Myelodysplastic syndromes: research and therapeutic landscape

12 genes recurrently implicated in myelodysplastic syndromes, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.

Of 12 recurrently implicated genes, 5 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Myelodysplastic syndromes does have labelled therapy — 9 drugs, listed in the next section.

Dataset evidence counts studies in the 237-study ranked set whose title or abstract names the gene; the bar is scaled to SF3B1. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-30. Clinical stage is the highest reached for any indication — see the per-drug indications noted in each row. Target-prioritisation reference only; not clinical or prescribing guidance.

9 drugs carry an FDA label naming myelodysplastic syndromes: Azacitidine, Azacitidine For, Cedazuridine And Decitabine, Decitabine, Imatinib, Imetelstat, Ivosidenib, Lenalidomide, Luspatercept. Separately, 5 of the drugs returned for the genes in the table above are approved only for other diseases and reach myelodysplastic syndromes through trials, not through their labels.

Every label that names myelodysplastic syndromes

99 labels carry a positive indication naming the disease under indications_and_usage:"myelodysplastic syndromes" OR indications_and_usage:"myelodysplastic syndrome" OR indications_and_usage:"myelodysplastic"; they collapse to 10 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-18. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.

Labels that name myelodysplastic syndromes without indicating for it

A label can name a disease under Limitations of Use, in a contraindication or as a risk. These were read and are not counted above.

hypomethylating is the busiest cell-therapy target among those this briefing searched in myelodysplastic syndromes: 3 registered trials naming it, 0 still active, 1 withdrawn before enrolling anyone.

3 of 3 shown, most recently active first. Source: ClinicalTrials.gov API v2, retrieved 2026-09-18. A trial counts here when its registered conditions name myelodysplastic syndromes or a tumour-agnostic basket, its title names an administered cell product, and the title names this target: a HER2 CAR-T trial that adds a PD-1 antibody is a HER2 trial. A trial stating only a product code is missed.

Every term below is a share of the myelodysplastic syndromes literature, not a count, because the field itself grew: 2015–2018 (n=1,961) against 2021–2025 (n=2,376). A topic whose papers doubled while the field doubled has not risen.

Rising, with a real baseline

Cooling

Biggest topics now

Publication mix

Query: Myelodysplastic Syndromes[MeSH Major Topic] NOT ("Anemia, Aplastic"[MeSH] OR "Leukemia, Myeloid, Acute"[MeSH] OR "Primary Myelofibrosis"[MeSH] OR "Polycythemia Vera"[MeSH] OR "Thrombocythemia, Essential"[MeSH]). Terms need at least 12 papers in the recent window and, in the rising table, at least 5 in the earlier one — a term going from 1 paper to 12 is a large fold change and no evidence of anything. Ubiquitous descriptors (Humans, Animals, age bands) and the disease’s own term are dropped. Source: PubMed MeSH, retrieved 2026-09-12.

What the public sources count, and how closely each category matches this disease. The same figures for every disease on the site are on one table.

Years of life lost: not computed. Years of life lost needs survival, age at diagnosis and a case count. five_year_relative_survival, median_age_at_diagnosis is not recorded for this disease.

NIH obligations naming myelodysplastic syndromes, after removing the 1,926 records across all years that matched the search but are about something else. That filter is not cosmetic: without it this section reports another field’s funding as this disease’s.

NIH obligations by fiscal year

Where FY2025 money went

Text search myelodysplastic syndromes over project title, terms and abstract, per fiscal year. Awards are individual funding actions; distinct projects collapse them by core project number, so a multi-year grant counts once. The most recent year may be incomplete. Source: NIH RePORTER, retrieved 2026-09-19.

Who funds it, FY2025

The total says how much. These say who from, through what kind of award, and to whom — which a dollar figure cannot.

597 human GEO series match myelodysplastic syndromes. Keyword relevance cannot tell a 320-sample patient cohort from a six-well cell-line experiment, so this ranking scores four things GEO does not expose: how often the accession is named in full-text papers, field-normalised citation impact, clinical annotation, and cohort type.

Composition of the 237-study ranked set

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-19. SubSeries are collapsed to one row per study by linked PMID. Of 597 series retrieved, 107 were dropped by the profile’s exclusion rules and 183 named the disease only in passing. The ranked set is a relevance-ranked sample, not a census, so the composition figures describe the sample only.

Each card below is a disagreement between two of the tables on this page — a drug that exists but was never tried here, a target that looks tested and was not, a gene with no drug whose product has a busy clinical programme. They are computed from the same facts as the tables, so they cannot contradict them.

This page is written to be quoted correctly by a model as much as read by a person. The same facts are available as JSON at the sibling URL below, which is what to cite. The cross-disease layer — burden, genes, drugs and antigens across every briefing — is described at /disease/api.json.

{
  "disease": "Myelodysplastic syndromes",
  "mesh": "Myelodysplastic Syndromes",
  "facts": "https://usebiotransfer.org/disease/myelodysplastic-syndromes.json",
  "methods": "https://usebiotransfer.org/methods/",
  "all_diseases": "https://usebiotransfer.org/disease/api.json",
  "note": "Every figure on the page is in that JSON with its source and retrieval date. Quote from it rather than from the HTML."
}