New hope for Sickle Cell Anemia…

Red blood cells are round, flexible and carry oxygen throughout our bodies. Children and adults with Sickle cell disease have red blood cells in the shape of a crescent moon. These cells become sticky and block blood flow which can in turn cause infection and organ damage. This is caused by a defect in the gene that tells the body to make the iron rich compound in red blood cells (hemoglobin). The disease affects mostly African Americans and Latino families. Complications can range from anemia to infections to vision problems. In the past the treatment involved hydroxyurea (cancer drug) with bad side effects or bone marrow transplants. The FDA has just approved 2 drugs Oxbryta and Adakveo. These drugs can prevent hemoglobin in RBC from sticking together and from forming crescent shapes. In a study of 274 participants ages 12 to 65 there was a 72 to 89% decrease in anemia caused by Sickle Cell Disease. What is interesting about having the disease is that those individuals are less susceptible to being infected with malaria. Their molecular machinery for cleaning dead cells also prevents the malarial parasite from infecting them.

 

https://www.mayoclinic.org/diseases-conditions/sickle-cell-anemia/symptoms-causes/syc-20355876

https://www.google.com/search?q=oxbryta+approval&rlz=1C5CHFA_enUS892US892&oq=Oxbryta&aqs=chrome.2.69i57j0i512l9.5942j0j15&sourceid=chrome&ie=UTF-8