Novel therapies in sickle cell disease

Abstract

Despite an increased understanding of the pathophysiology of sickle cell disease (SCD), there remains a paucity of available agents for the prevention and treatment of specific SCD-related complications. Recently, there has been significant progress in the development of novel drugs for this disease. These agents, which increase the production of fetal hemoglobin, improve red blood cell hydration, increase the availability of nitric oxide and possess anti-inflammatory effects, are in varying stages of clinical development. With the complex pathophysiology of SCD, it is unlikely that a single agent will prevent or treat all the sequelae of this disease. As a result, patients may benefit from treatment with a combination of agents that possess different mechanisms of action. This overview discusses selected novel agents that appear promising in SCD.