Myelodysplastic Syndromes (MDS) — RepurpOS

Disease overview

Alterations

178

Molecular (A)

100

Clinical (B–E)

Direct drugs

420

Via biomarker

158

Natural agents

Natural products

Merged ranked

576

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Examine.com, disease_db_100 · Pipeline phase 7 · Updated 2026-07-06

Remission & chronicity

Spontaneous remission

Essentially zero; progressive cytopaenia and transformation to AML in 30-40% of higher-risk MDS over 2-5 years; median survival low-risk MDS ~5 years, high-risk ~1 year without treatment

Best-intervention remission

Allogeneic HSCT: only curative option; 5-year OS 35-50% in fit patients; hypomethylating agents (azacitidine, decitabine): ORR 15-17%, overall survival improvement 9 months vs SC; oral azacitidine (CC-486): 10.7 vs 8.9 months OS in AML maintenance post-remission

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Large — MDS has limited treatment options; curative HSCT used in <15% of patients (age, comorbidity, donor availability); existing drugs are not curative

Primary barrier

biological + access

Barrier detail: MDS predominantly affects elderly patients who are often HSCT-ineligible; hypomethylating agents provide modest benefit without cure; luspatercept (TGF-β trap) approved for LR-MDS with ring sideroblasts 2020 — improved transfusion independence; imetelstat for LR-MDS — Phase 3 (IMerge) showed independence from transfusion in 40% vs 15% placebo

Last SoC change: 2023 — imetelstat (telomerase inhibitor) FDA approved for LR-MDS; luspatercept (2020) for RS+ LR-MDS; oral azacitidine (2020); parenteral azacitidine since 2004; EPO since mid-1990s

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

Identifiers: MONDO: MONDO:0018881

Right to Try Relevance Critical

Eligibility: This disease meets the federal and state right-to-try legal standard (21 U.S.C. § 360bbb-0a and Montana SB 535): it lacks disease-modifying therapy with indication-specific FDA approval. Patients with Myelodysplastic Syndrome may be eligible to access investigational therapies outside of clinical trials under right-to-try frameworks.

Investigational Agents

Phase 3: 40

Most Advanced Phase

Phase 3

Highest dev. stage

Gene Targets

178

Actionable

Pipeline Compounds (Top 6)

Alemtuzumab, Antilymphocyte Immunoglobulin (Horse), Apg-2575, Arsenic Trioxide, Asparaginase, Asparaginase Erwinia Chrysanthemi

Legal Standard: Right-to-try requires the disease lacks "adequate approved therapy for that specific disease" (not symptom-management drugs). This distinction means a disease with antidepressants available still qualifies if no FDA approval exists for THAT disease indication.

View Right-to-Try Compendium

Alterations

Showing 80 of 178 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Abnormal bleeding	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal cardiac ventricular function	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal circulating albumin concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Abnormal erythrocyte morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal facial shape	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal megakaryocyte morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal neutrophil morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal thorax morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormality of bone marrow stromal cells	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Acute myeloid leukemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anemia of inadequate production	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anisocytosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bone marrow hypercellularity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bone marrow hypocellularity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bone pain	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Brachydactyly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Broad clavicles	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cataract	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cerebral hypoplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Chronic infection	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Coarse facial features	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Congestive heart failure	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased total B cell count	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased total leukocyte count	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased total lymphocyte count	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased total neutrophil count	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Delayed eruption of teeth	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dry skin	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dysplastic granulopoesis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dyspnea	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Eczematoid dermatitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Erythroid dysplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Erythroid hypoplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Exertional dyspnea	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Fatigue	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Fever	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gingival overgrowth	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hearing impairment	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Increased circulating lactate dehydrogenase concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Increased mean corpuscular volume	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Increased total leukocyte count	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Intellectual disability	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Macrocytic anemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Megakaryocyte dysplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Megakaryocyte nucleus hypolobulation	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Midface retrusion	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Multiple lineage myelodysplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Myelodysplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Neurodevelopmental delay	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Noncompaction cardiomyopathy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Normochromic anemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Normocytic anemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Palpitations	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pancytopenia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Panniculitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pedal edema	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent lower respiratory tract infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent upper respiratory tract infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Refractory macrocytic anemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Reticulocytopenia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Rhizomelic arm shortening	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Short humerus	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Short metacarpal	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Short stature	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Single lineage myelodysplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Subcutaneous nodule	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Thrombocytopenia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Thrombocytosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Tricuspid regurgitation	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Trigonocephaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
ASXL1 ASXL transcriptional regulator 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BCL2 BCL2 apoptosis regulator	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BCR BCR activator of RhoGEF and GTPase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BLM BLM RecQ like helicase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CD33 CD33 molecule	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CD47 CD47 molecule	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CDA cytidine deaminase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CEP43 centrosomal protein 43	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CNTRL centriolin	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets

FDA-Approved Therapies

Phase 4 agents with regulatory approval for this indication

Drug Name	Mechanism	Type
4-PHENYLBUTYRIC ACID		Gene-targeted
ACETAZOLAMIDE	None	Gene-targeted
AMPHETAMINE	None	Gene-targeted
ASCIMINIB		Gene-targeted
ASCIMINIB HYDROCHLORIDE	inhibitor	Gene-targeted
AZACITIDINE		Gene-targeted
BEXAROTENE		Gene-targeted
BICALUTAMIDE	None	Gene-targeted
BLINATUMOMAB		Gene-targeted
BORTEZOMIB		Gene-targeted
BOSUTINIB		Gene-targeted
BOSUTINIB MONOHYDRATE	inhibitor	Gene-targeted
BRENTUXIMAB VEDOTIN		Gene-targeted
BUSULFAN		Gene-targeted
CARBACHOL	None	Gene-targeted
CARBOPLATIN		Gene-targeted
CEDAZURIDINE	inhibitor; inhibitor	Gene-targeted
CIPROFLOXACIN	None	Gene-targeted
CISPLATIN		Gene-targeted
CLOMIPRAMINE	None	Gene-targeted
CYTARABINE		Gene-targeted
DASATINIB ANHYDROUS	inhibitor	Gene-targeted
DAUNORUBICIN HYDROCHLORIDE		Gene-targeted
DAUNORUBICIN LIPOSOMAL		Gene-targeted
DIAZEPAM	None	Gene-targeted
DICHLORPHENAMIDE	None	Gene-targeted
DIETHYLSTILBESTROL		Gene-targeted
DIETHYLSTILBESTROL	None	Gene-targeted
DOCETAXEL ANHYDROUS		Gene-targeted
DOXORUBICIN HYDROCHLORIDE		Gene-targeted
EDARAVONE		Gene-targeted
EFBEMALENOGRASTIM ALFA	agonist	Gene-targeted
EFLAPEGRASTIM	agonist	Gene-targeted
EPIRUBICIN	None	Gene-targeted
EPIRUBICIN HYDROCHLORIDE		Gene-targeted
ERYTHROMYCIN		Gene-targeted
ESTRAMUSTINE		Gene-targeted
ETHOXZOLAMIDE	None	Gene-targeted
ETOPOSIDE		Gene-targeted
FENOLDOPAM MESYLATE		Gene-targeted

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, and merged ranking. Only agents with registry trials or published clinical literature are shown by default.

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
SIROLIMUS	Small molecule	Approved	Strong	105	8 trials 2 RCT View evidence Registry trials NCT01244906 — Post-transplant Cyclophosphamide and Sirolimus Following Reduced Intensity Conditioning (RIC) Transplant (COMPLETED) PHASE2 NCT02061800 — CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant (ACTIVE_NOT_RECRUITING) PHASE1, PHASE2 NCT00589563 — Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients Undergoing a Donor Stem Cell Transplant For Hematological Cancer (COMPLETED) PHASE2 NCT01488253 — Sirolimus/Tacrolimus Combination After HLA Matched Related Peripheral Blood Stem Cell Transplants (TERMINATED) PHASE2 NCT00105001 — Tacrolimus and Mycophenolate Mofetil With or Without Sirolimus in Preventing Acute Graft-Versus-Host Disease in Patients Who Are Undergoing Donor Stem Cell Transplant for Hematologic Cancer (COMPLETED) PHASE2 NCT00544115 — Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders (ACTIVE_NOT_RECRUITING) PHASE2 NCT05088356 — Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft (ACTIVE_NOT_RECRUITING) PHASE1 NCT00819546 — RAD001 in Combination With PKC412 in Patients With Relapsed, Refractory or Poor Prognosis AML or MDS (ACTIVE_NOT_RECRUITING) PHASE1 Published literature Randomized controlled trial Triple-Drug Graft-versus-Host Disease Prophylaxis after HLA-Matched Unrelated Donor Nonmyeloablative Allogenic Hematopoietic Stem Cell Transplantation (Transplantation and cellular therapy, 2023) Randomized controlled trial Effect of Sirolimus on Immune Reconstitution Following Myeloablative Allogeneic Stem Cell Transplantation: An Ancillary Analysis of a Randomized Controlled Trial Comparing Tacrolimus/Sirolimus and Tacrolimus/Methotrexate (Blood and Marrow Transplant Clinical Trials Network/BMT CTN 0402) (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2019) CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
TACROLIMUS ANHYDROUS	Small molecule	Approved	Strong	105	8 trials View evidence Registry trials NCT00402558 — Alloreactive NK Cells for Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) (COMPLETED) PHASE1 NCT05316701 — Precision-T: A Randomized Study of Orca-T in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies (ACTIVE_NOT_RECRUITING) PHASE3 NCT01518153 — Planned Donor Lymphocyte Infusion (DLI) After Allogeneic Stem Cell Transplantation (SCT) (TERMINATED) PHASE2 NCT01875237 — Donor Lymphocyte Infusion (DLI) of T-cells Genetically Modified With iCasp9 Suicide Gene (TERMINATED) PHASE1, PHASE2 NCT00818961 — Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Cancer (TERMINATED) PHASE2 NCT00322101 — Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia (COMPLETED) PHASE3 NCT04644016 — Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders (RECRUITING) PHASE2 NCT02566304 — Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
4-PHENYLBUTYRIC ACID Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ASCIMINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ASCIMINIB HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AZACITIDINE Repurposing	Small molecule	Approved	Strong	95	8 trials 6 Cochrane 11 Meta-analysis 89 Trial pubs View evidence Registry trials NCT01893372 — Eltrombopag With or Without Hypomethylating Agent After Hypomethylating Agent Failure For Patients With Myelodysplastic Syndrome (MDS) (COMPLETED) PHASE2 NCT01595295 — Registry on Hypomethylating Agents in Myeloid Neoplasms (COMPLETED) NCT03486353 — A Study of FF-10501-01 in Combination with Azacitidine in Patients with Myelodysplastic Syndrome (WITHDRAWN) PHASE2 NCT04638309 — APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) (TERMINATED) PHASE1 NCT03151304 — A Safety and Efficacy Study of Pracinostat and Azacitidine in Patients With High Risk Myelodysplastic Syndromes (TERMINATED) PHASE2 NCT01260714 — Azacitidine, Mitoxantrone Hydrochloride, and Etoposide in Treating Older Patients With Poor-Prognosis Acute Myeloid Leukemia (TERMINATED) PHASE1 NCT03613532 — Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN (ACTIVE_NOT_RECRUITING) PHASE1 NCT05829226 — A Phase 1 Study With LYT-200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS) (COMPLETED) PHASE1 Published literature Cochrane review The efficacy and safety of venetoclax and azacytidine combination treatment in patients with acute myeloid leukemia and myelodysplastic syndrome: systematic review and meta-analysis (Hematology (Amsterdam, Netherlands), 2023) Cochrane review Maintenance With Hypomethylating Agents After Allogeneic Stem Cell Transplantation in Acute Myeloid Leukemia and Myelodysplastic Syndrome: A Systematic Review and Meta-Analysis (Frontiers in medicine, 2022) Cochrane review Comparison Between Decitabine and Azacitidine for Patients With Acute Myeloid Leukemia and Higher-Risk Myelodysplastic Syndrome: A Systematic Review and Network Meta-Analysis (Frontiers in pharmacology, 2021) Cochrane review The efficacy and adverse events of venetoclax in combination with hypomethylating agents treatment for patients with acute myeloid leukemia and myelodysplastic syndrome: a systematic review and meta-analysis (Hematology (Amsterdam, Netherlands), 2020) Cochrane review A systematic review and network meta-analysis comparing azacitidine and decitabine for the treatment of myelodysplastic syndrome (Systematic reviews, 2018) Meta-analysis Emerging treatment approaches for VEXAS syndrome: a systematic review and meta-analysis (Annals of hematology, 2025) Meta-analysis Efficacy of epigenetic agents for older patients with acute myeloid leukemia and myelodysplastic syndrome in randomized controlled trials: a systematic review and network meta-analysis (Clinical and experimental medicine, 2023) Meta-analysis Azacitidine Monotherapy in Patients With Treatment-Naïve Higher-risk Myelodysplastic Syndrome: A Systematic Literature Review and Meta-analysis (Clinical lymphoma, myeloma & leukemia, 2023) Clinical trial publication Selinexor in combination with azacitidine or ruxolitinib in myelodysplastic/myeloproliferative neoplasm overlap syndromes: A multicenter prospective study (Cancer, 2026) Clinical trial publication Checkpoint immunotherapy is associated with preferential activation of tumor antigen-specific CD4(+) T cells in MDS (Blood neoplasia, 2025) Clinical trial publication Olutasidenib alone or combined with azacitidine in patients with mutant IDH1 myelodysplastic syndrome (Blood advances, 2025) Clinical trial publication Bexmarilimab plus azacitidine for high-risk myelodysplastic syndrome and relapsed or refractory acute myeloid leukaemia: results from the dose-escalation part of a multicentre, single-arm, phase 1/2 trial (The Lancet. Haematology, 2025) CT.gov search · Cochrane · PubMed	DGIdb	—
BEXAROTENE Repurposing	Small molecule	Approved	Strong	95	1 trials 1 Trial pubs View evidence Registry trials NCT00425477 — Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BLINATUMOMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BORTEZOMIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BOSUTINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BOSUTINIB MONOHYDRATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BRENTUXIMAB VEDOTIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BUSULFAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBOPLATIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEDAZURIDINE Repurposing	Small molecule	Approved	Strong	95	8 trials 3 Trial pubs View evidence Registry trials NCT03613532 — Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN (ACTIVE_NOT_RECRUITING) PHASE1 NCT06484062 — Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes (RECRUITING) PHASE1 NCT05201066 — Roll-over Study for Patients Who Have Completed a Prior Novartis-sponsored Sabatolimab (MBG453) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Sabatolimab. (ACTIVE_NOT_RECRUITING) PHASE2 NCT04013880 — ASTX727 and FT-2102 in Treating IDH1-Mutated Recurrent/Refractory Myelodysplastic Syndrome or Acute Myeloid Leukemia (WITHDRAWN) PHASE1, PHASE2 NCT05010122 — ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome (RECRUITING) PHASE1, PHASE2 NCT04655755 — Venetoclax in Combination With ASTX727 for the Treatment of Treatment-Naive High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia (ACTIVE_NOT_RECRUITING) PHASE1, PHASE2 NCT04953897 — Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Renal Impairment (RECRUITING) PHASE1 NCT03906695 — Phase 1 Trial of ASTX727 in Subjects With Lower-risk Myelodysplastic Syndromes (ACTIVE_NOT_RECRUITING) PHASE1 Published literature Systematic review Health-related quality of life of patients with acute myeloid leukemia and myelodysplastic syndromes/neoplasms treated with decitabine: a systematic literature review (Annals of hematology, 2026) Clinical trial publication Venetoclax/FluBu2 RIC transplant followed by all-oral venetoclax/decitabine maintenance for poor-risk MDS/AML (Blood advances, 2026) Clinical trial publication Oral decitabine and cedazuridine maintenance after haematopoietic stem-cell transplantation in very high-risk acute myeloid leukaemia or myelodysplastic syndrome (GFM-DACORAL-DLI): a multicentre, single-arm, phase 2 trial (The Lancet. Haematology, 2025) Clinical trial publication Oral decitabine and cedazuridine plus venetoclax for older or unfit patients with acute myeloid leukaemia: a phase 2 study (The Lancet. Haematology, 2024) CT.gov search · Cochrane · PubMed	DGIdb	—
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CYTARABINE Repurposing	Small molecule	Approved	Strong	95	8 trials 4 Meta-analysis 32 RCT View evidence Registry trials NCT00740181 — Decitabine, Cytarabine, GCSF for Refractory AML/MDS (TERMINATED) PHASE2 NCT00015951 — Bevacizumab, Cytarabine, and Mitoxantrone on Treating Patients With Hematologic Cancers (COMPLETED) PHASE2 NCT00002833 — Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia (COMPLETED) PHASE2 NCT01302106 — Study of Clofarabine in Combination With Low Dose Cytarabine to Treat Myelodysplastic Syndromes (WITHDRAWN) PHASE2 NCT01297543 — Safety Study of Human Myeloid Progenitor Cells (CLT-008) After Chemotherapy for Leukemia (COMPLETED) PHASE1, PHASE2 NCT01831232 — Idarubicin, Cytarabine, and Pravastatin Sodium in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes (COMPLETED) NA NCT00368355 — T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts (COMPLETED) PHASE2 NCT00046930 — Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia (COMPLETED) PHASE3 Published literature Meta-analysis [Efficacy and Safety of Decitabine Combined with CAG (Cytarabine, Aclarubicin, G-CSF) for Patients with Intermediate or High Risk Myelodysplastic Syndrome and Acute Myeloid Leukemia: a Meta-Analysis] (Zhongguo shi yan xue ye xue za zhi, 2019) Meta-analysis HAG (Homoharringtonine, Cytarabine, G-CSF) Regimen for the Treatment of Acute Myeloid Leukemia and Myelodysplastic Syndrome: A Meta-Analysis with 2,314 Participants (PloS one, 2016) Meta-analysis Outcomes after (90) Yttrium-ibritumomab tiuxetan-BEAM in diffuse large B-cell lymphoma: a meta-analysis (Cancer medicine, 2014) Meta-analysis A meta-analysis of CAG (cytarabine, aclarubicin, G-CSF) regimen for the treatment of 1029 patients with acute myeloid leukemia and myelodysplastic syndrome (Journal of hematology & oncology, 2011) Systematic review TKIs combined with chemotherapy followed by allo-HSCT in Philadelphia chromosome-positive myelodysplastic syndrome: A case report and literature review (Medicine, 2022) Systematic review Myeloid neoplasms with t(12;22)(p13;q12)/MN1-EVT6: a systematic review of 12 cases (Annals of hematology, 2018) Randomized controlled trial Patient-reported outcomes in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia receiving standard chemotherapy plus quizartinib or placebo (QuANTUM-First): a global, randomised, placebo-controlled, phase 3 trial (The Lancet. Haematology, 2026) Randomized controlled trial Addition of the nuclear export inhibitor selinexor to standard intensive treatment for elderly patients with acute myeloid leukemia and high risk myelodysplastic syndrome (Leukemia, 2022) Randomized controlled trial Subsequent malignant neoplasms among children with Hodgkin lymphoma: a report from the Children's Oncology Group (Blood, 2021) Randomized controlled trial [Clinical Efficacy of Low-Dose Decitabine alone for Treatment of Myelodysplastic Syndrome] (Zhongguo shi yan xue ye xue za zhi, 2019) Randomized controlled trial Glasdegib plus intensive/nonintensive chemotherapy in untreated acute myeloid leukemia: BRIGHT AML 1019 Phase III trials (Future oncology (London, England), 2019) CT.gov search · Cochrane · PubMed	DGIdb	—
DASATINIB ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DAUNORUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	8 trials View evidence Registry trials NCT00002833 — Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia (COMPLETED) PHASE2 NCT01831232 — Idarubicin, Cytarabine, and Pravastatin Sodium in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes (COMPLETED) NA NCT00046930 — Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia (COMPLETED) PHASE3 NCT02521493 — Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome (ACTIVE_NOT_RECRUITING) PHASE3 NCT01342692 — Best Promising Drug Association With Azacitidine in Higher Risk Myelodysplastic Syndromes (UNKNOWN) PHASE2 NCT00002517 — Combination Chemotherapy in Treating Children With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome (COMPLETED) PHASE3 NCT06050941 — Reduced Intensive Idarubicin and Cytarabine Plus Venetoclax as First-line Treatment for Adults AML and MDS (NOT_YET_RECRUITING) PHASE2 NCT07046078 — Combination Chemotherapy (FLAG-Ida) Followed Immediately by Reduced-Intensity Total Body Radiation Therapy and Donor Hematopoietic Cell Transplant for the Treatment of Adults Age 60 and Older With Newly Diagnosed Adverse-Risk Acute Myeloid Leukemia or Other High-Grade Myeloid Cancer (RECRUITING) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
DAUNORUBICIN LIPOSOMAL Repurposing	Small molecule	Approved	Strong	95	8 trials View evidence Registry trials NCT00002833 — Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia (COMPLETED) PHASE2 NCT01831232 — Idarubicin, Cytarabine, and Pravastatin Sodium in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes (COMPLETED) NA NCT00046930 — Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia (COMPLETED) PHASE3 NCT02521493 — Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome (ACTIVE_NOT_RECRUITING) PHASE3 NCT01342692 — Best Promising Drug Association With Azacitidine in Higher Risk Myelodysplastic Syndromes (UNKNOWN) PHASE2 NCT00002517 — Combination Chemotherapy in Treating Children With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome (COMPLETED) PHASE3 NCT06050941 — Reduced Intensive Idarubicin and Cytarabine Plus Venetoclax as First-line Treatment for Adults AML and MDS (NOT_YET_RECRUITING) PHASE2 NCT07046078 — Combination Chemotherapy (FLAG-Ida) Followed Immediately by Reduced-Intensity Total Body Radiation Therapy and Donor Hematopoietic Cell Transplant for the Treatment of Adults Age 60 and Older With Newly Diagnosed Adverse-Risk Acute Myeloid Leukemia or Other High-Grade Myeloid Cancer (RECRUITING) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DOXORUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	8 trials View evidence Registry trials NCT00237627 — Doxorubicin Hydrochloride Liposome and Bortezomib in Treating Patients With Refractory Hematologic Cancer or Malignant Solid Tumor or Metastatic Breast Cancer (COMPLETED) PHASE1, PHASE2 NCT00036790 — Motexafin Gadolinium and Doxorubicin in Treating Patients With Advanced Cancer (COMPLETED) PHASE1 NCT02356159 — Study of Palifermin (Kepivance) in Persons Undergoing Unrelated Donor Allogeneic Hematopoietic Cell Transplantation (COMPLETED) PHASE1, PHASE2 NCT03059615 — A Phase 2a, Open-Label, Two Stage Study of Nerofe or Nerofe With Doxorubicin in Subjects With AML or MDS (WITHDRAWN) PHASE2 NCT03878524 — Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial (TERMINATED) PHASE1 NCT00003243 — Combination Chemotherapy Plus Infusion of White Blood Cells in Treating Patients With Hematologic Cancer (COMPLETED) PHASE1 NCT00520130 — Chemotherapy and Unrelated Donor Stem Cell Transplantation for Patients With Cancers of the Blood and Immune System (COMPLETED) PHASE1, PHASE2 NCT00074490 — Donor Stem Cell Transplant With No or Low-Intensity Chemotherapy Using Sirolimus and Treated Immune Cells to Treat Blood and Lymph Cancers (TERMINATED) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
EDARAVONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
EFBEMALENOGRASTIM ALFA Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
EFLAPEGRASTIM Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
EPIRUBICIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
EPIRUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ERYTHROMYCIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ESTRAMUSTINE Repurposing	Small molecule	Approved	Strong	95	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ETHOXZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ETOPOSIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
FENOLDOPAM MESYLATE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
FLOXURIDINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
FLUMAZENIL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
GEMCITABINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
GEMTUZUMAB OZOGAMICIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
GENTIAN VIOLET CATION Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
HYDROQUINONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
HYDROXYUREA Repurposing	Small molecule	Approved	Strong	95	8 trials 11 Trial pubs View evidence Registry trials NCT02626715 — Reduced-Intensity Conditioning (RIC) and Myeloablative Conditioning (MAC) for HSCT in AML/MDS (COMPLETED) PHASE2 NCT00005823 — Intensive Compared With Nonintensive Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome (COMPLETED) PHASE3 NCT00083187 — VNP40101M in Treating Patients With Acute Myelogenous Leukemia or High-Risk Myelodysplasia (COMPLETED) PHASE2 NCT02017457 — Azacytidine and Lymphocytes in Relapse of AML or MDS After Allogeneic Stem Cell Transplantation. (COMPLETED) PHASE2 NCT02158858 — A Phase 1/2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Hematologic and Myeloproliferative Malignancies (COMPLETED) PHASE1, PHASE2 NCT06175923 — Role of BMP Pathway in MDS Progression (NOT_YET_RECRUITING) NCT01828619 — Study of New RIC Regimen of BuFlu in Older and/or Intolerable Patients (UNKNOWN) NCT06199557 — A Study to Investigate Treatment of HU and VPA, or 6-MP and VPA in Unfit AML/HR-MDS Patients (RECRUITING) PHASE1, PHASE2 Published literature Clinical trial publication Real-Life experience with hydroxyurea in patients with sickle cell disease: Results from the prospective ESCORT-HU cohort study (American journal of hematology, 2021) Clinical trial publication Busulfan in patients with polycythemia vera or essential thrombocythemia refractory or intolerant to hydroxyurea (Annals of hematology, 2014) Clinical trial publication Treatment of polycythemia vera with hydroxyurea and pipobroman: final results of a randomized trial initiated in 1980 (Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2011) Clinical trial publication A comparison of low-dose cytarabine and hydroxyurea with or without all-trans retinoic acid for acute myeloid leukemia and high-risk myelodysplastic syndrome in patients not considered fit for intensive treatment (Cancer, 2007) Clinical trial publication Clinical trial of valproic acid and all-trans retinoic acid in patients with poor-risk acute myeloid leukemia (Cancer, 2005) CT.gov search · Cochrane · PubMed	DGIdb	—
IBRUTINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—

Drug	Type	Phase	Evidence	Score	Sources	Links
ALEMTUZUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
APG-2575	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ARSENIC TRIOXIDE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ASPARAGINASE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ASPARAGINASE ERWINIA CHRYSANTHEMI	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BUSULFAN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CALCITRIOL	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CASPOFUNGIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CISPLATIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CYCLOPHOSPHAMIDE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CYCLOSPORINE	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CYTARABINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DARBEPOETIN ALFA	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DAUNORUBICIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DAUNORUBICIN HYDROCHLORIDE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DEXAMETHASONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DOCETAXEL	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ELRITERCEPT	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ENASIDENIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
EPOETIN BETA	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
EPRENETAPOPT	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ETELCALCETIDE	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ETOPOSIDE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
FLUDARABINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
FLUDARABINE PHOSPHATE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
GEMTUZUMAB OZOGAMICIN	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
GILTERITINIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
GLASDEGIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
GUADECITABINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
HYDROCORTISONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
HYDROXYUREA	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
IDARUBICIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ISOTRETINOIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
IVOSIDENIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LEMZOPARLIMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LENOGRASTIM	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LONAFARNIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LORNOXICAM	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
MAGROLIMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
4-PHENYLBUTYRIC ACID Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAZOLAMIDE Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMPHETAMINE Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ASCIMINIB Repurposing via BCR	Small molecule	Approved	Strong	95	—	DGIdb	—
ASCIMINIB HYDROCHLORIDE Repurposing via BCR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AZACITIDINE Repurposing via CDA	Small molecule	Approved	Strong	95	8 trials 6 Cochrane 11 Meta-analysis 89 Trial pubs View evidence Registry trials NCT01893372 — Eltrombopag With or Without Hypomethylating Agent After Hypomethylating Agent Failure For Patients With Myelodysplastic Syndrome (MDS) (COMPLETED) PHASE2 NCT01595295 — Registry on Hypomethylating Agents in Myeloid Neoplasms (COMPLETED) NCT03486353 — A Study of FF-10501-01 in Combination with Azacitidine in Patients with Myelodysplastic Syndrome (WITHDRAWN) PHASE2 NCT04638309 — APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) (TERMINATED) PHASE1 NCT03151304 — A Safety and Efficacy Study of Pracinostat and Azacitidine in Patients With High Risk Myelodysplastic Syndromes (TERMINATED) PHASE2 NCT01260714 — Azacitidine, Mitoxantrone Hydrochloride, and Etoposide in Treating Older Patients With Poor-Prognosis Acute Myeloid Leukemia (TERMINATED) PHASE1 NCT03613532 — Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN (ACTIVE_NOT_RECRUITING) PHASE1 NCT05829226 — A Phase 1 Study With LYT-200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS) (COMPLETED) PHASE1 Published literature Cochrane review The efficacy and safety of venetoclax and azacytidine combination treatment in patients with acute myeloid leukemia and myelodysplastic syndrome: systematic review and meta-analysis (Hematology (Amsterdam, Netherlands), 2023) Cochrane review Maintenance With Hypomethylating Agents After Allogeneic Stem Cell Transplantation in Acute Myeloid Leukemia and Myelodysplastic Syndrome: A Systematic Review and Meta-Analysis (Frontiers in medicine, 2022) Cochrane review Comparison Between Decitabine and Azacitidine for Patients With Acute Myeloid Leukemia and Higher-Risk Myelodysplastic Syndrome: A Systematic Review and Network Meta-Analysis (Frontiers in pharmacology, 2021) Cochrane review The efficacy and adverse events of venetoclax in combination with hypomethylating agents treatment for patients with acute myeloid leukemia and myelodysplastic syndrome: a systematic review and meta-analysis (Hematology (Amsterdam, Netherlands), 2020) Cochrane review A systematic review and network meta-analysis comparing azacitidine and decitabine for the treatment of myelodysplastic syndrome (Systematic reviews, 2018) Meta-analysis Emerging treatment approaches for VEXAS syndrome: a systematic review and meta-analysis (Annals of hematology, 2025) Meta-analysis Efficacy of epigenetic agents for older patients with acute myeloid leukemia and myelodysplastic syndrome in randomized controlled trials: a systematic review and network meta-analysis (Clinical and experimental medicine, 2023) Meta-analysis Azacitidine Monotherapy in Patients With Treatment-Naïve Higher-risk Myelodysplastic Syndrome: A Systematic Literature Review and Meta-analysis (Clinical lymphoma, myeloma & leukemia, 2023) Clinical trial publication Selinexor in combination with azacitidine or ruxolitinib in myelodysplastic/myeloproliferative neoplasm overlap syndromes: A multicenter prospective study (Cancer, 2026) Clinical trial publication Checkpoint immunotherapy is associated with preferential activation of tumor antigen-specific CD4(+) T cells in MDS (Blood neoplasia, 2025) Clinical trial publication Olutasidenib alone or combined with azacitidine in patients with mutant IDH1 myelodysplastic syndrome (Blood advances, 2025) Clinical trial publication Bexmarilimab plus azacitidine for high-risk myelodysplastic syndrome and relapsed or refractory acute myeloid leukaemia: results from the dose-escalation part of a multicentre, single-arm, phase 1/2 trial (The Lancet. Haematology, 2025) CT.gov search · Cochrane · PubMed	DGIdb	—
BEXAROTENE Repurposing via BLM	Small molecule	Approved	Strong	95	1 trials 1 Trial pubs View evidence Registry trials NCT00425477 — Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BLINATUMOMAB Repurposing via BCR	Small molecule	Approved	Strong	95	—	DGIdb	—
BORTEZOMIB Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
BOSUTINIB Repurposing via BCR	Small molecule	Approved	Strong	95	—	DGIdb	—
BOSUTINIB MONOHYDRATE Repurposing via BCR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BRENTUXIMAB VEDOTIN Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
BUSULFAN Repurposing via BCR	Small molecule	Approved	Strong	95	—	DGIdb	—
CARBACHOL Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBOPLATIN Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
CEDAZURIDINE Repurposing via CDA	Small molecule	Approved	Strong	95	8 trials 3 Trial pubs View evidence Registry trials NCT03613532 — Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN (ACTIVE_NOT_RECRUITING) PHASE1 NCT06484062 — Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes (RECRUITING) PHASE1 NCT05201066 — Roll-over Study for Patients Who Have Completed a Prior Novartis-sponsored Sabatolimab (MBG453) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Sabatolimab. (ACTIVE_NOT_RECRUITING) PHASE2 NCT04013880 — ASTX727 and FT-2102 in Treating IDH1-Mutated Recurrent/Refractory Myelodysplastic Syndrome or Acute Myeloid Leukemia (WITHDRAWN) PHASE1, PHASE2 NCT05010122 — ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome (RECRUITING) PHASE1, PHASE2 NCT04655755 — Venetoclax in Combination With ASTX727 for the Treatment of Treatment-Naive High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia (ACTIVE_NOT_RECRUITING) PHASE1, PHASE2 NCT04953897 — Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Renal Impairment (RECRUITING) PHASE1 NCT03906695 — Phase 1 Trial of ASTX727 in Subjects With Lower-risk Myelodysplastic Syndromes (ACTIVE_NOT_RECRUITING) PHASE1 Published literature Systematic review Health-related quality of life of patients with acute myeloid leukemia and myelodysplastic syndromes/neoplasms treated with decitabine: a systematic literature review (Annals of hematology, 2026) Clinical trial publication Venetoclax/FluBu2 RIC transplant followed by all-oral venetoclax/decitabine maintenance for poor-risk MDS/AML (Blood advances, 2026) Clinical trial publication Oral decitabine and cedazuridine maintenance after haematopoietic stem-cell transplantation in very high-risk acute myeloid leukaemia or myelodysplastic syndrome (GFM-DACORAL-DLI): a multicentre, single-arm, phase 2 trial (The Lancet. Haematology, 2025) Clinical trial publication Oral decitabine and cedazuridine plus venetoclax for older or unfit patients with acute myeloid leukaemia: a phase 2 study (The Lancet. Haematology, 2024) CT.gov search · Cochrane · PubMed	DGIdb	—
CIPROFLOXACIN Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOMIPRAMINE Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CYTARABINE Repurposing via CDA	Small molecule	Approved	Strong	95	8 trials 4 Meta-analysis 32 RCT View evidence Registry trials NCT00740181 — Decitabine, Cytarabine, GCSF for Refractory AML/MDS (TERMINATED) PHASE2 NCT00015951 — Bevacizumab, Cytarabine, and Mitoxantrone on Treating Patients With Hematologic Cancers (COMPLETED) PHASE2 NCT00002833 — Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia (COMPLETED) PHASE2 NCT01302106 — Study of Clofarabine in Combination With Low Dose Cytarabine to Treat Myelodysplastic Syndromes (WITHDRAWN) PHASE2 NCT01297543 — Safety Study of Human Myeloid Progenitor Cells (CLT-008) After Chemotherapy for Leukemia (COMPLETED) PHASE1, PHASE2 NCT01831232 — Idarubicin, Cytarabine, and Pravastatin Sodium in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes (COMPLETED) NA NCT00368355 — T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts (COMPLETED) PHASE2 NCT00046930 — Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia (COMPLETED) PHASE3 Published literature Meta-analysis [Efficacy and Safety of Decitabine Combined with CAG (Cytarabine, Aclarubicin, G-CSF) for Patients with Intermediate or High Risk Myelodysplastic Syndrome and Acute Myeloid Leukemia: a Meta-Analysis] (Zhongguo shi yan xue ye xue za zhi, 2019) Meta-analysis HAG (Homoharringtonine, Cytarabine, G-CSF) Regimen for the Treatment of Acute Myeloid Leukemia and Myelodysplastic Syndrome: A Meta-Analysis with 2,314 Participants (PloS one, 2016) Meta-analysis Outcomes after (90) Yttrium-ibritumomab tiuxetan-BEAM in diffuse large B-cell lymphoma: a meta-analysis (Cancer medicine, 2014) Meta-analysis A meta-analysis of CAG (cytarabine, aclarubicin, G-CSF) regimen for the treatment of 1029 patients with acute myeloid leukemia and myelodysplastic syndrome (Journal of hematology & oncology, 2011) Systematic review TKIs combined with chemotherapy followed by allo-HSCT in Philadelphia chromosome-positive myelodysplastic syndrome: A case report and literature review (Medicine, 2022) Systematic review Myeloid neoplasms with t(12;22)(p13;q12)/MN1-EVT6: a systematic review of 12 cases (Annals of hematology, 2018) Randomized controlled trial Patient-reported outcomes in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia receiving standard chemotherapy plus quizartinib or placebo (QuANTUM-First): a global, randomised, placebo-controlled, phase 3 trial (The Lancet. Haematology, 2026) Randomized controlled trial Addition of the nuclear export inhibitor selinexor to standard intensive treatment for elderly patients with acute myeloid leukemia and high risk myelodysplastic syndrome (Leukemia, 2022) Randomized controlled trial Subsequent malignant neoplasms among children with Hodgkin lymphoma: a report from the Children's Oncology Group (Blood, 2021) Randomized controlled trial [Clinical Efficacy of Low-Dose Decitabine alone for Treatment of Myelodysplastic Syndrome] (Zhongguo shi yan xue ye xue za zhi, 2019) Randomized controlled trial Glasdegib plus intensive/nonintensive chemotherapy in untreated acute myeloid leukemia: BRIGHT AML 1019 Phase III trials (Future oncology (London, England), 2019) CT.gov search · Cochrane · PubMed	DGIdb	—
DASATINIB ANHYDROUS Repurposing via BCR	Small molecule	Approved	Strong	95	—	DGIdb	—
DAUNORUBICIN HYDROCHLORIDE Repurposing via BLM	Small molecule	Approved	Strong	95	8 trials View evidence Registry trials NCT00002833 — Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia (COMPLETED) PHASE2 NCT01831232 — Idarubicin, Cytarabine, and Pravastatin Sodium in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes (COMPLETED) NA NCT00046930 — Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia (COMPLETED) PHASE3 NCT02521493 — Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome (ACTIVE_NOT_RECRUITING) PHASE3 NCT01342692 — Best Promising Drug Association With Azacitidine in Higher Risk Myelodysplastic Syndromes (UNKNOWN) PHASE2 NCT00002517 — Combination Chemotherapy in Treating Children With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome (COMPLETED) PHASE3 NCT06050941 — Reduced Intensive Idarubicin and Cytarabine Plus Venetoclax as First-line Treatment for Adults AML and MDS (NOT_YET_RECRUITING) PHASE2 NCT07046078 — Combination Chemotherapy (FLAG-Ida) Followed Immediately by Reduced-Intensity Total Body Radiation Therapy and Donor Hematopoietic Cell Transplant for the Treatment of Adults Age 60 and Older With Newly Diagnosed Adverse-Risk Acute Myeloid Leukemia or Other High-Grade Myeloid Cancer (RECRUITING) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
DAUNORUBICIN LIPOSOMAL Repurposing via BLM	Small molecule	Approved	Strong	95	8 trials View evidence Registry trials NCT00002833 — Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia (COMPLETED) PHASE2 NCT01831232 — Idarubicin, Cytarabine, and Pravastatin Sodium in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes (COMPLETED) NA NCT00046930 — Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia (COMPLETED) PHASE3 NCT02521493 — Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome (ACTIVE_NOT_RECRUITING) PHASE3 NCT01342692 — Best Promising Drug Association With Azacitidine in Higher Risk Myelodysplastic Syndromes (UNKNOWN) PHASE2 NCT00002517 — Combination Chemotherapy in Treating Children With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome (COMPLETED) PHASE3 NCT06050941 — Reduced Intensive Idarubicin and Cytarabine Plus Venetoclax as First-line Treatment for Adults AML and MDS (NOT_YET_RECRUITING) PHASE2 NCT07046078 — Combination Chemotherapy (FLAG-Ida) Followed Immediately by Reduced-Intensity Total Body Radiation Therapy and Donor Hematopoietic Cell Transplant for the Treatment of Adults Age 60 and Older With Newly Diagnosed Adverse-Risk Acute Myeloid Leukemia or Other High-Grade Myeloid Cancer (RECRUITING) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
DIETHYLSTILBESTROL Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
DOXORUBICIN HYDROCHLORIDE Repurposing via BLM	Small molecule	Approved	Strong	95	8 trials View evidence Registry trials NCT00237627 — Doxorubicin Hydrochloride Liposome and Bortezomib in Treating Patients With Refractory Hematologic Cancer or Malignant Solid Tumor or Metastatic Breast Cancer (COMPLETED) PHASE1, PHASE2 NCT00036790 — Motexafin Gadolinium and Doxorubicin in Treating Patients With Advanced Cancer (COMPLETED) PHASE1 NCT02356159 — Study of Palifermin (Kepivance) in Persons Undergoing Unrelated Donor Allogeneic Hematopoietic Cell Transplantation (COMPLETED) PHASE1, PHASE2 NCT03059615 — A Phase 2a, Open-Label, Two Stage Study of Nerofe or Nerofe With Doxorubicin in Subjects With AML or MDS (WITHDRAWN) PHASE2 NCT03878524 — Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial (TERMINATED) PHASE1 NCT00003243 — Combination Chemotherapy Plus Infusion of White Blood Cells in Treating Patients With Hematologic Cancer (COMPLETED) PHASE1 NCT00520130 — Chemotherapy and Unrelated Donor Stem Cell Transplantation for Patients With Cancers of the Blood and Immune System (COMPLETED) PHASE1, PHASE2 NCT00074490 — Donor Stem Cell Transplant With No or Low-Intensity Chemotherapy Using Sirolimus and Treated Immune Cells to Treat Blood and Lymph Cancers (TERMINATED) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
EDARAVONE Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
EFBEMALENOGRASTIM ALFA Repurposing via CSF3R	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
EFLAPEGRASTIM Repurposing via CSF3R	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
EPIRUBICIN Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
EPIRUBICIN HYDROCHLORIDE Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
ERYTHROMYCIN Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
ESTRAMUSTINE Repurposing via BLM	Small molecule	Approved	Strong	95	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ETHOXZOLAMIDE Repurposing via BCL2	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ETOPOSIDE Repurposing via BCL2	Small molecule	Approved	Strong	95	—	DGIdb	—
FENOLDOPAM MESYLATE Repurposing via BLM	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets

Natural Products

21 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked). Only agents with direct clinical evidence are shown by default.

Reference lookups: Examine.com

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Links
Vitamin K Natural product Meta-analysis The Efficacy of Vitamin K, A Member Of Naphthoquinones in the Treatment… Human Study Vitamin K2 appears effective in the treatment of myelodysplastic syndro… Human Study Vitamin K2 appears effective in the treatment of myelodysplastic syndro…	Nutraceutical	Preclinical	Emerging	10	1 Meta-analysis View evidence Published literature Meta-analysis The Efficacy of Vitamin K, A Member Of Naphthoquinones in the Treatment of Cancer: A Systematic Review and Meta-Analysis (Current cancer drug targets, 2021) CT.gov search · Cochrane · PubMed	PubChem
Vitamin D3 Natural product Clinical trial publication Survival improvement of poor-prognosis AML/MDS patients by maintenance… Clinical trial publication Long-term follow-up of myelodysplastic syndrome patients with moderate/… Human Study Vitamin D may be therapeutic in the treatment of myelodysplastic syndro…	Nutraceutical	Approved	Emerging	10	8 trials 2 Trial pubs View evidence Registry trials NCT06508099 — Vitamin A and D Supplementation in Allogeneic HCT (RECRUITING) PHASE2 NCT00068276 — Cholecalciferol in Treating Patients With Myelodysplastic Syndrome (COMPLETED) PHASE2 NCT00104806 — Arsenic Trioxide and Cholecalciferol (Vitamin D) in Treating Patients With Myelodysplastic Syndromes (TERMINATED) PHASE2 NCT03176849 — A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT (COMPLETED) PHASE4 NCT00804050 — Multicentric Study Comparison Between Erythropoietin and Erythropoietin Associated to Differentiating Therapy With Acid 13-cis-retinoic and Dihydroxyvitamin D3 in Myelodysplastic Syndromes Without Excess of Blasts (TERMINATED) PHASE3 NCT00057031 — Study of High-Dose Pulse Administration DN-101 (Calcitriol) in Patients With Myelodysplastic Syndrome (MDS) (UNKNOWN) PHASE2 NCT00321932 — Zoledronic Acid in Preventing Osteoporosis in Patients Undergoing Donor Stem Cell Transplant (COMPLETED) PHASE2 NCT00030069 — Calcitriol and Dexamethasone in Patients With Myelodysplastic Syndromes (COMPLETED) PHASE2 Published literature Clinical trial publication Survival improvement of poor-prognosis AML/MDS patients by maintenance treatment with low-dose chemotherapy and differentiating agents (Annals of hematology, 2014) Clinical trial publication Long-term follow-up of myelodysplastic syndrome patients with moderate/severe anaemia receiving human recombinant erythropoietin + 13-cis-retinoic acid and dihydroxylated vitamin D3: independent positive impact of erythroid response on survival (British journal of haematology, 2012) CT.gov search · Cochrane · PubMed	PubChem
Ginger Natural product Human Study 6-shogaol caused a decrease in serum ferritin levels in 3 of 6 patients…	Botanical	Preclinical	Emerging	10	2 pubs View evidence CT.gov search · Cochrane · PubMed	PubChem
Vitamin C Natural product Human Study l-Ascorbic acid may be of clinical benefit in Acute Myeloid Leukemia an… Human Study Vitamin D may be therapeutic in the treatment of myelodysplastic syndro… Human Study Vitamin E and a synthetic analogue of vitamin A improve the treatment o…	Nutraceutical	Phase II	Emerging	10	8 trials View evidence Registry trials NCT03397173 — TET2 Mutations in Myelodysplastic Syndromes and Acute Myeloid Leukemia With Azacitidine + Ascorbic Acid (COMPLETED) PHASE2 NCT03433781 — A Phase Ib Study Evaluating the Safety and Tolerability of Vitamin C in Patients With Intermediate or High Risk Myelodysplastic Syndrome With TET2 Mutations (COMPLETED) PHASE1, PHASE2 NCT00274820 — Arsenic Trioxide, Ascorbic Acid, Dexamethasone, and Thalidomide in Myelofibrosis/Myeloproliferative Disorder (COMPLETED) PHASE2 NCT02809222 — Plasmatic L-AScorbic Acid in MYelodyplastic Syndroms and Controls (COMPLETED) NA NCT03999723 — Combining Active and Passive DNA Hypomethylation (RECRUITING) PHASE2 NCT00671697 — Decitabine, Arsenic Trioxide and Ascorbic Acid for Myelodysplastic Syndromes and Acute Myeloid Leukemia (COMPLETED) PHASE1 NCT03613727 — Therapeutic Use of Intravenous Vitamin C in Allogeneic Stem Cell Transplant Recipients (COMPLETED) PHASE2 NCT03526666 — Ascorbic Acid Levels in MDS, AML, and CMML Patients (COMPLETED) CT.gov search · Cochrane · PubMed	PubChem
Curcumin Natural product Animal Study, In Vitro Study Anti-cancer effects of curcumin on myelodysplastic syndrome through the… Animal Study Curcumin as an epigenetic therapeutic agent in myelodysplastic syndrome… Animal Study Curcumin may represent a new horizon in managing iron overload-induced…	Food compound	Phase II	Emerging	7	2 trials View evidence Registry trials NCT00247026 — The Efficacy of Coenzyme Q10 And Curcumin in Patients With Myelodysplastic Syndromes (WITHDRAWN) PHASE1, PHASE2 NCT06063486 — Curcumin to Improve Inflammation and Symptoms in Patients With Clonal Cytopenia of Undetermined Significance, Low Risk Myelodysplastic Syndrome, and Myeloproliferative Neoplasms (RECRUITING) PHASE2 CT.gov search · Cochrane · PubMed	PubChem
Coenzyme Q10 Natural product Human Study Coenzyme Q10 may be of clinical benefit in a subset of myelodysplastic…	Nutraceutical	Phase III	Emerging	7	2 trials View evidence Registry trials NCT00247026 — The Efficacy of Coenzyme Q10 And Curcumin in Patients With Myelodysplastic Syndromes (WITHDRAWN) PHASE1, PHASE2 NCT02042482 — The Effect of Combination Ultra Q10 and L-carnitine on the Course of Myelodysplastic Syndrome (UNKNOWN) PHASE2, PHASE3 CT.gov search · Cochrane · PubMed	PubChem
Astragalus Natural product In Vitro Study Astragalus stimulates hematopoiesis and may attenuate the myelosuppress… Human Study The Chinese Herbal Formula Qinghuang is therapeutic in the treatment of…	TCM herb	Preclinical	Emerging	7	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
6-Shogaol Natural product Human Study 6-shogaol caused a decrease in serum ferritin levels in 3 of 6 patients…	Nutraceutical	Preclinical	Emerging	0	2 pubs View evidence CT.gov search · Cochrane · PubMed	—
Maitake Mushroom Natural product Human Study A maitake mushroom extract has beneficial immunomodulatory potential in…	Nutraceutical	Phase II	Emerging	0	1 trials View evidence Registry trials NCT01099917 — Does Maitake Mushroom Extract Enhance Hematopoiesis in Myelodysplastic Patients? (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	—
Chinese Herbal Formula: Qinghuang Natural product Human Study The Chinese Herbal Formula Qinghuang is therapeutic in the treatment of… Human Study The Chinese Herbal formula Yisuikang is effective in treating some form…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Chinense Herbal Formula: Yisuikang Natural product Human Study The Chinese Herbal Formula Qinghuang is therapeutic in the treatment of… Human Study The Chinese Herbal formula Yisuikang is effective in treating some form…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Tea Natural product Systematic review Medical Conditions and Modifiable Risk Factors for Myelodysplastic Synd… Human Study The study suggests that regular tea consumption may reduce the risk of…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence Published literature Systematic review Medical Conditions and Modifiable Risk Factors for Myelodysplastic Syndrome: A Systematic Review (Cancer epidemiology, biomarkers & prevention : a publication of the American Association for Cancer Research, cosponsored by the American Society of Preventive Oncology, 2019) CT.gov search · Cochrane · PubMed	—
Vitamin a Natural product Human Study Vitamin E and a synthetic analogue of vitamin A improve the treatment o… Human Study l-Ascorbic acid may be of clinical benefit in Acute Myeloid Leukemia an… Human Study Vitamin D may be therapeutic in the treatment of myelodysplastic syndro…	Food compound	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	PubChem
Vitamin E: Alpha Tocopherol Natural product Human Study Vitamin E and a synthetic analogue of vitamin A improve the treatment o… Human Study l-Ascorbic acid may be of clinical benefit in Acute Myeloid Leukemia an… Human Study Vitamin D may be therapeutic in the treatment of myelodysplastic syndro…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Cat's Claw Natural product Animal Study Cat's claw (Uncaria tomentosa) increases the number of myeloid pro…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Euphorbia Peplus Natural product Animal Study Euphorbia tirucalli has a myelopoietic effect which may contribute to i…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Woodfordia Fruticosa Natural product Animal Study Woodfordia fructicosa has an immunostimulatory effect and may protect t…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Stilbenes Natural product In Vitro Study Polydatin exerts therapeutic effects on myelodysplastic syndrome.	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Tripterygium Wilfordii Natural product In Vitro Study Triptolide inhibits transcription factor NF-kappa B, which translates i…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Triptolide Natural product In Vitro Study Triptolide inhibits transcription factor NF-kappa B, which translates i…	Nutraceutical	Preclinical	Emerging	0	4 pubs View evidence CT.gov search · Cochrane · PubMed	—
Wogonin Natural product In Vitro Study Wogonin inhibits the proliferation of myelodysplastic syndrome cells.	Nutraceutical	Preclinical	Emerging	0	—	—

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.