Biomarker & Intervention Discovery

Parkinson's Disease

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

Disease Overview

Spontaneous remission
Not established
Best-intervention remission
Gap size
Primary barrier
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
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ASENAPINE ? unclear Mechanistic
MEPHENTERMINE ? unclear Mechanistic
HALOPERIDOL LACTATE inhibits Mechanistic
BREXPIPRAZOLE activates Mechanistic
ANALGESIC AGENT ? unclear Mechanistic
AXITINIB inhibits Mechanistic
RG-1530 inhibits Mechanistic
LOXAPINE SUCCINATE inhibits Mechanistic
QUETIAPINE FUMARATE inhibits Mechanistic
PROCHLORPERAZINE inhibits Mechanistic
APOMORPHINE activates Mechanistic
PRAMIPEXOLE ? unclear Mechanistic
THIETHYLPERAZINE MALEATE inhibits Mechanistic
IBCASERTIB inhibits Mechanistic
FAMITINIB inhibits Mechanistic
SOXATALTINIB inhibits Mechanistic
SUMANIROLE activates Mechanistic 9.0 nM (Ki)
DOCARPAMINE ? unclear Mechanistic
PALIPERIDONE inhibits Mechanistic
PRIDOPIDINE ~ modulates Mechanistic
OLANZAPINE inhibits Mechanistic 20.0 nM (Ki)
PEGDINETANIB inhibits Mechanistic
RISPERIDONE inhibits Mechanistic
PERGOLIDE MESYLATE activates Mechanistic
LISURIDE ? unclear Mechanistic 0.5 nM (Ki)
ZICRONAPINE ? unclear Mechanistic
CLEBOPRIDE inhibits Mechanistic
LAZERTINIB inhibits Mechanistic
REMOXIPRIDE HYDROCHLORIDE inhibits Mechanistic
CABERGOLINE activates Mechanistic
JNJ-37822681 inhibits Mechanistic
ERGOT ALKALOIDS ? unclear Mechanistic
(-)-STEPHOLIDINE ? unclear Mechanistic
SARIZOTAN HYDROCHLORIDE activates Mechanistic
5-OH-DPAT ? unclear Mechanistic
LITHIUM ? unclear Mechanistic
DIHYDROERGOTOXINE ? unclear Mechanistic
REMOXIPRIDE inhibits Mechanistic
BROMPERIDOL inhibits Mechanistic
PHENTOLAMINE ? unclear Mechanistic

Showing top 40 of 427 agents ranked by evidence tier.

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

Showing top 40 of 156 agents ranked by evidence tier.

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

Showing top 40 of 206 agents ranked by evidence tier.

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

Showing top 40 of 218 agents ranked by evidence tier.

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

Showing top 40 of 176 agents ranked by evidence tier.

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

Showing top 40 of 221 agents ranked by evidence tier.

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

Clinical Trials

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

NCT07680725 Phase: N/A Started: 2025-10-01

Parkinson's disease (PD) is a progressive neurodegenerative disorder characterized by rigidity, tremor, postural instability, bradykinesia, and autonomic dysfunction. It is a common movement disorder worldwide. Motor impairments in PD patients are not limited to the muscles of the extremities; the n...

NCT07682311 Phase: NA Started: 2026-06

Parkinson's disease (PD) affects \~1% of people over 60 years old, is highly disabling and represents a large economic burden. While dopaminergic medications effectively treat motor symptoms early in the disease, most patients develop complications, including motor fluctuations and dyskinesias, whic...

NCT07115563 Phase: NA Started: 2025-10-31

The goal of this clinical trial is to learn if a tailored amino acid supplement works to help adults living with Parkinson's disease to improve nutrition, metabolic function, body composition, and physical and mental function. The main questions it aims to answer are: Does the tailored amino acid s...

NCT01496599 Phase: N/A Started: 2012-01-24

Background: \- Parkinson s disease (PD) causes slow movement, stiffness, and tremor. It results from the loss of a brain chemical called dopamine. PD gets worse over time, but researchers do not fully understand why the brain cells that produce dopamine stop working or die in people with PD. This s...

NCT07681609 Phase: NA Started: 2021-05-31

Parkinson's disease is frequently associated with impaired postural control, balance deficits, trunk misalignment, altered proprioception, and an increased risk of falls. These impairments may contribute to reduced mobility, pain, loss of independence, and decreased quality of life. Although rehabil...

NCT05510739 Phase: NA Started: 2022-09-19

This study aims to determine the effects of a motor-cognitive exercise intervention, delivered in the home environment using eHealth methods, among people with Parkinson's disease. The intervention will support and motivate motor training, combined with cognitive training, aimed at attentional and e...

NCT07428447 Phase: N/A Started: 2026-02-18

Background and Rationale Essential tremor (ET) affects over 6 million Americans and approximately 5% of adults over age 60. Patients with ET have a 10-20 times higher risk of developing Parkinson's disease (PD) compared to age-matched populations, with approximately 1% converting to PD annually. Pos...

NCT01621581 Phase: PHASE1 Started: 2013-03-13

Background: \- Glial cell line-derived neurotrophic factor (GDNF) is a chemical that may help protect and strengthen brain cells that produce dopamine. Dopamine is a chemical that affects brain function. People with Parkinson's disease (PD) have problems producing dopamine in the brain. Researchers...

NCT04396873 Phase: PHASE1 Started: 2021-08-17

Background: About 5 million adults in the U.S. have Alzheimer s disease or another adult-onset neurodegenerative disorder. Many studies have found that inflammation in the brain contributes to these diseases. Researchers want to find a better way to measure this inflammation. Objective: To learn ...

NCT06464029 Phase: N/A Started: 2024-06-01

The purpose of this project is to use transcranial magnetic stimulation (TMS) to explore the state of excitability of corticocortical and corticofugal (cortex to spinal cord, cortex to brainstem to spinal cord) pathways that project to muscles that control the legs and trunk in people with Parkinson...

NCT01019343 Phase: NA Started: 2009-12-08

Background: * Previous studies have given researchers information on how the brain controls movement, how people learn to make fine, skilled movements, and why some people have movement disorders. However, further research is needed to learn more about the causes of most movement disorders, such as...

NCT06680830 Phase: PHASE2 Started: 2025-01-17

The goal of this Phase 2 clinical trial is to investigate the efficacy and safety of NEU-411 in men and women aged 40-80 years with early Parkinson's Disease (PD) who have predicted elevations in the activity of the "leucine-rich repeat kinase 2" ("LRRK2" for short) pathway based on their genetic pr...

NCT05901818 Phase: PHASE1 Started: 2021-04-01

This is a phase I, interventional, single arm, open-label, clinical study to evaluate the safety and efficacy of the striatal transplantation of autologous induced neural stem cell-derived DA precursor cells in Parkinson's Disease patients.

NCT07679256 Phase: N/A Started: 2024-01-01

Based on the high-quality Parkinson's disease cohort population and biological sample database in the early stage of the team, multi-dimensional intelligent wearable device technology and machine learning were used to screen and classify digital biomarkers in the prodromal PD cohort, early PD cohort...

NCT07629115 Phase: N/A Started: 2026-05-29

The purpose of this study is to evaluate the long-term safety of IPS101A and to assess the durability of efficacy in subjects who received IPS101A.


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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