Novel agents in clinical trials
| Category* . | Therapeutic agent . | Mechanism of action . | Reference . |
|---|---|---|---|
| HbF augmentation | Vorinostat, panobinostat | HDAC inhibition | 43 |
| Sodium dimethylbutyrate | HDAC inhibition | 31 | |
| Decitabine | DNA demethylation | 33 | |
| Pomalidomide | Histone acetylation of γ-globin promoter | 30 | |
| Adhesion | GMI-1070 | Pan-selectin inhibitor | 21 |
| IVIG | Inhibits neutrophil activation and RBC capture | 22, 23, 25 | |
| SelG1 | Humanized anti-P-selectin monoclonal antibody | Selexys | |
| Heparin (tinzaparin) | Inhibits P-selectin | 37 | |
| Propranolol | Inhibits RBC adhesion to the endothelium | 7 | |
| Inflammation | Regadenoson | A2AR agonist, blocks iNKT activation | 39 |
| Statins | Anti-inflammatory | 44 | |
| Zileuton | 5-lipoxygenase inhibitor, used in asthma | 45 | |
| Fructose-1,6-diphosphate (FDP) | Reduces ischemia–induced tissue damage | 46 | |
| MP4CO | PEG carboxy-hemoglobin | 47 | |
| Antiplatelet therapy | Prasugrel | ADP receptor blockade | 42 |
| Eptifibatide | αIIb/β3 antagonist | 48 | |
| Oxidative injury | Omega-3 fatty acids | 40 | |
| Glutamine | Increases NADPH | 49 | |
| NAC | Increased glutathione | 41 | |
| Alpha-lipoic acid | Inhibits NF-κB, increases glutathione | 50 | |
| Acetyl-L-carnitine | Decreases lipid peroxidation | ||
| Antisickling agent | Aes-103 | Binds sickle hemoglobin and shifts oxyhemoglobin dissociation curve to the left | 51 |
| Viscosity | Poloxamer-188 | Non-ionic surfactant, improves microvascular flow | 52 |
| Vascular tone | IV magnesium | Vasodilatation | 53 |
| NO | L-arginine | Substrate for NO | 54 |
| Category* . | Therapeutic agent . | Mechanism of action . | Reference . |
|---|---|---|---|
| HbF augmentation | Vorinostat, panobinostat | HDAC inhibition | 43 |
| Sodium dimethylbutyrate | HDAC inhibition | 31 | |
| Decitabine | DNA demethylation | 33 | |
| Pomalidomide | Histone acetylation of γ-globin promoter | 30 | |
| Adhesion | GMI-1070 | Pan-selectin inhibitor | 21 |
| IVIG | Inhibits neutrophil activation and RBC capture | 22, 23, 25 | |
| SelG1 | Humanized anti-P-selectin monoclonal antibody | Selexys | |
| Heparin (tinzaparin) | Inhibits P-selectin | 37 | |
| Propranolol | Inhibits RBC adhesion to the endothelium | 7 | |
| Inflammation | Regadenoson | A2AR agonist, blocks iNKT activation | 39 |
| Statins | Anti-inflammatory | 44 | |
| Zileuton | 5-lipoxygenase inhibitor, used in asthma | 45 | |
| Fructose-1,6-diphosphate (FDP) | Reduces ischemia–induced tissue damage | 46 | |
| MP4CO | PEG carboxy-hemoglobin | 47 | |
| Antiplatelet therapy | Prasugrel | ADP receptor blockade | 42 |
| Eptifibatide | αIIb/β3 antagonist | 48 | |
| Oxidative injury | Omega-3 fatty acids | 40 | |
| Glutamine | Increases NADPH | 49 | |
| NAC | Increased glutathione | 41 | |
| Alpha-lipoic acid | Inhibits NF-κB, increases glutathione | 50 | |
| Acetyl-L-carnitine | Decreases lipid peroxidation | ||
| Antisickling agent | Aes-103 | Binds sickle hemoglobin and shifts oxyhemoglobin dissociation curve to the left | 51 |
| Viscosity | Poloxamer-188 | Non-ionic surfactant, improves microvascular flow | 52 |
| Vascular tone | IV magnesium | Vasodilatation | 53 |
| NO | L-arginine | Substrate for NO | 54 |
Some therapies have multiple mechanisms of action.