Biomarker & Intervention Discovery
Myelodysplastic Syndromes (MDS)
Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.
Spontaneous remissionEssentially zero; progressive cytopaenia and transformation to AML in 30-40% of higher-risk MDS over 2-5 years; median…
Best-intervention remissionAllogeneic HSCT: only curative option; 5-year OS 35-50% in fit patients; hypomethylating agents (azacitidine…
Gap sizeLarge — MDS has limited treatment options; curative HSCT used in <15% of patients (age, comorbidity, donor…
Primary barrierbiological + access
Full remission profile →
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
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
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
Biomarker Targets & Therapeutic Agents
Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC.
Agents ranked: ● Clinical >
● Mechanistic >
● Correlative.
Pipeline results not yet available. Run: python -m biomarker_pipeline.run_for_diseases --skip-llm
Clinical Trials
Recruiting and recently completed trials from ClinicalTrials.gov.
Data retrieved 2026-07-06.
No recent clinical trials found on ClinicalTrials.gov for this condition.
Agents Found By Disease-Level Search
Searched directly from “Myelodysplastic Syndromes (MDS)” via ClinicalTrials.gov interventions and Open Targets'
disease→drug data — independent of the 0-gene target panel above.
This surfaces agents whose mechanism doesn't route through a curated gene (combination
therapies, standard-of-care drugs, targets outside the panel).
Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."