{"title":"Preclinical efficacy of adipose-derived cell therapies for the treatment of myositis.","authors":"Baptiste Pileyre, Silvia Gandolfi, Catalina Abad, Thara Jaworski, Laurent Drouot, Laetitia Jean, Olivier Boyer, Isabelle Dubus, Jérémie Martinet","doi":"10.1093/stcltm/szaf038","DOIUrl":null,"url":null,"abstract":"<p><strong>Importance: </strong>Idiopathic inflammatory myopathies, commonly referred as myositis, are autoimmune diseases that cause muscle damage, progressive weakness, and disability. Current treatments, including corticosteroids and immunosuppressants, have significant limitations, highlighting the need for new therapies.</p><p><strong>Objective: </strong>This preclinical study explored the therapeutic potential of adipose tissue-derived cell therapies, specifically stromal vascular fraction (SVF) and adipose-derived stem cells (ADSC), using an Icos-/- NOD mouse model of spontaneous myositis.</p><p><strong>Design: </strong>SVF and ADSC were extracted from CD1 female mice adipose tissue and cultured. Various doses were injected intramuscularly into the right hind limb of 20- to 22-week-old female Icos-/- NOD mice with a control group. The therapeutic effects were assessed through clinical scoring, grip strength test, and motor function analysis using Catwalk system. Muscle atrophy was evidenced by histology, and systemic inflammation was analyzed by flow cytometry.</p><p><strong>Results: </strong>Mice treated with either SVF or ADSC showed a dose-dependent slowdown in disease progression and improvements in motor functions, such as gait, movement, speed, and weight distribution between the legs. Histological analysis showed a reduction in muscular atrophy, particularly in the injected limb. Flow cytometry analysis on lymph nodes showed shifts in leukocyte populations, with reduced expression of inflammatory and activation markers.</p><p><strong>Conclusions and relevance: </strong>Overall, this study demonstrated the therapeutic potential intramuscular injection of SVF or ADSC in the Icos-/- NOD mouse model of myositis, providing a proof-of-concept for the use of adipose tissue-derived cell therapies in the treatment of idiopathic inflammatory myopathies.</p>","PeriodicalId":21986,"journal":{"name":"Stem Cells Translational Medicine","volume":"14 9","pages":""},"PeriodicalIF":4.9000,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12445652/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Stem Cells Translational Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1093/stcltm/szaf038","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CELL & TISSUE ENGINEERING","Score":null,"Total":0}
引用次数: 0
Abstract
Importance: Idiopathic inflammatory myopathies, commonly referred as myositis, are autoimmune diseases that cause muscle damage, progressive weakness, and disability. Current treatments, including corticosteroids and immunosuppressants, have significant limitations, highlighting the need for new therapies.
Objective: This preclinical study explored the therapeutic potential of adipose tissue-derived cell therapies, specifically stromal vascular fraction (SVF) and adipose-derived stem cells (ADSC), using an Icos-/- NOD mouse model of spontaneous myositis.
Design: SVF and ADSC were extracted from CD1 female mice adipose tissue and cultured. Various doses were injected intramuscularly into the right hind limb of 20- to 22-week-old female Icos-/- NOD mice with a control group. The therapeutic effects were assessed through clinical scoring, grip strength test, and motor function analysis using Catwalk system. Muscle atrophy was evidenced by histology, and systemic inflammation was analyzed by flow cytometry.
Results: Mice treated with either SVF or ADSC showed a dose-dependent slowdown in disease progression and improvements in motor functions, such as gait, movement, speed, and weight distribution between the legs. Histological analysis showed a reduction in muscular atrophy, particularly in the injected limb. Flow cytometry analysis on lymph nodes showed shifts in leukocyte populations, with reduced expression of inflammatory and activation markers.
Conclusions and relevance: Overall, this study demonstrated the therapeutic potential intramuscular injection of SVF or ADSC in the Icos-/- NOD mouse model of myositis, providing a proof-of-concept for the use of adipose tissue-derived cell therapies in the treatment of idiopathic inflammatory myopathies.
期刊介绍：
STEM CELLS Translational Medicine is a monthly, peer-reviewed, largely online, open access journal.
STEM CELLS Translational Medicine works to advance the utilization of cells for clinical therapy. By bridging stem cell molecular and biological research and helping speed translations of emerging lab discoveries into clinical trials, STEM CELLS Translational Medicine will help move applications of these critical investigations closer to accepted best patient practices and ultimately improve outcomes.
The journal encourages original research articles and concise reviews describing laboratory investigations of stem cells, including their characterization and manipulation, and the translation of their clinical aspects of from the bench to patient care. STEM CELLS Translational Medicine covers all aspects of translational cell studies, including bench research, first-in-human case studies, and relevant clinical trials.
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