease, Duchenne muscle distrophy. This covers one of 3500 boys; life expectancy is 30 years. Clinical trials are now under way.
Based on these earlier promising results, scientists around Leonardo Scapozza now experience the effectiveness of tamoxifen in myotubular myopathy mouse models. The results are reported in the "Nature Communications" magazine.
However, both diseases lead to muscle weakness in different ways: in myotubular myopathy, there is no enzyme of myotubularin and myotubularin. Without it, a protein of the dynamic 2 name builds up, causing the muscle to perform. Tamoxifen starts with Dynamics 2.
Mice with the same symptoms as myotathy myopathy babies lived twice as long as the lowest dose tested, with the maximum dose even more than seven times longer than untreated mice. The upper two were – which is equivalent to the mouse organism – used in breast cancer.
In addition, the drug slows down the progressive muscle paralysis or even has ended. The muscle strength also increased significantly.
Early approach prevents palsy
The researchers began treatment with tamoxifen when the first symptoms in the mice appeared around three weeks. Canada's Canadian research group started even earlier, and the mice had not developed any signs of barálysis, written by the University of Geneva.
In the meantime, there are also research attempts to treat the serious inherited disease through gen therapy. Its effectiveness and safety, however, take years, mentioning the author of the study of Olivier Dorchies at the University of Geneva.
Because tamoxifen has already been approved for human use, and as clinical trials for Duchenne muscular dystrophy are already underway, researchers hope that tamoxifen treatment will receive much faster approval in myopathy myotubular