Journal
STEM CELLS TRANSLATIONAL MEDICINE
Volume 6, Issue 2, Pages 539-565Publisher
OXFORD UNIV PRESS
DOI: 10.5966/sctm.2015-0427
Keywords
Mesenchymal stem cells; Pediatric diseases; Bronchopulmonary dysplasia; Autism; Osteogenesis imperfecta; Graft versus host disease
Categories
Funding
- David and Virginia Baldwin Fund
- Cord Blood Registry
- Rainbow Babies and Children's Foundation
- Little Giraffe Foundation
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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. Wethen 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.
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