Single Stem Cell Infusion Shows Promise in Advanced Osteoporosis, Suggests Study
Spain: Researchers have found in a small study published in Cell that a single stem cell infusion was associated with a 94% reduction in fragility fractures over two years among 10 women with advanced osteoporosis. The findings suggest a potential regenerative approach for severe osteoporosis, but the very small sample size warrants confirmation in larger, controlled studies.
Osteoporosis is characterized by progressive loss of bone strength and increased susceptibility to fractures. Although mesenchymal stem/stromal cells (MSCs) have regenerative potential, their clinical use for skeletal disorders has been limited by their relatively poor ability to reach and remain at bone tissue. Jose M. Moraleda, Hematopoietic Cell Transplant and Cellular Therapy Unit, Instituto Murciano de Investigación Biosanitaria, Murcia, Spain, and colleagues investigated whether modifying the cell surface glycocalyx could improve this bone-targeting property.
The first-in-human clinical trial evaluated exofucosylated autologous bone marrow-derived MSCs (Fuc-autoBM-MSCs), which were modified to express sialylated Lewis X (sLeX). This glycocalyx modification was designed to enhance the cells' ability to home toward bone tissue. Ten women with advanced osteoporosis received a single intravenous infusion and underwent protocol-specified assessments for two years.
The researchers monitored participants clinically and through radiographic evaluations, while also assessing bone turnover markers (BTMs), bone tissue area (BTA), and bone mineral density (BMD). Fracture and safety surveillance was subsequently extended for more than three additional years for each participant.
Key findings:
- No serious adverse events were reported following the intravenous administration of Fuc-autoBM-MSCs.
- Fragility fracture incidence decreased markedly and remained reduced during follow-up.
- Bone tissue area and volumetric bone mineral density increased after treatment.
- Changes in bone turnover markers indicated enhanced osteoanabolic activity, consistent with an osteoregenerative response.
- The findings suggested that modifying the MSC glycocalyx may improve the cells' bone-targeting properties and therapeutic potential.
The investigators noted that the results also challenge the assumption that MSCs obtained from older individuals or diseased tissues necessarily have substantially impaired regenerative capabilities. By modifying the cells to improve osteotropism, the approach may allow their intrinsic regenerative properties to be harnessed more effectively in skeletal disease.
The authors concluded that a single intravenous infusion of glycocalyx-edited autologous MSCs was safe in this small group and was associated with sustained reductions in fragility fractures alongside improvements in bone-related measures. However, the study involved only 10 patients and lacked a larger controlled comparison, meaning the observed fracture reduction cannot yet establish treatment efficacy. Larger clinical trials will be required to determine whether this regenerative strategy can provide a durable therapeutic option for patients with advanced osteoporosis.
Reference:
Moraleda, J. M., Linares, L. F., García-Bernal, D., Lozano-Rivas, N., Ruiz-Sará, J., Blanquer, M., López-Lucas, M. D., Sánchez-Salinas, D., Algueró, M. C., Molina-Molina, M. M., Ruiz-Espejo, F., Peris-Serra, J. L., Arrabal, P. M., Becerra, J., Kiwitt-Cárdenas, J., Marras, C., García-Hernández, A. M., & Sackstein, R. (2026). Glycocalyx-edited mesenchymal stem/stromal cell therapy in advanced osteoporosis. Cell. https://doi.org/10.1016/j.cell.2026.08.017
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