Disease Briefing

Myeloproliferative neoplasms: research and therapeutic landscape

A live synthesis of what is being targeted, what is being trialled, which datasets exist, and where the actionable gaps sit — assembled from public APIs, with every figure traceable to its source.

Generated 2026-09-17Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 109 studies · 7,059 GEO samples
Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in myeloproliferative neoplasms — JAK2, CALR, MPL, TET2, ASXL1, EZH2, SRSF2, U2AF1, IDH2, TP53, SH2B3, NFE2 — triaged for what can actually be aimed at them, alongside the trials, public datasets, literature and funding around the disease. Every figure below carries its source and retrieval date in the section it comes from.

7
drugs carry an FDA label naming myeloproliferative neoplasms: Fedratinib, Luspatercept, Momelotinib, Pacritinib, Ropeginterferon Alfa-2B, Rusfertide and 1 more. This counts labels, not treatment — the disease is also treated with agents approved under broader indications
1
of the 12 genes above carry a drug that is approved in myeloproliferative neoplasms itself — JAK2. Across all of them 56 drug entries reach these genes, 47 distinct once salt forms are merged
6
registered hydroxyurea cell-therapy trials in myeloproliferative neoplasms, 2 active. Counted from ClinicalTrials.gov across 3 synonyms, deduplicated by NCT id, interventional studies only — the query is published in the JSON beside this page so the number can be re-derived
8
targets carry an Open Targets tractability signal and have no clinical programme of any kind: CALR, TET2, ASXL1, EZH2, SRSF2, U2AF1, SH2B3, NFE2
758
human GEO series match the disease; 109 survive on-topic filtering, and only 7 are patient cohorts of 100+ samples
31
Europe PMC full-text papers name GSE53482 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$6.7M
NIH obligations in FY2025, up -3% since 2013 — while distinct core projects went 7 to 5. Larger awards rather than more distinct core projects; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-17 · weekly

12 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.

GeneDrugsApproval, and whether it reached this diseaseOpen Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it worksCell 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 · weekly

7 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.

7Labelled for myeloproliferative neoplasmsFDA INDICATIONS AND USAGE names the disease
19Approved, but for another diseasereturned for the genes in the table above
0Backbone agents listing itbroad cytotoxics whose labels name many tumours
2Active hydroxyurea cell-therapy trialsof 6 registered

Every label that names myeloproliferative neoplasms

DrugRoleWhat the label says
FedratinibInrebicLabelled hereINREBIC 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 ).
LuspaterceptReblozylLabelled hereMyelodysplastic 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...
MomelotinibOjjaaraLabelled hereOJJAARA 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.
PacritinibVonjoLabelled hereVONJO 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 PenLabelled herePolycythemia Vera BESREMi is indicated for the treatment of adults with polycythemia vera.
RusfertideMIMRYLOLabelled hereMIMRYLO is a hepcidin mimetic indicated for the treatment of erythrocytosis in adults with polycythemia vera (PV).
RuxolitinibJAKAFI, JAKAFI XRLabelled herepolycythemia 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 · weekly

hydroxyurea is the busiest cell-therapy antigen in myeloproliferative neoplasms: 6 registered trials, 2 still active.

TrialPhaseStatusTitleLast update
NCT03810196no phaseRecruitingCD45RA Depleted Peripheral Stem Cell Addback for Viral or Fungal Infections Post TCRαβ/CD19 Depleted HSCT2026-07-08
NCT027278032RecruitingPersonalized NK Cell Therapy in CBT2026-07-30
NCT008067672CompletedFludarabine, Busulfan, and Antilymphocyte Globulin Followed by Donor Stem Cell Transplant in Treating Older Patients With Hematological Cancer2011-05-17
NCT000056412TerminatedRemoval of T Cells to Prevent Graft-Versus-Host Disease in Patients Undergoing Bone Marrow Transplantation2012-12-11
NCT004025581CompletedAlloreactive NK Cells for Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)2015-05-08
NCT036151052TerminatedDonor Stem Cell Transplantation Using α/β+ T-lymphocyte Depleted Grafts From HLA Mismatched Donors2025-04-06

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 · monthly

Every 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 term2015–182021–25ChangeShare now
Janus Kinase Inhibitors1.61%7.73%4.8×126 papers
Neoplasms0.69%3.13%4.54×51 papers
Venous Thrombosis0.54%1.84%3.43×30 papers
Neutrophils0.54%1.78%3.32×29 papers
Polycythemia1.0%3.19%3.2×52 papers
Pyrrolidines0.69%2.08%3.02×34 papers
Sulfonamides0.69%1.9%2.76×31 papers
Interferon alpha-21.0%2.39%2.4×39 papers
Benzenesulfonamides0.61%1.47%2.4×24 papers
Graft vs Host Disease1.53%3.56%2.32×58 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Biopsy3.6%0.74%0.2×12 papers
Mutation, Missense3.52%0.74%0.21×12 papers
Diagnosis, Differential4.21%0.92%0.22×15 papers
Quinazolines2.83%0.74%0.26×12 papers
Disease-Free Survival2.91%0.86%0.3×14 papers
Genetic Predisposition to Disease2.76%0.86%0.31×14 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Primary Myelofibrosis59.42%55.86%0.94×911 papers
Polycythemia Vera31.93%36.97%1.16×603 papers
Janus Kinase 233.08%29.68%0.9×484 papers
Thrombocythemia, Essential33.46%28.57%0.85×466 papers
Mutation26.72%22.62%0.85×369 papers
Pyrimidines17.61%17.84%1.01×291 papers
Nitriles16.0%16.49%1.03×269 papers
Pyrazoles16.77%15.63%0.93×255 papers
Treatment Outcome17.53%11.59%0.66×189 papers
Thrombosis7.96%11.28%1.42×184 papers

Publication mix

Type2015–182021–25
Clinical Trial0.0%0.1%
Randomized Controlled Trial2.6%1.8%
Review14.9%14.7%
Meta-Analysis0.9%0.8%
Case Reports0.0%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.

MeasureValueMatchWhat it counts
New cases each yearnot publishedNo 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 ratenot publishedPublished 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 yearnot publishedNot published for the group; deaths are recorded as AML when the disease transforms and as thrombosis when it does not.
Five-year relative survivalnot publishedNot 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 diagnosisnot publishedNot 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 · quarterly

NIH 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.

$6.7MNIH obligations, FY2025from $7.0M in FY2013 · -3%
5distinct projects funded7 in FY2013
$10.1Mpeak year was FY2015obligations, all institutes
67%of FY2025 awards from NHLBI4 of 6

NIH obligations by fiscal year

$3M$5M$8M$10M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$7.0M971
FY2014$5.5M971
FY2015$10.1M1171
FY2016$2.4M980
FY2017$1.9M870
FY2018$6.3M870
FY2019$4.8M651
FY2020$6.7M11101
FY2021$6.3M980
FY2022$6.9M11101
FY2023$7.3M1091
FY2024$7.1M981
FY2025$6.7M651

Where FY2025 money went

InstitutionObligationsAwards
Icahn School Of Medicine At Mount Sinai$4.6M3
Aplexis, Inc.$1.1M1
University Of Virginia$0.6M1
Utah State Higher Education System--University Of Utah$0.5M1

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

NHLBI4 · 67%
NCI2 · 33%

Projects by administering institute. The rows above are the top 2 and account for 6 of 6.

Award mechanisms

P012 · 33%
R012 · 33%
R441 · 17%
K081 · 17%

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

Icahn School Of Medicine At Mount Sinai$4.6M · 67.9%
Aplexis, Inc.$1.1M · 16.3%
University Of Virginia$0.6M · 8.9%
Utah State Higher Education System--Univ$0.5M · 6.9%

Share of $6.7M in FY2025. The top three hold 93%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

758 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

patient 63unspecified 20mixed 17cell line 7xenograft 2
63 patient20 unspecified17 mixed7 cell line2 xenograft83 carry clinical annotation20 carry survival7 patient cohorts ≥100 GEO samples7,059 GEO samples totalin 12 single-cell series a GEO sample is one cell, not one patient

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE41812Gene expression profile of mutated and wild-type PMF CD34+ cells2013 · array20817.1
patient cohortAPT 0.95molecularASXL1EZH2IDH2627 cites
GSE1221983'-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-cell2,79846.6
patient cohortAPT 0.75molecularJAK2240 cites
GSE53482Integrative Analysis of Gene and miRNA expression profiles in Primary Myelofibrosis CD34+ cells2014 · array146312.8
patient cohortAPT 0.75survivalmolecular105 cites
GSE54646Integrated genomic analysis illustrates the central role of JAK-STAT pathway activation in myeloproliferative neoplasm pathogenesis2014 · array116139.7
APT 0.95molecularJAK356 cites
GSE55976Gene expression profile in myeloproliferative neoplasms2015 · array4572.3
patient cohortAPT 0.5survivalmolecularJAKJAK275 cites
GSE47018Gene Expression Profiling in Polycythemia Vera (PV)2014 · array27101.5
patient cohortAPT 0.75survivalmolecularJAK254 cites
GSE26049Expression data from patients with Essentiel Thrombocythemia (ET), Polycythemia Vera (PV), Primary Myelofibrosis (PMF) and control subjects2012 · array181251.4
patient cohortAPT 0.556 cites
GSE19647SNP data of MPD/MPN samples2010 · array15912.3
patient cohortAPT 0.75survivalstagemolecularTP53109 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE277354Genetic charcterization of myeloproliferative neoplasms (MPN)2024 · sequencing40223.0
patient cohortAPT 0.75molecular17 cites
GSE190517DUSP6 mediates resistance to JAK2 inhibition and drives leukemic progression (RNA-Seq)2022 · sequencing2014.3
patient cohortAPT 0.75survivalmolecularJAKJAK2MPL51 cites
GSE214914DUSP6 mediates resistance to JAK2 inhibition and drives leukemic progression (single-cell RNA-seq)2022 · single-cell414.0
patient cohortAPT 0.5survivalmolecularJAKJAK2MPL48 cites
GSE277035Pre-clinical efficacy of CDK7 inhibitor-based combinations against myeloproliferative neoplasms (MPN) transformed to AML2025 · sequencing1012.6
mixedAPT 0.25survivalmolecularJAKPELABRESIBRUXOLITINIB10 cites
GSE206768Inflammatory gene expression associated with advanced myelofibrosis2022 · array2915.3
patient cohortAPT 0.5molecular73 cites
GSE183374HMGA1 Chromatin Regulators Drive MPN Progression [RNA-Seq]2023 · sequencing1222.2
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 refresh

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.

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

Static

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.

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."
}