Biomarker & Intervention Discovery

Alzheimer's Disease and Other Dementias

Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.

Research Tracker / Chronic Disease Interventions / Alzheimer's Disease and Other Dementias / Biomarker Atlas →

Disease Overview

Spontaneous remission
Not established
Best-intervention remission
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Gap size
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Primary barrier
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Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: ● Clinical > ● Mechanistic > ● Correlative.

ACHE 243APOE 58APP 218BACE1 206BCHE 198CHRNA7 145GRIN1 183MAPT 605PSEN1 136TREM2 6

ACHE

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
EDROPHONIUM CATION ? unclear Mechanistic DGIdb
PYRIDOSTIGMINE ? unclear Mechanistic DGIdb
ACOTIAMIDE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
EPTASTIGMINE ? unclear Mechanistic 290.0 nM (IC50) DGIdb
ISOFLUROPHATE ↓ inhibits Mechanistic DGIdb
METHADONE HYDROCHLORIDE ? unclear Mechanistic DGIdb
AMBENONIUM CHLORIDE ↓ inhibits Mechanistic DGIdb
AMBENONIUM ? unclear Mechanistic DGIdb
PRALIDOXIME CHLORIDE ↑ activates Mechanistic DGIdb
NEOSTIGMINE ↓ inhibits Mechanistic DGIdb
MIMOPEZIL ? unclear Mechanistic DGIdb
SKIMMIANINE ? unclear Mechanistic DGIdb
ADRENOMEDULLIN ↑ activates Mechanistic DGIdb
TACRINE ? unclear Mechanistic 218.78 nM (IC50) DGIdb
HUMAN CALCITONIN ↑ activates Mechanistic DGIdb
HUPERZINE A ↓ inhibits Mechanistic 5.0 nM (IC50) DGIdb
Β-CGRP ↑ activates Mechanistic DGIdb
SALMON CALCITONIN ↑ activates Mechanistic DGIdb
GALACTITOL ? unclear Mechanistic DGIdb
GALANTAMINE ↓ inhibits Mechanistic DGIdb
ECHOTHIOPHATE IODIDE ↓ inhibits Mechanistic DGIdb
AMYLIN ↑ activates Mechanistic DGIdb
TURBINATINE ? unclear Mechanistic DGIdb
EDROPHONIUM CHLORIDE ↓ inhibits Mechanistic DGIdb
LATREPIRDINE ? unclear Mechanistic DGIdb
PYRIDOSTIGMINE BROMIDE ↓ inhibits Mechanistic DGIdb
DONEPEZIL ? unclear Mechanistic 2.33 nM (IC50) DGIdb
8-AZAGUANINE ? unclear Mechanistic DGIdb
DONEPEZIL HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
PHYSOSTIGMINE SALICYLATE ↓ inhibits Mechanistic DGIdb
MINAPRINE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
ADRENOMEDULLIN 2/INTERMEDIN ↑ activates Mechanistic DGIdb
AC187 ↓ inhibits Mechanistic DGIdb
PRAMLINTIDE ↑ activates Mechanistic DGIdb
METHANESULFONYL FLUORIDE ? unclear Mechanistic DGIdb
SURONACRINE MALEATE ? unclear Mechanistic DGIdb
RIVASTIGMINE TARTRATE ↓ inhibits Mechanistic DGIdb
KBP-088 ↑ activates Mechanistic DGIdb
DESOXYCORDIFOLINE ? unclear Mechanistic DGIdb
HEXAFLUORENIUM BROMIDE ↓ inhibits Mechanistic DGIdb

Showing top 40 of 243 agents ranked by evidence tier.

APOE

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TRIAMCINOLONE ? unclear Mechanistic DGIdb
AZ7188 ↓ inhibits Mechanistic DGIdb
PRAVASTATIN SODIUM ? unclear Mechanistic DGIdb
LUTEIN ? unclear Mechanistic DGIdb
TRETINOIN ? unclear Mechanistic DGIdb
LECANEMAB ? unclear Mechanistic DGIdb
PHYTOSTEROL ? unclear Mechanistic DGIdb
BUTHIONINE SULFOXIMINE ? unclear Mechanistic DGIdb
P2PAL18S ↓ inhibits Mechanistic DGIdb
THERAPEUTIC CORTICOSTEROID ? unclear Mechanistic DGIdb
VITAMIN E ? unclear Mechanistic DGIdb
AZ2429 ↑ activates Mechanistic DGIdb
GB88 ↓ inhibits Mechanistic DGIdb
IRBESARTAN ? unclear Mechanistic DGIdb
SLIGKV-NH2 ↑ activates Mechanistic DGIdb
STAUROSPORINE ? unclear Mechanistic DGIdb
TROGLITAZONE ? unclear Mechanistic DGIdb
AEM-28 ? unclear Mechanistic DGIdb
TRANS-CINNAMOYL-LIGRLO [N-[3H]PROPIONYL]-NH2 ↑ activates Mechanistic DGIdb
ACE INHIBITOR ? unclear Mechanistic DGIdb
I-191 ↓ inhibits Mechanistic DGIdb
AZ3451 ~ modulates Mechanistic DGIdb
CN-105 ? unclear Mechanistic DGIdb
RECOMBINANT CYTOKINE ? unclear Mechanistic DGIdb
2-FUROYL-LIGRL[N[3H]PROPIONYL]-O-NH2 ↑ activates Mechanistic DGIdb
FENOFIBRATE MICRONIZED ? unclear Mechanistic DGIdb
DONEPEZIL ? unclear Mechanistic DGIdb
AC-98170 ↑ activates Mechanistic DGIdb
HUMAN CHORIONIC GONADOTROPIN ? unclear Mechanistic DGIdb
GALANTAMINE ? unclear Mechanistic DGIdb
NICOTINE POLACRILEX ? unclear Mechanistic DGIdb
I-287 ~ modulates Mechanistic DGIdb
ANTIDEPRESSANT AGENT ? unclear Mechanistic DGIdb
ISOX-CHA-CHG-ALA-ARG-DPR(4FB)-NH2 ↑ activates Mechanistic DGIdb
GANCICLOVIR ? unclear Mechanistic DGIdb
C391 ↓ inhibits Mechanistic DGIdb
IMMUNOSUPPRESSANT ? unclear Mechanistic DGIdb
AZ8838 ↓ inhibits Mechanistic DGIdb
2-FUROYL-LIGRL[N-(ALEXA FLUOR 594)-O]-NH2 ↑ activates Mechanistic DGIdb
ISOX-CHA-CHG-ARK(SULFO-CY5)-NH2 ↑ activates Mechanistic DGIdb

Showing top 40 of 58 agents ranked by evidence tier.

APP

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
VALILTRAMIPROSATE ? unclear Mechanistic DGIdb
CLIOQUINOL ? unclear Mechanistic DGIdb
PROPOFOL ? unclear Mechanistic DGIdb
NIROGACESTAT ? unclear Mechanistic DGIdb
BAPINEUZUMAB ~ modulates Mechanistic DGIdb
TRIAZINATE ? unclear Mechanistic DGIdb
GANTENERUMAB ~ modulates Mechanistic DGIdb
NANOLUCIFERASE-LABELLED RELAXIN ↑ activates Mechanistic DGIdb
ML290 ~ modulates Mechanistic DGIdb
ADUCANUMAB ~ modulates Mechanistic DGIdb
TAMRLX ↑ activates Mechanistic DGIdb
AZD5462 ~ modulates Mechanistic DGIdb
A(4-24)(B7-24)H2 ↑ activates Mechanistic DGIdb
SOLANEZUMAB ~ modulates Mechanistic DGIdb
GSK933776 ~ modulates Mechanistic DGIdb
CAPROSPINOL ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL477841 ? unclear Mechanistic DGIdb
(B7-33)H2 ↑ activates Mechanistic DGIdb
METHYLINOSITOL ? unclear Mechanistic DGIdb
3,5-DICAFFEOYLQUINIC ACID ? unclear Mechanistic DGIdb
TRAMIPROSATE ~ modulates Mechanistic DGIdb
RESVERATROL ? unclear Mechanistic DGIdb
INSL3 ↑ activates Mechanistic DGIdb
EUROPIUM-LABELLED RELAXIN ↑ activates Mechanistic DGIdb
SCYLLITOL ? unclear Mechanistic DGIdb
RELAXIN ↑ activates Mechanistic DGIdb
AMILOMOTIDE ? unclear Mechanistic DGIdb
CURCUMIN ? unclear Mechanistic 0.208 nM (Ki) DGIdb
2-CHLOROADENOSINE-5-TRIPHOSPHATE ? unclear Mechanistic DGIdb
DAUNORUBICIN LIPOSOMAL ? unclear Mechanistic DGIdb
COMPOUND 54 ↑ activates Mechanistic DGIdb
INOSITOL ? unclear Mechanistic DGIdb
LECANEMAB ? unclear Mechanistic DGIdb
AMPHOTERICIN B LIPOSOMAL ? unclear Mechanistic DGIdb
GENTIAN VIOLET CATION ? unclear Mechanistic DGIdb
[125I]RELAXIN (HUMAN) ↑ activates Mechanistic DGIdb
[33P]RELAXIN (HUMAN) ↑ activates Mechanistic DGIdb
B-R13/17K H2 RELAXIN ↓ inhibits Mechanistic DGIdb
HYDROXYCHLOROQUINE ? unclear Mechanistic DGIdb
FERULIC ACID ? unclear Mechanistic DGIdb

Showing top 40 of 218 agents ranked by evidence tier.

BACE1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
SOPHORAFLAVANONE G ? unclear Mechanistic DGIdb
AZ-4217 ? unclear Mechanistic DGIdb
LY2886721 ? unclear Mechanistic DGIdb
BYAKANGELICIN ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL496451 ? unclear Mechanistic DGIdb
AZD-3839 ? unclear Mechanistic DGIdb
TAK-070 ? unclear Mechanistic DGIdb
ELENBECESTAT ↓ inhibits Mechanistic DGIdb
KUSHENOL A ? unclear Mechanistic DGIdb
LY3202626 ? unclear Mechanistic DGIdb
VERUBECESTAT ↓ inhibits Mechanistic DGIdb
BUNTANETAP ? unclear Mechanistic DGIdb
CTS-21166 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1412710 ? unclear Mechanistic DGIdb
BERGAMOTTIN ? unclear Mechanistic DGIdb
LANABECESTAT ? unclear Mechanistic DGIdb
OXAZINE 89 ? unclear Mechanistic DGIdb
RO5508887 ? unclear Mechanistic DGIdb
LY-2811376 ? unclear Mechanistic DGIdb
ATABECESTAT ↓ inhibits Mechanistic DGIdb
EPIGALLOCATECHIN-3-GALLATE ? unclear Mechanistic DGIdb
KURARINONE ? unclear Mechanistic DGIdb
TARENFLURBIL ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL2047666 ? unclear Mechanistic DGIdb
HGLUVALLEUPNSASPALAGLUPHEOH ↓ inhibits Mechanistic 413.0 nM (IC50) ChEMBL
CHEMBL302651 ↓ inhibits Mechanistic 10491.0 nM (Ki) ChEMBL
CHEMBL305381 ↓ inhibits Mechanistic 3134.0 nM (Ki) ChEMBL
CHEMBL431917 ↓ inhibits Mechanistic 22423.0 nM (Ki) ChEMBL
CHEMBL302833 ↓ inhibits Mechanistic 1129.0 nM (Ki) ChEMBL
CHEMBL78946 ↓ inhibits Mechanistic 1.6 nM (Ki) ChEMBL
CHEMBL65752 ↓ inhibits Mechanistic 50.1 nM (Ki) ChEMBL
CHEMBL65981 ↓ inhibits Mechanistic 61.4 nM (Ki) ChEMBL
CHEMBL66595 ↓ inhibits Mechanistic 5808.0 nM (Ki) ChEMBL
CHEMBL293611 ↓ inhibits Mechanistic 2.5 nM (Ki) ChEMBL
CHEMBL294274 ↓ inhibits Mechanistic 9.4 nM (Ki) ChEMBL
CHEMBL304010 ↓ inhibits Mechanistic 5.9 nM (Ki) ChEMBL
CHEMBL264379 ↓ inhibits Mechanistic 0.32 nM (Ki) ChEMBL
CHEMBL440490 ↓ inhibits Mechanistic 8.0 nM (Ki) ChEMBL
CHEMBL324109 ↓ inhibits Mechanistic 460.0 nM (IC50) ChEMBL
CHEMBL114147 ↓ inhibits Mechanistic 9000.0 nM (IC50) ChEMBL

Showing top 40 of 206 agents ranked by evidence tier.

BCHE

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
SN-38 ? unclear Mechanistic DGIdb
BMS753 ↑ activates Mechanistic DGIdb
BMS-189532 ↓ inhibits Mechanistic DGIdb
IRINOTECAN HYDROCHLORIDE ? unclear Mechanistic DGIdb
AROTINOID ACID ↑ activates Mechanistic DGIdb
DEMECARIUM BROMIDE ? unclear Mechanistic DGIdb
ECHOTHIOPHATE IODIDE ? unclear Mechanistic DGIdb
IMMUNOTOXIN ? unclear Mechanistic DGIdb
ISOFLUROPHATE ? unclear Mechanistic DGIdb
BUPIVACAINE ? unclear Mechanistic DGIdb
HEXAFLUORENIUM ? unclear Mechanistic DGIdb
RO 41-5253 ↓ inhibits Mechanistic DGIdb
TAZAROTENE ↑ activates Mechanistic DGIdb
TAMIBAROTENE ↑ activates Mechanistic DGIdb
MEPTAZINOL ? unclear Mechanistic DGIdb
ALITRETINOIN ↑ activates Mechanistic DGIdb
TACRINE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
AGN193836 ↑ activates Mechanistic DGIdb
RIVASTIGMINE ↓ inhibits Mechanistic DGIdb
VOLANESORSEN ? unclear Mechanistic DGIdb
BMS614 ↓ inhibits Mechanistic DGIdb
PHENSERINE ? unclear Mechanistic 2500.0 nM (IC50) DGIdb
MEMANTINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
EPTASTIGMINE ? unclear Mechanistic 0.69 nM (IC50) DGIdb
BMS493 ↓ inhibits Mechanistic DGIdb
ADAPALENE ↑ activates Mechanistic DGIdb
RO 40-6055 ↑ activates Mechanistic DGIdb
AGN193109 ↓ inhibits Mechanistic DGIdb
PROPANIDID ↓ inhibits Mechanistic DGIdb
RIVASTIGMINE TARTRATE ↓ inhibits Mechanistic DGIdb
CD666 ↑ activates Mechanistic DGIdb
DONEPEZIL ? unclear Mechanistic 8200.0 nM (IC50) DGIdb
TRETINOIN ↑ activates Mechanistic DGIdb
TRIAMCINOLONE ? unclear Mechanistic DGIdb
ANESTHETIC AGENT ? unclear Mechanistic DGIdb
GALANTAMINE ? unclear Mechanistic 18000.0 nM (IC50) DGIdb
MIVACURIUM ? unclear Mechanistic DGIdb
PHENOTHIAZINE ? unclear Mechanistic DGIdb
TRIFAROTENE ↑ activates Mechanistic DGIdb
SUCCINYLCHOLINE ? unclear Mechanistic DGIdb

Showing top 40 of 198 agents ranked by evidence tier.

CHRNA7

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
FACINICLINE HYDROCHLORIDE ↑ activates Mechanistic DGIdb
TALBUTAL ? unclear Mechanistic DGIdb
PNU-120596 ? unclear Mechanistic DGIdb
ENCENICLINE ↑ activates Mechanistic DGIdb
VARENICLINE ? unclear Mechanistic DGIdb
CYTISINE ? unclear Mechanistic DGIdb
PRIMIDONE ? unclear Mechanistic DGIdb
SSR180711 ↑ activates Mechanistic DGIdb
ECULIZUMAB ? unclear Mechanistic DGIdb
ZILUCOPLAN ? unclear Mechanistic DGIdb
BNC375 ? unclear Mechanistic DGIdb
ASM-024 ~ modulates Mechanistic DGIdb
TC-6987 ↑ activates Mechanistic DGIdb
ALCURONIUM ? unclear Mechanistic DGIdb
BRADANICLINE ↑ activates Mechanistic DGIdb
ABT-126 ↑ activates Mechanistic DGIdb
AZD0328 ↑ activates Mechanistic DGIdb
DONEPEZIL ? unclear Mechanistic DGIdb
THIOPENTAL ? unclear Mechanistic DGIdb
PHA-543613 ↑ activates Mechanistic DGIdb
GTS-21 ↑ activates Mechanistic DGIdb
SECOBARBITAL ? unclear Mechanistic DGIdb
BUTOBARBITAL ? unclear Mechanistic DGIdb
METHARBITAL ? unclear Mechanistic DGIdb
AQW051 ↑ activates Mechanistic DGIdb
ROZANOLIXIZUMAB ? unclear Mechanistic DGIdb
HEXOBARBITAL ? unclear Mechanistic DGIdb
PHENOBARBITAL ? unclear Mechanistic DGIdb
TREBANANIB ? unclear Mechanistic DGIdb
APN1125 ↑ activates Mechanistic DGIdb
GALANTAMINE ? unclear Mechanistic DGIdb
ND0801 ↑ activates Mechanistic DGIdb
NICOTINE POLACRILEX ? unclear Mechanistic DGIdb
EFGARTIGIMOD ALFA ? unclear Mechanistic DGIdb
ZIPRASIDONE ? unclear Mechanistic DGIdb
BUTABARBITAL ? unclear Mechanistic DGIdb
BUTALBITAL ? unclear Mechanistic DGIdb
RIVASTIGMINE ? unclear Mechanistic DGIdb
MEPHOBARBITAL ? unclear Mechanistic DGIdb
AVL-3288 ~ modulates Mechanistic DGIdb

Showing top 40 of 145 agents ranked by evidence tier.

GRIN1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
COMPOUND 53 [PMID: 33038794] ↑ activates Mechanistic DGIdb
PAREGORIC ? unclear Mechanistic DGIdb
STING AGONIST 2 ↑ activates Mechanistic DGIdb
LATREPIRDINE ? unclear Mechanistic DGIdb
CNS-5161 ↓ inhibits Mechanistic DGIdb
MEMANTINE HYDROCHLORIDE ~ modulates Mechanistic DGIdb
LANICEMINE ↓ inhibits Mechanistic DGIdb
ULEVOSTINAG ↑ activates Mechanistic DGIdb
ELIPRODIL ? unclear Mechanistic DGIdb
NBQX ? unclear Mechanistic DGIdb
RAPASTINEL ? unclear Mechanistic DGIdb
DEXANABINOL ? unclear Mechanistic DGIdb
NEBOGLAMINE ~ modulates Mechanistic DGIdb
HALOPERIDOL DECANOATE ? unclear Mechanistic DGIdb
ORPHENADRINE ? unclear Mechanistic DGIdb
GW468816 ↓ inhibits Mechanistic DGIdb
ACAMPROSATE CALCIUM ↓ inhibits Mechanistic DGIdb
AMANTADINE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
H-151 ↓ inhibits Mechanistic DGIdb
D-SERINE ? unclear Mechanistic DGIdb
AZD8108 ↓ inhibits Mechanistic DGIdb
KETAMINE HYDROCHLORIDE ~ modulates Mechanistic DGIdb
DELUCEMINE ↓ inhibits Mechanistic DGIdb
EVT-101 ↓ inhibits Mechanistic DGIdb
MSA-2 ↑ activates Mechanistic DGIdb
ESKETAMINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
2'3'-CGAMP ↑ activates Mechanistic DGIdb
COMPOUND 40 [PMID: 33470814] ↑ activates Mechanistic DGIdb
PHILANTHOTOXIN 343 ? unclear Mechanistic DGIdb
FELBAMATE ↓ inhibits Mechanistic DGIdb
INDANTADOL ↓ inhibits Mechanistic DGIdb
STING AGONIST 3 ↑ activates Mechanistic DGIdb
ORPHENADRINE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
CYCLOSERINE ? unclear Mechanistic DGIdb
RADIPRODIL ↓ inhibits Mechanistic DGIdb
PERZINFOTEL ↓ inhibits Mechanistic DGIdb
ESKETAMINE ~ modulates Mechanistic DGIdb
ADU-S100 ↑ activates Mechanistic DGIdb
RALFINAMIDE ↓ inhibits Mechanistic DGIdb
SNX281 ↑ activates Mechanistic DGIdb

Showing top 40 of 183 agents ranked by evidence tier.

MAPT

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHYSERGIDE MALEATE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL582473 ? unclear Mechanistic DGIdb
CHELERYTHRINE CHLORIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL606167 ? unclear Mechanistic DGIdb
GRINIMIBINE ? unclear Mechanistic DGIdb
GOSURANEMAB ↓ inhibits Mechanistic DGIdb
PLUMBAGIN ? unclear Mechanistic DGIdb
WORTMANNIN ? unclear Mechanistic DGIdb
HEXACHLOROPHENE ? unclear Mechanistic DGIdb
ACID BLUE 129 ? unclear Mechanistic DGIdb
CHLORANIL ? unclear Mechanistic DGIdb
INDIRUBIN-3'-MONOXIME ? unclear Mechanistic DGIdb
NSC-663284 ? unclear Mechanistic DGIdb
CARBIDOPA ANHYDROUS ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL601618 ? unclear Mechanistic DGIdb
BROMOENOL LACTONE ? unclear Mechanistic DGIdb
METHYLENE BLUE ANHYDROUS ? unclear Mechanistic DGIdb
DIHYDROERGOCRISTINE MESYLATE ? unclear Mechanistic DGIdb
ANGUSTIBALIN ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL310934 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL528181 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL609606 ? unclear Mechanistic DGIdb
CALCIMYCIN ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1989853 ? unclear Mechanistic DGIdb
DOCEBENONE ? unclear Mechanistic DGIdb
NPPB ? unclear Mechanistic DGIdb
AG 9 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL601353 ? unclear Mechanistic DGIdb
3,4-DICHLOROISOCOUMARIN ? unclear Mechanistic DGIdb
CEAROIN ? unclear Mechanistic DGIdb
SULFURETIN ? unclear Mechanistic DGIdb
2,3-DIHYDROXY-4-METHOXY-4'-ETHOXYBENZOPHENONE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL211670 ? unclear Mechanistic DGIdb
DIHYDROERGOCRISTINE ? unclear Mechanistic DGIdb
NIFEDIPINE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL339587 ? unclear Mechanistic DGIdb
EPINEPHRINE BITARTRATE ? unclear Mechanistic DGIdb
RABEPRAZOLE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL578905 ? unclear Mechanistic DGIdb
PICEATANNOL ? unclear Mechanistic DGIdb

Showing top 40 of 605 agents ranked by evidence tier.

PSEN1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
AVAGACESTAT ↓ inhibits Mechanistic DGIdb
RG-4733 ↓ inhibits Mechanistic DGIdb
TETRADECANOYLPHORBOL ACETATE ? unclear Mechanistic DGIdb
NIROGACESTAT ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL247471 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL392068 ? unclear Mechanistic DGIdb
SEMAGACESTAT ↓ inhibits Mechanistic DGIdb
BEGACESTAT ↓ inhibits Mechanistic DGIdb
TARENFLURBIL ~ modulates Mechanistic DGIdb
IC261 ? unclear Mechanistic DGIdb
NOTCH SIGNALING PATHWAY INHIBITOR MK0752 ? unclear Mechanistic DGIdb
E2012 ? unclear Mechanistic DGIdb
GSI-136 ? unclear Mechanistic DGIdb
NIROGACESTAT HYDROBROMIDE ? unclear Mechanistic Open Targets
CHEMBL392068 ↑ activates Mechanistic 0.114 nM (EC50) ChEMBL
CHEMBL2159511 ↓ inhibits Mechanistic 46.0 nM (IC50) ChEMBL
CHEMBL2159510 ↓ inhibits Mechanistic 5.0 nM (IC50) ChEMBL
CHEMBL2159509 ↓ inhibits Mechanistic 8.0 nM (IC50) ChEMBL
CHEMBL2159691 ↓ inhibits Mechanistic 37.0 nM (IC50) ChEMBL
CHEMBL2159690 ↓ inhibits Mechanistic 48.0 nM (IC50) ChEMBL
CHEMBL2159689 ↓ inhibits Mechanistic 8.0 nM (IC50) ChEMBL
CHEMBL2159687 ↓ inhibits Mechanistic 18.0 nM (IC50) ChEMBL
CHEMBL2159686 ↓ inhibits Mechanistic 377.0 nM (IC50) ChEMBL
CHEMBL2159688 ↓ inhibits Mechanistic 320.0 nM (IC50) ChEMBL
CHEMBL2398571 ↓ inhibits Mechanistic 4000.0 nM (IC50) ChEMBL
CHEMBL2398570 ↓ inhibits Mechanistic 39810.72 nM (IC50) ChEMBL
CHEMBL2398569 ↓ inhibits Mechanistic 39810.72 nM (IC50) ChEMBL
CHEMBL2398568 ↓ inhibits Mechanistic 39810.72 nM (IC50) ChEMBL
CHEMBL2398567 ↓ inhibits Mechanistic 31622.78 nM (IC50) ChEMBL
CHEMBL2398579 ↓ inhibits Mechanistic 31622.78 nM (IC50) ChEMBL
CHEMBL2398578 ↓ inhibits Mechanistic 31622.78 nM (IC50) ChEMBL
CHEMBL2396675 ↓ inhibits Mechanistic 39810.72 nM (IC50) ChEMBL
CHEMBL2398577 ↓ inhibits Mechanistic 31622.78 nM (IC50) ChEMBL
CHEMBL2398576 ↓ inhibits Mechanistic 31622.78 nM (IC50) ChEMBL
CHEMBL2398575 ↓ inhibits Mechanistic 25118.86 nM (IC50) ChEMBL
CHEMBL2398574 ↓ inhibits Mechanistic 31622.78 nM (IC50) ChEMBL
CHEMBL2398573 ↓ inhibits Mechanistic 31622.78 nM (IC50) ChEMBL
CHEMBL2398572 ↓ inhibits Mechanistic 39810.72 nM (IC50) ChEMBL
CHEMBL3397716 ↑ activates Mechanistic 3200.0 nM (EC50) ChEMBL
CHEMBL3397715 ↑ activates Mechanistic 13800.0 nM (EC50) ChEMBL

Showing top 40 of 136 agents ranked by evidence tier.

TREM2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL6174051 ↑ activates Mechanistic 3.0 nM (EC50) ChEMBL
CHEMBL6165036 ↑ activates Mechanistic 2.0 nM (EC50) ChEMBL
CHEMBL6172797 ↑ activates Mechanistic 1.0 nM (EC50) ChEMBL
CHEMBL6166220 ↑ activates Mechanistic 5.0 nM (EC50) ChEMBL
CHEMBL6165261 ↑ activates Mechanistic 4.0 nM (EC50) ChEMBL
CHEMBL6166127 ↑ activates Mechanistic 2.0 nM (EC50) ChEMBL
Clinical Trials

Recruiting and recently completed trials from ClinicalTrials.gov. Data retrieved 2026-07-03.

InRAD Observational Study Recruiting
NCT07213700 Phase: N/A Started: 2026-03-30

The goal of this international observational study is to evaluate long-term disease outcomes and treatment safety in people with Alzheimer's disease (PwAD), by collecting real-world data from routine clinical practice across global clinical centers. The InRAD Registry Observational Study has severa...

A Study to Evaluate Safety and Efficacy of KarXT + KarX-EC as a Treatment for Psychosis Associated With Alzheimer's Disease (ADEPT-5) Recruiting
NCT06947941 Phase: PHASE3 Started: 2026-03-25

The purpose of this study is to evaluate KarXT + KarX-EC as a treatment for psychosis associated with Alzheimer's disease.

Stress and Blood Pressure Management for Caregivers Active Not Recruiting
NCT05734638 Phase: NA Started: 2023-09-11

Due to health and wealth disparities, no demographic group is more at risk than African American women for the double jeopardy of stress from caregiving for persons living with dementia (PLWD) and stress associated with hypertension (HTN). This double jeopardy puts those they care for in jeopardy as...

A Study to Evaluate KarXT as a Treatment for Psychosis Associated With Alzheimer's Disease (ADEPT-4) Recruiting
NCT06585787 Phase: PHASE3 Started: 2024-09-26

The purpose of this study is to evaluate the safety and efficacy of KarXT in adult participants with mild to severe Alzheimer's Disease (AD) with moderate to severe psychosis related to AD.

Changing Talk Online Training (CHATO) National Trial Completed
NCT04610944 Phase: NA Started: 2021-04-26

The National Plan to Address Alzheimer's disease has identified education of dementia care providers as a top priority to address the need for quality care for the population of persons with dementia that will triple in the next 30 years. This study will test new online interactive training for nurs...

Human Mesenchymal Stem Cells (hMSC) in Behavioral Problems Due to Alzheimer's Disease. Recruiting
NCT06781333 Phase: PHASE2 Started: 2025-04-29

The purpose of this research study is to test if adding one infusion of mesenchymal stem cells to the current treatment with antipsychotic medication may help control behavioral problems in people with a diagnosis of moderate to severe Alzheimer's disease.

A Study of Caregiver Connections Via Technology in Dementia Active Not Recruiting
NCT05779839 Phase: NA Started: 2023-02-22

This research is being done to develop a unique matching process for caregivers of persons living with dementia, such as Alzheimer's disease, Lewy body dementia, frontotemporal degeneration, or other dementia syndromes. Dementia caregivers often assume greater caregiving burden than do non-dementia ...

A Study to Assess the Efficacy and Safety of ML-007C-MA for the Treatment of Alzheimer's Disease Psychosis Recruiting
NCT06887192 Phase: PHASE2 Started: 2025-08-15

ML-007C-MA-221 is a Phase 2, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of ML-007C-MA in male and female participants aged 55 to 90 years with hallucinations and delusions associated with Alzheimer's Disease Psychosis (ADP). The primary objective is to ev...

An Open-Label Study of ML-007C-MA in Adults With Alzheimer's Disease Psychosis Recruiting
NCT07459660 Phase: PHASE2 Started: 2026-03-25

ML-007C-MA-222 is a 52-week, flexible-dose, open-label extension study designed to evaluate the long-term safety, tolerability, and effectiveness of ML007C-MA in participants with ADP who have completed the antecedent study (ie, Study ML-007C-MA-221).

Ultra-High Resolution PET in Aging, Neurodegeneration and Psychotic Disorders Recruiting
NCT07509125 Phase: NA Started: 2026-02-13

The goal of this study is to use ultra-high-resolution (UHR) PET imaging to better understand how the brain and spinal cord change in healthy aging and in neurological and psychiatric disorders such as Alzheimer's disease (AD), Parkinson's disease and related movement disorders, amyotrophic lateral ...

Brain Effects of Gamma Frequency Sensory Stimulation (40Hz Light, Sound and Other Devices) Recruiting
NCT07496918 Phase: PHASE2 Started: 2018-02-20

Despite the huge healthcare and economic impact of AD, there is still no disease modifying therapeutics available. In fact, the available therapeutics show low efficacy at best in the treatment of cognitive impairment in dementia. Development of a non-invasive medical device that is effective in slo...

A Research Study Looking at the Effect of Semaglutide on the Immune System and Other Biological Processes in People With Alzheimer's Disease Completed
NCT05891496 Phase: PHASE3 Started: 2023-06-20

The study is being conducted to understand how the medicine, semaglutide, affects the immune system and other biological processes in people with Alzheimer's disease. Semaglutide is a medicine that doctors can prescribe in some countries for the treatment of type 2 diabetes and excess body weight. T...

Circadian Rhythm and Other Factors in Memory Clinic Patients Recruiting
NCT05977712 Phase: N/A Started: 2024-03-06

The CIRCAME study is a bicentric study of patients from 2 memory clinics in Paris. The main objective is to identify circadian rhythm components and other individual risk factors (sociodemographic, behavioral, and health related factors) associated with the diagnosis of subtypes (AD, Lewy bodies, va...

PET Brain Imaging in Multiple Sclerosis, Alzheimer's Disease, and Other Neurological and Neuropsychiatric Diseases Recruiting
NCT02649985 Phase: PHASE1, PHASE2 Started: 2016-05-02

The specific aims of the study are: Primary: To determine the presence and regional distribution of microglial activation, as assessed by Fluorine-18 (18F) labeled "Peripheral Benzodiazepine Receptor 06" (PBR06) -PET, in subjects with active Relapsing Remitting Multiple Sclerosis (RRMS), Secondary ...

Trial to Reduce Antimicrobial Use in Nursing Home Residents With Alzheimer's Disease and Other Dementias 2.0 Recruiting
NCT05950607 Phase: NA Started: 2023-11-13

The goal of this pragmatic cluster randomized clinical trial is to compare management of suspected infection in nursing home residents with dementia The main questions it aims to answer whether residents with dementia in nursing homes randomized to use a multicomponent intervention to optimize suspe...

Agents Found By Disease-Level Search

Searched directly from “Alzheimer's Disease and Other Dementias” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 10-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

56 agents shown (approved or with trial evidence) — 56 not found by the gene-target search above.

risperidoneNOT in gene-target list

· 6 trials · source: ClinicalTrials.gov
NCT00417482 NCT00208819 NCT00034762

OlanzapineNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT00015548 NCT00245206 NCT00208819

ML-007C-MANOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT07459660 NCT06887192

KarXTNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06585787 NCT06947941

QuetiapineNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00015548 NCT00208819

active tDCSNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT05478681 NCT05730023

Nautilus NeuroWaveTM SystemNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02333942

[18F]THK-5351NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02656498

NSA-789NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00795730

Photo elicitationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04103580

LithiumNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02129348

Building Better Caregivers Online WorkshopNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00799786

TRAIN-ADNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03244917

hMSCNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06781333

SemaglutideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05891496

ValproateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00071721

Placebo matching BI 409306 25 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02392468

Placebo matching BI 409306 50 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02392468

BI 409306 25 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02392468

BI 409306 50 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02392468

Haloperidol-HaloperidolNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00009217

Haloperidol-PlaceboNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00009217

Social DancingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03475316

Treadmill WalkingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03475316

[F-18]PBR06NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02649985

[C-11]PBR28NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02649985

CitalopramNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00015548

Speeko for ElderspeakNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04064164

Blood samplingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00880347

GSK1034702NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00937846

MIM-DASHNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05734638

MIM OnlyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05734638

DASH OnlyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05734638

blood sample takenNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04946916

divalproexNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00208819

EscitalopramNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01119638

AlgorithmNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05779839

Random MatchNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05779839

Connect-Home PlusNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05887388

Changing Talk Online (CHATO)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04610944

KarX-ECNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06947941

KarXT + KarX-EC Arm Matching PlaceboNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06947941

Pimavanserin 34 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03325556

Pimavanserin 20 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03325556

individualized language trainingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05730023

Placebo tDCSNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05730023

F-18NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03625128

AripiprazoleNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00245206

Transcranial direct current stimulationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02453763

MRINOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02453763

GENUSNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07496918

Tactile GENUSNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07496918

Empatica EmbracePlusNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06326424

[18F]MNI-815 (MNI-815)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02531360

TRAIN AD 2.0NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05950607

Active tDCS paired with active online chair yogaNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07303998

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