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Table 3 Repurposed agents currently in Phase II in the Alzheimer’s disease development pipeline (as of February 27, 2020)

From: Repurposed agents in the Alzheimer’s disease drug development pipeline

Agent name

Phase

Agent mechanism class

Therapeutic purpose under study

Drug class

FDA-approved indication(s)

Therapeutic field

ClinicalTrials.gov ID(s) (EudraCT ID)

Benfotiamine

II

Metabolic

Improve multiple cellular processes; minimize decline in glucose utilization (DMT)

Synthetic thiamine

Diabetic nephropathy; type 2 diabetes

Antidiabetic

NCT02292238

Candesartan

II

Anti-amyloid, neuroprotective, metabolic

Improve vascular function and reduce amyloid (DMT)

Angiotensin II Receptor Blocker

Hypertension; heart failure

Cardiovascular

NCT02646982

Cilostazol

II

Neuroprotective

Reduce accumulation of amyloid and reduce tau phosphorylation; improve cerebral circulation (DMT)

Antiplatelet

Intermittent claudication

Hematologic-oncologic

NCT02491268

Dapagliflozin

I/II

Metabolic

Improve insulin sensitivity (DMT)

Sodium-glucose co-transporter 2 inhibitor

Adjunct therapy for type 2 diabetes

Antidiabetic

NCT03801642

Daratumumab

II

Anti-inflammatory

Broad range immunomodulatory effects; regulate microglia (DMT)

Human antibody targeting CD38

Multiple myeloma

Hematologic-oncologic

NCT04070378

Dasatinib + Quercetin (Combo)

I/II

Anti-inflammatory

Senolytic therapy approach to reduce senescent cells and tau aggregation (DMT)

Tyrosine kinase inhibitor + antioxidant/anti-inflammatory (Flavonoid)

Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase

Hematologic-oncologic

NCT04063124

Deferiprone

II

Anti-Amyloid, neuroprotective

Reduce reactive oxygen species that damage neurons; effect on amyloid and BACE pathology (DMT)

Iron chelator

Iron overload (thalassemia syndromes)

Hematologic-oncologic

NCT03234686

Dronabinol

II

Neurotransmitter-based

Improve neuropsychiatric symptoms (agitation)

Cannabinoid

Anorexia (associated with AIDS); nausea/vomiting (associated with chemotherapy)

Cannabinoid

NCT02792257

Insulin glulisine

II

Metabolic

Enhance cell signaling and growth; promote neuronal metabolism (DMT)

Insulin

Diabetes

Antidiabetic

NCT02503501

Lenolidomide

II

Anti-inflammatory

Reduce inflammatory cytokines (TNF-a, IL-6, IL-8); reduce inflammatory and AD-associated biomarkers; improve cognition (DMT)

Anti-neoplastic

Multiple myeloma

Hematologic-oncologic

NCT04032626

Levetiracetam

II

Neuroprotective

Reduce amyloid induced neuronal hyperactivity (DMT)

Anticonvulsant

Adjunct therapy for partial-onset seizures; juvenile myoclonic epilepsy; primary generalized tonic-clonic seizures

Neurologic

NCT03489044

(2016–003109-32)

Levetiracetam

II

Neuroprotective

Reduce amyloid induced neuronal hyperactivity (DMT)

Anticonvulsant

Adjunct therapy for partial-onset seizures; juvenile myoclonic epilepsy; primary generalized tonic-clonic seizures

Neurologic

NCT02002819

Levetiracetam

II

Neuroprotective

Reduce amyloid induced neuronal hyperactivity (DMT)

Anticonvulsant

Adjunct therapy for partial-onset seizures; juvenile myoclonic epilepsy; primary generalized tonic-clonic seizures

Neurologic

NCT03875638

Levetiracetam (AGB101)

II

Neuroprotective

Reduce amyloid induced neuronal hyperactivity (DMT)

Anticonvulsant

Adjunct therapy for partial-onset seizures; juvenile myoclonic epilepsy; primary generalized tonic-clonic seizures

Neurologic

NCT03461861

Liraglutide

II

Metabolic

Enhance cell signaling; improve CNS glucose metabolism (DMT)

Glucagon-like peptide-1 agonist

Adjunct therapy for type 2 diabetes; risk reduction of major cardiovascular events

Antidiabetic

NCT01843075 (2013-000962-13)

Lithium

II

Neurotransmitter-based

Improve neuropsychiatric symptoms (agitation/aggression with or without psychosis)

Antimanic

Bipolar disorder

Psychiatric

NCT02129348

Leuprolide depot

II

Metabolic

Alleviates negative effects of elevated GnRH and gonadtropins on the brain (DMT)

Gonadotropin-releasing hormone agonist

Endometriosis; uterine leiomyomata

Hormonal

NCT03649724

Montelukast (buccal film)

II

Anti-inflammatory

Reduce inflammatory pathways (neuronal injury, blood-brain barrier integrity, amyloid-ß accumulation); effect on cognition and AD biomarkers (DMT)

Leukotriene receptor antagonist

Asthma; exercise-induced bronchoconstriction; allergic rhinitis

Anti-inflammatory

NCT03402503

Montelukast (tablet)

II

Anti-inflammatory

Reduce inflammatory pathways that mediate neuronal injury, blood-brain barrier integrity, amyloid-ß accumulation (DMT)

Leukotriene receptor antagonist

Asthma; exercise-induced bronchoconstriction; allergic rhinitis

Anti-inflammatory

NCT03991988

Nicotinamide

II

Anti-tau, neuroprotective

Reduce tau-induced microtubule depolymerization; reduce phosphorylation of tau (DMT)

Vitamin B3

Pellagra

Vitamin

NCT03061474

Nicotine (transdermal patch)

II

Neurotransmitter-based

Enhance acetylcholine signaling (cognitive enhancer)

Alkaloid

Smoking cessation withdrawal symptoms

Alkaloid

NCT02720445

Nilotinib

II

Anti-amyloid, anti-tau

Autophagy enhancer; promotes clearance of amyloid and tau proteins (DMT)

Tyrosine kinase inhibitor

Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase

Hematologic-oncologic

NCT02947893

Perindopril

II

Neuroprotective, anti-inflammatory

Improves vascular functioning (DMT)

Angiotensin-converting-enzyme inhibitor

Essential hypertension; stable coronary artery disease

Cardiovascular

NCT02085265

Prazosin

II

Neurotransmitter-based

Improve neuropsychiatric symptoms (agitation)

Alpha-receptor antagonist

Hypertension

Cardiovascular

NCT03710642

Rifaximin

II

Anti-inflammatory

Improve cognition and function by lowering blood ammonia; lower circulatory pro-inflammatory cytokines secreted by harmful gut bacteria (DMT)

Non-systemic antibiotic

Traveler’s diarrhea; overt hepatic encephalopathy; irritable bowel syndrome (diarrhea subtype)

Infectious disease

NCT03856359

Riluzole

II

Neuroprotective

Reduce glutamate-mediated excitotoxicity (DMT)

Benzothiazole

Amyotrophic lateral sclerosis

Neurologic

NCT01703117

Sagramostim

II

Anti-amyloid, neuroprotective

Stimulates innate immune system to remove amyloid pathology; increase neuronal connectivity (DMT)

Human granulocyte-macrophage colony-stimulating factor

Bone marrow stimulation

Hematologic-oncologic

NCT01409915

Sodium phenylbutyrate

II

Neuroprotective

Blocks nerve cell death and modulates neuroinflammation (DMT)

Anti-hyperammonemia

Adjunct for chronic urea cycle disorders

Genetics

NCT03533257

Tacrolimus

II

Neuroprotective

Prevents amyloid-ß dendritic spine loss; normalizes cranial nerve activity in the brain (DMT)

Calcineurin inhibitor

Prevent rejection of organ transplants

Immunologic

NCT04263519

Telmisartan

II

Neuroprotective, anti-inflammatory

Improves vascular functioning (DMT)

Angiotensin II Receptor Blocker

Hypertension; diabetic neuropathy; risk reduction of stroke

Cardiovascular

NCT02085265

Thiethylperazine

II

Anti-amyloid

Activates transport protein ABCC1 and enhances transport amyloid-ß peptides from the brain into the blood (DMT)

Phenothiazine

Nausea/vomiting

Neurologic

NCT03417986 (2014-000870-20)

Valacyclovir

II

Neuroprotective, anti-inflammatory

Reduce amyloid-ß aggregation by preventing overproduction of amyloid in response to infection (DMT)

Antiviral (nucleoside analog)

Herpes labialis; genital herpes

Infectious disease

NCT03282916

Valacyclovir

II

Neuroprotective, anti-inflammatory

Reduce amyloid-ß aggregation by preventing overproduction of amyloid in response to infection (DMT)

Antiviral (nucleoside analog)

Herpes labialis; genital herpes

Infectious disease

NCT02997982

  1. Abbreviation: DMT disease-modifying therapy