Neuroprotection - Drugs, Markets and Companies

0. Executive Summary ..........17

1. Introduction .....................21

Definitions ......21

Historical development of neuroprotection ...21

Intrinsic neuroprotective factors ..................22

Neuroprotective gene expression ..................23

Neurotrophic factors .............23

Intrinsic non-enzymatic antioxidants .............24

Intrinsic neuroprotective proteins..................24

Excitatory amino acid transporters (EAATs) ......................24

Extracellular serine protease thrombin ......24

Neuroglobin....................25

Galanin ...25

Prion protein...................26

Rai (Shc C) adaptor protein .....................26

Stem cell factor ...................26

Role of the immune system in neuroprotection ......................26

Pathomechanisms of CNS injury as basis for neuroprotection ................... 27

Role of neuroprotection in various neurological disorders ...28

Neuroprotection and neuroregeneration.......29

Acute versus chronic neuroprotection...........29

Discovery and evaluation of neuroprotective agents............29

Neuroprotective drug discovery.....................29

Discovery of CNS drugs that penetrate the blood-brain barrier.30

In vitro assays for the evaluation of neuroprotective agents ....30

Oxidative injury model to test neuroprotective drugs ..............31

Apoptosis model for designing neuroprotective drugs..............31

Transgenic mouse models of neurological disorders................32

Brain-on-a-Chip™ technology 32

Role of brain imaging in neuroprotective drug discovery and development .......32

Role of single photon emission computed tomography .......32

Functional CT scanning to evaluate cerebrovascular protection ..................32

Magnetic resonance imaging for the evaluation of neuroprotectives ............33

Application of nanotechnology to neuroprotection...................33

Evaluation criteria for potential neuroprotective agents...........34

Evaluation of neuroprotective effect of drugs approved for other indications ....34

2. Neuroprotective Agents....35

Classification of neuroprotective agents .......35

Activated protein C ................39

Activity-dependent neuroprotective protein .40

Adenosine analogs.................40

Propentofylline....................40

Andepressants 41

Antiepileptic drugs as neuroprotectives........41

Phenytoin....42

Valproic acid.42

Levetiracetam......................42

Antiinflammatory agents........43

Aspirin........43

Interleukin-1 antagonists ......43

COX-2 inhibitors ..................43

Nimesulide .....................44

NS-398...44

Minocycline ..44

VP015.........46

Anti-apoptosis agents ............46

Activated protein C...............46

Calpain inhibitors .................47

Caspase inhibitors ................47

Clusterin ......47

DNA binding drugs ...............48

Lithium ........48

Omega-3 fatty acids .............48

Docosahexaenoic acid ......48

Poly (ADP-ribose) polymerase (PARP) inhibitors .....................49

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Prevention of apoptosis by binding of proNGF to Sortilin .........50

TRO19622....50

Antioxidants/free radical scavengers ...........51

Free radical generation .........51

Natural defenses against oxidative stress .......51

Effects of oxidative damage...51

Oxidative damage and aging ...................51

Neuronal damage by free radicals ............52

Oxidative damage and neurodegenerative disorders ..........52

Measures to control oxidative stress ..............52

Categories of therapeutic antioxidants ......53

Alpha-phenyl-tert-butylnitrone.................53

Coenzyme Q10 ...............54

Dihydroergocryptine ........54

Flavonoids......................55

Mitochondria-targeted antioxidants...........55

Nanoparticles as neuroprotective antioxidants ..................55

Neuroleptics as antioxidants ....................56

Nitrones..56

Carbon monoxide and heme oxygenase ........57

Cell transplants secreting neuroprotective substances ........57

Cytokines ........58

Erythropoietin.....................58

Granulocyte colony-stimulating factor ............59

Delta-opioid receptor agonists......................60

FK960 .............60

GABA agonists.60

Gene therapy ..61

Glucagon-like peptide ............62

Glatiramer acetate .................62

Glutamate antagonists...........62

Neuroprotection by scavenging blood glutamate ....................63

N-acylethanolamines for protection against glutamatergic excitotoxicity...........64

Glutamate transporters.........64

Glutamate transporter-mediated neuroprotective effect of drugs.....................64

Glycine-proline-glutamate analogs ...............65

Herbal preparations ...............65

Flavonoid wogonin................66

Ginseng .......66

NMDA receptor ion channel complex.............66

NMDA receptor antagonists ...68

NPS 150668

NMDA NR2B subunit receptor antagonists .68

Ifenprodil 68

Memantine .....................68

Magnesium.....................69

NAALADase inhibitors ......69

3,3-bis (3-fluorophenyl) propylamine........69

Gacyclidine (GK-11) ........69

N-alkylglycines................70

Partial NMDA agonists at glycine site .............70

AMPA receptor modulators ....70

Metabotropic glutamate receptor modulators ..71

Cannabinoids71

Dexanabinol (HU-211) ..........72

Glutathione .....72

Heat shock proteins ...............73

Hormones .......73

Estrogen and neuroprotection73

Neuroprotective effect of estrogen receptor ligands ...........74

Selective estrogen receptor modulators ....74

Receptor-independent neuroprotective effects of estrogens 75

Insulin .........75

Ion Channel modulators.........75

Calcium channel blockers. .....76

Ziconotide ......................76

Na+ channel blockers. ...........76

Neuroprotective potassium channel inhibitors .76

Kynurenine inhibitors.............77

Leukocyte adhesion inhibitors77

Modafinil .........78

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Neural regeneration protein...78

Neurite outgrowth-promoting prostaglandin compounds.....79

Neuroimmunophilins..............79

Cyclosporin ..79

FK506.........80

GPI 1485 .....80

Rapamycin ...80

Neuropeptides81

Corticotropin-releasing hormone ...................81

Neuroprotective peptide NAP .81

Thyrotropin-releasing hormone .....................82

Vasoactive intestinal peptide (VIP). ...............82

Neurotrophic factors ..............83

Activity-dependent neurotrophic factor ..........83

Angiogenesis growth factor-1 83

Bone morphogenetic proteins 83

Brain-derived neurotrophic factor..................84

Ciliary neurotrophic factor .....84

Fibroblast growth factors.......85

Glial cell line-derived neurotrophic factor .......85

Insulin-like growth factor ......86

Nerve growth factor..............86

Neurotrophins......................87

Osteogenic protein-1 ............87

Pigment epithelium-derived factor.................87

Transforming growth factor-b1......................88

Vascular endothelial growth factor.................88

Neurotrophic factor-related neuroprotective agents ................88

Colivelin..88

Gambogic amide .............88

Inosine ...89

Meteorin .89

Oxygen-regulated protein 150 kD (ORP150) .....................89

Prosaptide ......................89

Siagoside (GM1 ganglioside)....................90

Nicotine and nicotinic receptor agonists .......90

Nitric oxide-based Neuroprotection ..............92

Nitric oxide synthase inhibitors .....................92

Nitric oxide mimetics ............92

Nootropics ......93

Piracetam94

Nutraceuticals and food constituents............94

Creatine......94

Curcumrin/ curry..................95

Glyceryltriacetate.................95

Green tea.....96

Nicotinamide 96

Resveratrol/ red wine ...........96

Osmotic diuretics ...................97

Mannitol......97

Osteopontin.98

Oxygen therapeutics ..............98

Oxygen carriers ...................98

Hemoglobin-based oxygen carriers................99

Perfluorocarbons ..................99

Hyperbaric oxygen therapy...100

PPARs as drug targets for neuroprotection .100

Riluzole .........101

Role of RNA interference in neuroprotection......................101

Signaling pathway activator.101

Fructose-1,6-bisphosphate ...101

Sir 2 group of proteins .........102

Statins ..........102

Steroids ........103

Dehydroepiandrosterone ......103

HF0220.......103

Sulforaphane.104

Tauroursodeoxycholic acid...104

Tetanus toxin as a neuroprotective agent ...104

Uncoupling protein 2............105

Vaccines as neuroprotectives......................105

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Non-pharmacological approaches to neuroprotection........105

Environmental enrichment....105

Hypothermia106

Limitations of hypothermia .....................106

Hypothermic neuroprotection in hypoxia-ischemia............107

Hibernation induced by hydrogen sulfide .......107

Ketogenic diet.....................107

Preconditioning-induced neuroprotection.......108

Hypoxic preconditioning ..108

Moderate stimulation of reactive oxygen species ..............109

Chemical preconditioning 109

Transcranial magnetic stimulation ................109

Electrical fields for improvement of cerebral function in neurodegeneration .... 110

Neuroprotective effect of exercise ................110

Hibernation and neuroprotection ..................111

Suspended animation and neuroprotection ....111

3. Neuroprotection in Cerebrovascular Disease ............... 113

Introduction.113

Epidemiology of stroke ........113

Pathophysiology of cerebral ischemia .........114

Calcium overload ................114

Ion channel dysfunction in stroke .................115

Role of oxygen free radicals in cerebral ischemia...................115

Role of Nitric oxide (NO) in cerebral ischemia 115

Glutamate as a marker of stroke ..................115

Cerebral edema in stroke .....116

Gene expression in response to cerebral ischemia .................116

Induction of heat shock proteins in stroke .....116

Role of cytokines and adhesion molecules in stroke ...............117

Tumor necrosis factor (TNF)-a ................117

Interleukin (IL)-1 and IL-6 .....................117

Adhesion molecules ........117

DNA damage and repair in cerebral ischemia .118

Role of neurotrophic factors..118

Problems requiring investigation of the role of NTFs in stroke .................. 119

Role of Poly(ADP-ribose) polymerase (PARP) gene.................119

Role of protease-activated receptor 1 ...........119

Reperfusion injury after cerebral ischemia .119

Neuroprotection according to zones in cerebral infarction.120

Zone of ischemic infarction...120

Penumbra ...120

Current management of stroke ...................121

Neuroprotection in stenosis of intracranial arteries ................121

Neuroprotective therapies for stroke ..........122

Agents in research/preclinical stage for neuroprotection in stroke.................. 123

Acid-sensing ion channel blockers ................124

AMPA receptor antagonists as neuroprotectives for stroke ......125

Zonampanel ..................125

Anti-apoptotic neuroprotectives ...................125

NIM811..125

Creatine as neuroprotective in stroke.......126

Lithium as a neuroprotective in stroke .....126

TUDCA as a neuroprotective in stroke......126

Anti-HMGB1 monoclonal antibody.................127

Antioxidant approaches........127

Dehydroascorbic acid......127

Tocotrienols...................128

Carnosine as a neuroprotective in stroke..128

Antiglutamate compounds....129

202W92.129

AR-R15896AR................129

MRZ 2/576 ....................129

L-Phenylalanine .............130

Arimoclomol for stroke.........130

Cardiac glycosides as neuroprotectives in stroke ...................130

Clenbuterol .130

Cox-2 inhibitors for ischemic stroke ..............131

Enoxaparin..131

Estrogen for stroke..............131

Granulocyte-macrophage colony-stimulating factor for cerebral ischemia ....... 133

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Histamine H2-receptor antagonists ...............133

Inosine for stroke................133

Intravenous immunoglobulin as neuroprotective in stroke .134

Insulin-like growth factor-I...134

Ischemic preconditioning for neuroprotection in stroke ..........134

Ketone bodies.....................135

Minalocorticoid receptor blockade for neuroprotection............136

Multifunctional neuroprotective agents..........136

Neuroserpin as a neuroprotective in stroke....136

N-2-mercaptopropionyl glycine ....................137

NeuroAid.....137

Neurotrophic factors as neuroprotectives for stroke ...............137

AX200 ...137

Brain-derived neurotrophic factor ............138

Neuregulin-1 .................138

NO-based strategies for neuroprotection in cerebral ischemia .138

Peroxisome proliferator-activated receptor-g agonists ............139

PGE2 EP2 receptor activation 139

Pioglitazone for reduction of stroke risk in diabetes ...............139

Progesterone ......................139

Proteosome inhibitors ..........140

Statins for stroke prevention 140

Thrombosis inhibitors ..........140

Aspirin ...141

Clopidogrel....................141

Dipyridamole .................141

CBL1309 141

SB-239063..142

Sildenafil....142

Src receptor blockade ..........142

Stroke vaccine ....................143

SUN N4057 .143

Tiagabine ....143

Topiramate..143

Zonisamide .144

Neuroprotectives in reperfusion injury ..........144

Non-pharmacological neuroprotective therapies for stroke......145

Preconditioning for neuroprotection against cerebral ischemia.................. 145

Hypothermia for neuroprotection in acute stroke ..............145

Hyperbaric oxygen therapy for neuroprotection in acute stroke................ 146

Infrared laser therapy for ischemic stroke 147

Neurorehabilitation in relation to neuroprotection in stroke147

Cell therapy for stroke .........147

Gene therapy for neuroprotection in cerebrovascular disease..148

Neuroprotective genetic vaccine..............149

COX-1/PGIS and COX-1 gene transfer in cerebral ischemia ..................... 150

Transfer of the bcl-2 gene for neuroprotection in cerebral ischemia .......... 150

Neuroglobin gene therapy ......................150

Neuroprotective therapies for cerebral ischemia: clinical trials 150

Albumin .152

Free radical scavengers...152

DP-b99 ..154

Erythropoietin as a neuroprotective in stroke ...................154

MaxiPost (BMS-204352)..154

Minocycline for neuroprotection in stroke ......154

Perindopril.......