Recent developments on using pyruvate kinase activators as a new approach for treating sickle cell disease

Abstract

Pyruvate kinase (PK) is a key enzyme in glycolysis, the sole source of ATP, which is essential for all energy-dependent activities of red blood cells. Activating PK shows great potential for treating a broad range of hemolytic anemias beyond PK deficiency, as they also enhance activity of wild-type PK. Motivated by observations of sickle cell complications in sickle trait individuals with concomitant PK deficiency, activating endogenous PK offers a novel and promising approach for treating patients with sickle cell disease.