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Published on: September 11, 2018
Mesenchymal stem cells for the prevention of bronchopulmonary dysplasia
1Department of Pediatrics, Saitama Medical Center, Saitama Medical University, Kawagoe, Saitama, Japan.
Insights
Mesenchymal stem cells (MSC) show promise for treating bronchopulmonary dysplasia (BPD) in premature infants. Early trials indicate MSC therapy is safe and feasible, with ongoing studies evaluating its efficacy in reducing BPD rates.
Area of Science:
- Neonatal Medicine
- Regenerative Medicine
- Pulmonology
Background:
- Bronchopulmonary dysplasia (BPD) is a chronic lung disease affecting preterm infants, with limited therapeutic advancements despite medical progress.
- Mesenchymal stem cells (MSC) offer potential due to their low immunogenicity, anti-inflammatory, and regenerative properties.
- Pre-clinical studies suggest MSC exert therapeutic effects in BPD models primarily through paracrine factors, not direct cell regeneration.
Purpose of the Study:
- To evaluate the safety and feasibility of mesenchymal stem cell (MSC) therapy for bronchopulmonary dysplasia (BPD) in preterm infants.
- To explore the potential of MSC-derived paracrine factors in managing BPD.
- To inform ongoing and future clinical trials investigating MSC for BPD prevention and treatment.
Main Methods:
- A first-in-human, phase I dose-escalation clinical trial was conducted using umbilical cord blood-derived MSC.
- Tracheal aspirates were analyzed to assess inflammatory marker expression post-treatment.
- Ongoing and planned clinical trials are investigating the efficacy of MSC in BPD.
Main Results:
- The initial clinical trial demonstrated the short- and long-term safety and feasibility of MSC administration in preterm infants.
- A significant reduction in inflammatory marker expression was observed in tracheal aspirates following MSC treatment.
- Several clinical trials are currently underway to further assess MSC efficacy for BPD.
Conclusions:
- Mesenchymal stem cell (MSC) therapy is a safe and feasible option for preterm infants with bronchopulmonary dysplasia (BPD).
- MSC's therapeutic effects in BPD appear to be mediated by paracrine signaling, reducing inflammation.
- Further clinical trials are crucial to establish the efficacy of MSC for BPD treatment and prevention.
Abstract:
Bronchopulmonary dysplasia (BPD) is a chronic lung disease in preterm infants who have been treated with supplemental oxygen and mechanical ventilation. Despite major advances in perinatal and neonatal medicine, limited progress has been made in reducing BPD rates. The use of mesenchymal stem cells (MSC) is a promising and innovative therapy for several diseases because they are easy to extract and they have low immunogenicity, anti-inflammatory properties, and regenerative ability. According to several pre-clinical studies that have used BPD animal models, one mechanism of action for MSC in BPD is mainly due to the paracrine effects of MSC-derived humoral factors, such as interleukin (IL)-6, IL-8, vascular endothelial growth factor, collagen, and elastin, rather than the multilineage and regenerative capacities of MSC. Cell-free preparations derived from MSC, including conditioned media and exosomes, remain a pre-clinical technology despite their great clinical potential. A first-in-human clinical trial of MSC treatment for BPD was performed as a phase I dose-escalation trial using umbilical cord blood-derived MSC. That trial demonstrated the short- and long-term safety and feasibility of MSC, given that significantly reduced inflammatory marker expression was observed in tracheal aspirates. As of recently, several clinical trials of MSC use for BPD are ongoing or are planned in some countries to investigate the efficacy of MSC in the prevention or treatment of BPD in premature infants. Many clinicians are currently awaiting the results from these trials so that MSC can be used clinically for human BPD.
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