Disease overview

Alterations

126

Molecular (A)

100

Clinical (B–E)

Direct drugs

160

Via biomarker

504

Natural agents

Natural products

Merged ranked

662

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Seeds, GreenMedInfo · Pipeline phase 7 · Updated 2026-07-06

Remission & chronicity

Spontaneous remission

NMIBC: 50-70% recurrence within 5 years; MIBC: progression to metastatic in 50% without radical treatment; spontaneous remission essentially zero

Best-intervention remission

NMIBC: BCG intravesical immunotherapy: 70% reduction in recurrence, 30-40% reduction in progression; MIBC with neoadjuvant cisplatin + radical cystectomy: 5-year survival 50-60% (vs 45% cystectomy alone); metastatic with enfortumab vedotin + pembrolizumab: 31.5 months OS vs 16.1 months cisplatin (EV-302/KEYNOTE-A39)

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Large — EV + pembrolizumab is a major advance but not yet universally standard; BCG supply shortage is a persistent clinical crisis in NMIBC

Primary barrier

access

Barrier detail: BCG shortage (since 2012, persistent) means many NMIBC patients cannot receive standard treatment; gemcitabine + mitomycin as alternative to BCG established as FDA-cleared 2024 (CISTO trial); EV + pembrolizumab is a genuine paradigm change for metastatic disease but requires PD-L1 testing integration

Last SoC change: 2023 — EV + pembrolizumab (EV-302) established as first-line for cisplatin-eligible metastatic UC; 2022 pembrolizumab second-line established; 2019 enfortumab vedotin approved; BCG standard for NMIBC since 1990

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0004986

Alterations

Showing 80 of 126 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Abdominal pain	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bladder neoplasm	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dysuria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hematuria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypercalcemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent urinary tract infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
%AE,ur(0-12) (Amount of Drug Excreted Via Urine During the Collection Interval 0 - 12 Hours Post Administration) of BAY1163877	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
ALK ALK receptor tyrosine kinase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
APC APC regulator of Wnt signaling pathway	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AR androgen receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ARID1A AT-rich interaction domain 1A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ATM ATM serine/threonine kinase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AUC(0-12) (Area Under the Plasma Concentration vs Time Curve From Time Zero to 12 Hours) of BAY1163877 After Single Dose Administration on Cycle 1, Day -3	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
AUC(0-12) (Area Under the Plasma Concentration vs Time Curve From Time Zero to 12 Hours) of BAY1163877 After Single Dose Administration on Cycle 1, Day 1	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
AUC(0-12)/D (Area Under the Plasma Concentration vs Time Curve From Time Zero to 12 Hours Divided by Dose) of BAY1163877 After Single Dose Administration on Cycle 1, Day -3	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
AUC(0-12)/D (Area Under the Plasma Concentration vs Time Curve From Time Zero to 12 Hours Divided by Dose) of BAY1163877 After Single Dose Administration on Cycle 1, Day 1	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
AUC(0-12)/Dmd (AUC(0-12) Divided by Dose After Multiple-dose Administration) of BAY1163877 on Cycle 1, Day 15	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
BRAF B-Raf proto-oncogene, serine/threonine kinase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BRCA1 BRCA1 DNA repair associated	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BRCA2 BRCA2 DNA repair associated	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
C11orf65 chromosome 11 open reading frame 65	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CCND1 cyclin D1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CCNE1 cyclin E1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CD274 CD274 molecule	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CDH1 cadherin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CDK12 cyclin dependent kinase 12	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CDK6 cyclin dependent kinase 6	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CDKN1A cyclin dependent kinase inhibitor 1A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CDKN2A cyclin dependent kinase inhibitor 2A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CES-D scale Illness Perception Questionnaire (IPQ)	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
CIC capicua transcriptional repressor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CREBBP CREB binding lysine acetyltransferase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CSF3R colony stimulating factor 3 receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CTNNB1 catenin beta 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Cancer worries scale	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
Comprehension of informed consent	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Decisional Regret Scale	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
Decisional Self-Efficacy Scale	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
Duration of Response (DOR)	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
EGFR epidermal growth factor receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
EML4 EMAP like 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
EP300 EP300 lysine acetyltransferase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ERBB2 erb-b2 receptor tyrosine kinase 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ERBB3 erb-b2 receptor tyrosine kinase 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ERCC2 ERCC excision repair 2, TFIIH core complex helicase subunit	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ESR1 estrogen receptor 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
EZH2 enhancer of zeste 2 polycomb repressive complex 2 subunit	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FANCC FA complementation group C	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FAT1 FAT atypical cadherin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FBXW7 F-box and WD repeat domain containing 7	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FGFR1 fibroblast growth factor receptor 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FGFR2 fibroblast growth factor receptor 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FGFR3 fibroblast growth factor receptor 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FGFR4 fibroblast growth factor receptor 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FLT4 fms related receptor tyrosine kinase 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FOXA1 forkhead box A1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
GATA3 GATA binding protein 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
GNAS GNAS complex locus	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HRAS HRas proto-oncogene, GTPase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KDM6A lysine demethylase 6A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KDR kinase insert domain receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KIT KIT proto-oncogene, receptor tyrosine kinase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KMT2C lysine methyltransferase 2C	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KMT2D lysine methyltransferase 2D	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KRAS KRas proto-oncogene, GTPase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Local Control	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
MDM2 MDM2 proto-oncogene	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MET MET proto-oncogene, receptor tyrosine kinase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MSH2 mutS homolog 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MTOR mechanistic target of rapamycin kinase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MYC MYC proto-oncogene, bHLH transcription factor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NCOR1 nuclear receptor corepressor 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NF1 neurofibromin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NFE2L2 NFE2 like bZIP transcription factor 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NOTCH1 notch receptor 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Number of Participants With Treatment-Emergent Laboratory Abnormalities (Hematology)	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Overall Survival	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
PDGFRA platelet derived growth factor receptor alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PIK3CA phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
POLD1 DNA polymerase delta 1, catalytic subunit	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, merged ranking, and clinical trial / literature evidence (top agents)

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
EPIRUBICIN	Small molecule	Approved	Strong	100	8 trials View evidence Registry trials NCT01094964 — Hyperthermia and Mitomycin C, Bacillus Calmette-Guerin, or Standard Therapy as Second-Line Therapy in Treating Patients With Recurrent Bladder Cancer (UNKNOWN) PHASE3 NCT06227065 — Precise Neoadjuvant Chemoresection of Low Grade NMIBC (NOT_YET_RECRUITING) PHASE2 NCT02760953 — TURBt With Adjuvant Cryoablation to Treat Bladder Cancer (UNKNOWN) NA NCT04736394 — A Phase 3 Study to Evaluate the Safety and Efficacy of APL-1202 as a Single-agent Oral Treatment Versus Intravesical Instillation of Epirubicin Hydrochloride in naïve Intermediate-risk NMIBC Patients (UNKNOWN) PHASE3 NCT04490993 — Safety and Efficacy of APL-1202 in Combination With Epirubicin Hydrochloride Versus Epirubicin Hydrochloride Alone in Intermediate and High-risk Chemo-refractory Non-muscle Invasive Bladder Cancer (NMIBC) Patients (UNKNOWN) PHASE3 NCT02438865 — Prophylactic Intravesical Chemotherapy After Radical Nephroureterectomy for Upper Tract Urothelial Carcinoma: a Randomized Controlled Trial Between Single Postoperative Dose Versus Maintenance Therapy. (COMPLETED) PHASE2 NCT05547516 — Blue Laser -5ala Photodynamic Therapy (PDT) in High-Risk Non-muscle Invasive Bladder Cancer (NMIBC) Patients (UNKNOWN) NA NCT05975151 — Efficacy and Safety of Pseudomonas Aeruginosa for Intermediate and High-risk Non-muscle Invasive Bladder Cancer (UNKNOWN) PHASE2 CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
SIROLIMUS	Small molecule	Approved	Strong	100	8 trials View evidence Registry trials NCT00375245 — Rapamycin With Grapefruit Juice for Advanced Malignancies (COMPLETED) PHASE1 NCT01938573 — Sirolimus, Cisplatin, and Gemcitabine Hydrochloride in Treating Patients With Bladder Cancer (COMPLETED) PHASE1, PHASE2 NCT01090466 — Gemcitabine Hydrochloride, Cisplatin, and Temsirolimus as First-Line Therapy in Treating Patients With Locally Advanced and/or Metastatic Transitional Cell Cancer of the Urothelium (COMPLETED) PHASE1, PHASE2 NCT02753309 — A Study of Rapamycin Combined With Intravesical BCG in Patients With Non-muscle Invasive Bladder Cancer (COMPLETED) EARLY_PHASE1 NCT01215136 — First-line Everolimus +/- Paclitaxel for Cisplatin-ineligible Patients With Advanced Urothelial Carcinoma (TERMINATED) PHASE2 NCT01522820 — Vaccine Therapy With or Without Sirolimus in Treating Patients With NY-ESO-1 Expressing Solid Tumors (COMPLETED) PHASE1 NCT04375813 — Trial of Encapsulated Rapamycin (eRapa) for Bladder Cancer Prevention (ACTIVE_NOT_RECRUITING) PHASE2 NCT00933374 — Trial to Evaluate Paclitaxel Plus RAD001 in Urothelial Carcinoma (TERMINATED) PHASE2 CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
3,3',4',5-TETRACHLOROSALICYLANILIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ABEMACICLIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ABIRATERONE ACETATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAMINOPHEN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ACETOPHENAZINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AFATINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ALCLOFENAC Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALCLOMETASONE DIPROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALECTINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ALECTINIB HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALPELISIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMCINONIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMDINOCILLIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMIODARONE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMLEXANOX Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMLODIPINE BESYLATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANHYDROUS TACROLIMUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
APALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
APIGENIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
APOMORPHINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
APREMILAST Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ARSENIC TRIOXIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ASTEMIZOLE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATALUREN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATEZOLIZUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATRACURIUM BESYLATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATROPINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AVELUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AXITINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AZACITIDINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AZARIBINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BECLOMETHASONE DIPROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BENAZEPRIL HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BENDAMUSTINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BENORILATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BETAMETHASONE DIPROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE SODIUM PHOSPHATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE VALERATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BEVACIZUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BIFONAZOLE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BINIMETINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BISACODYL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BITHIONOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—

Drug	Type	Phase	Evidence	Score	Sources	Links
APAZIQUONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
APROTININ	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ATEZOLIZUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BCG LIVE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CISPLATIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DURVALUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ENFORTUMAB VEDOTIN	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
FLUOROURACIL	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
GEMCITABINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
KEYHOLE LIMPET HEMOCYANIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LAROTAXEL	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
MITOMYCIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
NADOFARAGENE FIRADENOVEC	Gene therapy	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
OPORTUZUMAB MONATOX	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
PEMBROLIZUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
RAMUCIRUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ANTINEOPLASTON A10	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
APATORSEN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
APATORSEN SODIUM	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
AVELUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
AZD-4877	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BRIGIMADLIN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BRIVANIB ALANINATE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CABAZITAXEL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CABOZANTINIB	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CADONILIMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CARBOPLATIN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CEMIPLIMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CRETOSTIMOGENE GRENADENOREPVEC	Gene therapy	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CYCLOSPORINE	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DARGISTOTUG	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DEMPLATIN PEGRAGLUMER	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DIENESTROL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DISITAMAB VEDOTIN	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DOCETAXEL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DOMVANALIMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DOSTARLIMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DOXECITINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DOXORUBICIN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
EGANELISIB	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
3,3',4',5-TETRACHLOROSALICYLANILIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ABEMACICLIB Repurposing via CCND1	Small molecule	Approved	Strong	95	—	DGIdb	—
ABIRATERONE ACETATE Repurposing via APC	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAMINOPHEN Repurposing via CCND1	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAZOLAMIDE Repurposing via ALK	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ACETOPHENAZINE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AFATINIB Repurposing via BRAF	Small molecule	Approved	Strong	95	—	DGIdb	—
ALCLOFENAC Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALCLOMETASONE DIPROPIONATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALECTINIB Repurposing via BRAF	Small molecule	Approved	Strong	95	—	DGIdb	—
ALECTINIB HYDROCHLORIDE Repurposing via ALK	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALPELISIB Repurposing via BRAF	Small molecule	Approved	Strong	95	—	DGIdb	—
AMCINONIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AMDINOCILLIN Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AMIODARONE HYDROCHLORIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMLEXANOX Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AMLODIPINE BESYLATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMPHETAMINE Repurposing via ALK	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANHYDROUS TACROLIMUS Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
APALUTAMIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
APIGENIN Repurposing via BRCA1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
APOMORPHINE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
APREMILAST Repurposing via CDK6	Small molecule	Approved	Strong	95	—	DGIdb	—
ARSENIC TRIOXIDE Repurposing via CCND1	Small molecule	Approved	Strong	95	—	DGIdb	—
ASTEMIZOLE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ATALUREN Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ATEZOLIZUMAB Repurposing via ARID1A	Small molecule	Approved	Strong	95	—	DGIdb	—
ATRACURIUM BESYLATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ATROPINE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AVELUMAB Repurposing via CD274	Small molecule	Approved	Strong	95	—	DGIdb	—
AXITINIB Repurposing via BRAF	Small molecule	Approved	Strong	95	—	DGIdb	—
AZACITIDINE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AZARIBINE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BECLOMETHASONE DIPROPIONATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
BENAZEPRIL HYDROCHLORIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BENDAMUSTINE Repurposing via ATM	Small molecule	Approved	Strong	95	—	DGIdb	—
BENORILATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
BETAMETHASONE DIPROPIONATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE SODIUM PHOSPHATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets

Natural Products

3 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked)

Reference lookups: GreenMedInfo · Examine.com

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
Curcumin Natural product Animal Study Curcumin may increase muscle tone in urinary bladder.	Food compound	Preclinical	Emerging	7	No indexed hits View evidence CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Cannabidiol Natural product Review Cannabinoids are promising candidates for gastrointestinal and urinary…	Food compound	Preclinical	Emerging	2	No indexed hits View evidence CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Cannabinoids Natural product Review Cannabinoids are promising candidates for gastrointestinal and urinary…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	GreenMedInfo	GreenMedInfo

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.

Disease overview

Remission & chronicity

Alterations

Therapeutics

Natural Products

Part of the Open Source Medicine Foundation Network