The Therapeutic Potential of Stem Cells for Bronchopulmonary Dysplasia: "It's About Time" or "Not so Fast" ?
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Mong Tieng Ee1, Bernard Thébaud1,2,3
1Division of Neonatology, Department of Pediatrics, Children's Hospital of Eastern Ontario (CHEO) and CHEO Research Institute, Ottawa, ON, Canada.
Insights
Mesenchymal Stromal Cells (MSCs) show promise in treating bronchopulmonary dysplasia (BPD) in premature infants. Early clinical trials are evaluating MSCs
Area of Science:
- Neonatal Medicine
- Pulmonology
- Regenerative Medicine
Background:
- Bronchopulmonary dysplasia (BPD) remains a significant complication in extremely premature infants, with high rates of morbidity.
- Despite advances in neonatal care, effective therapies to prevent or treat BPD are lacking.
- The multifactorial nature and increasing lung immaturity contribute to BPD's persistent challenge.
Purpose of the Study:
- To review current knowledge on Mesenchymal Stromal Cells (MSCs) for BPD treatment.
- To summarize preclinical findings supporting MSCs' therapeutic potential in neonatal lung injury.
- To discuss the rationale and ongoing early-phase clinical trials of MSCs in extremely premature infants.
Main Methods:
- Review of existing literature on Mesenchymal Stromal Cells (MSCs).
- Analysis of preclinical studies in neonatal rodent models of lung injury.
- Examination of data from early-phase clinical trials investigating MSCs for BPD.
Main Results:
- MSCs exhibit pleiotropic effects beneficial for lung injury.
- Preclinical studies demonstrate MSCs can prevent or rescue lung injury in neonatal rodents without adverse effects.
- Early clinical trials are exploring the safety and efficacy of MSC therapy in premature infants.
Conclusions:
- Preclinical data provide a strong rationale for investigating MSCs in clinical settings.
- Further research is needed to address remaining knowledge gaps regarding MSC therapy for BPD.
- Early phase clinical trials are crucial for determining the safety and efficacy of MSCs in this vulnerable population.
Objective:
While the survival of extremely premature infants has improved over the past decades, the rate of complications - especially for bronchopulmonary dysplasia (BPD) - remains unacceptably high. Over the past 50 years, no safe therapy has had a substantial impact on the incidence and severity of BPD.
Methods:
This may stem from the multifactorial disease pathogenesis and the increasing lung immaturity. Mesenchymal Stromal Cells (MSCs) display pleiotropic effects and show promising results in neonatal rodents in preventing or rescuing lung injury without adverse effects. Early phase clinical trials are now underway to determine the safety and efficacy of this therapy in extremely premature infants.
Results And Conclusion:
This review summarizes our current knowledge about MSCs, their mechanism of action and the results of preclinical studies that provide the rationale for early phase clinical trials and discuss remaining gaps in our knowledge.