Nitric Oxide - Therapeutics, Markets and Companies

0. Executive Summary ................. 11

1. Introduction ....... 13

Free radicals ............... 13

Nitric oxide ................. 13

Historical aspects .......13

Role of NO in biology and medicine...14

Nitric oxide synthase .. 15

Structure and function of NOS .........15

Inducible nitric oxide synthase.........17

iNOS gene............17

Regulation of iNOS 17

Regulation of endothelial nitric oxide synthase ........17

Interaction between eNOS and other proteins....18

Tetrahydrobiopterin...................18

Methods of study of NO and NOS .....19

Bioimaging of NO ..19

Assays of NO in tissues ..............19

Metabolomics approach to study of NO metabolism ................20

2. Nitric Oxide Pathways ............. 21

Introduction............... 21

Mechanisms action of NO ................ 22

NO-cGMP pathway ...... 22

Soluble guanylyl cyclase as the NO receptor ...........23

Oxidative stress pathways .............. 23

NO and oxidative stress ..................23

Oxidative stress and the NO-cyclic GMP signal transduction pathway ..................24

NO and platelets ........26

Mitochondrial NO-cytochrome c oxidase signaling pathway...........26

Dual role of NO as a free radical and a scavenger....27

NO and carbon monoxide ................ 28

3. Role of NO in Physiology.......... 31

Homeostasis of NO...... 31

NO as a biomarker ...... 31

Functions of NO in various systems of the body... 31

NO and proteins.........32

A proteomic method for identification of cysteine S-nitrosylation sites ...........32

Protein S-nitrosylation and intracellular transport processes ....33

NO and mitochondria..33

Mitochondrial permeability and reperfusion injury...................34

Role of NO in the cardiovascular system.................34

NO and atrial natriuretic peptide .35

NOS in the cardiac myocyte........35

NO and the autonomic control of the heart rate .36

NO and vasodilatation................37

Role of NO in the plasma compartment .............38

Measurement of NO as a biomarker of cardiovascular function.38

Hemoglobin, oxygen and nitric oxide 38

Myoglobin and NO .40

NO and pulmonary circulation..........40

Role of NO in the regulation of hypoxic pulmonary vasoconstriction...............41

Role of NO in the nervous system ....41

Neurovascular coupling of COX-2 and nNOS ......42

Neuroglobin..........42

Acute actions of NO in the CNS pathways..........43

Role of NO in memory and learning ..................43

Role of NO in synaptic plasticity ..43

Role of NO in the peripheral nervous system .....43

Role of NO in the cochlea ................44

NO and neuroendocrine function ......44

NO and pregnancy......44

Role of NO in penile erection............44

Role of NO in immune regulation......45

Role of NO in temperature regulation46

Role of NO in gastrointestinal system46

Role of NO in kidney function...........46

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Role of NO in liver ......47

NO and hypertension .................47

Role of NO in the skin .47

4. Role of NO in Diseases............. 49

Introduction............... 49

Cytotoxicity of reactive nitrogen species.................49

Peroxynitrite, mitochondria and cell death..............49

Diseases involving oxidative stress and nitric oxide ................ 51

Stress-related disorders.................. 52

Role of NO in allergic disorders....... 52

Inflammatory diseases ................... 52

Autoimmune disorders 53

Role of NO in rheumatoid arthritis ....54

Role of NO in infections................... 54

NO-mediated cytoprotection in bacteria .................55

Trypanosomiasis ........55

Malaria and iNOS polymorphism.......56

Susceptibility of Mycobacterium leprae to NO..........56

Inhaled NO for tuberculosis .............56

Septic shock ..............57

Viral infections...........57

Role of NO in anaphylactic shock .... 58

Role of NO in neurological disorders  59

Neurodegenerative diseases ............59

NO-induced mitochondrial dysfunction in neurodegeneration ...59

White matter disorders ..............59

Amyotrophic lateral sclerosis ......59

Alzheimer's disease ...................60

Role of NO in pathophysiology of Alzheimer's disease .............60

Role of ApoE genotype ...............63

Parkinson's disease ...................63

Traumatic brain injury 65

Epilepsy 65

Stroke .66

Pathophysiology of cerebral ischemia................66

Role of NO in cerebral ischemia...67

eNOS gene polymorphisms as predictor of cerebral aneurysm rupture ...........68

Role of NO in assessment of cerebral and retinal blood flow.....68

Role of NO in neuroprotection .....69

Stroke and heart disease............69

Role of NO in peripheral neuropathy .69

iNOS induction in experimental allergic neuritis..69

Role of NO in sciatica.................69

Role of NO in the pathogenesis of muscular dystrophy .................70

Role of NO in psychiatric disorders . 70

NO-dysregulation in schizophrenia....70

Role of NO in pathomechanism of cardiovascular disorders..... 71

Oxidative stress as a cause of cardiovascular disease ..................71

Role of NO in pathomechanism of cardiovascular diseases............71

Role of iNOS in cardiovascular disease ..............72

Role of eNOS in cardiovascular disease .............72

Role nNOS in cardiac arrhythmia and sudden death................73

NO and atherosclerosis ...................73

Role of NO in cardiopulmonary disorders................74

Role of NO in disturbances of vasodilation ..............75

Role of NO in hypercholesterolemia ..75

Pulmonary hypertension..................76

NO and systemic hypertension.........77

Coronary artery disease ..................77

Role of NO in the pathophysiology of angina pectoris...................78

Congestive heart failure ..................78

Myocardial ischemia/reperfusion injury ..................78

Role of NO in sickle cell disease ...... 80

Role of NO in respiratory disorders . 80

Role of NO in the pathophysiology of asthma ..........80

iNOS gene polymorphisms in asthma................81

Role of S-nitrosoglutathione in bronchodilation in asthma.............82

Monitoring of exhaled NO ................82

Nasal NO as a biomarker of response to rhinosinusitis therapy......83

Elevated urinary NO as a biomarker of improved survival in ARDS 83

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Role of NO in renal disorders .......... 84

Role of NOS in diabetic nephropathy.84

Role of NO in cancer.... 84

Tumor hypoxia and NO ...................85

NO and p53 mutations86

NO and matrix metalloproteinase .....86

Role of NO in angiogenesis in cancer 86

Role of NO in diseases of the eye .... 88

Glaucoma.................88

Role of NO in metabolic disorders ... 88

Obesity 88

Diabetes mellitus .......88

Role of NO in gastrointestinal disorders............... 89

Role of L-arginine in intestinal adaptation...............89

Role of NO in irritable bowel syndrome ..................89

Role of NO in inflammatory bowel diseases.............89

Role of NO in celiac disease .............90

Role of NO in diabetic gastroparesis..90

NO and diseases of the liver............ 90

Cirrhosis of liver.........90

Hepatic encephalopathy ..................91

Role of NO in skin disorders ............ 91

Role of NO and oxidative stress in the aging skin ....91

Role of NO in wound healing............91

Role of NO in pain ....... 92

NO and pain of spinal cord origin......92

NO interaction with other receptors in pain.............92

nNOS and pain...........92

Role of NO in various types of pain...93

Neuropathic pain...93

Role of NO in diabetic neuropathy 93

NO in oral and facial pain ...........93

Role of NO in migraine ...............94

Role of NO in osteoarthritis.........94

NO and Fibromyalgia syndrome ..95

Role of spinal NO in analgesic action.................95

Role of NO in nicotine addiction ...... 96

Role of NO in carbon monoxide poisoning............ 96

Role of NO in radiation damage....... 96

5. Pharmacology of Nitric Oxide .. 99

Introduction............... 99

Cytoxic vs cytoprotective role of NO  99

Antioxidants............... 99

Ebselen.............. 100

Nicaraven........... 100

Nitroxides .......... 101

Antioxidants in relation to NO ........ 101

Limitation of antioxidant therapy in congestive heart failure ....... 102

NO-related drugs ...... 102

Drugs that activate endothelial NO production ...... 103

Dehydroepiandrosterone .......... 103

Drugs that scavenge free radicals/NO .................. 104

Peroxynitrite scavengers .......... 104

Ruthenium (III) polyaminocarboxylates .......... 104

Nitrones............. 104

Drugs that inhibit NO 105

Ginko biloba ....... 105

Epigallocatechin .. 105

NO Therapeutics ....... 105

Nitric oxide as an antioxidant......... 105

Nitric oxide donors ... 106

Nitroglycerine/glycerine trinitrate ................... 107

Isosorbide dinitrate ................. 107

Sodium nitrite..... 107

Organic nitrites ... 108

NO-releasing NSAIDs............... 108

COX-inhibiting NO-donors ........ 109

Grafting of NO-releasing structures on to existing drugs........ 111

Mesoionic Oxatriazoles............. 113

Adding NO-donating structures to extend life cycle of existing drugs............ 113

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Cysteine-containing NO donors . 113

Ferrous nitrosyl complexes ....... 114

Syndnonimines ... 114

S-Nitrosothiols .... 114

Diazeniumdiolates ................... 115

COX-2 inhibitors.. 115

NO hydrogels...... 116

Novel NO donors . 116

NO mimetics....... 116

Comparison of classical nitrates, grafted NO donors, and NO mimetics......... 117

NO donors and soluble guanylate cyclase activation.............. 117

NO donors for increasing the efficacy of chemotherapy ......... 118

Modulators of cyclic guanosine-3',5'-monophosphate-dependent protein kinases ................... 118

L-Arginine ............... 119

Delivery of nitric oxide .................. 119

Targeted delivery of NO donors. 120

Nitric oxide delivery by encapsulated cells ....... 120

NO-lipid combination ............... 120

NO-releasing coating to prevent infection of implanted devices ................... 121

Factors that enhance availability of NO.............. 121

NOS-modulating drugs.................. 121

Drugs that activate eNOS.............. 122

Statins............... 122

Angiotensin converting enzyme inhibitors........ 123

17 Beta-estradiol  123

C-2431 .............. 123

NOS inhibitors ......... 123

Rationale of NOS inhibitors....... 123

Design of NOS inhibitors .......... 125

Selective iNOS inhibitors .......... 125

Non-amino acid-based inhibitors  126

Aminoguanidine .. 126

Heme ligands...... 127

Pterin antagonists ................... 127

Fused-ring bio-isoteric models of arginine as NOS inhibitors .. 127

nNOS inhibitors... 127

Lubeluzole.......... 129

Neurotrophic factors ................ 129

Concluding remarks about NOS inhibiting drugs ................... 129

NO and stem cell-based therapy ... 130

Nitric oxide and gene therapy ....... 131

NOS gene transfer.... 131

Inhibition of NOS by antisense technology............ 132

Drugs that modulate NO action on platelets....... 132

Action of NO and NO donors on platelets.............. 133

NOS inhibitors and NO scavengers . 133

Phosphodiesterase inhibitors........ 133

Activators of soluble guanylate cyclase.............. 133

YC-1.. 133

A-350619................ 134

Bay 41-2272 ........... 134

Secondary role of NO in the action of drugs ....... 134

Selective serotonin reuptake inhibitors................. 134

P2Y receptors and NO ................... 135

Calcium channel blockers and NO... 135

Nitric oxide-based transdermal drug delivery ........ 135

NO and nutraceuticals................... 135

L-arginine as a nutraceutical.......... 136

Oleuropein .............. 136

Role of NO in beneficial effects of chocolate.......... 136

6. Therapeutic Applications ....... 139

Introduction............. 139

Role of NO in the management of pulmonary disorders ......... 139

Manufacture of NO gas for inhalation ................... 139

NO inhalation for acute respiratory distress syndrome ............... 139

NO inhalation for premature children with pulmonary dysplasia................... 140

NO inhalation for premature children with respiratory failure . 140

Pulmonary hypertension................ 141

NO-based treatment of pulmonary hypertension .................. 141

Inhaled nebulized nitrite for neonatal pulmonary hypertension 142

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Gene therapy for pulmonary hypertension....... 142

Asthma................... 142

iNOS inhibitors for asthma........ 143

NO for bronchodilation in asthma ................... 143

Role of NO in acute lung injury after smoke inhalation ............... 143

Cardiovascular disorders............... 144

Role of NO in cardioprotection........ 144

Role of NO in the management of angina pectoris . 144

Role of NO in therapy of coronary heart disease.... 145

NO-releasing aspirin in patients undergoing CABG. 145

Management of coronary restenosis  146

Modified NO donors ................. 146

NO-generating stent for coronary restenosis.... 146

eNOS gene therapy for restenosis .................. 147

Congestive heart failure ................ 148

NO-based therapy for congestive heart failure . 148

eNOS gene therapy for congestive heart failure ................... 149

Gene transfer of nNOS in congestive heart failure ................ 149

NO-based therapy for management of cardiogenic shock ........... 149

Prophylaxis of cardiovascular disorders ................ 150

Prevention of atherosclerosis with aging.......... 150

Peripheral vascular disorders ......... 150

Peripheral atherosclerotic arterial disease........ 150

Peripheral ischemic disease ...... 151

An eNOS mutant as therapeutic for peripheral vascular ischemia................. 151

Raynaud's phenomenon ........... 152

Neurological disorders .................. 152

Cerebrovascular ischemic disorders  152

Attenuation of NO for neuroprotection in cerebral ischemia.... 153

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