Biomarker & Intervention Discovery

Parkinson's Disease

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

NIH funding$255MFY2025 · RCDC estimate
Funding levelModerate
Spontaneous remissionProgressive neurodegeneration — no spontaneous remission; untreated motor decline averages 3-5 UPDRS points/year…
Best-intervention remissionLevodopa/carbidopa: 40-50% motor improvement in early disease but efficacy wanes (motor fluctuations in 50% at 5…
Gap sizeLarge — DBS significantly underused; no disease-modifying therapy exists; current treatments are purely symptomatic
Primary barrierbiological + practice
Full remission profile →
Research Tracker / Chronic Disease Interventions / Parkinson's Disease

Remission & chronicity

Spontaneous remission
Progressive neurodegeneration — no spontaneous remission; untreated motor decline averages 3-5 UPDRS points/year; non-motor symptoms (depression, cognitive decline, autonomic dysfunction) also progressive
Best-intervention remission
Levodopa/carbidopa: 40-50% motor improvement in early disease but efficacy wanes (motor fluctuations in 50% at 5 years, dyskinesias in 30%); deep brain stimulation (DBS): 50-60% reduction in off-time, 60-70% reduction in dyskinesias at 5 years; LCIG (levodopa-carbidopa intestinal gel infusion): near-continuous on-time in advanced patients
Gap size
Large — DBS significantly underused; no disease-modifying therapy exists; current treatments are purely symptomatic
Primary barrier
biological + practice
No neuroprotective or disease-modifying treatment is approved despite decades of research; DBS is offered too late in disease course (often 10+ years after diagnosis vs evidence for benefit in earlier disease — EARLYSTIM trial); focused ultrasound (FDA cleared 2021) underused as non-invasive alternative
Biomarker Targets & Therapeutic Agents

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

DRD2 427GBA 156LRRK2 206MAOB 218SLC6A3 176SNCA 221TH 29

DRD2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ASENAPINE ? unclear Mechanistic DGIdb
MEPHENTERMINE ? unclear Mechanistic DGIdb
HALOPERIDOL LACTATE ↓ inhibits Mechanistic DGIdb
BREXPIPRAZOLE ↑ activates Mechanistic DGIdb
ANALGESIC AGENT ? unclear Mechanistic DGIdb
AXITINIB ↓ inhibits Mechanistic DGIdb
RG-1530 ↓ inhibits Mechanistic DGIdb
LOXAPINE SUCCINATE ↓ inhibits Mechanistic DGIdb
QUETIAPINE FUMARATE ↓ inhibits Mechanistic DGIdb
PROCHLORPERAZINE ↓ inhibits Mechanistic DGIdb
APOMORPHINE ↑ activates Mechanistic DGIdb
PRAMIPEXOLE ? unclear Mechanistic DGIdb
THIETHYLPERAZINE MALEATE ↓ inhibits Mechanistic DGIdb
IBCASERTIB ↓ inhibits Mechanistic DGIdb
FAMITINIB ↓ inhibits Mechanistic DGIdb
SOXATALTINIB ↓ inhibits Mechanistic DGIdb
SUMANIROLE ↑ activates Mechanistic 9.0 nM (Ki) DGIdb
DOCARPAMINE ? unclear Mechanistic DGIdb
PALIPERIDONE ↓ inhibits Mechanistic DGIdb
PRIDOPIDINE ~ modulates Mechanistic DGIdb
OLANZAPINE ↓ inhibits Mechanistic 20.0 nM (Ki) DGIdb
PEGDINETANIB ↓ inhibits Mechanistic DGIdb
RISPERIDONE ↓ inhibits Mechanistic DGIdb
PERGOLIDE MESYLATE ↑ activates Mechanistic DGIdb
LISURIDE ? unclear Mechanistic 0.5 nM (Ki) DGIdb
ZICRONAPINE ? unclear Mechanistic DGIdb
CLEBOPRIDE ↓ inhibits Mechanistic DGIdb
LAZERTINIB ↓ inhibits Mechanistic DGIdb
REMOXIPRIDE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
CABERGOLINE ↑ activates Mechanistic DGIdb
JNJ-37822681 ↓ inhibits Mechanistic DGIdb
ERGOT ALKALOIDS ? unclear Mechanistic DGIdb
(-)-STEPHOLIDINE ? unclear Mechanistic DGIdb
SARIZOTAN HYDROCHLORIDE ↑ activates Mechanistic DGIdb
5-OH-DPAT ? unclear Mechanistic DGIdb
LITHIUM ? unclear Mechanistic DGIdb
DIHYDROERGOTOXINE ? unclear Mechanistic DGIdb
REMOXIPRIDE ↓ inhibits Mechanistic DGIdb
BROMPERIDOL ↓ inhibits Mechanistic DGIdb
PHENTOLAMINE ? unclear Mechanistic DGIdb

Showing top 40 of 427 agents ranked by evidence tier.

GBA

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
NCGC607 ? unclear Mechanistic DGIdb
AFEGOSTAT ? unclear Mechanistic 60.0 nM (IC50) DGIdb
GENTIAN VIOLET CATION ? unclear Mechanistic DGIdb
VELAGLUCERASE ALFA ? unclear Mechanistic DGIdb
RALOXIFENE HYDROCHLORIDE ? unclear Mechanistic DGIdb
CIRSIMARITIN ? unclear Mechanistic DGIdb
DOMIPHEN BROMIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1169388 ? unclear Mechanistic DGIdb
2,3-DIHYDROXY-6,7-DICHLOROQUINOXALINE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL579837 ? unclear Mechanistic DGIdb
METITEPINE ? unclear Mechanistic DGIdb
TALIGLUCERASE ALFA ? unclear Mechanistic DGIdb
APTIGANEL HYDROCHLORIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL588327 ? unclear Mechanistic DGIdb
MIGALASTAT ? unclear Mechanistic DGIdb
CHLORHEXIDINE GLUCONATE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL528181 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1379900 ? unclear Mechanistic DGIdb
L 755507 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL73711 ? unclear Mechanistic DGIdb
DNQX ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL408500 ? unclear Mechanistic DGIdb
CASTANOSPERMINE ? unclear Mechanistic DGIdb
SULFANITRAN ? unclear Mechanistic DGIdb
AMBROXOL ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL578928 ? unclear Mechanistic DGIdb
PHLORETIN ? unclear Mechanistic DGIdb
GENISTEIN ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL603154 ? unclear Mechanistic DGIdb
AFEGOSTAT TARTRATE ~ modulates Mechanistic DGIdb
CHEMBL:CHEMBL529939 ? unclear Mechanistic DGIdb
ISOLIQUIRITIGENIN ? unclear Mechanistic DGIdb
TRICHOSTATIN A ? unclear Mechanistic DGIdb
EBSELEN ? unclear Mechanistic DGIdb
CHEMBL3350088 ↓ inhibits Mechanistic 150.0 nM (Ki) ChEMBL
CHEMBL3350093 ↓ inhibits Mechanistic 550.0 nM (Ki) ChEMBL
CHEMBL3350086 ↓ inhibits Mechanistic 25.1 nM (Ki) ChEMBL
CHEMBL3350089 ↓ inhibits Mechanistic 100.0 nM (Ki) ChEMBL
CHEMBL3350092 ↓ inhibits Mechanistic 1.2 nM (Ki) ChEMBL
CHEMBL469844 ↓ inhibits Mechanistic 200.0 nM (Ki) ChEMBL

Showing top 40 of 156 agents ranked by evidence tier.

LRRK2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
LRRK2 INHIBITOR 6 [PMID: 40353293] ↓ inhibits Mechanistic DGIdb
LIRADASERTIB ↓ inhibits Mechanistic DGIdb
ADAVOSERTIB ? unclear Mechanistic DGIdb
PF-562271 ? unclear Mechanistic DGIdb
NIK INHIBITOR 12F ↓ inhibits Mechanistic DGIdb
DCLK1-IN-1 ↓ inhibits Mechanistic DGIdb
DACTOLISIB ? unclear Mechanistic DGIdb
VANDETANIB ? unclear Mechanistic 1995.26 nM (Ki) DGIdb
LRRK2-IN-1 ↓ inhibits Mechanistic DGIdb
DOVITINIB ? unclear Mechanistic 1500.0 nM (Kd) DGIdb
BOS172722 ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL2204495 ? unclear Mechanistic DGIdb
TOZASERTIB ? unclear Mechanistic 630.96 nM (Ki) DGIdb
MLI-2 ↓ inhibits Mechanistic DGIdb
PALBOCICLIB ? unclear Mechanistic 2511.89 nM (Ki) DGIdb
ENTRECTINIB ? unclear Mechanistic 2511.89 nM (Ki) DGIdb
HG-10-102-01 ↓ inhibits Mechanistic DGIdb
RG-1530 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL379975 ? unclear Mechanistic DGIdb
GSK2646264 ↓ inhibits Mechanistic DGIdb
PD166285 ? unclear Mechanistic DGIdb
GNE-9605 ↓ inhibits Mechanistic DGIdb
PELERASERTIB ↓ inhibits Mechanistic DGIdb
ALISERTIB ? unclear Mechanistic 794.33 nM (Ki) DGIdb
AST-487 ? unclear Mechanistic 950.0 nM (Kd) DGIdb
PF-06447475 ↓ inhibits Mechanistic DGIdb
URMC-099 ↓ inhibits Mechanistic DGIdb
PICTILISIB ? unclear Mechanistic DGIdb
NVP-TAE684 ? unclear Mechanistic DGIdb
XMD-12 ↓ inhibits Mechanistic DGIdb
EB-42486 ↓ inhibits Mechanistic DGIdb
PF-06454589 ↓ inhibits Mechanistic DGIdb
NIK SMI1 ↓ inhibits Mechanistic DGIdb
JAK3 INHIBITOR VI ↓ inhibits Mechanistic DGIdb
TTT-3002 ↓ inhibits Mechanistic DGIdb
XL01126 ↓ inhibits Mechanistic DGIdb
COMPOUND 8 [PMID: 17480064] ? unclear Mechanistic DGIdb
ILORASERTIB ? unclear Mechanistic DGIdb
CENISERTIB ? unclear Mechanistic 0.631 nM (Ki) DGIdb
FASUDIL ? unclear Mechanistic 1995.26 nM (Ki) DGIdb

Showing top 40 of 206 agents ranked by evidence tier.

MAOB

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
SAFINAMIDE ↓ inhibits Mechanistic 450.0 nM (Ki) DGIdb
LEVODOPA ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1927670 ? unclear Mechanistic DGIdb
MOCLOBEMIDE ↓ inhibits Mechanistic 100000.0 nM (Ki) DGIdb
CHEMBL:CHEMBL1927684 ? unclear Mechanistic DGIdb
SELEGILINE ↓ inhibits Mechanistic 970.0 nM (Ki) DGIdb
BIFEMELANE ↓ inhibits Mechanistic DGIdb
MEBANAZINE ↓ inhibits Mechanistic DGIdb
MELATONIN ? unclear Mechanistic DGIdb
PARGYLINE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL602260 ? unclear Mechanistic DGIdb
ISOCARBOXAZID ↓ inhibits Mechanistic DGIdb
SEROTONIN ? unclear Mechanistic DGIdb
[18F]FLUORORASAGILINE ? unclear Mechanistic DGIdb
RASAGILINE ↓ inhibits Mechanistic 700.0 nM (Ki) DGIdb
CHEMBL:CHEMBL1642672 ? unclear Mechanistic DGIdb
PHENOXYPROPAZINE ↓ inhibits Mechanistic DGIdb
LAZABEMIDE ↓ inhibits Mechanistic 84.0 nM (Ki) DGIdb
NIALAMIDE ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL1813518 ? unclear Mechanistic DGIdb
SAFINAMIDE MESYLATE ↓ inhibits Mechanistic DGIdb
COBALAMIN ? unclear Mechanistic DGIdb
PHENELZINE ↓ inhibits Mechanistic DGIdb
RASAGILINE TARTRATE ↓ inhibits Mechanistic DGIdb
RALFINAMIDE ↓ inhibits Mechanistic DGIdb
SELEGILINE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
VITAMIN K3 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL2088303 ? unclear Mechanistic DGIdb
TRANYLCYPROMINE ↓ inhibits Mechanistic DGIdb
PARGYLINE ? unclear Mechanistic 1800.0 nM (Ki) DGIdb
ANTIDEPRESSANT AGENT ? unclear Mechanistic DGIdb
MOFEGILINE ? unclear Mechanistic DGIdb
INDANTADOL ? unclear Mechanistic DGIdb
NAPHTHOQUINONE ? unclear Mechanistic DGIdb
METHAMPHETAMINE ? unclear Mechanistic DGIdb
HALOPERIDOL DECANOATE ? unclear Mechanistic DGIdb
PHENELZINE SULFATE ↓ inhibits Mechanistic DGIdb
IPRONIAZID ↓ inhibits Mechanistic 7540.0 nM (IC50) DGIdb
LINEZOLID ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL602466 ? unclear Mechanistic DGIdb

Showing top 40 of 218 agents ranked by evidence tier.

SLC6A3

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ALTROPANE I-123 ? unclear Mechanistic DGIdb
BUPROPION HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
DEXMETHYLPHENIDATE ↓ inhibits Mechanistic DGIdb
AMPHETAMINE ADIPATE ? unclear Mechanistic DGIdb
DEXTROAMPHETAMINE SACCHARATE ? unclear Mechanistic DGIdb
LIAFENSINE ↓ inhibits Mechanistic DGIdb
LISDEXAMFETAMINE DIMESYLATE ? unclear Mechanistic DGIdb
METHAMPHETAMINE ↓ inhibits Mechanistic DGIdb
SIBUTRAMINE ? unclear Mechanistic DGIdb
METHYLPHENIDATE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
3,4-METHYLENEDIOXYMETHAMPHETAMINE ? unclear Mechanistic DGIdb
LEVODOPA ? unclear Mechanistic DGIdb
DEXTROAMPHETAMINE ADIPATE ? unclear Mechanistic DGIdb
METYROSINE ? unclear Mechanistic DGIdb
ANESTHETIC AGENT ? unclear Mechanistic DGIdb
SERTRALINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
BENZTROPINE ? unclear Mechanistic 312.0 nM (IC50) DGIdb
BICIFADINE ↓ inhibits Mechanistic DGIdb
ALTROPANE ? unclear Mechanistic DGIdb
DASOTRALINE ↓ inhibits Mechanistic DGIdb
DISULFIRAM ? unclear Mechanistic DGIdb
AMITIFADINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
AMPHETAMINE ASPARTATE ? unclear Mechanistic DGIdb
NICOTINE POLACRILEX ? unclear Mechanistic DGIdb
SPIROGLUMIDE ? unclear Mechanistic DGIdb
HINOKININ ? unclear Mechanistic DGIdb
DIETHYLPROPION HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
SERDEXMETHYLPHENIDATE CHLORIDE ↓ inhibits Mechanistic DGIdb
PHENTERMINE RESIN ? unclear Mechanistic DGIdb
PSEUDOEPHEDRINE ? unclear Mechanistic DGIdb
PHENMETRAZINE ? unclear Mechanistic DGIdb
CENTANAFADINE ↓ inhibits Mechanistic DGIdb
BUPRENORPHINE ? unclear Mechanistic DGIdb
PYROVALERONE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
SERDEXMETHYLPHENIDATE ↓ inhibits Mechanistic DGIdb
PHENMETRAZINE HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
FENCAMFAMIN ? unclear Mechanistic DGIdb
IOFLUPANE I-123 ? unclear Mechanistic DGIdb
PHENDIMETRAZINE ↓ inhibits Mechanistic DGIdb
MAZINDOL ↓ inhibits Mechanistic 42.6 nM (IC50) DGIdb

Showing top 40 of 176 agents ranked by evidence tier.

SNCA

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL:CHEMBL1521156 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL599100 ? unclear Mechanistic DGIdb
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic DGIdb
LEVOKETOCONAZOLE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL450802 ? unclear Mechanistic DGIdb
CALCDPWW ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL580727 ? unclear Mechanistic DGIdb
PRASINEZUMAB ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL505670 ? unclear Mechanistic DGIdb
CONGO RED FREE ACID ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL548540 ? unclear Mechanistic DGIdb
GARDENIN ? unclear Mechanistic DGIdb
6-AZAURIDINE ? unclear Mechanistic DGIdb
PARAROSANILINE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL534353 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL2094833 ? unclear Mechanistic DGIdb
GENTIAN VIOLET CATION ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL2094567 ? unclear Mechanistic DGIdb
2-CHLOROADENOSINE-5-TRIPHOSPHATE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL442906 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1534035 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1526167 ? unclear Mechanistic DGIdb
PP2 ? unclear Mechanistic DGIdb
MYCOPHENOLATE ? unclear Mechanistic DGIdb
ALTENUSIN ? unclear Mechanistic DGIdb
GW305074X ? unclear Mechanistic DGIdb
INDENEBART ? unclear Mechanistic DGIdb
ABT702 ? unclear Mechanistic DGIdb
CINPANEMAB ↓ inhibits Mechanistic DGIdb
LICARIN A ? unclear Mechanistic DGIdb
LITHOCHOLIC ACID ? unclear Mechanistic DGIdb
CAFFEIC ACID PHENETHYL ESTER ? unclear Mechanistic DGIdb
CHEMBL1594514 ~ modulates Mechanistic ChEMBL
CHEMBL1411318 ~ modulates Mechanistic ChEMBL
CHEMBL1379677 ~ modulates Mechanistic ChEMBL
CHEMBL1476741 ~ modulates Mechanistic ChEMBL
CHEMBL1565212 ~ modulates Mechanistic ChEMBL
CHEMBL1331245 ~ modulates Mechanistic ChEMBL
CHEMBL1411120 ~ modulates Mechanistic ChEMBL
CHEMBL1459532 ~ modulates Mechanistic ChEMBL

Showing top 40 of 221 agents ranked by evidence tier.

TH

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
METYROSINE ↓ inhibits Mechanistic DGIdb
STREPTOZOCIN ? unclear Mechanistic DGIdb
SAPONIN ? unclear Mechanistic DGIdb
PROSAVIN ? unclear Mechanistic DGIdb
OPIATE ? unclear Mechanistic DGIdb
FLUTAMIDE ? unclear Mechanistic DGIdb
3-IODOBENZYLGUANIDINE ? unclear Mechanistic DGIdb
PRASTERONE ? unclear Mechanistic DGIdb
METHYLPHENIDATE ? unclear Mechanistic DGIdb
RECOMBINANT INTERFERON ? unclear Mechanistic DGIdb
SERTRALINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
INDOMETHACIN ? unclear Mechanistic DGIdb
3-IODOTYROSINE ↓ inhibits Mechanistic DGIdb
3-CHLOROTYROSINE ↓ inhibits Mechanistic DGIdb
SAPROPTERIN ? unclear Mechanistic DGIdb
DOXYCYCLINE ANHYDROUS ? unclear Mechanistic DGIdb
TRIAMTERENE ? unclear Mechanistic DGIdb
ALPHA-TOCOPHEROL ? unclear Mechanistic DGIdb
Α-PROPYLDOPACETAMIDE ↓ inhibits Mechanistic DGIdb
TYROSINE ? unclear Mechanistic DGIdb
PHENYLALANINE ? unclear Mechanistic DGIdb
METHADONE HYDROCHLORIDE ? unclear Mechanistic DGIdb
ANTIESTROGEN ? unclear Mechanistic DGIdb
EPOETIN ALFA ? unclear Mechanistic DGIdb
CHEMBL27559 ↓ inhibits Mechanistic 42000.0 nM (IC50) ChEMBL
CHEMBL330274 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
R-N-PROPYLNORAPOMORPHINE ↓ inhibits Mechanistic 300.0 nM (IC50) ChEMBL
CHEMBL283489 ↓ inhibits Mechanistic 87000.0 nM (IC50) ChEMBL
IOTYROSINE ↓ inhibits Mechanistic 720.0 nM (IC50) ChEMBL
Clinical Trials

Recruiting and recently completed trials from ClinicalTrials.gov. Data retrieved 2026-07-06.

No recent clinical trials found on ClinicalTrials.gov for this condition.

Agents Found By Disease-Level Search

Searched directly from “Parkinson's Disease” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 7-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).

Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."

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