Peptide drugs are expected to treat rare anemia and polycythemia at the same time

  A new study has found that a synthetic peptide (minihepcidins) may have potential therapeutic effects on β - thalassemia and polycythemia vera, two serious blood diseases Although these two diseases have opposite effects on erythropoiesis, minihepcidins can help to restore the normal level of erythrocytes and relieve the abnormal increase of spleen; in β - thalassemia, the drug can also control the excessive accumulation of iron ions and avoid the serious toxic effects caused by excessive iron ions Stefano rivella, the author of the paper, said: "it seems counterintuitive for us to treat two different diseases at the same time with one compound, but by limiting the absorption of iron ions, this compound can indeed normalize the level of red blood cells in animal models If these preclinical results can be transformed into human beings, it will provide new treatment options for the treatment of these two blood diseases " In this study, the researchers used a mouse model of β - thalassemia and polycythemia vera to study the therapeutic effect of minihepcidins on these two diseases In β - thalassemia, gene mutation impairs hemoglobin synthesis, affects the ability of red blood cells to carry oxygen and leads to anemia However, due to the low content of ferritin, the body continues to accumulate iron ions, which forms a vicious circle - more and more red blood cells are damaged, which may also cause serious damage to the liver and heart In polycythemia vera, the rare gene mutation drives the over production of red blood cells, and gradually forms tomato sauce like thick state This increases the risk of high blood pressure and thrombosis, and may cause stroke After careful study, the researchers found that minihepcidins can normalize the level of red blood cells and relieve anemia and iron overload in young β - thalassemia mice In older mice, the compound improves red blood cell production and does not interfere with the effect of a chelating drug used to remove excess iron deposition In the mouse model of polycythemia vera, minihepcidins can weaken the absorption of iron ions, reduce the number of red blood cells, and also alleviate the abnormal increase of spleen Minihepcidins is a modified ferritin analogue, which can simulate the function of ferritin to regulate iron ions Minihepcidins is smaller than the full-length ferritin, but it has long-term stability and long-term biological activity in animals Previous studies have found that minihepcidins treatment can prevent iron overload in the mouse model of hemochromatosis The results were published in the international academic journal blood July 25, 2019