POEMS Syndrome is a rare, paraneoplastic (cancer-related) multisystem disorder characterized by a constellation of symptoms: Polyneuropathy, Organomegaly (enlarged organs), Endocrinopathy (hormone abnormalities), Monoclonal gammopathy (presence of abnormal antibodies), and Skin changes. Protheragen provides an end-to-end preclinical drug and therapy development focus on solving the dilemmas associated with POEMS Syndrome.
POEMS Syndrome (Polyneuropathy, Organomegaly, Endocrinopathy, M-protein, Skin changes) is a rare multisystem disorder driven by VEGF-secreting plasma cell dyscrasias. Characterized by disabling neuropathy, organ enlargement, and hyperpigmented skin lesions, its incidence is estimated at 0.3 per 100,000 with 5-year survival <60% without treatment.
POEMS Syndrome pathogenesis is characterized by the overproduction of Vascular Endothelial Growth Factor (VEGF) by a clonal population of plasma cells. This elevated VEGF leads to widespread endothelial dysfunction, increasing vascular permeability and contributing to polyneuropathy, organomegaly, edema, and diverse skin changes. An inflammatory cytokine milieu further exacerbates these multisystem manifestations.
Fig.1 Bone lesions in POEMS Syndrome. (Jaccard, et al., 2020)| Category | Drug Name | Target / Intervention | Mechanistic Validation/Model Data | Development Stage |
| Anti-CD38 mAb | Daratumumab | CD38 (plasma cell surface antigen) | Depletes VEGF-secreting plasma cells in bone marrow xenografts; reduces microvascular leakage | Clinical use |
| Proteasome Inhibitor | Bortezomib | NF-κB pathway suppression | Induces apoptosis in VEGFhi plasma cells; suppresses IL-6/VEGF crosstalk | Clinical use |
| Anti-VEGF Biologics | Bevacizumab | VEGF-A neutralization | Blocks VEGF-induced angiogenesis in 3D neurovascular models; reduces edema | Salvage therapy |
| JAK Inhibitor | Ruxolitinib | JAK1/2 inhibition (IFN signaling blockade) | Normalizes IFN-γ/IL-6 ratios in Castleman-like lesions; reduces inflammatory markers | Case reports |
| Dual-Target CAR-T | KQ-2003 | BCMA/CD19 dual targeting | Eliminates pathogenic plasma cells and modulates B-cell microenvironment in humanized mice | Phase I |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Protheragen offers a comprehensive suite of services to advance POEMS Syndrome therapeutics. We are dedicated to supporting your projects with a team of expert scientists, dermatologists, and geneticists, driving progress in therapeutic development, diagnostics development as well as disease model development.
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References
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