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Concise Review: Mesenchymal Stem Cell Therapy for Pediatric Disease: Perspectives on Success and Potential
Christopher R Nitkin1, Tracey L Bonfield2,3
1Division of Neonatology, Rainbow Babies and Children's Hospital, Cleveland, Ohio, USA.
Insights
Mesenchymal stem cells (MSCs) show promise for pediatric diseases, but optimizing their use requires further research. This review highlights key factors for enhancing MSC therapy efficacy in children.
Area of Science:
- Regenerative Medicine
- Pediatric Therapeutics
- Stem Cell Biology
Background:
- Mesenchymal stem cells (MSCs) offer potential for diverse pediatric diseases.
- Existing clinical trials demonstrate MSC efficacy but show significant variability in approaches.
Purpose of the Study:
- To review current MSC clinical trials in pediatric populations.
- To identify factors crucial for optimizing MSC therapeutic efficacy in children.
- To guide future preclinical and clinical research in pediatric regenerative medicine.
Main Methods:
- Review of published clinical trials involving MSCs for pediatric diseases.
- Analysis of MSC sources: bone marrow, umbilical cord, placenta, amniotic fluid, adipose tissue.
- Identification of key therapeutic variables influencing MSC efficacy.
Main Results:
- MSCs are efficacious across various pediatric conditions (pulmonary, cardiac, orthopedic, endocrine, neurologic, hematologic).
- Significant heterogeneity exists in administration routes, dosage, timing, and ex vivo manipulation.
- Donor, host, and immunologic factors (allogeneic therapy) require further investigation.
Conclusions:
- Optimizing MSC therapy in pediatrics necessitates examining specific factors like administration, dose, timing, and cell processing.
- Addressing these variables is essential for maximizing therapeutic benefits for each child and disease.
- Further research and standardized approaches are needed to translate MSC potential into effective clinical practice.
Abstract:
Mesenchymal stem cells (MSCs) represent a potentially revolutionary therapy for a wide variety of pediatric diseases, but the optimal cell-based therapeutics for such diversity have not yet been specified. The published clinical trials for pediatric pulmonary, cardiac, orthopedic, endocrine, neurologic, and hematologic diseases provide evidence that MSCs are indeed efficacious, but the significant heterogeneity in therapeutic approaches between studies raises new questions. The purpose of this review is to stimulate new preclinical and clinical trials to investigate these factors. First, we discuss recent clinical trials for pediatric diseases studying MSCs obtained from bone marrow, umbilical cord and umbilical cord blood, placenta, amniotic fluid, and adipose tissue. We then identify factors, some unique to pediatrics, which must be examined to optimize therapeutic efficacy, including route of administration, dose, timing of administration, the role of ex vivo differentiation, cell culture techniques, donor factors, host factors, and the immunologic implications of allogeneic therapy. Finally, we discuss some of the practicalities of bringing cell-based therapy into the clinic, including regulatory and manufacturing considerations. The aim of this review is to inform future studies seeking to maximize therapeutic efficacy for each disease and for each patient. Stem Cells Translational Medicine 2017;6:539-565.
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