DeCure for Myelodysplastic/Myeloproliferative Neoplasm
DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for Myelodysplastic/Myeloproliferative Neoplasm — screening already-approved drugs against its 47-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMyelodysplastic/Myeloproliferative Neoplasm maps to a 47-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.
Research record
01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Current lead
No approved-drug candidate for myelodysplastic/myeloproliferative neoplasm 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.
Molecular view
isocitrate dehydrogenase (NADP(+)) 2 (IDH2) — IDH2 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.
Loading structure…
helix sheet ndpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5I96 · 1.55 Å · ligand NADPH DIHYDRO-NICOTINAMIDE-ADENINE-DINUCLEOTIDE PHOSPHATE (NDP). Experimental structure, not a prediction.
What the evidence adds up to
Recurrent mutations in calreticulin are present in about 20% of patients with myeloproliferative neoplasms. Mutant CALR, an endoplasmic reticulum chaperone protein, activates the JAK/STAT signalling pathway through a pathogenic binding interaction with the thrombopoietin receptor MPL. This is a review of the biology of mutant CALR-driven MPNs and highlights future therapeutic approaches; no clinical trial results are reported.
The WHO classification includes the category "Myelodysplastic/myeloproliferative neoplasms" for clonal myeloid neoplasms that at initial presentation have features of both myelodysplastic syndrome and myeloproliferative neoplasm. These disorders comprise chronic myelomonocytic leukaemia, atypical chronic myeloid leukaemia (BCR-ABL1 negative), juvenile myelomonocytic leukaemia, and MDS/MPN unclassifiable. The best characterised unclassifiable condition is refractory anaemia with ringed sideroblasts associated with marked thrombocytosis.
A cross-sectional study from South India reported 51 cases of myeloid neoplasms over 18 months. Among these, 28 (55%) were MPN, 14 (27%) were acute myeloid leukaemia, five (10%) were MDS, and four (8%) were MPN/MDS. Age range was 14 to 74 years, with most cases between 51 and 60 years, and a male-to-female ratio of 1:1.25. The most common symptoms were constitutional: generalised weakness, easy fatigability, and fever. No treatment outcomes or survival data are given.
A 2013 review notes that three drugs were approved for MDS in the preceding decade and that novel sequencing has identified recurrent gene mutations, but states plainly that treatment options remain limited and novel therapeutic strategies are needed. The review asks how to manage the challenging group of patients with MDS/MPN overlap and what is on the horizon for novel therapies. What is still missing are prospective trials that test specific repurposed drugs in MDS/MPN overlap patients, adequate funding for such trials, and validated biomarkers to stratify patients by molecular subtype.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Blood · 2019 · 83 citations · open access
Mutant calreticulin in myeloproliferative neoplasms
AbstractRecurrent mutations in calreticulin are present in ∼20% of patients with myeloproliferative neoplasms (MPNs). Since its discovery in 2013, we now have a more precise understanding of how mutant CALR, an endoplasmic reticulum chaperone protein, activates the JAK/STAT signaling pathway via a pathogenic binding interaction with the thrombopoietin receptor MPL to induce MPNs. In this Spotlight article, we review the current understanding of the biology underpinning mutant CALR-driven MPNs, discuss clinical implications, and highlight future therapeutic approaches.
Molecular basis of myelodysplastic/myeloproliferative neoplasms
AbstractThe World Health Organization (WHO) classification of tumors of hematopoietic and lymphoid tissues1 includes within myeloid neoplasms the category "Myelodysplastic/myeloproliferative neoplasms". According to Vardiman et al.,2 these are "clonal myeloid neoplasms that at the time of initial presentation have some clinical, laboratory or morphologic findings that support a diagnosis of myelodysplastic syndrome (MDS), and other findings more consistent with myeloproliferative neoplasm (MPN)". These disorders comprise chronic myelomonocytic leukemia (CMML),3 atypical chronic myeloid leukemia (aCML, BCR-ABL1 negative),4 juvenile myelomonocytic leukemia (JMML),5 and myelodysplastic/myeloproliferative neoplasms, unclassifiable (MDS/MPN, U).6 The best characterized of these unclassifiable conditions is the provisional entity defined as refractory anemia with ringed sideroblasts (RARS) associated with marked thrombocytosis (RARS-T).7
American Society of Clinical Oncology Educational Book · 2013 · 13 citations
Myelodysplastic Syndromes: Recent Advancements in Risk Stratification and Unmet Therapeutic Challenges
AbstractSignificant advances have been achieved in understanding and treating myelodysplastic syndromes (MDS) in the past decade. For the first time, three drugs were approved specifically for this disease. Novel sequencing techniques have expanded our understanding of the molecular basis of MDS. Several clinically significant recurrent gene mutations have been identified. The classification and risk stratification of MDS continues to evolve in light of such advances. However, treatment options remain limited and novel therapeutic strategies are needed. In this review we address key questions for management of MDS. How do we better classify and risk stratify MDS, tailoring treatment accordingly? How do we diagnose and manage the challenging group of patients with MDS/myeloproliferative neoplasms (MPN) overlap? And finally, what is on the horizon for novel therapies?
American Society of Clinical Oncology Educational Book · 2013 · 6 citations
Myelodysplastic Syndromes: Recent Advancements in Risk Stratification and Unmet Therapeutic Challenges
AbstractSignificant advances have been achieved in understanding and treating myelodysplastic syndromes (MDS) in the past decade. For the first time, three drugs were approved specifically for this disease. Novel sequencing techniques have expanded our understanding of the molecular basis of MDS. Several clinically significant recurrent gene mutations have been identified. The classification and risk stratification of MDS continues to evolve in light of such advances. However, treatment options remain limited and novel therapeutic strategies are needed. In this review we address key questions for management of MDS. How do we better classify and risk stratify MDS, tailoring treatment accordingly? How do we diagnose and manage the challenging group of patients with MDS/myeloproliferative neoplasms (MPN) overlap? And finally, what is on the horizon for novel therapies?
Medical Journal of Dr D Y Patil Vidyapeeth · 2025 · 0 citations · open access
Clinico-hematological and Cytogenetic Study of Myeloid Neoplasms: A cross-sectional Study from South India
AbstractA BSTRACT Background: Myeloid neoplasms are clonal diseases arising in hematopoietic stem or progenitor cells consisting of myeloproliferative neoplasm (MPN), myelodysplastic syndrome (MDS), and acute myeloid leukemia (AML). Aim: To re-classify the myeloid neoplasm as per the World Health Organization (WHO)-5 classification and to study the frequency of occurrence and clinical, hematological, and cytogenetic profile of myeloid neoplasms. Settings and Design: A cross-sectional study was conducted for a period of 1 and a 1/2 years from January 2019 to June 2020. Materials and Methods: All the cases newly diagnosed as myeloid neoplasms during this period were included in the study. Clinical features, and hematological and cytogenetic findings were compiled. Statistical Analysis: For categorical data, the number and percentage were used. The Chi-square (χ2) test was used for association between two categorical variables. If the P value was < 0.05, then the results were considered to be statistically significant. Data were analyzed using the Statistical Package for the Social Sciences (SPSS) software v. 23 (IBM Statistics, Chicago, USA) and Microsoft Office 2007. Results and Conclusion: A total of 51 cases were observed during the study period. The cases ranged from 14 to 74 years with maximum cases occurring between 51 and 60 years with a male-to-female (M: F) ratio of 1:1.25. Among the 51 cases of myeloid neoplasms, 28 (55%) cases of MPN, 14 (27%) cases of AML, five (10%) cases of MDS, and four (8%) cases of MPN/MDS were observed in their decreasing frequency. The most common symptoms were constitutional symptoms, such as generalized weakness, easy fatigability, and fever. Bone marrow study played a major role in diagnosis. The cytogenetic study complemented the diagnosis. A high clinical suspicion along with a good morphological diagnosis using peripheral smear, bone marrow aspiration, and biopsy supplemented by special stains and molecular studies will enable early and efficient diagnosis of myeloid neoplasms.
Disease 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.
