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Published on: May 21, 2013
Reiterative infusions of MSCs improve pediatric osteogenesis imperfecta eliciting a pro-osteogenic paracrine
Arantza Infante1, Blanca Gener1,2, Miguel Vázquez3
1Stem Cells and Cell Therapy Laboratory, Biocruces Bizkaia Health Research Institute, Cruces University Hospital, Barakaldo, Spain.
Background:
Osteogenesis imperfecta (OI) is a rare genetic disease characterized by bone fragility, with a wide range in the severity of clinical manifestations. The majority of cases are due to mutations in the COL1A1 or COL1A2 genes, which encode type I collagen. Mesenchymal stem cells (MSCs), as the progenitors of the osteoblasts, the main type I collagen secreting cell type in the bone, have been proposed and tested as an innovative therapy for OI with promising but transient outcomes.
Methods:
To overcome the short-term effect of MSCs therapy, we performed a phase I clinical trial based on reiterative infusions of histocompatible MSCs, administered in a 2.5-year period, in two pediatric patients affected by severe and moderate OI. The aim of this study was to assess the safety and effectiveness of this cell therapy in nonimmunosuppressed OI patients. The host response to MSCs was studied by analyzing the sera from OI patients, collected before, during, and after the cell therapy.
Results:
We first demonstrated that the sequential administration of MSCs was safe and improved the bone parameters and quality of life of OI patients along the cell treatment plus 2-year follow-up period. Moreover, the study of the mechanism of action indicated that MSCs therapy elicited a pro-osteogenic paracrine response in patients, especially noticeable in the patient affected by severe OI.
Conclusions:
Our results demonstrate the feasibility and potential of reiterative MSCs infusion for two pediatric OI and highlight the paracrine response shown by patients as a consequence of MSCs treatment.
Insights
Repeated infusions of mesenchymal stem cells (MSCs) are safe and effective for treating osteogenesis imperfecta (OI) in children. This cell therapy improved bone health and quality of life by stimulating a beneficial paracrine response.
Area of Science:
- Regenerative Medicine
- Pediatric Orthopedics
- Genetics
Background:
- Osteogenesis imperfecta (OI) is a rare genetic bone fragility disorder, often caused by COL1A1/COL1A2 gene mutations affecting type I collagen.
- Mesenchymal stem cells (MSCs) show promise for OI therapy but have historically provided only transient benefits.
Purpose of the Study:
- To evaluate the safety and efficacy of repeated mesenchymal stem cell (MSC) infusions in pediatric patients with osteogenesis imperfecta (OI).
- To investigate the host response to MSC therapy in non-immunosuppressed OI patients.
Main Methods:
- A Phase I clinical trial involving repeated, histocompatible MSC infusions over 2.5 years in two pediatric OI patients.
- Analysis of patient sera before, during, and after cell therapy to assess host response.
- Evaluation of bone parameters and quality of life.
Main Results:
- Sequential MSC administration was safe and improved bone parameters and quality of life in OI patients throughout treatment and a 2-year follow-up.
- MSC therapy induced a pro-osteogenic paracrine response in patients, particularly evident in the severe OI case.
Conclusions:
- Reiterative MSC infusions are feasible and demonstrate potential for treating pediatric OI.
- The study highlights the significant paracrine response elicited by MSC treatment in OI patients.
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