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One drug in 100 years? Metformin Brings New Hope!

2017-01-18 来源:转载自第三方
18 January 2017
  Metformin can inhibit liver gluconeogenesis, increased peripheral tissue insulin sensitivity, in order to reduce blood glucose and insulin concentration, it is the world's most widely used oral hypoglycemic agents. In recent years, more and more studies have also found that metformin also has the function of anti-cancer, anti-aging, prolong life and so on. Recently, researchers from Baylor College of Medicine and the Children's Cancer and Hematology Center in Texas found that metformin might be the first drug to treat drepanocytosis in 30 years.
  Drepanocytosis, also known as hemoglobin S disease, is a recurrent acute exacerbation, organ damage of the multi-system disease, it is the world's most common single-gene genetic disease, millions of people worldwide suffer drepanocytosis. However, there are few breakthroughs in the treatment of drepanocytosis, in 100 years, there is only one drug——hydroxyurea. But the disease-specific targeted therapy is still no breakthrough.
  The pathophysiology of drepanocytosis involves not only red blood cells, but also vascular endothelial cells, inflammation and coagulation disorders. It is Mainly caused by mutations in β-hemoglobin gene, the 17th nucleotide change from thymine into adenine, leading to the 6th amino acid, glutamic acid is replaced by valine, and forming abnormal hemoglobin. This alternative structure leads to deoxidized hemoglobin easy to dissolve and gather and cause vascular obstruction. In addition, sickle-shaped hemoglobin can cause cell membrane distortion, leading to iron overload, so that red blood cell dehydration, stiffness and obstruction.
One drug in 100 years? Metformin Brings New Hope!
  Researchers at Baylor College of Medicine and the Children's Cancer and Hematology Center in Texas launched a study on drepanocytosis in 2011 to identify new drug targets that help patients produce more fetal hemoglobin. Fetal hemoglobin present in the newborn for about 6 months and is replaced by adult hemoglobin. Children with drepanocytosis develop "defective" versions of adult hemoglobin. This defective version of protein can cause red blood cells to become sickle-like, stuck in the blood vessels, causing pain and other symptoms.
  Using full exon sequencing, the researchers found that the FOXO3 gene appears to control fetal hemoglobin formation, and patients with FOXO3 mutations produce fewer fetal hemoglobin. Because metformin is able to induce FOXO3, the researchers used metformin to increase FOXO3 levels in bone marrow cells in patients with drepanocytosis, and the cells produced more fetal hemoglobin. bone marrow cells can produce more fetal hemoglobin when combined with metformin and hydroxyurea.
  At present, the researchers have launched a clinical trial to further investigate the efficacy of metformin in the treatment of drepanocytosis. The major breakthrough shows that metformin may be the first new treatment of drepanocytosis in decades.
Edited by the Editorial Office of Suzhou Yacoo Science Co., Ltd.