PK-R allosteric activator AG-348

{
  "id": "MLC-T-ONC-016100",
  "term": "PK-R allosteric activator AG-348",
  "field": "Oncology and biomedicine",
  "definition": "An orally available, small-molecule, allosteric activator of the red cell isoform of pyruvate kinase (PK-R), with potential to improve hemolytic anemia in patients with pyruvate kinase deficiency (PKD). Upon oral administration, AG-348 binds to and activates PK-R, thereby enhancing glycolytic pathway activity, improving adenosine triphosphate (ATP) levels and reducing 2,3-diphosphoglycerate (2,3-DPG) levels. Mutations in PK-R cause deficiency in PK-R which prevents adequate red blood cell (RBC) glycolysis, leading to a buildup of the upstream glycolytic intermediate 2,3-DPG and deficiency in the PK-R product ATP.",
  "synonyms": [
    "PK-R activator AG-348",
    "red cell pyruvate kinase activator AG-348"
  ],
  "references": [
    "National Cancer Institute Thesaurus"
  ],
  "url": "https://mlchart.com/terminology/oncology/pk-r-allosteric-activator-ag-348/"
}