Target landscape
Open Targets · retrieved 2026-09-17 · weekly12 genes recurrently implicated in myeloproliferative neoplasms, 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: Myeloproliferative neoplasms does have labelled therapy — 7 drugs, listed in the next section.
Two different questions
The table below asks what can be aimed at 12 recurrently implicated genes. That is narrower than what is approved for the disease, and narrower again than what myeloproliferative neoplasms is actually treated with, which includes agents approved under broader indications. All three are on this page and they are kept apart deliberately.
| Gene | Drugs | Approval, and whether it reached this disease | Open Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it works | Cell therapy here |
|---|---|---|---|---|
| JAK2 across cancers → | 26 | 13 approvedBaricitinib, Delgocitinib, Deuruxolitinib, Fedratinib, Filgotinib, Lestaurtinib, Momelotinib, Momelotinib Dihydrochloride, Pacritinib, Ruxolitinib, Tofacitinib, Upadacitinib, Upadacitinib HemihydrateApproved in myeloproliferative neoplasms: Fedratinib, Momelotinib, Pacritinib, Ruxolitinib.90 active of 229 myeloproliferative neoplasms trials | antibody, protein degrader, small molecule | — |
| CALR | 0 | No drug— | antibody, protein degrader, small molecule | — |
| MPL | 8 | 6 approvedAvatrombopag, Eltrombopag, Eltrombopag Choline, Eltrombopag Olamine, Lusutrombopag, RomiplostimApproved in hemorrhage, Thrombocytopenia, liver disorder and 3 other indications. No myeloproliferative neoplasms indication appears on these drugs’ labels.2 active of 15 myeloproliferative neoplasms trials | antibody, other clinical modality, protein degrader, small molecule | — |
| TET2 across cancers → | 0 | No drug— | protein degrader, small molecule | — |
| ASXL1 across cancers → | 0 | No drug— | protein degrader | — |
| EZH2 across cancers → | 3 | 2 approvedTazemetostat, Tazemetostat HydrobromideApproved in follicular lymphoma, sarcoma. No myeloproliferative neoplasms indication appears on these drugs’ labels.No myeloproliferative neoplasms trial of any of these drugs | protein degrader, small molecule | — |
| SRSF2 across cancers → | 0 | No drug— | protein degrader | — |
| U2AF1 across cancers → | 0 | No drug— | protein degrader | — |
| IDH2 across cancers → | 2 | 2 approvedEnasidenib, VorasidenibApproved in acute myeloid leukemia by FAB classification, astrocytoma (excluding glioblastoma), oligodendroglioma. No myeloproliferative neoplasms indication appears on these drugs’ labels.5 active of 10 myeloproliferative neoplasms trials | protein degrader, small molecule | — |
| TP53 across cancers → | 8 | Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, Teprasiran2 active of 13 myeloproliferative neoplasms trials | other clinical modality, protein degrader, small molecule | — |
| SH2B3 | 0 | No drug— | antibody, protein degrader | — |
| NFE2 | 0 | No drug— | protein degrader | — |
Dataset evidence counts studies in the 109-study ranked set whose title or abstract names the gene; the bar is scaled to JAK2. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-17. 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.
What is actually approved here
openFDA drug labels · weekly7 drugs carry an FDA label naming myeloproliferative neoplasms: Fedratinib, Luspatercept, Momelotinib, Pacritinib, Ropeginterferon Alfa-2B, Rusfertide, Ruxolitinib. Separately, 19 of the drugs returned for the genes in the table above are approved only for other diseases and reach myeloproliferative neoplasms through trials, not through their labels.
Every label that names myeloproliferative neoplasms
| Drug | Role | What the label says |
|---|---|---|
| FedratinibInrebic | Labelled here | INREBIC is a kinase inhibitor indicated for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF) ( 1 ). |
| LuspaterceptReblozyl | Labelled here | Myelodysplastic Syndromes with Ring Sideroblasts or Myelodysplastic/ Myeloproliferative Neoplasm with Ring Sideroblasts and Thrombocytosis Associated Anemia REBLOZYL is indicated for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with rin... |
| MomelotinibOjjaara | Labelled here | OJJAARA is a kinase inhibitor indicated for the treatment of intermediate or high‑risk myelofibrosis (MF), including primary MF or secondary MF [post‑polycythemia vera (PV) and post‑essential thrombocythemia (ET)], in adults with anemia. |
| PacritinibVonjo | Labelled here | VONJO is a kinase inhibitor indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 10 9 /L ( 1 ). |
| Ropeginterferon Alfa-2BBESREMi, BESREMi Pen | Labelled here | Polycythemia Vera BESREMi is indicated for the treatment of adults with polycythemia vera. |
| RusfertideMIMRYLO | Labelled here | MIMRYLO is a hepcidin mimetic indicated for the treatment of erythrocytosis in adults with polycythemia vera (PV). |
| RuxolitinibJAKAFI, JAKAFI XR | Labelled here | polycythemia vera in adults who have had an inadequate response to or are intolerant of hydroxyurea. |
1 labels match indications_and_usage:"polycythemia vera" OR indications_and_usage:"myelofibrosis" OR indications_and_usage:"essential thrombocythemia" OR indications_and_usage:"myeloproliferative neoplasm"; they collapse to 7 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-17. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.
Cell therapy against hydroxyurea
ClinicalTrials.gov · retrieved 2026-09-17 · weeklyhydroxyurea is the busiest cell-therapy antigen in myeloproliferative neoplasms: 6 registered trials, 2 still active.
| Trial | Phase | Status | Title | Last update |
|---|---|---|---|---|
| NCT03810196 | no phase | Recruiting | CD45RA Depleted Peripheral Stem Cell Addback for Viral or Fungal Infections Post TCRαβ/CD19 Depleted HSCT | 2026-07-08 |
| NCT02727803 | 2 | Recruiting | Personalized NK Cell Therapy in CBT | 2026-07-30 |
| NCT00806767 | 2 | Completed | Fludarabine, Busulfan, and Antilymphocyte Globulin Followed by Donor Stem Cell Transplant in Treating Older Patients With Hematological Cancer | 2011-05-17 |
| NCT00005641 | 2 | Terminated | Removal of T Cells to Prevent Graft-Versus-Host Disease in Patients Undergoing Bone Marrow Transplantation | 2012-12-11 |
| NCT00402558 | 1 | Completed | Alloreactive NK Cells for Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) | 2015-05-08 |
| NCT03615105 | 2 | Terminated | Donor Stem Cell Transplantation Using α/β+ T-lymphocyte Depleted Grafts From HLA Mismatched Donors | 2025-04-06 |
6 of 6 shown, most recently active first. Source: ClinicalTrials.gov API v2, retrieved 2026-09-17. Trials are matched on the antigen named in the title or intervention, so a trial stating only a product code is missed.
Research momentum
PubMed MeSH · monthlyEvery term below is a share of the myeloproliferative neoplasms literature, not a count, because the field itself grew: 2015–2018 (n=1,306) against 2021–2025 (n=1,631). A topic whose papers doubled while the field doubled has not risen.
Rising, with a real baseline
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Janus Kinase Inhibitors | 1.61% | 7.73% | 4.8× | 126 papers |
| Neoplasms | 0.69% | 3.13% | 4.54× | 51 papers |
| Venous Thrombosis | 0.54% | 1.84% | 3.43× | 30 papers |
| Neutrophils | 0.54% | 1.78% | 3.32× | 29 papers |
| Polycythemia | 1.0% | 3.19% | 3.2× | 52 papers |
| Pyrrolidines | 0.69% | 2.08% | 3.02× | 34 papers |
| Sulfonamides | 0.69% | 1.9% | 2.76× | 31 papers |
| Interferon alpha-2 | 1.0% | 2.39% | 2.4× | 39 papers |
| Benzenesulfonamides | 0.61% | 1.47% | 2.4× | 24 papers |
| Graft vs Host Disease | 1.53% | 3.56% | 2.32× | 58 papers |
Cooling
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Biopsy | 3.6% | 0.74% | 0.2× | 12 papers |
| Mutation, Missense | 3.52% | 0.74% | 0.21× | 12 papers |
| Diagnosis, Differential | 4.21% | 0.92% | 0.22× | 15 papers |
| Quinazolines | 2.83% | 0.74% | 0.26× | 12 papers |
| Disease-Free Survival | 2.91% | 0.86% | 0.3× | 14 papers |
| Genetic Predisposition to Disease | 2.76% | 0.86% | 0.31× | 14 papers |
Biggest topics now
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Primary Myelofibrosis | 59.42% | 55.86% | 0.94× | 911 papers |
| Polycythemia Vera | 31.93% | 36.97% | 1.16× | 603 papers |
| Janus Kinase 2 | 33.08% | 29.68% | 0.9× | 484 papers |
| Thrombocythemia, Essential | 33.46% | 28.57% | 0.85× | 466 papers |
| Mutation | 26.72% | 22.62% | 0.85× | 369 papers |
| Pyrimidines | 17.61% | 17.84% | 1.01× | 291 papers |
| Nitriles | 16.0% | 16.49% | 1.03× | 269 papers |
| Pyrazoles | 16.77% | 15.63% | 0.93× | 255 papers |
| Treatment Outcome | 17.53% | 11.59% | 0.66× | 189 papers |
| Thrombosis | 7.96% | 11.28% | 1.42× | 184 papers |
Publication mix
| Type | 2015–18 | 2021–25 |
|---|---|---|
| Clinical Trial | 0.0% | 0.1% |
| Randomized Controlled Trial | 2.6% | 1.8% |
| Review | 14.9% | 14.7% |
| Meta-Analysis | 0.9% | 0.8% |
| Case Reports | 0.0% | 3.2% |
Query: ("Polycythemia Vera"[Majr] OR "Thrombocythemia, Essential"[Majr] OR "Primary Myelofibrosis"[Majr]) NOT ("Leukemia, Myelogenous, Chronic, BCR-ABL Positive"[MeSH] OR "Myelodysplastic Syndromes"[MeSH] OR "Mastocytosis"[MeSH] OR "Hypereosinophilic Syndrome"[MeSH] OR "Leukemia, Myelomonocytic, Chronic"[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 2026-09-17.
Disease burden
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.
| Measure | Value | Match | What it counts |
|---|---|---|---|
| New cases each year | — | not published | No registry publishes a count: SEER has no Stat Facts page for polycythaemia vera, essential thrombocythaemia or myelofibrosis, and the American Cancer Society has no page for the group. The cited SEER-based review gives incidence as ranges, and a count from a range would be a derivation the source does not make. |
| Incidence rate | — | not published | Published as ranges, not a figure: "the incidences of CML, PV, and ET are relatively similar at 1.0-2.0 per 100,000 person-years in the United States, while PMF is rarer with an incidence of 0.3 per 100,000 person-years" (SEER data through 2016). |
| Deaths each year | — | not published | Not published for the group; deaths are recorded as AML when the disease transforms and as thrombosis when it does not. |
| Five-year relative survival | — | not published | Not published as one figure; the three diseases differ by an order of magnitude in median survival (polycythaemia vera and essential thrombocythaemia near-normal, primary myelofibrosis about six years). |
| Median age at diagnosis | — | not published | Not published for the group as a median; the review gives older age as the main risk factor. |
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, incident cases is not recorded for this disease.
Funding
NIH RePORTER · quarterlyNIH obligations naming myeloproliferative neoplasms, after removing the 9 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
| Year | Obligations | Awards | Distinct projects | Dropped as off-topic |
|---|---|---|---|---|
| FY2013 | $7.0M | 9 | 7 | 1 |
| FY2014 | $5.5M | 9 | 7 | 1 |
| FY2015 | $10.1M | 11 | 7 | 1 |
| FY2016 | $2.4M | 9 | 8 | 0 |
| FY2017 | $1.9M | 8 | 7 | 0 |
| FY2018 | $6.3M | 8 | 7 | 0 |
| FY2019 | $4.8M | 6 | 5 | 1 |
| FY2020 | $6.7M | 11 | 10 | 1 |
| FY2021 | $6.3M | 9 | 8 | 0 |
| FY2022 | $6.9M | 11 | 10 | 1 |
| FY2023 | $7.3M | 10 | 9 | 1 |
| FY2024 | $7.1M | 9 | 8 | 1 |
| FY2025 | $6.7M | 6 | 5 | 1 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| Icahn School Of Medicine At Mount Sinai | $4.6M | 3 |
| Aplexis, Inc. | $1.1M | 1 |
| University Of Virginia | $0.6M | 1 |
| Utah State Higher Education System--University Of Utah | $0.5M | 1 |
Text search myeloproliferative neoplasm OR polycythemia vera OR essential thrombocythemia OR myelofibrosis 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 2026-09-17.
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.
NIH institutes
Projects by administering institute. The rows above are the top 2 and account for 6 of 6.
Award mechanisms
R01 is an investigator-initiated grant; ZIA is NIH intramural, meaning the work happens inside NIH rather than being funded outside it. The rows above are the top 4 and account for 6 of 6.
Where it lands
Share of $6.7M in FY2025. The top three hold 93%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly758 human GEO series match myeloproliferative neoplasms. Keyword relevance cannot tell a 2,798-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 109-study ranked set
Established
high reuse, older| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE41812 | Gene expression profile of mutated and wild-type PMF CD34+ cells2013 · array | 20 | 8 | 17.1 | patient cohortAPT 0.95molecularASXL1EZH2IDH2627 cites |
| GSE122198 | 3'-TARGET-seq: a novel method for high-sensitivity single-cell mutational analysis and parallel high throughput RNA-sequencing [Figures 5 and 6, Figure S7]2019 · single-cell | 2,798 | 4 | 6.6 | patient cohortAPT 0.75molecularJAK2240 cites |
| GSE53482 | Integrative Analysis of Gene and miRNA expression profiles in Primary Myelofibrosis CD34+ cells2014 · array | 146 | 31 | 2.8 | patient cohortAPT 0.75survivalmolecular105 cites |
| GSE54646 | Integrated genomic analysis illustrates the central role of JAK-STAT pathway activation in myeloproliferative neoplasm pathogenesis2014 · array | 116 | 13 | 9.7 | APT 0.95molecularJAK356 cites |
| GSE55976 | Gene expression profile in myeloproliferative neoplasms2015 · array | 45 | 7 | 2.3 | patient cohortAPT 0.5survivalmolecularJAKJAK275 cites |
| GSE47018 | Gene Expression Profiling in Polycythemia Vera (PV)2014 · array | 27 | 10 | 1.5 | patient cohortAPT 0.75survivalmolecularJAK254 cites |
| GSE26049 | Expression data from patients with Essentiel Thrombocythemia (ET), Polycythemia Vera (PV), Primary Myelofibrosis (PMF) and control subjects2012 · array | 181 | 25 | 1.4 | patient cohortAPT 0.556 cites |
| GSE19647 | SNP data of MPD/MPN samples2010 · array | 159 | 1 | 2.3 | patient cohortAPT 0.75survivalstagemolecularTP53109 cites |
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE277354 | Genetic charcterization of myeloproliferative neoplasms (MPN)2024 · sequencing | 402 | 2 | 3.0 | patient cohortAPT 0.75molecular17 cites |
| GSE190517 | DUSP6 mediates resistance to JAK2 inhibition and drives leukemic progression (RNA-Seq)2022 · sequencing | 20 | 1 | 4.3 | patient cohortAPT 0.75survivalmolecularJAKJAK2MPL51 cites |
| GSE214914 | DUSP6 mediates resistance to JAK2 inhibition and drives leukemic progression (single-cell RNA-seq)2022 · single-cell | 4 | 1 | 4.0 | patient cohortAPT 0.5survivalmolecularJAKJAK2MPL48 cites |
| GSE277035 | Pre-clinical efficacy of CDK7 inhibitor-based combinations against myeloproliferative neoplasms (MPN) transformed to AML2025 · sequencing | 10 | 1 | 2.6 | mixedAPT 0.25survivalmolecularJAKPELABRESIBRUXOLITINIB10 cites |
| GSE206768 | Inflammatory gene expression associated with advanced myelofibrosis2022 · array | 29 | 1 | 5.3 | patient cohortAPT 0.5molecular73 cites |
| GSE183374 | HMGA1 Chromatin Regulators Drive MPN Progression [RNA-Seq]2023 · sequencing | 12 | 2 | 2.2 | cell lineAPT 0.5survivalmolecularJAK2RUXOLITINIB36 cites |
Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-17. SubSeries are collapsed to one row per study by linked PMID. Of 758 series retrieved, 107 were dropped by the profile’s exclusion rules and 506 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.
Where the gaps are
Derived from the sections above · recomputed on every refreshEach 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.
CALR
No drug in any database targets CALR, while 10 trials target CALR. A gene-centric search finds nothing here and concludes wrongly.
TET2
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
ASXL1
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
EZH2
2 approved drugs (Tazemetostat, Tazemetostat Hydrobromide) and no registered trial in myeloproliferative neoplasms. The molecules exist; nobody has tested them here.
SRSF2
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
U2AF1
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
SH2B3
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
NFE2
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
How a machine reads this page
StaticThis 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.
Fully server-rendered
Every figure is in the HTML at first byte. No JavaScript runs, no figure is fetched after load, and nothing on this page requires a renderer to see.
Every number carries a source
Each section names the API it came from and the date it was retrieved: Open Targets (2026-09-17), ClinicalTrials.gov (2026-09-17), openFDA (2026-09-17), NCBI GEO (2026-09-17), PubMed (2026-09-17), NIH RePORTER (2026-09-17).
Negatives stated explicitly
Where nothing exists for myeloproliferative neoplasms — no drug, no trial, no approval — the page says so in those words rather than omitting the row. An absent row is unquotable; a stated negative is the finding.
Denominators, not just percentages
Every share is printed with the count and the total it came from, so a figure can be checked rather than taken.
The filters are published
6 exclusion patterns are applied to free text before anything is ranked, because HEL and Ba/F3 (general-purpose lines) is used as a model system in HEL: erythroid biology and much else; Ba/F3: the IL-3-dependent line every kinase inhibitor is tested in. What was removed and why is stated in each section rather than silently applied.
{
"disease": "Myeloproliferative neoplasms",
"mesh": "Myeloproliferative Disorders",
"facts": "https://usebiotransfer.org/disease/myeloproliferative-neoplasms.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."
}