Research Progress of Mesenchymal Stem/Stromal Cell Therapy Medicinal Product

MSCs, with their diverse biological properties, have become a focal point in both basic and clinical research. Their immune-regulating and regenerative capabilities are widely utilized in clinical cell therapies and studies. Over the past decade, clinical trials involving MSCs have surged dramatically, with over 1 000 trials either completed or ongoing. However, developing MSCs into safe, effective, quality-controlled, and process-stable living cell therapies remains challenging, necessitating preclinical drugability evaluations. Concurrently, preclinical pharmacological studies, pharmacokinetic research, and non-clinical safety assessments must be conducted. To date, more than a dozen MSC-based drugs have been approved globally. In January 2025, NMPA (National Medical Products Administration) conditionally approved umbilical cord-derived MSCs through expedited review and approval procedures, marking the country’s entry into the commercialization phase of MSC therapies. Despite these achievements, the number of approved drugs remains disproportionately low compared with the volume of clinical trials. This imbalance primarily stems from incomplete understanding of MSC mechanisms, leading to imprecise patient stratification in trials and suboptimal outcomes across multiple studies. In the future, more research efforts are required in areas such as mechanism exploration, PK-PD studies, and large-scale preparation technology development. With emerging knowledge and methodologies, the development of MSC extracellular vesicles, genetically modified MSCs, and composite products combining cells with biomaterials has gained significant attention. Furthermore, exploratory studies on clinical efficacy-related biomarkers and combination therapies are advancing the development of mesenchymal stem/stromal cell-based drugs, paving the way for their broader application in treating patients.

CSTR: 32200.14.cjcb.2026.01.0005