DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for POEMS syndrome — screening already-approved drugs against its 5-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePOEMS syndrome maps to a 5-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 poems syndrome 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
cereblon (CRBN) — CRBN 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 3sdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8TNQ · 2.41 Å · ligand 2-[(3S)-2,6-dioxopiperidin-3-yl]-5-(morpholin-4-yl)-1H-isoindole-1,3(2H)-dione (MIQ). Experimental structure, not a prediction.
What the evidence adds up to
In a retrospective longitudinal cohort of 100 POEMS patients followed for a median of 59 months, mean time from symptom onset to diagnosis was 15 months and 54% were initially misdiagnosed with chronic inflammatory demyelinating polyneuropathy. At diagnosis 35% were wheelchair or bedbound; the median Overall Neuropathy Limitation Score was 6 and improved to 3 after treatment. Five-year overall survival was 90%, ten-year survival 82%; five-year progression-free survival was 65% and ten-year 53%. Nontreatment with autologous stem cell transplantation, nonhematologic response, and non–vascular endothelial growth factor response were significant risk factors for progression or death in multivariate analysis.
A separate clinicopathologic analysis of 9 cases found that POEMS patients had lower serum M-protein and bone marrow plasma cell levels than multiple myeloma patients, and that sclerotic bone lesions were a distinctive feature. The 2021 case report describes a 23-year-old woman with a 9-year history of type I diabetes and primary amenorrhea who developed congestive heart failure with left ventricular ejection fraction of 38%. After one month of dexamethasone and thalidomide her symptoms did not improve; adding twice-weekly bortezomib led to significant improvement in heart function within two weeks. A 2024 case report describes an M protein-negative POEMS patient with membranoproliferative glomerulonephritis and thrombotic microangiopathic changes who was treated successfully by comparing serum VEGF levels and polyserositis changes before and after treatment.
The 2018 review states there is no unified standard treatment and lists general symptomatic therapy, drug treatment, radiotherapy, and autologous haematopoietic stem cell transplantation as approaches. The 2020 cohort found that nontreatment with autologous stem cell transplantation was a risk factor for worse outcomes, but the 2021 case report notes that a patient failed to improve on dexamethasone and thalidomide and required bortezomib. The evidence base remains limited to retrospective series and single case reports.
What is still missing: prospective randomised trials comparing treatment regimens, validated stratification tools beyond the single-centre risk score, and systematic data on heart failure management in POEMS. The rarity of the disease makes adequately powered trials difficult to fund and complete.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Neurology · 2020 · 50 citations
Clinical characteristics, risk factors, and outcomes of POEMS syndrome
Abstract<h3>Objective</h3> POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin lesions) is a paraneoplastic disorder resulting in severe neurologic disability. Understanding the clinical, laboratory, neurophysiologic, and histopathologic features as well as treatment responses of POEMS will assist in more accurate and timely diagnosis, risk stratification, and effective management. <h3>Methods</h3> This was a retrospective longitudinal cohort study from 1998 to March 2019, with 7,184 person-months of follow-up time. Hospital databases were used to collate presenting features, investigations, therapies, and response. <h3>Results</h3> One hundred patients were included with a median follow-up time of 59 months (range, 1–252). Mean symptom onset to diagnosis was 15 months (range, 1–77), with 54% of patients initially misdiagnosed with chronic inflammatory demyelinating polyneuropathy. Median number of multisystem features at diagnosis was 7. Ninety-six (96%) presented with neuropathy, which was length-dependent in 93 (93%) and painful in 75 (75%). At diagnosis, 35% of patients were wheelchair or bedbound, with median Overall Neuropathy Limitation Score of 6, improving to 3 following treatment (<i>p</i> < 0.05). Five-year survival was 90% and 82% at 10 years, with 5- and 10-year progression-free survival of 65% and 53%. Nontreatment with autologous stem cell transplantation, nonhematologic response, and non–vascular endothelial growth factor response are significant risk factors in multivariate analysis to predict progression or death. Risk factors are incorporated to develop a risk score enabling stratification of high- and low-risk cases. <h3>Conclusions</h3> POEMS syndrome is a rare multisystem condition with delayed diagnosis and poor neurologic function at presentation. Therapy has favorable outcomes. Patients at high risk of death or progression can be identified, which may allow for more active monitoring and influence management.
Clinicopathologic Analysis of POEMS Syndrome and Related Diseases
AbstractBACKGROUND: POEMS syndrome, a rare paraneoplastic disease, is related to multiple organs, multiple systems, and multiple disciplines and can be mistaken for other disorders. Consequently, the diagnoses are often delayed. In this work we studied the clinicopathologic characteristics of the POEMS syndrome to improve early diagnosis to prevent irreversible damage. PATIENTS AND METHODS: We conducted a clinicopathologic analysis of 9 cases of POEMS and made a differential diagnosis with related diseases. RESULTS: The patients with POEMS syndrome were shown to have complicated clinical characteristics, including peripheral neuropathy, organomegaly, endocrinopathy, monoclonal plasma proliferative disorder, skin changes, extravascular volume overload, sclerotic bone lesions, thrombocytosis, and Castleman disease. POEMS syndrome shared many elements with other diseases and the key way to differentiate them was to determine whether there were other fundamental POEMS syndrome symptoms or signs. The level of M-protein in serum and plasma cells in bone marrow of POEMS patients was lower than that of patients with multiple myeloma (MM). Sclerotic bone lesions were a distinctive feature in patients with POEMS, compared with in those with MM. CONCLUSION: Some unique clinicopathologic characteristics of POEMS syndrome can be used for differential diagnosis. This study provides increased awareness of POEMS syndrome.
World Journal of Clinical Cases · 2021 · 2 citations · open access
Effective treatment of polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes syndrome with congestive heart failure: A case report
AbstractBACKGROUND: Polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes (POEMS) syndrome is a rare paraneoplastic syndrome caused by a plasma cell proliferative disorder. The syndrome is characterized by elevated plasma cells, platelets, and vascular endothelial growth factor levels. Although heart disease rarely occurs in POEMS syndrome, the death rate increases sharply after heart failure. We report a patient who initially presented with an endocrine disease and developed congestive heart failure related to POEMS syndrome 9 years later. CASE SUMMARY: A 23-year-old woman with no history of menstruation and a 9-year history of type I diabetes reported feeling breathless after activities. She could not lie down and rest at night. Three months prior, she experienced pain and increased tension in her left thigh accompanied by tenderness and edema in both lower extremities. The chief complaint upon hospital admission was that blood sugar has increased for more than 9 years, pain in the left thigh, and edema in both legs for more than 2 mo. After a multisystem evaluation, she was diagnosed with POEMS syndrome. Her echocardiogram showed left ventricular dilation with systolic dysfunction, and the left ventricular ejection fraction was only 38% with severely elevated brain natriuretic peptide. She received a combination of dexamethasone and thalidomide for 1 mo, but her symptoms did not improve. Therefore, we added a two-per-week bortezomib injection. After 2 wk, the patient's heart function had improved significantly. CONCLUSION: This case provides information about the treatment of POEMS syndrome with complications and highlights the challenges of developing a standardized treatment.
AbstractPOEMS syndrome is a disease associated with plasma cell paraneoplastic syndrome. There is no unified standard treatment, and often general symptomatic treatment, drug treatment, radiotherapy, autologous hematopoietic stem cell transplantation, etc. is used. The latest treatment progress of POEMS syndrome is reviewed.
Key words:
POEMS syndrome; Clinical protocols; Review
A case report of an M protein-negative patient with POEMS syndrome associated with renal involvement
AbstractBACKGROUND: POEMS syndrome with polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes is an uncommon plasma cell paraneoplastic syndrome involving multiple system. It is relatively rare in clinical practice, and renal involvement is a usual yet easily overlooked symptom. CASE PRESENTATION: We successfully treated a patient with M protein-negative POEMS syndrome with membranoproliferative glomerulonephritis (MPGN) findings and thrombotic microangiopathic changes by comparing the level of Vascular endothelial growth factor (VEGF) in the serum and the changes in polyserositis before and after the patient's treatment. CONCLUSION: POEMS syndrome clinically involves multiple systems and has complex symptoms. Because of the diversity of the disease manifestations, identification of atypical POEMS syndrome and timely intervention are important for patient survival and prognosis.
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.
