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Table 1 Drug candidates exhibiting benefits in sickle cell disease

From: Precipitating factors and targeted therapies in combating the perils of sickle cell disease--- A special nutritional consideration

Drug name Action mechanism Reference
Butyrate HDAC inhibition, mood stabilization [110, 111]
Decitabine DNA demethylation [112]
Trichostatin A Increases HbF level; decrease adhesion of cells to vessel wall [113]
Pomalidomide Gammaglobin activation by Histone deacetylase [114]
Senicapoc Improves RBC hydration [115]
Nitric Oxide Increase NO [116]
Tinzaparin Decreases P-selectin-mediated acute pain episodes [117]
6R-BH4 Increases NO; improve endothelial function [118, 119]
Sidenafil Increases NO [116]
Eptifibatide Decreases platelet aggregation and decreases CD40 ligand release [120]
Statins Improves endothelial function [121]
Dexmethasone Decrease inflammation [122]
Nix-0699 Uncertain, but inhibits acute painful crisis [44, 123, 124]
Intravenous immunoglobulin (IVIG) Decreases the number of leukocytes & acute pain episodes [44, 123, 124]
Vorinostat, panobinostat HDAC inhibition [113]
GMI-1070 Pan-selectin inhibitor [45]
Propranolol Inhibits RBC adhesion to the endothelium [125]
Regadenoson A2AR agonist, blocks iNKT activation [126]
Zileuton 5-lipoxygenase inhibitor, used in asthma [127]
Fructose-1,6-diphosphate (FDP) Reduces ischemia–induced tissue damage [128]
Prasugrel ADP receptor blockade [129]
MP4CO PEG carboxy-hemoglobin [130]
Acetyl-L-carnitine Decreases lipid peroxidation [90]
Alpha-lipoic acid Inhibits NFkB, increases glutathione [131]
NAC Increased glutathione [132]
Omega-3 fatty acids Decreases VOC events [18]
Glutamine Increases NADPH [133]
IV magnesium Vasodilatation [134]
Aes-103 Binds sickle hemoglobin and shifts oxy-hemoglobin dissociation curve to the left [90]
Poloxamer-188 Non-ionic surfactant, improves micro-vascular flow [135]
L-arginine Substrate for NO [116]
  1. HbF fetal hemoglobin, NO nitric oxide, RBC red blood cell, IVIG intravenous immunoglobulin, HDAC Histone deacetylases, Nix-0699 Niprisan, GMI-1070 Rivipansel, FDP Fructose-1,6-diphosphate, NADPH Nicotinamide adenine dinucleotide phosphate, A2AR Adenosine A2A receptor, PEG Polyethylene glycol, ADP Adenosine diphosphate, iNKT Invariant natural killer T cells
  2. Few drug candidates may exhibit multiple mechanisms of action