Rare & Orphan Lab · DeCure for X
DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for polycythemia vera — screening already-approved drugs against its 45-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
No approved-drug candidate for polycythemia vera is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
endothelial PAS domain protein 1 (EPAS1) — EPAS1 is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
RCSB Protein Data Bank · entry 3H82 · 1.5 Å · ligand N-(furan-2-ylmethyl)-2-nitro-4-(trifluoromethyl)aniline (020). Experimental structure, not a prediction.
Polycythemia vera is a myeloproliferative neoplasm that increases haematopoiesis, mainly affecting the erythroid lineage. More than 95% of cases carry a JAK2 mutation, most commonly the V617F variant in exon 14, and the absence of CALR mutations in JAK2-negative polycythemia vera was confirmed in a 2016 study. A 1968 report on 210 cases observed over 15 years described treatment with radioactive phosphorus (P32) and, more recently, with Myleran; a small number of patients received chemotherapy alternating with P32. The authors noted differing response patterns to each treatment and possible interference of therapy with the natural disease course, but provided no response rates, survival figures, or sample sizes for individual regimens.
A 2023 nationwide cross-sectional study from Taiwan examined real-world patient characteristics and treatment patterns for polycythemia vera between 2016 and 2017. The supplemental material for that study is available but the abstract itself gives no concrete numbers on patient counts, treatments used, or outcomes such as haematocrit control, thrombosis rates, or survival. No drug repurposing data, no novel agents, and no comparative efficacy results appear in any of these abstracts.
The evidence base for polycythemia vera remains dominated by JAK2 mutation diagnostics and older therapies such as P32 and Myleran, with no modern randomised trials of repurposed drugs reported in these abstracts. What is missing is any prospective trial design that tests a repurposed agent against standard cytoreduction, adequate funding for such a trial, and patient stratification by mutation status or prior treatment history.
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
AbstractPolycythemia vera (PV) is a myeloproliferative neoplasm (MPN) responsible for increased hematopoiesis, mainly affecting the erythroid lineage. The diagnosis of PV has been simplified enormously by the description of JAK2 mutations, affecting the canonical V617 in exon 14 in more than 95% of cases,
https://doi.org/10.3324/haematol.2016.154799AbstractPURPOSE OF REVIEW: Polycythemia vera is a relatively common myeloproliferative neoplasm (MPN) molecularly defined by the presence of mutations in the janus kinase (JAK2) gene. Yet, many aspects of pathogenesis remain to be ascertained and no effective treatment for curing the disease or preventing major cardiovascular events and progression to myelofibrosis or acute leukemia exists. The objective of this report is to review recent advances in the field and discuss the potential of novel therapeutic approaches. RECENT FINDINGS: The discovery of a specific germline haplotype in JAK2 provided an explanation for the well known phenomenon of familial clustering of MPN. Clinical trials with JAK2 inhibitors, either specific or not, have been initiated and first results are available. However, the expectation that these drugs could selectively target mutant cells and cause a molecular remission, similar to the experience with imatinib in chronic myelogenous leukemia, has been largely unmet. With the aim to develop common tools for clinicians involved in conventional and experimental therapies, a set of criteria for evaluating response to treatment have been developed. SUMMARY: The last couple of years have witnessed significant improvements in understanding polycythemia vera biology and management, and the activation or completion of novel trials are expected to provide further information to improve the treatment.
https://doi.org/10.1097/cco.0b013e32833ed81cAbstractRopeginterferon alfa-2b (RopegIFN) enables effective cytoreduction in polycythemia vera (PV). Recent analyses suggest that long-term RopegIFN therapy fulfills treatment goals important to patients with PV including good quality of life, the slowing of disease progression, and long event-free survival. Data support the use of RopegIFN in both early PV therapy and second-line and beyond.
https://doi.org/10.4274/tjh.galenos.2023.2023.0419AbstractSupplemental material, sj-docx-1-tah-10.1177_20406207231179331 for Real-world patient characteristics and treatment patterns of polycythemia vera in Taiwan between 2016 and 2017: a nationwide cross-sectional study by Tsung-Hsien Tsai, Lennex Hsueh-Lin Yu, Ming-Sun Yu, Shih-Hao Huang, Alex Jia-Hong Lin, Kuan-Der Lee and Min-Chi Chen in Therapeutic Advances in Hematology
https://doi.org/10.25384/sage.23528481AbstractSupplemental material, sj-docx-1-tah-10.1177_20406207231179331 for Real-world patient characteristics and treatment patterns of polycythemia vera in Taiwan between 2016 and 2017: a nationwide cross-sectional study by Tsung-Hsien Tsai, Lennex Hsueh-Lin Yu, Ming-Sun Yu, Shih-Hao Huang, Alex Jia-Hong Lin, Kuan-Der Lee and Min-Chi Chen in Therapeutic Advances in Hematology
https://doi.org/10.25384/sage.23528481.v1AbstractSupplemental material, sj-docx-2-tah-10.1177_20406207231179331 for Real-world patient characteristics and treatment patterns of polycythemia vera in Taiwan between 2016 and 2017: a nationwide cross-sectional study by Tsung-Hsien Tsai, Lennex Hsueh-Lin Yu, Ming-Sun Yu, Shih-Hao Huang, Alex Jia-Hong Lin, Kuan-Der Lee and Min-Chi Chen in Therapeutic Advances in Hematology
https://doi.org/10.25384/sage.23528484.v1AbstractSummary 210 cases of polycythemia vera were observed over a period of 15 years. The majority of them received P 32 ; treatment with Myleran instead of with radioactive phosphorus was instituted only recently. To a small number of patients chemotherapy was given alternately with P 32 . The differing response pattern to either kind of treatment and the possible interference of therapy with the natural development of the disease process are demonstrated in a few selected cases.
https://doi.org/10.1017/s1120962300026548Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
